Evaluating and Valuing Drugs for Rare Conditions: No Easy Answers

Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
Daniel A OllendorfSteven D Pearson

Abstract

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.

Citations

Feb 21, 2019·Journal of Managed Care & Specialty Pharmacy·Louis P GarrisonMike Kenston
Apr 16, 2020·Journal of General Internal Medicine·James D ChambersPeter J Neumann
Dec 8, 2020·Genetics in Medicine : Official Journal of the American College of Medical Genetics·John S RichardsonLisa A Prosser
Feb 2, 2021·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Sandra M SirrsCarla Hollak
Nov 27, 2020·International Journal of Neonatal Screening·Scott D Grosse, Guy Van Vliet
Mar 2, 2021·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Gerasimos KonidarisShannon Cope
Mar 2, 2021·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Isaac Aranda-ReneoJulio López-Bastida
Jun 1, 2021·Frontiers in Pharmacology·Alessandra BlondaSteven Simoens
Jun 14, 2021·Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research·Richard H ChapmanSteven D Pearson
Jul 2, 2021·Orphanet Journal of Rare Diseases·Bettina M ZimmermannMatthias R Baumgartner
Apr 17, 2020·Neuroendocrinology·Leticia P LeonartRoberto Pontarolo

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