Public health
How the US made orphan drugs a market
Open access · cc by · source: Europe PMC
From Congressional hearings and interviews, Mikami shows the 1983 Orphan Drug Act’s market incentives were a contingent American choice, not the only way to supply rare-disease drugs.
Study at a glance
- Design
- Qualitative / archival — Congressional/regulatory archives plus participant interviews on the Orphan Drug Act’s origins
- N
- Policy history from 1962 reforms through 1983 Act and later amendments; interview count not primary in stored text
- Population
- US (and comparative UK) orphan-drug policy actors and rare-disease drug regulation
- Outcome
- Market exclusivity/tax credits as a contingent American choice versus alternative supply paths
Structured fields used in claim comparison tables when every cited study has a complete layer.
Key findings
Market exclusivity and tax credits were not inevitable: Britain’s Medicines Act path (named-patient supply, state product licences) offered an alternative. Patient visibility, generic-industry politics, and FDA insistence on unchanged safety standards locked in a market-based US model that later produced high-priced “true orphans.”
Methodology
The author read Congressional hearings, FDA and UK regulatory records, and secondary literature, and interviewed participants, to follow the word “orphan” from the 1962 Kefauver–Harris reforms through the 1983 Act, the 1984–85 amendments, and 1990s fights over “billion-dollar orphans.”
Limitations
It does not measure today’s orphan-drug prices or prove how many 350 post-1983 approvals would have appeared without the Act; interviews cover participants, not patients living with current access barriers.
How this study connects
Role on claims
Each row is a claim on a concept or method page where this paper supports, challenges, or qualifies the statement. Roles are hand-checked — not a model guess.
The 1983 US Orphan Drug Act’s market incentives were a contingent American choice, not the only way to supply rare-disease drugs.
Evidence for the claim as stated.
Interviews with policymakers, responders and scientists reconstruct why decisions were taken. More than 70 US interviews underpin the finding that HIV prevention policy swung with party ideology; interviews with WHO responders show Ebola being demoted to a neglected African fever inside pandemic triage; an interview with Grahame Bulfield helps explain how ABRO survived cuts by promising biotechnology; participant interviews show the Orphan Drug Act's market model was a contingent American choice.
Evidence for the claim as stated.
In practice oral history is almost always combined with documents rather than used alone. Padamsee pairs interviews with legislative histories and press; Honigsbaum with internal WHO documents; Myelnikov with Roslin/BBSRC archives and press; Mikami with Congressional hearings and FDA and UK regulatory records; Winters with archives and grey literature.
Evidence for the claim as stated.
Who gets interviewed shapes what the method can show. Padamsee's and Mikami's samples are elite policymakers, advocates and participants, and both note they do not cover patients living with current barriers. Hay and the Hungarian polio study instead centre patients, women and families. Elite interviews explain policy; patient interviews explain experience; neither does both.
Evidence for the claim as stated.
Open questions
Tensions this paper is part of
From concept pages' “where studies disagree.” Disagreement means the same question; scope means different assays, populations, or outcomes.
Who gets interviewed shapes what the method can show. Padamsee's and Mikami's samples are elite policymakers, advocates and participants, and both note they do not cover patients living with current barriers. Hay and the Hungarian polio study instead centre patients, women and families. Elite interviews explain policy; patient interviews explain experience; neither does both.
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- Supports · How did a Hungarian polio hospital work?
Discoveries this paper informs or conflicts with
- The orphan drug market was a policy choice, and Britain shows what the alternative looked like
This paper informs this development.
Related papers in this topic
Same topic cluster — not a recommendation engine.