Evaluating and Valuing Drugs for Rare Conditions: No Easy Answers

• Published in: Value in health 2018-05, Vol.21 (5), p.547-552
• Main Authors: Ollendorf, Daniel A., Chapman, Richard H., Pearson, Steven D.
• Format: Article
• Language: English
• Subjects: Cost analysis; Decision makers; Drug development; Drugs; Economics; Ethical dilemmas; Evaluation; financing; Health care; Incentives; Medical technology; Medical treatment; neglected diseases; orphan diseases; orphan drug production; Pharmaceutical industry; Pharmacoeconomics; Rare diseases; Technology assessment; Valuation
• ISSN: 1098-3015; 1524-4733
• DOI: 10.1016/j.jval.2018.01.008

We find ourselves in an era of unprecedented growth in the development and use of so-called “orphan” drugs to treat rare diseases, which are poised to represent more than one-fifth of pharmaceutical expenditures by 2022. This widespread use has been facilitated by legislative and regulatory incentives in both the United States and abroad, yet US payers and health systems have not yet made a concerted effort to understand whether and how rare diseases require special considerations on their part and how to adapt traditional methods of health technology assessment and economic evaluation to accommodate these situations. In this article, we explore the general ethical dilemmas that rare diseases present, steps taken by health technology assessment bodies worldwide to define the level of rarity that would necessitate special measures and the modifications to their assessment and valuation processes needed, and the contextual components for rare-disease evaluation that lie outside of the assessment framework as a guide to US decision makers on constructing a formal and relevant process stateside. •This article is an overview of the societal, ethical, and coverage/reimbursement landscape for consideration of novel treatments for rare diseases and highlights the following:•Confirms the ethical struggle and tradeoffs associated with making special arrangements to consider the value of new treatments for rare diseases and the high degree of variation in how payer and health technology assessment organizations deploy such arrangements;•Adds a discussion of the contextual elements necessary for developing rare-disease policy, highlighting those that fall outside the traditional boundaries of economic assessment and evidence synthesis;•Documents the current gaps in US payer understanding of these issues and outlines the steps necessary to create an internationally harmonious approach to rare-disease policy.•Confirms the ethical struggle and tradeoffs associated with making special arrangements to consider the value of new treatments for rare diseases and the high degree of variation in how payer and health technology assessment organizations deploy such arrangements;•Adds a discussion of the contextual elements necessary for developing rare-disease policy, highlighting those that fall outside the traditional boundaries of economic assessment and evidence synthesis;•Documents the current gaps in US payer understanding of these issues and outlines the steps necessary to