HMPI

Pharma Prices Are Not Too High (Usually)

Will Mitchell, Anthony S. Fell Chair in New Technologies and Commercialization, Rotman School of Management, University of Toronto

Contact: Will Mitchell, william.mitchell@Rotman.Utoronto.Ca

Abstract

What is the message?

Although drug pricing is highly contentious around the world, with frequent claims of overcharging, average profitability in the pharmaceutical industry is not excessive. Companies need to achieve prices above total average costs if they are to cover the fixed costs of successful and failed development efforts. The most efficient way to accomplish the dual goal of incenting ongoing innovation while also achieving cost effectiveness and broad-based access is to price drugs at different prices in different markets, based on some combination of ability and willingness to pay.

What is the evidence?

Assessment and evaluation of current bio-pharmaceutical industry data, trends, and strategies.

Disclosure: Some of the academic health management programs that I have taught in and several of my research projects have received programmatic support from life sciences companies. This article received no financial or editorial support. All information in the article is based on public sources.

Submitted: October 1, 2018; accepted after review: October 31, 2018.

Cite as: Will Mitchell. 2018. Pharma Prices Are Not Too High (Usually). Health Management Policy and Innovation, Volume 3, Issue 2.

Drug pricing has been controversial in the U.S. and elsewhere essentially as long as drugs have been sold. In 1959-1960, the Kefauver Drug Hearings conducted by the U.S. Senate Subcommittee on Antitrust and Monopoly concluded that pharmaceutical firms did not merit the prices they were charging; [1] moreover, as William Comanor put it in 1966, “the committee charged that little of social value came from industry laboratories”. [2] Scrolling forward to the present, in the past decade or so, prices have escalated far higher, particularly as the biological revolution has taken hold, with some drugs now having list prices in the tens and even hundreds of thousands of dollars.

With increased drug prices has come scrutiny and debate. Perhaps the only thing that the three highest profile candidates in the 2016 U.S. Presidential primaries and election – Donald J. Trump, Hillary Rodham Clinton, and Bernard Sanders – agreed on was that drug prices are far too high. Article after article in the press and media in the U.S. and other countries, as well as highly publicized Congressional hearings, have reported claims of high prices and massive price increases, sometimes targeting individual executives as responsible for price gouging. The current U.S. administration has announced an ongoing sequence of potential initiatives directed at what it believes are excessive prices. [3] And in the 2018 Gallup poll of industry reputation, the pharmaceutical industry finished 29th of 30, with a net negative rating of 23% [30% positive; 53% negative], slightly ahead of only the U.S. federal government. [4] Clearly, drug prices must be too high.

Yet what would it mean for prices to be “too high”? The simplest conceptual case would be that value received by patients and other stakeholders in the healthcare system does not justify the prices charged by pharmaceutical companies and intermediaries such as distributors, presumably because the companies have market power that allows them to price above marginal cost. Yet, as I will argue in this article, a close look at relevant data does not support this conclusion.

Instead, thoughtful assessment suggests that average profits in the pharmaceutical industry are largely in line with the companies’ needs to support ongoing development and commercialization of new drugs and related services. Although some individual cases may be questionable, the overall pattern is one of an industry that typically acts responsibly in supporting necessary business activities while seeking to provide value for patients.

I will start with the assumption that recent advances in drug therapies are making important contributions to healthcare and human health. While there are credible debates about marginal value and concerns about side effects of some drugs and, especially, which patients they might be suited to, there are undoubted major contributions in areas ranging from multiple types of cancer, to Hepatitis C, to a broad set of immunological diseases, to ophthalmic needs, to HIV/AIDS, and a host of other conditions. [5] Moreover, the companies that market these drugs also are increasingly providing a range of support such as infusion services, patient and provider education, patient financial support, nutrition and life style counselling, pay for performance contracts, and other services that provide far more encompassing value than simply a core pill or injection.

Some of these advances of drugs and complementary services serve tens of thousands of people. Others serve only a few individuals who suffer from orphan diseases. Again, while it is entirely reasonable to question whether a particular therapy suits an individual patient in a given context, the overall impact is contributing to solving real human medical needs

Rather than a single price, drugs have multiple prices

Of course, improved health by itself is not enough to end an argument about the industry. There needs to be a corresponding judgement about cost effectiveness [6], as reflected in the price that drug companies receive from the myriad types of payers. This is where the controversy arises. Yet payers, particularly third party payers such as pharmaceutical benefit management companies (PBMs) with substantial market power of their own, have substantial ability to negotiate discounts and rebates, manage formularies, and shape whether drugs achieve market access. [7]

In practice, actual prices to most payers typically are far below list prices that show up in public reports such as Average Wholesale Price (AWP), which one of my colleagues informally likes to refer to as “Ain’t What’s Paid”. Indeed, for almost all drugs, there is no one price – instead, there are multiple prices, sometimes even to the same payer, based on negotiations, public mandates, pressure from patients and patient advocates, and market conditions.

It is not my intent here to argue that any one price to any one payer is “too high” or “too low”. It is the job of any payer to negotiate a price that meets its own definition of value. And there may well be appropriate opportunities to help some payers increase their negotiating strength and sophistication, and so help the healthcare system meet the goals of cost effectiveness and broad access.

Rather, my aim is to argue that the overall revenue that drug companies receive from payers aligns with the health system’s complementary goals of creating incentives for ongoing innovation and for providing as broad access as possible to appropriate health services. Several metrics and practical conditions of the industry underlie this conclusion. In the next sections, I will discuss profitability, the need for average prices of pharmaceuticals to exceed variable costs, and the increasingly short time window for companies to recoup fixed costs of developing and bringing drugs to market before they face generic competition.

Average profitability grew through the early 2000s and then declined

Using companies’ financial statements, I have collected data on more than 70 major pharmaceutical firms that have sold branded pharmaceuticals, including long established companies and new biological entrants based in Western Europe, North America, and Japan, with data for most firms going back into the 1970s or earlier. The most complete period, 1990 to 2017, includes 33 to 50 firms per year, with the numbers varying due to consolidation. Where appropriate, I also draw data on the more than 500 public pharmaceutical and biological firms listed in the Compustat data base. This section and the discussion of costs that follow will be a bit numbers heavy, because it is necessary to use real data to provide an accurate picture of the industry and relevant trends.

The simple story of pharma profitability is that it grew and then declined. In the 1980s and early 1990s, bio-pharma firms’ median profitability based on return on sales (ROS) was about 7% to 11%, a comfortable but not particularly high rate of return. During the 2000s, with the introduction of new generations of large market drugs, median ROS grew to a maximum of 17% in 2009.

Then, between 2010 and 2017, list prices of many high profile new drugs, particularly newly-approved biologicals, increased substantially. One might expect corporate profits to have grown even higher in the past decade.

However, rather than continuing to increase, median return on sales in the industry has declined: falling to 13% to 14% in each year from 2014 to 2017 (the trends are similar if we base profitability on return on assets). Within the average, there is substantial variance. In 2017, for instance, the profitability of 33 companies with a median ROS of 13% ranged from a high of 41% (reflecting gains from the sale of a major business unit) to a low of –9% (reflecting ongoing losses at a biological specialist). In 2016, with median ROS of 14%, the maximum and minimum ranged from 45% (a win from a blockbuster biological) to –56% (the same financially struggling biological specialist as in 2017).

While some firms in some years have achieved particularly high profitability, occasionally even with ROS of 40% or higher – 1.5% of the cases among 1,193 years of observations for 61 unique firms in my data from 1990 to 2017 exceed 40% ROS; 5% exceed 30% – such cases typically last at most for a few years and fall again as competing products enter the market. Hence, even as list prices have increased, average profits in the industry have fallen.

Overall, the bio-pharma industry is now comfortably profitable on average, but far from levels that suggest massive systemic price-gouging. Indeed, major ongoing price reductions on the scale that some critics appear to believe appropriate would quickly lead to untenable financial levels for most or all firms.

Consider the simple math: if the median firm with 13% ROS in 2017 was forced to cut its net prices by 10% with no other changes to its business model, it would barely clear break even.  And most of the half of its competitors with ROS below the median value would fall to break even or below. The arithmetic becomes a bit more complicated if the price cut only applied to U.S. sales, which typically mark well over 40% of a U.S. based company’s revenue (and often much higher) and lesser but still substantial proportions for European and Japanese pharma firms, but the financial impact would quickly be unsustainable. Yet a 10% price cut is well below what the debate would suggest is needed.

Cross-industry comparisons of multiple profitability metrics (e.g., return on sales, assets, capital, and equity) based on reports from the industry analyst firm CapitalIQ show that the pharmaceutical and biological industry categories tend to be somewhat more profitable on average than the S&P 500 list of the largest public firms, commonly at about the level of technology and computer hardware companies, but again well within a range of normal profitability. Why, then, have bio-pharma profits declined as list prices – and controversy about prices –have increased?

The costs of doing business have increased

I will highlight four reasons for declining profitability during the past decade: discounts, production costs, R&D expenses, and marketing expenditures. First, part of the cause for the decline in average profitability is the issue I noted earlier: list prices are largely meaningless. During the past decade, third-party payers in the U.S. have become increasingly aggressive about demanding discounts in return for agreeing to include drugs on their formularies of drugs approved for their covered lives. Particularly as competitor products enter a therapeutic class, negotiated discounts commonly increase and net prices fall.

While most discounts are confidential, reports from Kaiser Permanente, investigative journalists, and others suggest that rebates can reach 25% to 50% or more of list price. Hence, while a company with a first-to-market blockbuster may enjoy a few years of high profitability, competition and reactions by payers tend to bring it back down.

Second, many of the newer biological drugs are more expensive to produce than earlier generations of small-cell pharmaceuticals. Median “cost of goods sold” (COGS), i.e., production costs of the drugs, of two dozen major firms I am tracking has grown from about 21% in 2000 to 25% in 2017. While not a huge increase, the extra costs have been enough to affect profits.

Third, the costs of developing and obtaining new drugs have increased due to increasing need for multi-source development activities. It simply is not possible for a single company to possess all the skills needed to bring a full portfolio of drugs to market by relying solely on internal development. Instead, bio-pharma firms now employ a sophisticated set of build, borrow, and buy strategies to source new drugs, including a complex mix of internal R&D, alliances, and acquisitions.

The extent of both acquisitions and alliances has grown strikingly according to figures reported by data bases such as ReCap, Cortellis, CapitalIQ, and SDC Platinum. The annual number of M&A deals has grown from 150 to 200 in the late 1990s, to more than 500 in 2017. Some M&A deal values reach billions of dollars: Cortellis reports more than $250 billion in global bio-pharma acquisition value in each of 2016 and 2017.

Inter-firm alliances in the sector also have become increasingly common. In 1990, fewer than 350 alliances were reported in industry data bases. In 2017, depending on the data source, the reported number had grown to somewhere between 2,500 and 4,000 partnerships. While alliances tend to have lower deal value than acquisitions, annual expenditures now total multiple billions of dollars.

Internal R&D costs, meanwhile, rather than decline as acquisitions and alliances have increased, have also grown. Annual R&D expenditures by 22 major bio-pharma firms in 2000 reached about $32 billion; in 2017, the top 17 bio-pharma companies spent about $73 billion. Across the full set of publically traded bio-pharma firms reported by Compustat, R&D expenditures in 2000 and 2014 grew from $51 billion to $118 billion. In addition to sheer magnitude, R&D has also grown as a percentage of sales: among the leading firms, the average ratio grew from 15% in 2000 to 18% in 2018.

Fourth, selling, general, and administration (SG&A) expenditures also have grown, though at a slower rate than R&D. SG&A is an indicator of marketing and other commercialization activities, including the costs of patient support that are now important parts of the suite of services that complement a core drug. Compustat data for public bio-pharma firms report SG&A growth from about $150 billion in 2000 to $280 billion in 2014.

Despite the growth in magnitude, average SG&A as a percentage of sales has remained stable or even fallen; among the top 30 to 40 firms, the ratio fell from 35% in 2000 to 28% in 2017. The reduction in the marketing cost ratio reflects the shift in strategy during the period, from emphasizing large market drugs such as cardiovascular statins and gastro-intestinal proton pump inhibitors, which require large sales forces, to placing greater emphasis on products such as immuno-oncology drugs prescribed by specialist health care providers, which require more targeted commercial support. At the same time, though, the newer drugs have required substantial patient support as well as market access expenditures in negotiations with third-party payers, which limits the ability to undertake further reductions in expenditure.

The core point here is that multiple aspects of development and commercialization costs have grown more quickly than revenue during the past decade or so, despite the frequent claims of excessive price increases. The expenditures reflect the real costs of doing business in the bio-pharma sector: obtaining and creating new drugs, conducting trials, manufacturing the drugs, gaining market access, and supporting them in the market. Currently, the industry is not under a threat of failure but also is not at any obvious level of excessive profitability that would support extensive price cuts. While individual firms may enjoy very high profits for a few years, they typically return to earth; the overall profile is reasonable. In parallel, firms may suffer low or even negative profits in some years – 14% of the annual observations for the major firms in my data from 1990-2017 report losses – but typically return to reasonable levels, or are purchased by competitors who can use their resources more effectively.

Fixed costs are high, with a substantial gap between variable costs and total average costs

Now let’s leave the deep dive into data and consider the nature of costs in the industry. Bio-pharma is marked by high fixed costs in R&D, whether done internally or paid for via alliances and acquisitions. Successful projects typically require many years and many millions of dollars in lab work, clinical trials, and regulatory expenses, whether initially sourced internally or externally. And many projects fail – that is the very nature of experimentation – sometimes early in the development cycle after a few million dollars but occasionally reaching into the hundreds of millions or more if a drug proceeds to large scale Phase 3 trials before failing.

Figure 1 builds on this point. Development costs, including the costs of failures, are fixed costs. As such, they do not show up in the average variable costs required to produce and support a drug and its associated services in the market. The high relevance of fixed costs creates a substantial gap between “Variable cost to produce” and “Total average cost”, as the horizontal red lines in the figure depict.

Figure 1. Bio-pharma value, costs, and prices

The gap between variable costs and average total costs introduces a major issue in bio-pharma price negotiations. The goal of a pharmaceutical firms’ negotiator is to gain a price as close as possible to a buyer’s value ceiling (the horizontal green dashes in the figure). Yet few buyers will simply pay for the full value they receive from any product – including products that produce health value – if they can negotiate a lower price.

Instead, the goal of any payer is to negotiate price down as close as possible to its estimate of the seller’s average variable cost. That is the minimum price below which a producer cannot cover its operating expenses. And, if pushed to the limit, the variable cost floor is the price that a seller will settle for: this rate at least covers the costs of producing and selling the drug. Where the price ends – up near the value ceiling or down on the variable cost floor – depends on the comparative bargaining power and negotiation skill of the buyer and seller.

This is a challenging negotiating calculus. All payers in the health sector face real pressure on their budgets, with increased prices for pharmaceutical products creating part of those pressures. In the U.S., prescription pharmaceuticals accounted for almost $325 billion in 2015, about 10.1% of national health expenditures (up from 8.8% in 2000), behind the hospital (32%) and physician/clinical (20%) shares. [8] In Canada, drug expenditures reached about 16.2% of national health expenditures in 2016, behind only hospitals (29%), up slightly from 15.4% in 2000. [9] Hence, even though a payer may recognize and even embrace the high health value ceiling of a pharma product, it needs to push as hard as possible to bring prices down toward the variable cost floor.

The individual buyer’s goal to push prices to the floor in turn creates the rub for the seller. If every payer successfully negotiated a price that just met a drug’s average variable cost, then the company producing the drug would fail because it was not covering its fixed costs of development. Instead, a bio-pharma firm requires some degree of negotiating power to charge at least some buyers prices that exceed the minimum market clearing price, shifting up toward the value ceiling.

Indeed, this is one of the purposes of the patent system: to provide a successful innovator with a period of exclusivity in which it can recover the costs of creating the innovation. Then, once a patent ends or, equally powerfully, once a product with similar therapeutic value enters the market, competition will drive prices down toward marginal costs. This balance – of incentive to innovate and competition to bring on subsequent price pressure – is central to patent policy and law.

Now let’s use Figure 1 to make things a bit more complicated, while introducing a necessary part of pharmaceutical pricing strategy. The bio-pharma market, like almost all markets, has multiple segments, with different customers who have differing ability and willingness to pay for a product. In Figure 1, these segments are depicted as Markets A, B, C, and D. For a pharmaceutical company, the goal is to identify the combination of value and ability to pay for each segment and, ideally, settle on prices that come close to that value ceiling for each market, while surpassing variable costs. Such price discrimination strategies are profit maximizing.

Market segmentation introduces pricing variation across and within countries. For instance, prices are commonly lower in lower income countries such as Greece and Spain, versus higher prices in higher income countries such as Germany and the U.S. Even within countries, different payers commonly have different ability to pay. In the U.S., for example, the state-based Medicaid systems are mandated to receive the lowest price negotiated by any other actor, while other payers such as employment-based PBMs commonly have greater latitude and financial resources.

In such cases of multiple market segments with different ability to pay, a pharma company’s dominant strategy is to set different pricing points for each segment. To be sustainable, the strategy needs to result in some prices being high enough above the variable cost floor, in aggregate across the company’s portfolio of products, to cover the gap between that floor and total average costs.

Market D in Figure 1, where buyers are not able or willing to pay enough to cover even the variable costs of production, introduces a further complication. In most industries, sellers would ignore this segment. Yet in healthcare markets, most people – including most people who work in pharmaceutical companies – feel a responsibility to reach as many patients as possible, including those who cannot pay enough to cover operating costs. While no public or private actor can afford infinite below cost contributions, there is real need and desire to provide access to as much of the population as possible. For pharma companies, this is part of the basis of patient assistance programs, which provide subsidized or free drugs and services when people in market D lack insurance or personal resources. In turn, though, such below cost strategies place even more pressure to move well above the variable cost floor when negotiating with buyers in market segments A, B, and C.

Quite simply, “too much” pressure to drive prices down will lead to two negative consequences. In the short term, the pressure will drive out a company’s ability to provide cross-subsidized services below the variable cost floor. In the longer term, the pressure will drive innovative firms out of the market because they cannot cover the gap between variable and total costs.

Time windows before competitors enter have become shorter

Now consider time windows for pharmaceutical companies to earn high prices, even after discounts. The key issue here is penetration of the market by generic drugs and “biosimilars” of biologics. Before the 1984 Hatch-Waxman legislation facilitated entry of generic competitors when drugs came off patent, generic drugs made up about 20% of prescriptions in the U.S. Generic penetration grew to about 40% in 2000. Today, as generic and biosimilar competition has become much more active, and as payers respond to pricing pressure by mandating generics on formularies whenever possible, the generic prescription rate in the U.S. is about 90%. Most traditional developed markets also are at substantial, if somewhat lower, levels; Canada, for instance, has a generic prescription rate of about 70%.

Moreover, under the rules of Hatch-Waxman and similar policies around the world, generic approvals have grown exponentially. In 1984, the U.S. FDA reported 66 approvals; in 2017, there were 847 generic approvals. [10] Generic competitors are commonly lined up to enter the market immediately when a drug goes off patent, particularly if the drug had achieved a substantial market size.

Once generic competitors enter, prices fall, sometimes drastically. Prices for traditional large market drugs with multiple generic competitors commonly face price reductions of as much as 90%. Price reductions for the specialized biologicals that have begun to go off patent and face biosimilar competition have been less striking, because so far there are fewer competitors. Nonetheless, the reductions are substantial, with discounting in the range of 35% commonly being reported.

Once generic competition becomes active, prices move, often rapidly, toward the variable cost floor in Figure 1. From the point of view of payers, this is a good outcome. And, again, this is one of the goals of the patent system: provide a period of exclusivity as an incentive to innovate, then open the doors to competition to create incentives to innovate again as well as providing cost effectiveness in the market.

From the point of view of a bio-pharma innovator, though, this increasingly striking combination of early generic entry and strong price reductions means that there are far fewer years than there once were to earn the profits needed to cover the gap between variable and fixed costs. If the firm is going to survive, it needs active strategies to deal with the shorter window.

Time window strategies have had two major components, both of which we have noted earlier. First, companies are increasingly emphasizing specialty drugs. This is partly a feature of the biological revolution, which has created opportunities for major contributions to health for targeted medical needs. In addition, drugs for specialty market segments such as oncology and immunology, whether based on biological or older science, typically face lower rates of post-patent generic competition, both because only a few firms currently have the skills needed to compete effectively and because specialty prescribers and patients are often reluctant to switch away from drugs that they have come to understand and rely on. Hence, even after patents expire, specialty drugs commonly achieve prices above the variable cost floor, sometimes for multiple years.

Second, firms have increased the initial prices they charge during the shorter protected window, in order to generate income as quickly as possible before facing generic or biosimilar competition. Thus, the policy changes that have encouraged price competition from generics – as desirable as these policies undoubtedly are for payers, patients, and the health system – have also created strong pressures to increase prices that policy makers are now complaining about. The quid of competition and long term price reductions has induced a pro quo of initial price increases.

Where does this leave us?

The key question is whether healthcare systems in countries such as the U.S. are now reasonably close to achieving a balance of innovation and cost effectiveness or, as some voices implicitly claim, can we continue to maintain incentives to innovate while drastically bringing down pre-generic prices? My interpretation of the data and observation of strategies and incentives is that we are fortunate to have an actively innovative bio-pharma sector, made up of a complex and dynamic mix of established firms, new ventures, academic and government scientists, complementary firms, and regulatory bodies, paralleled by a critically important set of generic manufacturers (some of which are the same companies) that help provide discipline in the system. This quasi-market is far from fully efficient – no market is – but it has evolved to a point of generating and commercializing new bio-pharmaceutical products and supporting services at a rate that is higher than at any point in the history of the industry.

Consider recent innovation. Just as approval of generics is increasing, so is approval of new drugs. In 2017, the U.S. FDA reported granting 54 new approvals, including 21 biologicals. This was the highest rate ever (other than a 1996 clean out of the approval pipeline), up from 29 (2 biologicals) in 2000. The companies that received the approvals included a wide mix of established pharmaceutical firms from Western Europe, Japan, and the U.S., plus an even wider mix of new ventures and smaller specialty companies.

Now consider market entry. Innovator companies commonly apply for FDA approval and introduce their drugs to the U.S. market before entering other countries. The prices available in the U.S. are not as much higher than those in other traditional developed markets as reported list prices would suggest, but on average are somewhat higher, due in large part to the multiple segments available for price discrimination. In turn, profitability in the U.S. is higher: the few firms that report geographic profit margins commonly recognize operating profits as a percentage of sales that are 15% to 25% higher in the U.S than in Europe. Higher prices and profits typically lead to faster entry, earlier access to novel treatments, and ultimately, earlier access to lower-priced generics once the innovators’ patents expire.

It is important to recognize that there are outliers in the system. Some companies earn very high profits, though typically for only a few years. In part, the hope of such profits can be viewed as a lottery that incents entry to the industry. Indeed, there are far more companies with very negative profitability than very positive results – the median return on sales of all bio-pharma companies in the Compustat data base (now more than 500 firms) has been negative each year since 1989. The lure of a payoff is a necessary complement to the uncertainties of experimentation.

There are also outliers in pricing strategy that on the face, and likely even the depths, of it do appear unreasonable. Massive increases in prices of sole-source generic drugs that can be maintained until competitors gain approval and enter, which typically takes several years, stick in many craws. But it is important to recognize that these are outliers and not to develop general policies targeted at extreme cases.

A key point here is that it is equally important to recognize that lower prices typically lead to later entry and sometimes no entry at all. Introduction rates to lower priced southern European markets, for instance, are substantially lower than in North America or much of northern Europe. Simply forcing prices to a lower level in any given country, whether the U.S. or elsewhere, would almost certainly lead to reduced entry in that country.

Nonetheless, there is an active policy and health question here, of when higher initial prices in a market – and consequent more active entry – are balanced by greater health benefits. The answer to that question requires engagement of multiple stakeholders in the health system, including payers, prescribers, regulators, bio-pharma companies, and patients. Currently, once a drug has received market approval, payers such as PBMs have become increasingly powerful gate keepers in access, with their role in setting formularies. There is real strength in that role, but there are also opportunities for more effective engagement of the other stakeholders in assessing costs and benefits.

If there is a need for policy initiatives, the goal of integrating insights across the fragmented silos of the healthcare system in the U.S. and elsewhere is far more of a priority than the marginal reductions in prices that could be accomplished without drastically damaging the ability of innovator companies and their generic followers to provide continuing health value. Rather than the current debate about costs, we would be much better served by a debate – and action – about appropriate value.

References

  1. Daniel C. Morgan, and Samuel E. Allison. 1964. The Kefauver Drug Hearings in Perspective. Southwestern Social Science Quarterly, 45 (1): pp. 59-68. https://news.gallup.com/poll/12748/business-industry-sector-ratings.aspx
  2. William S. Comanor. 1966. The Drug Industry and Medical Research: The Economics of the Kefauver Committee Investigations, The Journal of Business, 39 (1): pp. 12-18. https://www.jstor.org/stable/2352011?seq=3#metadata_info_tab_contents
  3. Yasmeen Abutaleb and Michael Erman. 2018. Trump seeks to base Medicare drug prices on lower overseas rates. Health News, October 25. https://www.reuters.com/article/us-usa-trump-drugpricing/trump-seeks-to-base-medicare-drug-prices-on-lower-overseas-rates-idUSKCN1MZ2SF
  4. https://news.gallup.com/poll/12748/business-industry-sector-ratings.aspx
  5. As one of many examples of studies of health benefits, see a discussion of health benefits for rheumatoid arthritis see: John J. Cush and Kathryn H. Dao. 2007. Perspectives on Safety vs. Benefits of Biologic Therapies. Medscape Rheumatology. https://www.medscape.org/viewarticle/553515_4
  6. Robert S. Kaplan, Michael E. Porter, Mark L. Frigo. 2017. Managing Healthcare Costs and Value. Strategic Finance 98 (no. 7): pp. 24–33.
  7.  Cole Werble. 2017. “Health Policy Brief: Pharmacy Benefit Managers,” Health Affairs, September 14, 2017. DOI: 10.1377/hpb20170914.000178
  8. Centers for Medicare & Medicaid Services, Office of the Actuary, National Health Statistics Group.
  9. Canadian Institute for Health Information (CIHI): https://apps.cihi.ca/mstrapp/asp/Main.aspx
  10. Drugs@FDA: http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm?fuseaction=Reports.NewOriginal_ANDA

Value-Based Pricing as a Signal for Drug Innovation

 Jennifer Ohn, MPH, Anna Kaltenboeck, MS, Memorial Sloan Kettering Cancer Center

Contact: Anna Kaltenboeck, kaltenba@mskcc.org

Abstract

What is the message?

In today’s pharmaceutical drug market, revenue is not directly associated with value. As drug prices increase, the financial burden is felt by payers and patients alike. Various strategies are undertaken to address this issue, but none addresses the conflicting market signals for what types of innovation are valuable. Incorporating analytic evaluations of value, value-based pricing takes aim at the increasing prices for the healthcare system and for patients.

What is the evidence?

Assessment and evaluation of current market strategies among stakeholders involved in the drug pricing supply and reimbursement chain.

Submitted: September 9, 2018; accepted after review: September 17, 2018

Cite as: Anna Kaltenboeck, Jennifer Ohn. 2018. Value-Based Pricing as a Signal for Drug Innovation. Health Management Policy and Innovation, Volume 3, Issue 2.

Introduction

By announcing that it would require new drugs to meet a threshold of $100,000 per quality adjusted life year (QALY) or face exclusion from their formulary, CVS Caremark, one of the largest pharmacy benefit managers (PBM) in the US, fired the latest salvo in the debate about the price of drugs in relation to their value.1 For years, the prices of specialty branded drugs have commanded headlines with their continued ascent, both for drugs dispensed by pharmacies and for those administered by physicians. This trend has highlighted the need for an analytic alternative to current approaches – an alternative by which a drug’s attributes and outcomes are systematically evaluated by the many stakeholders involved. Financial pressures due to the rise in prices have raised questions about what we are willing to trade off to pay for new drugs. The controversy has become particularly pressing at the state level, where drug coverage can significantly affect Medicaid spending, which comprises 28.7% of all total state budgets, as well as among patients who struggle to afford the out-of-pocket payments for their prescriptions.2 Beyond affordability concerns, compromises made to afford high-priced drugs often result in access restrictions that hamper the ability of patients to use therapies from which they might otherwise benefit.

Revenue as a Driver of Innovation: An Imperfect Signal of Value

By definition, affordability and access are linked to price and volume, the two variables that make up the revenue equation for drug manufacturers. These revenues act as the reward for innovation and commercial success, funding ongoing R&D efforts in hopes of developing innovative therapies and allowing manufacturers to recoup the expenses of clinical development. Formalized under the Drug Price Competition and Patent Term Restoration Act of 1984 (Hatch-Waxman Amendments), this arrangement grants branded drugs a period of time on the market during which they are protected from competition by generics or biosimilar equivalents of biological drugs. Underlying this approach is the assumption that revenue potential for new drugs offers a good signal about what type of innovation is valued, and that manufacturers will invest accordingly. Where this alone fails to offer sufficient inducement, such as with rare diseases with small populations, further provisions are offered to improve the payoff for innovation through tax credits, additional periods of exclusivity, and less burdensome regulatory review.

In an ideal market, greater revenue would be associated with greater value; however, for drugs, the market often fails to provide such clear direction. The current system of purchasing and reimbursement operates on transaction fees, spread pricing, and markups that generally have the effect of increasing pressure on prices. A well-known example is the gap between the list and net prices of drugs dispensed by pharmacies. Known as the “gross to net differential”, this disconnect arises from discounts and rebates provided by manufacturers throughout the supply chain to encourage preference for their drug, often at the expense of access to competitors’ drugs. Another is the practice of “buy-and-bill”, in which physicians are reimbursed for administering a drug plus its cost and a percentage markup, creating incentives to prefer higher priced drugs when the opportunity arises. For example, prescribing tendencies in oncology show a bias toward higher priced drugs; all other things being equal, drugs being offered at a lower price could find themselves at a competitive disadvantage compared to their more expensive peers.3

These practices result, among other things, in directionally problematic signals to the market about what types of innovation are valued.  For example, as of 2017, there were over 5,789 therapies in pipeline for oncology, more than 7 times the number of therapies in development for cardiovascular disease;5 yet the health impact of the two disease categories is similar – 1.2 years of life are lost due to malignancies for every one lost to cardiovascular disease.6 Prices of oncology drugs have been increasing over time, even when adjusted for life year gained, begging the question of how well existing incentives perform in driving drug development.7

Solutions to Date: Limited Impact

There has been no lack of proposed solutions to these problems, and many have already been implemented (see Table 1). One approach is to modify incentives within the supply chain, built on the thesis that addressing these financial interests reduces their inflationary pressure on drug prices. Efforts such as these may offer financial enticements for granting access to treatments that are known to provide certain health outcomes. This is a frequent feature of risk-sharing agreements, such as those in which health plans and providers are paid at least in part according to the number of patients receiving preventative care and screening or how many with diabetes achieve blood sugar control targets.8,9 Centers for Medicare and Medicaid Services (CMS) created the Star Rating System in 2007 with the goal of increasing payer accountability among their Medicare plans. Plans are rewarded through quality bonus payments (QBP) and Medicare Advantage rebate percentages based on the Star Rating and its bid. In the Medicare Shared Savings Program Accountable Care Organization model, participating provider organizations are rewarded when costs in Medicare Part A and B are lowered. Provider risk-sharing is also the rationale behind bundled payments; providers receive a fixed reimbursement for a particular bundle of care, with the choice of treatment left up to them. However, there is little or no evidence to suggest that these solutions have changed formulary placement and treatment selection in ways that reward value.

Table 1. Approaches for mitigating incentives for high drug prices

 

Approach

 

Premise

Changing payment practices Risk-sharing: By rewarding health plans and providers for performance on population-level outcomes measures, they  are incentivized to prefer treatments that improve outcomes
Bundled payment: By paying for a bundle of care or for meeting certain performance metrics, providers are incentivized to select treatments that improve care quality
Competitive acquisition & white bagging: By disintermediating providers from the purchasing process, payers can mitigate markup incentives that promote inflation
Changing patient incentives Cost-sharing: Increasing the patient’s share of a drug’s cost, they create downwards pressure on prices of drugs
Value-based insurance design (VBID): Matching the cost-sharing levels with evidence-based measures, health plans are able to  direct patient decision-making to reflect value
Value-based pricing Systematic analyses to determine a price based on a drug’s attributes and stakeholder preferences

Another approach to the problem is to change patient incentives in an effort to leverage their sensitivity to cost. Health plans are able to do so by shifting payment responsibility for a greater portion of the cost to beneficiaries through both high deductibles and higher co-pay and co-insurance rates. Value-based insurance design (VBID) also takes aim at this price sensitivity but instead harnesses it by lowering out-of-pocket payments for drugs that health plans would like to encourage them to use.

These approaches face a major practical barrier:  manufacturers can counteract this shift by providing copayment support to offset the out-of-pocket spending, making the tool moot. Copay assistance programs prevent patients from acting as consumers and undermine insurance companies’ leverage. Absent copay support, many patients are effectively priced out of the market, directly affecting adherence to potentially beneficial drugs. One study found that patients facing higher copayments for imatinib (Gleevec), a drug used to treat chronic myeloid leukemia and a number of other cancers, were significantly more likely to discontinue treatment than those with lower out-of-pocket expenses, putting them at risk of disease progression.10

The overarching drawback in these two approaches is that they do not address the issue of the unclear signal in the market of what treatments have value. None do away with confidential net price concessions in exchange for patient access. This makes it unclear what price would be acceptable for extending the incremental benefits of a new therapy to as many patients as could use it.

Potential Solution: Value-based Pricing

Benefits of value-based pricing

Value-based pricing aims to solve the problem of conflicting signals by offering a transparent and replicable analysis of the available evidence that reflects public input.11 The resulting price captures only the value of a drug’s incremental benefits over existing options, that is, it quantifies the price at which the financial trade-offs for offering treatment to all eligible patients would be net beneficial to patients and a good value for the healthcare system as a whole. In addition to providing a clearer understanding of what trade-offs must be made to maximize the health gains of new therapies, this model also gives manufacturers a clear line of sight of the desired attributes of new therapies without including the additional distortions that are added through purchasing and reimbursement transactions.

Overcoming implementation challenges

The challenge of value-based pricing lies in its implementation. To work effectively over a drug’s life cycle, pricing must be set at market entry. As post-approval evidence becomes available, the price should be adjusted accordingly to ensure that health gains continue to be captured and innovation incentivized accordingly; yet, current payment practice is not yet equipped for this. To be successful, this approach also requires that access be granted to those drugs that are priced at or below value, and penalizes those that are not.

This also brings up major challenges in multi-stakeholder buy-in. In the immediate term, systematic and complete implementation of such policies would be a heavy lift for payers. The challenge is especially striking for novel drugs with few competitors. New York State’s experience illustrated this challenge when Vertex12 , the manufacturer of the cystic fibrosis drug Orkambi, dismissed calls to bring its price in line with the drug’s value.

CVS acknowledges this limitation by exempting drugs with breakthrough designation from their program. But the industry, particularly health plans and PBMs, can gain practice in less contentious areas. Most branded drugs with multiple competitors that have similar benefits already compete heavily on net price to capture market share through formulary placement. By predicating coverage on their ability to meet or beat a value-based price, they would be forced to compete for patient preference or face exclusion from the formulary.

Outpatient drugs pose a different problem as neither list nor net price are incentivized to be lower. Unlike pharmacy benefits, medical benefits have no formulary, so there is no existing pathway for creating preference on the part of health plans. Here, plans can turn to what is known as “white bagging”, or competitive acquisition, an alternative system of reimbursement under which physicians order drugs on demand from a specialty pharmacy or distributor that will deliver it to the provider on behalf of the patient. This arrangement allows the health plan to pay the pharmacy or manufacturer for the drug, circumventing the markup to physicians and paying them in other ways to encourage value-based care. Already being used by a number of plans, this approach enables them to begin managing access according to whether or not a drug meets a value-based price.13

Looking Forward

Still in its infancy, the shift to value-based pricing will require changes to how the healthcare industry conducts its business. Conditioning formulary placement and provider payment on whether a drug has a value-based price are important first steps.14 Until recently, health plans’ coverage policies were driven primarily by pharmacy and therapeutics committee  reviews that focused on efficacy and safety; adding the assessment of value will require, at a minimum, new processes and skills. In the long run, extending the approach to branded drugs with limited competition and following through by managing according to value across benefit type will likely alter the financial relationships between health plans and providers, manufacturers, and employers.

In the short term, changes that are already underway as part of separate efforts to control costs or improve quality may offer opportunities to provide broader and more affordable access to drugs with value-based prices. Manufacturers have begun to place bets that this evolution will continue.  Novartis priced tisagenlecluecel (Kymriah) separately for its adult and pediatric uses to reflect differences in its value for each indication.15 The success of this strategy has yet to be determined, but they may hope for success similar to Regeneron’s, which was rewarded for improved access and exclusive formulary position by Express Scripts when it repriced alirocumab (Praluent) to align with ICER’s assessment of PCSK9 inhibitors.16

 

References

  1. Silverman E. CVS exec: Out new reliance on cost effectiveness should make drug makers ‘think about launch prices’. STAT News.  Published August 31, 2018. https://www.statnews.com/pharmalot/2018/08/31/cvs-troyen-brennan-cost/?utm_campaign=KHN%3A+First+Edition&utm_source=hs_email&utm_medium=email&utm_content=65598216&_hsenc=p2ANqtz–NTtY3W-R5v7dtq5HrdoC2MYdHLRWOqhPNO86IYFSXRfySqLaWKDVnJMl1VH58Z0vNC6vE_ZX8fL_28wEfKllglUjkjA&_hsmi=65598216.
  2. The Medicaid and CHIP Payment and Access Commission. Medicaid’s share of state budgets. https://www.macpac.gov/subtopic/medicaids-share-of-state-budgets/. Published 2017. Accessed September 10, 2018.
  3. Bach PB, Ohn J. Does the 6% in Medicare Part B drug reimbursement affect prescribing? Drug Pricing Lab. https://drugpricinglab.org/wp-content/uploads/2018/05/Part-B-Reimbursement-and-Prescribing.pdf. Published May 9, 2018. Accessed August 21, 2018.
  4. Global Oncology Trends 2018. IQVIA Institute. https://www.iqvia.com/-/media/iqvia/pdfs/institute-reports/global-oncology-trends-2018.pdf?_=1536696423000. Published May 24, 2018. Accessed September 11, 2018.
  5. Long G. The Biopharmaceutical Pipeline: Innovative Therapies in Clinical Development. Analysis Group, Inc. http://www.analysisgroup.com/uploadedfiles/content/insights/publishing/the_biopharmaceutical_pipeline_report_2017.pdf.  Published July 2017. Accessed September 12, 2018.
  6. National Cancer Institute. Person Years of Life Lost. https://progressreport.cancer.gov/end/life_lost. Updated February 2018. Accessed September 12, 2018.
  7. Howard DH, Bach PB, Berndt ER, Conti RM. Pricing in the Market for Anticancer Drugs. J Econ Perspect. 2015;29(1):139-162.
  8. Centers for Medicare and Medicaid Services. Medicare 2018 Part C & D Star Ratings Technical Notes. https://www.cms.gov/Medicare/Prescription-Drug-Coverage/PrescriptionDrugCovGenIn/Downloads/2018-Star-Ratings-Technical-Notes-2017_09_06.pdf. Published September 6, 2017. Accessed September 9, 2018.
  9. Centers for Medicare and Medicaid Services. Medicare Shared Savings Program Shared Savings and Losses and Assignment Methodology Specifications. https://www.cms.gov/Medicare/Medicare-Fee-for-Service-Payment/sharedsavingsprogram/Downloads/Shared-Savings-Losses-Assignment-Spec-V4.pdf. Published December 2015. Accessed September 9, 2018.
  10. Dusetzina SB, Winn AN, Able GA, et al. Cost Sharing and Adherence to Tyrosine Kinase Inhibitors for Patients With Chronic Myeloid Leukemia. J Clin Oncol. 2014;32(4):306-11. doi: 10.1200/JCO.2013.52.9123.
  11. Kaltenboeck A, Bach PB. Value-Based Pricing for Drugs: Themes and Variations.  JAMA.  2018;319(21):2165–2166.  doi:10.1001/jama.2018.4871
  12. Thomas K. A Drug Costs $272,000 a Year. Not So Fast, Says New York State. New York Times.  Published June 24, 2018. https://www.nytimes.com/2018/06/24/health/drug-prices-orkambi-new-york.html.
  13. Boekell DD. The Evolving Use of White Bagging in Oncology. OncLive. https://www.onclive.com/publications/oncology-business-news/2014/june-2013/the-evolving-use-of-white-bagging-in-oncology. Published July 9, 2014.
  14. Bach PB, Pearson SD. Payer and policy maker steps to support value-based pricing for drugs. JAMA. 2015;314(23):2503-2504. doi: 10.1001/jama.2015.16843.
  15. Ramsey L. Indication based pricing for Novartis. Business Insider. Published May 7, 2018. https://www.businessinsider.com/indication-based-pricing-for-novartis-car-t-cell-therapy-kymriah-2018-5.
  16. Terry M. Sanofi and Regeneron Slash Cost of New Cholesterol Drug Praluent. Biospace. Published March 12, 2018. https://www.biospace.com/article/sanofi-and-regeneron-slash-cost-of-new-cholesterol-drug-praluent/.

High-Cost U.S. Drugs: A Tale of Unhealthy Markets

David Wohlever Sánchez, Jackie Xu, and Qiang Zhang, Duke University

Abstract

What is the message?

Behind the high-cost drug industry lies a complex environment with many stakeholders with different incentives and strategies. In this article, we cover these major stakeholders, and their role, incentive, notable strategies, and relationship to drug prices to gain a comprehensive outlook of the healthcare market structure. We find that the healthcare system represents an unbalanced market with information asymmetry, a lack of competition, and exploitative practices. Existing solutions include different government policies and work from direct-action NGOs and advocacy NGOs. However, there remains a lack of integration between the private, public, and non-profit sectors, which can represent a next step in addressing this problem.

What is the evidence?

Our research is based on over 45+ academic sources and multiple interviews with stakeholders and industry experts, including a pharmaceutical executive, NGO founder, and Duke business and medical professors.

Submitted: December 1, 2018; accepted after review: July 21, 2018

Cite as: David Wohlever Sánchez, Jackie Xu, Qiang Zhang. 2018. High-Cost U.S. Drugs: A Tale of Unhealthy Markets. Health Management Policy and Innovation, Volume 3, Issue 2.

Introduction

While one might expect that high prices in an industry indicate a functioning market system, the US drug market is anything but ideal, characterized by a lack of competition, asymmetric information, and misaligned incentive structures.

From 2013 to 2015, net spending on prescription drugs increased approximately 20% in the United States, outpacing a forecast 11% increase in aggregate healthcare expenditures. In 2013, per capita spending on prescription drugs was $858, compared to an average of $400 for 19 advanced industrialized nations.

This problem has a tangible human cost. High costs are often passed to patients through higher copays, deductibles, and premiums for those on insurance and higher out-of-pocket expenses for those without coverage.1 Research suggests that as many as one in four patients cannot afford and do not fill their prescriptions, and the elderly and patients with chronic conditions are the most affected.

In many cases (including Medicare, Medicaid, and subsidized insurance plans), healthcare is a public venture, making price hikes a “tragedy of the commons.”2 This concentration of interests makes legislative change difficult; accordingly, we must search for solutions beyond only government policy.

Additionally, international trends demonstrate that high prices in the US put distorting pressures on global drug prices.3 Since U.S. drug markets and international drug markets are so intertwined, it is important that any changes in the U.S. market are carefully considered, given the potential implications to international markets. Accordingly, it is critical that we avoid blunt force or overly distortive economic policy. Holistic, market-driven, and competition-inducing reform is necessary to properly address this global challenge.

History

Starting in the 1990s, in a spur of scientific innovation, the pharmaceutical industry developed blockbuster drugs, extremely popular and profitable compounds. However, patents only last so long—at least, in theory.

As drug compounds became more complex, marginal pharmaceutical improvements became more difficult, meaning manufacturers had to find new sources of revenue. This meant increased costs of R&D for new drugs, as well as efforts by firms to protect their market exclusivities. The combination of direct-to-consumer advertising, loose patent law, and unparalleled lobbying put stress on the market, contributing to the high prices patients see today.

Core Realities: A Model to Contextualize High Prices

In order to contextualize the problem, we must understand four core realities.

First, drugs, especially life-saving drugs, are generally price inelastic; demand will change little despite price hikes.

Second, science and technology are complicated and expensive. Research and development costs make it difficult for firms to enter the market.

Third, the U.S. government is only partly responsive. At the macro level, government responds to popular demand for healthcare provisions (e.g. Medicare/Medicaid/subsidies) and drug safety regulations (i.e. FDA). At the micro level, however, politicians react to lobbyists who help reelect them.

Fourth, we are in a profit-driven market system. Accordingly, we should not expect firms to neglect profit maximization insofar as we wouldn’t expect firms in other industries to do so.

These core realities lead to a system where market realities inform actor strategies, and vice versa. This creates a feedback loop that results in high prices.

Core Realities in Practice: Factors that Drive Up Prices

These core realities create an unhealthy market characterized by the following:

Regulation: The FDA process creates a barrier to entry by increasing production costs and putting downward pressure on competition.4

Lack of transparency5: There is little transparency with R&D and production costs, since firms are not required to release this data. Thus, firms can easily “justify” high prices by claiming high costs.

Market uncertainty: Since this space is complex, suboptimal pricing schemes can emerge.

Types of High Priced Drugs:

In the prescription drug market, there are three main categories of high-cost drugs:

  1. Patented pharmaceuticals6: Once approved by the FDA, drugs can be sold at any price that a payer agrees to cover. These drugs stay high-priced, so long as no adequate substitutes exist. This state-granted monopoly administered via patent laws limits competition. However, these laws do spur innovation by creating incentives to develop new drugs.
  2. Specialty generics (“orphan drugs”)7: On-patent and off-patent specialty drugs that a small number of people need. However, since they are often life-saving, demand is inelastic.8 Even when patents expire, the lack of competition is caused by the high cost of entry and the low demand.9
  3. “Super generics10: New generics with more convenient methods of administration (e.g., nasal vs. injection) or combination of multiple pills into one.11 Slightly more effective, much more expensive. High cost of entry decreases competition.12

Stakeholder Analysis & Strategies

Stakeholders employ strategies to “maintain the loop” and their marketplace position, having direct and indirect impact on the problem landscape.

 

Stakeholder Role Incentive Strategy Relationship to price
Pharmaceutical  Manufacturers Manufacture drugs; R&D High prices; strict patent law Evergreening, lobbying, information asymmetry, strategic payouts “Price-makers” in problematic cases, “price-takers” in competitive cases
Hospitals / Physicians Receives drugs from manufacturers at discount; drug vendors that can pocket profit Generally profit-driven Utilize price mark-ups when selling drugs to patients Intermediary that drives up prices for the patient or payer
Pharmacy Benefit Managers Mediators between insurance companies and pharmaceuticals, negotiate prices and coverage options Profit-driven Receive discounts from manufacturers to promote their product over competitors’ Act as mediators and drive up transactions costs; negotiate discounts and rebates
Patient Advocacy Groups Advocates for and educates patients Profit-driven given relationship with manufacturers Provide educational materials that are not value-based Contribute to information asymmetry that drives up prices
Government at the micro level Public expectation to provide for high-quality, low-cost, and safe healthcare system Hold interest in lower drug costs as funder of Medicare/Medicaid Subsidize R&D; regulate drug manufacturers via FDA As regulators, drive prices up (FDA); subsidize costs of production via research grants
Health insurance providers Compete with other insurance providers to include many treatments in their plans but constrained by costs Generally want lower costs for themselves so their share of the payout is lower Negotiate group discounts Historically price-takers; now generally attempt to negotiate down prices
Universities Fuel basic science for pharmaceutical drug R&D, often funded by government Seek grant money to fund research and other activities Do not have the capacity to manufacture drugs, sell royalty rights to pharmaceutical companies13;

receive grants and donations from pharmaceuticals

Currently do little to influence pricing schemes

 

Notable Strategies

Pharmaceuticals/Manufacturers: 

  • “Evergreening”14: Involves tweaking a small aspect of a drug’s formula or delivery method to extend patent by 20 years.
  • Lobbying: In 2016, pharmaceuticals spent $244 million lobbying, the most of any US industry.15
  • Paying generic manufacturers to drop patent challenges16: 2005 Federal Trade Commission decision allows manufacturers to pay generic companies to drop patent challenges.
  • Lack of transparency on internal finances17: Cost of R&D is often a justification for exorbitant prices18. However, manufacturers spend nearly twice as much on marketing products as on R&D.

 

Hospitals: Charity hospitals (serving underprivileged neighborhoods) receive discounted drugs and sell them for profit. Through the 340b program, Medicare/Medicaid exclude these discounted rates when establishing payouts, which allows hospitals and manufacturers to profit heavily. An expanding number of hospitals now qualify for this status.

Patient Advocacy Groups (PAGs): PAGs receive donations and drug royalties from pharmaceutical companies. Accordingly, they provide “education” to patients about the drugs that are most effective/efficient, without any consideration of value-pricing.19

Universities: More than half of the 26 most transformative drugs of the last 25 years originated in publicly funded research.20 They also often receive grants and donations from manufacturers. However, they do not have the capacity to manufacture drugs, so they sell royalty rights to pharmaceutical companies.21 They play very little role in determining price schemes.

The Solution Landscape

At the core of the problem lies an unbalanced market, characterized by information asymmetry, lack of competition, and exploitative practices. Below are some existing solutions that have improved market systems in the U.S. and other countries.

Current Solutions Landscape

  R&D, Intellectual Property Direct pricing negotiation Increase competition Increase transparency
Government Bayh-Dohl Act (patenting by federal research grantees) Value-based pricing Hatch-Waxman Act (facilitate generic entry) Sunshine laws
Direct action NGOs Public-private partnerships Generic manufacturers Organize into networks to collect market information for drug manufacturer

 

Advocacy NGOs Advocacy with pharmaceuticals and PBMs: release price and production costs

 

Solutions for R&D/Intellectual Property Rights:

  • Bayh-Dohl Act of 198022:
    • Section 202 requires federal research grantees to confer a nonexclusive, royalty-free license on their patents to the government
    • Government has “march-in rights” to demand a patented drug be manufactured on its behalf23
  • Public-Private Partnerships:
    • Public (NGO, government) and private (companies) actors co-finance R&D for diseases affecting developing countries that would not otherwise be attractive markets24
    • Private companies receive PR benefits

Solutions for Direct Pricing Negotiation:

  • Value-based pricing25:
  • UK’s central advisory board calculates value of drug based on efficacy, safety, and total benefits to the healthcare system, setting prices accordingly

Solutions for Increasing Competition:

  • Hatch Waxman Act of 1984
    • Decreased the price of FDA generic drug applications26
    • Granted a period of market exclusivity to generic manufacturers who challenged patents before they expired27
    • Enabled, in part, the increase from 36% to 84% of generic product’s share of total prescriptions market in US28 (about 90% in 2018)
  • Promote generic manufacturing by nonprofits
    • Increased competition among drug manufacturers reduces prices
    • The Drew Quality Group
      • First approved 501(c)(3) to manufacture drugs, developing off-patent generic drugs and eventually legacy drugs29
      • Must publicly disclose all financial information

Solutions for Increasing Transparency:

  • Physician Payments Sunshine Act30
    • Requires drug and medical device manufacturers to disclose payments made to physicians31
  • Pharmaceutical companies market products by giving physicians free drug samples or gifts, skewing prescribing habits.32
  • NGO Networks
    • Create local networks of NGOs to gather market data on drug demand
    • Build reliable forecasting, convincing pharmaceutical companies to lower drug prices to reach a wider market.33
  • NGO Advocacy34
    • Universities Allied for Independent NGOs35 have mitigated information asymmetry between patients and pharmaceuticals.
    • Essential Medicines empowers students to petition their universities for better drug access policies.36

Lessons & Levers of Change

Media coverage of this issue has spotlighted manufacturers, but structural barriers within the healthcare system challenge reform.

At present, only the public and nonprofit sectors are direct actors in the solution landscape. Yet to build a more balanced market system, the private sector needs increased opportunities and stronger incentives to price products sustainably. This sector has the most leveraging power to change the pricing ecosystem.

Private Sector: Market governance

  1. Provide more robust opportunities for drug manufacturers to decrease R&D costs, encouraging price reductions
  2. Increase government grant funding and open knowledge collaboration with private manufacturing companies

Non-Profit: Informal governance

  1. Creation of NGO network: Pool local demands as negotiating power to reduce drug prices, or assume risk of drug manufacturers by holding excess stock; NGOs can leverage local knowledge; incentivize state governments to create NGO networks through common interest of providing medication for populations of need; advocacy NGOs can partner with NGO drug manufacturers, providing market information
  2. Venture-capital and government co-investment in NGO drug manufacturers to decrease capital barriers to entry.
  3. Incentivize and empower universities to leverage their position as the innovators of the science on which drug manufacturers rely; charitable social mission to educate individuals in service of society
  4. Publicize third-party, research findings on the value of drugs based on efficacy, safety, and overall societal benefits.

Public: Official governance

  1. Leverage Bayh-Dohl Section 202 provision: Inform NGOs of existing institutional pathway; allow government to pass license to NGO drug manufacturers, who can then compete with pharmaceuticals in patented market
  2. Include accessibility requirements on existing R&D cost-sharing agreements through public-private partnerships

Conclusion

The question of “fair” pricing remains open. While we do not want to ignore the contributions of profit-seeking firms, we cannot bear exorbitant prices forever. We believe a middle ground exists where manufacturers can pocket a profit and patients can afford their drugs.

Governments, manufacturers, and NGOs ought to remember the human costs of restricted access. Solutions will only be found when stakeholder incentives align with reasonable pricing. This can be achieved; whether or not we follow through remains to be seen.

Appendix – Bibliography

Endnotes

[1] Studies have found that people who see rises in their drug costs spend less on their families, other expenses, and sometimes even postpone retirement to keep their employer’s health insurance so they can afford the drug.

[2] This means that a few firms can internalize huge gains at the expense of the rest of society, the members of which each bear a tiny fraction of the true cost: a “death by a thousand papercuts.”

[3] For example, Canadian internet pharmacies, also dubbed mail-order pharmacies, enable Americans to purchase drugs from Canada, putting strong upward pressure on Canadian retail drug prices.

[4] There is a tradeoff here: the FDA keeps drugs safe, but also increases prices. This is not an all-or-nothing binary; we believe that there is a middle ground between drug safety and producer-side cost of regulation that would lead to optimal outcomes.

[5] This allows the pharmaceutical lobby to repeat the oft-given response that high prices are justified by cost of research. However, our research suggests cost of research and development is not as high as one might think.

[6] Example: Hepatitis C drug Sovaldi was originally planned to sell at $34,000. Gilead now sells Sovaldi at $84,000 for a 12 week treatment, or $1,000/pill.

[7] Example 1: Turing Pharmaceutical’s (former CEO Martin Shkreli) Daraprim jumped from $13.50 to $750/pill overnight. Daraprim treats a parasitic infection called toxoplasmosis that targets people with compromised immune systems, certain cancer patients. Toxoplasmosis infects an estimated 4,000 individuals in the US each year.

Example 2: Rodelis Therapeutic’s acquisition of cycloserine resulted in increase from $500 to $10,800 for 30 pills. Cycloserine treats multi-drug resistant tuberculosis (MDR-TB). In 2015, the US had 89 cases of MDR-TB.

[8] Inelastic meaning people will continue to buy regardless of price, since they need the drug.

[9] Firms that manufacture and sell these specialty drugs are able to set the price high enough so they are able to make a huge profit, but low enough such that new firms won’t be incentivized to enter the market, given cost of entry.

[10] Example: Amphastar Pharmaceutical is the only company to manufacture intranasal naloxone, which experienced a 100% price increase in 5 months.

[11] The existence of these effective generics improves patient health outcomes, but the difference between the more effective and less effective generics is likely not large enough to justify the huge price discrepancies.

[12] This leaves one or two firms at the top with the more effective, much more expensive generics.

[13] Example: Xtandi (prostate cancer blockbuster drug) will generate $33.3 million in royalties and other income for University of California.

[14] Example: Pfizer’s Caduet is a simple combination of Norvasc and Lipitor, which expired in 2007and 2011, respectively. Pfizer’s creation of Caduet when Norvasc and Lipitor were due to expire prevented other manufacturers from producing generic versions of these drugs.

[15] Example: Pharma lobbied Congress in 2003 to prevent Medicare from negotiating prices with pharmaceuticals for its new Part D program. Wholesale prices of brand-name drugs have increased average of 3.6 percent since the establishment of Medicare Part D.

[16] Example: In a patent challenge case against Cipro, a potential generic manufacturer received upfront and quarterly payments totaling $398 million and agreed to wait until patent expiration to market its product.

[17] In December 2016, 20 states filed complaints against pharmaceutical companies conspiring to artificially inflate prices generic drugs, coordinating through informal industry gatherings and personal calls/text messages.

[18] Example: Johnson & Johnson and Pfizer spent about 13 percent and 16 percent on R&D, respectively. At the same time, both companies spent about 30 percent of revenue on selling, marketing, and administrative expenses.

[19] 67% of patient advocacy groups receive funding from for-profit companies.

[20] Example: Sanofi’s collaboration with Harvard University is part of corporate strategy to fill pipeline with innovative drugs.

[21] Example: Xtandi (prostate cancer blockbuster drug) will generate $33.3 million in royalties and other income for University of California.

[22] Originally, this Act allowed federally-funded inventors and their employers to retain patent ownerships, incentivizing the commercialization of government funded R&D.

[23] However, note that all six petitions to the National Institutes of Health (NIH) to exercise march-in rights have been denied. The NIH claimed that drug pricing itself was not sufficient to provoke march-in rights.

[24] Examples include the International AIDS Vaccine Initiative, Global Alliance for TB Drugs Development (Stop TB), and Medicines for Malaria Venture (MMV). The Stop TB Partnership led to an agreement signed by Janssen Therapeutics to provide medication free for eligible MDR-TB patients—a promised donation of 30 mil over a 4 year period to low/middle income countries.

[25] In Australia, the Pharmaceutical Benefits Advisory Committee reviews the comparative effectiveness of various drugs. Similar patterns are also seen in Germany under the Federal Joint Committee, Canada under the Patented Medicines Prices Review Board, and the UK under the National Institute for Health and Clinical Excellence. In all these nations, the price of a drug is determined by the value it will bring.

[26] An Abbreviated New Drug Application (ANDA) process granted by the Act reduced the cost of completing an FDA application for approval of a generic drug.

[27] The first generic manufacturer(s) to file a Paragraph IV challenge against a brand-name patent is granted a 180-day period of exclusivity on the market before patent expiration. A Paragraph IV challenge is a claim to the FDA that the generic product does not infringe on the listed patent of a brand name drug, or that the brand-name patent is not valid.

[28] Other proposed reforms included the Greater Access to Affordable Pharmaceuticals Act in 2003, the Pharmaceutical Market Access Act of 2003, and the Pharmaceutical Market Access and Drug Safety Act of 2011. While these acts slightly differ in detailed nuances, their ultimate intent was to break down barriers of entry for generic drugs within the drug market.

[29] Legacy drugs are patented products that companies no longer desire in their product lineup. Drew Quality Group has two classifications of drugs. 1) Surplus drugs generate capital for future growth. 2) Service drugs may be sold at or below production cost to increase access of medications to vulnerable populations.

[30] Signed into the Affordable Care Act of 2010.

[31] All reports made to Centers for Medicare and Medicaid Services.

[32] Doctors may begin prescribing medications driven by personal motivations. In 2014, nearly 40% of the 50 largest pharmaceutical companies had academic medical center leaders on their Board of Directors, giving these individuals with significant weight on directions of medical research a financial responsibility to generate profits to shareholders.

[33] Example: AA&D is working with cities around world to quantify demand for TB drugs, creating NGO coalitions to streamline and pool this demand and use as negotiating power. These NGO coalitions can also assume risks from pharmaceutical companies by holding excess drug stock.

[34] Examples: NeedyMeds.org, a national non-profit, maintains a website of free information on programs that help people who can’t afford their medications or other health-care costs. It also offers a free drug discount card. The patient advocacy groups Campaign for Personal Prescription Importation, PharmacyChecker.com, Prescription Justice Action Group, RxRights.org, and the publisher of TodaysSeniorsNetwork.com, together representing more than four million Americans, advocated for the need of political action on high drug costs. Organizations such as Pharmacists United for Truth and Transparency, comprised of more than 1,000 pharmacists and pharmacy owners, aim to expose the intricate business model of PBMs designed to exploit the other players within the prescription drug market. Other examples include the National Community Pharmacists Association and PBMwatch.com.

[35] More advocacy NGOs: Sites like GoodRx offer information on retail costs of medications at local pharmacies. FamilyWize offers free prescription drug savings cards, allowing patients to negotiate discounts with pharmacies.

[36] This group was founded by successful Yale student advocates who reduced price and opened generic manufacturing of Bristol-Meyers Squibb’s antiretroviral d4t (discovered at Yale).

The Supply-Side Effects of Moral Hazard on Drug Prices

Kevin A. Schulman, MD, Clinical Excellence Research Center, Stanford University

Contact: Kevin A. Schulman, kevin.schulman@standford.edu

Abstract

What is the message?

This paper analyzes the impact of moral hazard on prices established by pharmaceutical manufacturers and the implications for policy makers. The findings show that the escalation of drug prices will likely continue unabated in the absence of significant mechanisms to induce restraint and discipline in this market.

What is the evidence?

Moral hazard is a powerful theory of how health insurance influences the delivery of healthcare. While moral hazard has been used to understand changes in demand for services through insurance, a literature review shows that until now, its impact on pharmaceutical prices has not been well developed. The findings documented in this paper should therefore help spark a vigorous debate on the implications of different pharmaceutical pricing models for the US market.

Submitted: December 18, 2017; accepted after review: July 21, 2018

Cite as: Kevin A. Schulman. 2018. The Supply-Side Effects of Moral Hazard on Drug Prices. Health Management Policy and Innovation, Volume 3, Issue 2.

Unit prices for pharmaceutical products are reaching ranges never before experienced.  Prices for cancer products are routinely over $100,000 per patient per annum, while novel CAR-T therapies are being offered for $475,000 per patient. These prices are a dramatic increase over the prices of the most expensive drugs only a decade ago.[1]

Prices are set by pharmaceutical manufacturers in negotiation with payers, and are set to reflect the fact that these products have a value based in intellectual property that is far greater than the marginal cost of production. Historically, the pharmaceutical industry has argued that prices are justified on the basis of research and development costs, the high risk of drug failure[2], and the value of new products.[3] Rarely are the prices ascribed to the actual price of manufacturing the products. At the same time, industry critics have highlighted the role of public funding in biomedical sciences as directly or indirectly supporting the development of many products, and the changing nature and risk of drug development characterized by much smaller and speedier clinical development programs and accelerated review times at FDA.[4]

While these arguments have existed for many years, the nature of biomedical research has changed.  The industry has moved development of products from broad, mass-market molecules to niche investments in cancer and orphan diseases.  The concern over high drug prices in these markets has been met with additional arguments for high prices based on limited market sizes in many of these product niches.[5]

In the current market environment, there is limited direct pressure on manufacturers setting these unprecedented prices. In contrast to many other countries, the US government does not directly negotiate over pharmaceutical prices except within the Veteran’s Administration (VA) system.  Even when direct negotiation is possible, the lack of competition within market segments limits the bargaining power of private payers. As a result, high and increasing prices directly impact health insurance premiums across the market, and the cost of the federal health plans, Medicare and Medicaid.[6] [7]

The Supply Effects of Moral Hazard

The extraordinary prices for pharmaceutical products can only be imagined in a world where patients have health insurance. Health economists have long been worried about the economic impact of health insurance on the patterns of consumption of healthcare due to a concept called “moral hazard.” Moral hazard describes the change in individual behavior between conditions of self-pay and conditions of third party payment. Kenneth Arrow was awarded the Nobel prize in economics for developing this framework[8]. Mark Pauly further developed the theory to focus on demand.[9]

The basic framework is easy to understand.  We all make purchases based on our concept of value.  We generally make purchases of goods or products for $1.00 when we perceive that they offer $1.00 worth of value. This concept of value is an individual determination – we all have our own tastes, preferences, and needs which form our assessment of value.

Third-party payment alters this fundamental calculus. Consider going out to dinner with a group of friends.  After the menu is passed around, you notice items of lower and higher price, salad, and steak. You can approach payment in one of two ways: individual checks or splitting the check.  If you all decide on individual checks before you order, you may decide to purchase the lower-cost salad since you are on a budget.  However, what happens if you decide to split the check after you order? You may be worried that everyone else at the table is likely to order the higher-priced steak, and you will have to pay your share of their higher-priced meals.  Since you are paying for their steak, why not order your own steak so at least you get the benefit of the higher price you will pay for dinner.  In this simple illustration, your behavior changes between self-payment and third-party payment models.

Health insurance is one form of third-party payment. Under health insurance, rather than paying the full cost of medical products, you pay only a co-payment (fixed amount), or co-insurance (a percentage payment) for medical products.  As illustrated in Exhibit 1, products 1-3 offer at least $1.00 of value for $1.00 of cost.  In a self-payment model, you would be expected to purchase only products 1-3 since only these products have a value of $1.00.  In an insurance model, however, you only pay the copayment of $0.20.  Now, products 1-6 offer value equal to or greater than the $0.20 copayment, so using the same rule (only buying products that offer value greater than or equal to the price you pay), you would purchase products 1-6.  Again, behavior changes under conditions of third-party payment.

Exhibit 1: Hypothetical Set of Products Based on Value, Cost and Insurance Status

Legend: Value-perception of value to the patient (in dollar equivalents). Cost (No insurance)-assumes only cash payments for the product. Cost (insurance)-assumes the product is covered by an insurance policy with a 20% coinsurance requirement.

 

While many economists have argued that health insurance increases the overall cost of healthcare due to these changes in demand,[10] there is also the concept of good moral hazard where people can purchase goods or products through insurance that would otherwise be unaffordable.[11]

To this point, the discussion of moral hazard has focused on the impact of moral hazard on the demand for healthcare products. However, the impact of moral hazard also extends to the supply side of health care.[12] [13] [14] While much of the literature examines the impact of moral hazard on the provision of services and technology, there is also an impact on the price of products. Given insurance, the suppliers of high-value products can realize that products are perceived as being significantly underpriced since insured patients only consider the out-of-pocket costs and not the full cost of therapy.  Applying a value framework to pricing can lead manufacturers to raise their prices to meet the value threshold rather than simply developing a price to meet their internal financial expectations. This supply-side moral hazard effect on the price of pharmaceutical products has been much less discussed in the literature.[15] [16]

Again, going back to the basic example of product 1 in Exhibit 1, this product provides great value to patients under conditions of self-payment and even more under conditions of third-party payment. Sophisticated suppliers will notice these conditions. In a competitive market, suppliers will have little ability to influence the welfare surplus enjoyed by patients in this example since the price is determined by the market and is driven by the entry and exit of firms. However, there are circumstances when suppliers have power to influence prices, especially in healthcare.

Suppliers can have market power when they have a barrier to market entry such as a patent awarded to a pharmaceutical manufacturer or a product developed for a niche category which is too small to attract competition.  In these cases, suppliers can increase the price of product 1 based on value.  If they decide to price at the total value of the product, they could raise the price from $1.00 to $1.50 to capture the full value to patients. Under our conceptual model, this pricing strategy would be attractive to patients even in a cash pay market.

However, under conditions of third-party payment, suppliers can consider an even more aggressive pricing strategy by considering that patients measure value against their co-insurance, not the full cost of the product.  Under these conditions, suppliers can raise the price to $7.50 while consumers would have a cost-share of $1.50, or an amount equal to the value they expect to receive from the therapy. As a result of supply-side moral hazard, the cost increased from $1.00 to $7.50 in this simple example.

The supply side implications of moral hazard are potentially significant. Beyond the short-term impacts on patients, this effect can have longer term effects by distorting the drug development portfolio. In Exhibit 2, we imagine a manufacturer with a simple two product portfolio, with each product having equal development costs and market price. In analyzing their options, the firm invests in the opportunity with the largest market size.[1] However, in Exhibit 3, under conditions of market power, they can consider the question of value of the therapy to patients in setting a price.  In this case, they chose to undertake development of product B despite its smaller market size.

Thus, the supply-side effects of moral hazard can be seen in both the prices of products in the marketplace, and in the portfolio of drug products available on the market.

Exhibit 2: Optimal Drug Portfolio without Moral Hazard

Product Cost of Development Size of Target Market Price Revenue
A $50,000,000 20,000 $10,000 $200 M
B $50,000,000 10,000 $10,000 $100 M

Legend: Cost of Development-out of pocket dollar costs of development (assumption).  Size of Target Market: number of accessible candidates for therapy considering incidence and prevalence of underlying condition.  Price-market price for the product (net price to manufacturer). Revenue-net revenue from the product (price times market size).

 

Exhibit 3: Optimal Drug Portfolio with Moral Hazard

Product Cost of Development Size of Target Market Value of Therapy Price Revenue
A $50,000,000 20,000 1 $10,000 $200 M
B $50,000,000 10,000 5 $50,000 $500 M


Legend: Cost of Development-out of pocket dollar costs of development (assumption).  Size of Target Market: number of accessible candidates for therapy considering incidence and prevalence of underlying condition.  Value-perception of value to the patient (in dollar equivalents). Price-value price for the product (net price to manufacturer). Revenue-net revenue from the product (price times market size).

 

Possible Solutions

Four frameworks can be considered as part of a regime to set appropriate market prices: market competition, cost-effectiveness analysis, prizes, and profit regulation.

1. Market Competition

As suggested earlier, in competitive markets with free entry of firms, the supply-side effect of moral hazard is not an issue. Firms that attempt to extract a high price from the market will be quickly met with competitors.  To a great degree, market competition has worked in the pharmaceutical market. When several firms with products enter a class, they often face price competition (although, this being healthcare, not all of this price competition is transparent and can occur in the form of a PBM rebate[17]).

A recent example is the cost of Hepatitis C treatment in the United States.  The payer community was in shock over the original price of Solvaldi at $84,000 per patient for a 12-week course of therapy (or double that amount for 24-weeks).[18] This set off a debate around the price of the therapy, and led to significant challenges for many public and private health insurers.[19] However, the entry of additional novel therapies for patients with Hepatitis C led to price competition in the marketplace, with prices ranging from $26,400 to $62,500 per treatment course by 2017.[20]

While this may be seen as a success of the market model, we do not have a good understanding of which product categories will remain competitive in an era of precision medicine. As indications for products become more targeted at a molecular level, it may be difficult for fast-followers to enter specific niche markets, leading to the failure of this mechanism to address the pricing impact of moral hazard.

2. a. Economic Analysis

Over the last several years, we have seen the re-emergence of products’ economic value as a consideration for supporting prices. This concept was developed in the 1970s and 1980s as a tool to help understand the value of investments in new pharmaceutical therapies.  Outside of the United States, regimes incorporating economic analysis have helped to set national formulary decisions in the UK, Germany, and Australia. These regimes can consider the patient population impacted by the condition, the potential outcomes of the therapy, and the net cost of the intervention (the net cost considers the cost of the new therapy, the cost of administration such as hospitalization for supportive care or side effects, less costs avoided as a result of the treatment). Through this analysis, products can be found to be cost saving (those rare products that reduce overall costs), or cost effective, requiring additional spending but providing additional value to patients.

Providing coverage for cost-effectiveness therapies is predicated on an interest in an investment in new therapies by everyone in the insurance pool or by taxpayers if addressing a publicly funded program. Generally, there is no direct process for such a determination, so this process is left to proxies such as a pharmacy and a therapeutic committee or some other type of formulary review committee.

Economic evaluation can be used to assess the value of therapies once a price is determined, or it can be used to set the price in advance to ensure “access” or uptake into the marketplace. The use of economic analysis before a product is marketed can help understand the potential value of the therapy in practice.[21] If economic evaluation is conducted once a product is on the market, findings that therapies are not cost-saving or that they do not meet a value threshold could reduce spending by limiting access to low-value therapies. At present, there is no consistent application of a value threshold in the United States.

2.b. Considerations for the Specialty Pharmaceutical Marketplace

Applications of the economic analysis framework to high-cost therapies could be problematic.

Economic evaluation can be used by manufacturers to help set a price for a product. Using this framework, high prices can be justified by expected benefits using outcome measures such as years of life gained for therapies that have a survival advantage, or quality-adjusted life years considering an impact on both length and quality of life.

One means of enhancing the “value” of a therapy is to limit the indication to those that would perform best under this framework to support a high price.  Therapies that have a large impact on pediatric cancer, for example, would offer the potential for large denominators in a cost-effectiveness framework since patients who benefit from therapy would have substantial remaining life expectancy. Thus, bringing a technology to market first for a pediatric indication would allow a price that would be considered poor value in other indications.

Application-specific pricing is a remedy to address this strategy where prices would be adjusted based on clinical indication. However, there is no evidence that these schemes have been successfully adopted and enforced.  If enforcement of this method fails, then the “benchmark” price for a technology could be established by the best-case scenario rather than the expected use in the market.

Market “guarantees” could be another mechanism to enhance the effectiveness of therapies.  Again, the issue would be the ability and cost to track “effectiveness” over time to enforce these contracts.  Not only is there potential for significant disputes over patients who have adverse outcomes for a variety of reasons unrelated to the treatment (car accidents for example),  significant administrative costs would be associated with the resulting “manual” billing process.  Further, if a manufacturer offers this framework in advance of going to market, they could include their estimate of likely clinical effectiveness in setting their price (i.e., they raise the price above their initial consideration to account for the cost of the guarantee).

If patient perspective is considered in assessing value, there is significant concern that conditions involving highly emotional situations (such as a life-threatening illness or genetic diseases) or loss (a new diagnosis of cancer) lead to very high value on any potential benefits of therapy, and patients may appear to be risk-seeking in making treatment choices. [6], [21] This value framework of patients is expected to differ significantly from the value framework of people in the insurance pool not impacted by life-threatening illness.[22]

Cost-effectiveness of therapies is not tied to the overall cost in the market since total budget is tied to the price and the number of patients treated, not the value of a therapy.[21] Cost-effectiveness analysis can be used to prioritize new investments in pharmaceutical therapy, but will lead by definition to increases in spending.[23]

3. Prizes for Drug Discovery

Nobel Prize-winning economist Joseph Stiglitz developed the concept of a prize for drug discovery that would compensate inventors for their efforts while providing the public access to novel therapies closer to their marginal cost.[23] However, the mechanics of such an approach are challenging.

First, new drugs enter to market as a combination of both drug discovery and drug development.  Discovery is the early-stage basic science work in a laboratory, while development requires teams of people to develop a formulation for use in humans, optimize drug manufacturing, test for toxicity, and undertake clinical testing.  Discovery often occurs through public funding, while development requires private funding.  A prize awarded for discovery would be very difficult to administer; many new targets and biologic mechanisms are discovered, only to later fall out of the drug development pipeline.

Similarly, a prize for drug discovery could create significant challenges in funding drug development, with no private incentive to invest in clinical research since post-prize research would be a public good. A prize awarded after drug development runs the risk of skewing development to tasks required to access the prize rather than to optimize the development program for market impact.

Finally, given the tremendous output of basic science in the United States, there may be a subjective component to the process of awarding prizes which could create significant uncertainty in the marketplace and reduce incentives for drug development.

4. Profit Regulation

In some European markets, price negotiation used to include profit regulation. Government purchasers developed a framework considering that the patent holder was a monopolist that needed to be regulated (like a utility). Profit regulation schemes considered the costs of drug development and manufacturing in setting price and market access, with additional consideration for manufacturers’ production capacity within a market. The manufacturer would be allowed a price that offered a return on these demonstrated costs. For example, in the UK, profits were limited to 21% until 1998, rising to 29%.[24]

Profit regulation allows for a separation of the research costs (supported by the public) and drug development costs incurred by the private sector.  In addition to direct costs in clinical development, profit regulation schemes can consider the time cost of an investment. This mechanism was largely applied to the consideration of self-originated portfolios.  It is not clear how these schemes would evaluate in-licensed products and whether there would be any consideration of the costs of acquiring molecules in calculating the allowable profit for firms. A decision to exclude acquisition costs from pricing considerations could have a significant negative impact on firms paying high prices to acquire novel therapies, but could potentially lead to a reduction in market prices for new products.[25] [26]

Under a profit framework, manufacturers would have incentives to develop products for a wide variety of indications.  This could broaden the portfolio beyond the narrow niche of oncology and orphan products currently in development.  While manufacturers might have an incentive to increase development costs under this framework to enable a higher nominal profit level,[27] in practice, the high cost of venture capital may limit the impact of this perverse incentive.

Given the fixed cost of drug development, this concept of profit regulation entails a scale problem. The market may have to facilitate purchase of a sufficient quantity of a novel therapy before this profit cap would apply.

One approach to profit regulation could be to provide a pathway to using less private capital in drug development.  Leveraging private capital with public grant funding could allow private investors to achieve their expected returns while providing a pathway for lower drug prices post-approval.[28] The NIH’s recent announcement of a novel co-development program with industry would be a perfect opportunity to test such a program.[29]

Final Considerations

There is significant anecdotal evidence of an impact of supply-side moral hazard on specialty drug prices. In the face of this effect, public constraints on drug pricing may be required:  “To avoid breaking the government’s budget, this would require some type of restraint on manufacturer prices, such as price-regulation or direct price controls.”[14]

Currently, there is no mechanism in the US market to address this issue in product categories with single solutions such as many specialty pharmaceutical categories.  This paper developed the theory of why this is an issue, and examined how four currently available strategies can be used to assess the appropriate price for products for the US market.  Each framework, the market, value, prize, and profit frameworks, have significant strengths and significant limitations. Each approach provides different incentives to shape the portfolio of products in development, and may result in different pricing frameworks for products that either the market (see Exhibit 4). Consideration of each of these schemes should include considerations of access, innovation and affordability of new drug products [21] At some level, all of these mechanisms are designed to ensure some conflict in setting prices within this market.

The escalation of drug prices will likely continue unabated in the absence of significant mechanisms to induce restraint and discipline into this market.  Thus suggests the need for a vigorous debate on the implications of these different pricing models in the US market, a need recently highlighted by the National Academy of Medicine.[31]

Exhibit 4: Policy Solutions to the Effects of Moral Hazard on Price

Scheme Unit Price Utilization Total cost
Market Competition High until competitors enter the market May result in barriers to market access Potential to increase the total cost
Economic Analysis May increase prices for high value therapies May enable market access Potential to increase the total cost
Prize Low marginal cost per patient Enables increased use with little cost barrier Total cost depends on how the prize scheme is established
Profit Regulation Price cap based on investment May enable market access Total cost depends on regulation scheme

 

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