The Orphan Drug Act at 35: Observations and an Outlook for the Twenty-First Century

Bagley, Nicholas; Berger, Benjamin; Chandra, Amitabh; Garthwaite, Craig; Stern, Ariel Dora

doi:10.1086/699934

Cited by 28 publications

(22 citation statements)

References 26 publications

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“…Humira, for example, had minimal utilization among patients with hidradenitis suppurativa before FDA approval. 29 FDA approval resulted in an almost 10-fold increase in sales revenue for Humira among this patient population, suggesting that offlabel use is not a substitute for FDA-approved use.…”

Section: Discussionmentioning

confidence: 96%

Modifying the Criteria for Granting Orphan Drug Market Exclusivity

2020

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Section: Discussionmentioning

confidence: 96%

Modifying the Criteria for Granting Orphan Drug Market Exclusivity

2020

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“…"Evergreening"-the attempt to extend a product's exclusivity by patenting minor improvements-is especially criticized (Hemphill andSampat 2012 andEuropean Commission Competition DG 2009). Bagley et al (2019) argue that the Orphan Drug Act disproportionately rewards producers of inframarginal products. In some cases, the protection generates no innovation benefits.…”

Section: Discussionmentioning

confidence: 99%

The Alignment of Innovation Policy and Social Welfare: Evidence from Pharmaceuticals

Kyle

2020

Innovation Policy and the Economy

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This chapter provides an overview of different innovation policies and their performance in the pharmaceutical sector. I emphasize three points. First, both push and pull policies have generally promoted pharmaceutical research for diseases with large burdens. Second, imperfections in product and capital markets undermine the efficiency of pull policies. Similarly, the allocation of public funds is not always optimal, which limits the efficacy of push policies. Finally, interactions with other domestic policies and with policies in other countries are often overlooked in both economic studies of pharmaceutical research and development as well as policy choices.

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“…Some of the recently approved drugs are much more likely to include those that are intended for use in much smaller populations, many of which meet the definition of rare disease. The number of "orphan drugs" -a US Food and Drug Administration (FDA) designation for drugs for rare diseases -approvals have increased five-fold from an average of 15 approvals per year in the 1990s to over 80 in 2017 alone (Bagley et al 2018;CADTH 2016). Given that these drugs target smaller populations, the efficacy and safety evidence for these drugs tends to be developed in relatively smaller studies, which can undermine their reliability.…”

Section: Challenges and Opportunitiesmentioning

confidence: 99%