Capitalizing on Hope: Questionable Marketing Approval and Pricing of a New ALS Drug.
| Autor: | Flynn MB; Department of Political Science and International Studies, Georgia Southern University College of Behavioural and Social Sciences, Statesboro, GA, USA., Flynn JF; 425 E 54 St, Savannah, GA, USA., Palacios AM; Department of Health Policy and Community Health, Georgia Southern University Jiann-Ping Hsu College of Public Health, Savannah, GA, USA. |
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| Jazyk: | angličtina |
| Zdroj: | International journal of social determinants of health and health services [Int J Soc Determinants Health Health Serv] 2024 Oct; Vol. 54 (4), pp. 405-411. Date of Electronic Publication: 2024 Apr 22. |
| DOI: | 10.1177/27551938241247778 |
| Abstrakt: | Regulatory agencies must balance patient demands to access new treatments for fatal diseases with limited treatment options while ensuring drug safety and efficacy. However, questionable U.S. regulatory actions resulted in the early approval of AMX0035 to treat amyotrophic lateral sclerosis (ALS) by reconvening advisory commissions to obtain positive decisions and designating the drug as a new molecular entity. Data from one randomized clinical trial suggests minimal delays in disease progression and longer survivability, but debate remains about the lack of confirmatory evidence of effectiveness owing to study limitations. A patient's decision-making process details the experience of using the drug, including perspectives on access, cost, effectiveness, and adverse effects. In line with the "nichebuster" business model, the drugmaker, Amylyx Pharmaceuticals, is charging US$158,000/year/patient and thus forecast to turn a profit on a drug with debatable clinical effectiveness prior to completing a Phase 3 trial. Early marketing approval, despite community demands, is unnecessary and may have reduced access because of the end of a compassionate use program, and the high price tag results in restricted coverage and high out-of-pocket costs. Also, the drug's key ingredients are available as a generic and a supplement. Competing Interests: Declaration of Conflicting InterestsThe authors declared the following potential conflicts of interest with respect to the research, authorship, and/or publication of this article. M.B.F. and A.M.P. are partners and caretakers to J.F.F. who is a sibling of M.B.F. and participated in a compassionate care trial sponsored by Amylyx Pharmaceuticals. |
| Databáze: | MEDLINE |
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