The next generation of rare disease drug policy: ensuring both innovation and affordability

Autor:	Caroline Pearson, Lindsey Schapiro, Steven D Pearson
Rok vydání:	2022
Předmět:	Policy Rare Diseases Orphan Drug Production Health Policy Costs and Cost Analysis Humans Drug Approval United States
Zdroj:	Journal of Comparative Effectiveness Research. 11:999-1010
ISSN:	2042-6313 2042-6305
DOI:	10.2217/cer-2022-0120
Popis:	Scientific advancements, new US FDA approval pathways and limited competition have contributed to rapid growth in the number of approved rare disease treatments in recent years. While the rising numbers of orphan drug approvals are a sign of success, the rapid growth in approved rare disease treatments has created concerns about the pricing of orphan drugs and their cumulative affordability to the health system. To support efforts to build a policy and practice infrastructure that drives innovation within a platform that is affordable to patients and the health system, this paper provides an analysis of potential risks as well as advantages of reform options related to drug development, pricing and coverage.
Databáze:	OpenAIRE
Externí odkaz:	https://explore.openaire.eu/search/publication?articleId=doi_dedup___::754629e3a92ffa1a305866329bfc3f95 https://doi.org/10.2217/cer-2022-0120 Zobrazit plný text záznamu