Orphan drug development: Challenges, regulation, and success stories

Chirmule, N and Feng, H and Cyril, E and Ghalsasi, VV and Choudhury, MC (2024) Orphan drug development: Challenges, regulation, and success stories. In: Journal of Biosciences, 49 (1).

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Abstract

Rare diseases, also known as orphan diseases, are diseases with low occurrence in the population. Developing orphan drugs is challenging because of inadequate financial and scientific resources and insufficient subjects to run clinical trials. With advances in genome sequencing technologies, emergence of cell and gene therapies, and the latest developments in regulatory pathways, some orphan drugs that have curative potential have been approved. In India, due to its large population and resource crunch, developing orphan drugs is phenomenally challenging. After adopting the Orphan Drug Act, the US-FDA has continuously made advances in regulatory pathways for orphan drugs. Particularly, n-of-one clinical trials have been successful in some cases. India has recently adopted policies that have impacted the long-neglected rare-disease ecosystem; however, there is no clear regulatory path for orphan drug development in India. We have proposed a multi-pronged approach involving close collaboration between the government, regulatory bodies, industries, and patient advocacy groups to boost orphan drug development in India. We believe that rapidly evolving technologies and business models can enable better and faster development of novel orphan drugs in India and other resource-constrained countries. © Indian Academy of Sciences 2024.

Item Type:	Journal Article
Publication:	Journal of Biosciences
Publisher:	Springer
Additional Information:	The copyright for this article belongs to the Springer.
Keywords:	casimersen; elivaldogene autotemcel; etranacogene dezaparvovec; fidanacogene elaparvovec; ifezuntirgene inilparvovec; onasemnogene abeparvovec; orphan drug; sonpiretigene isteparvovec; voretigene neparvovec; orphan drug, disease incidence; disease treatment; drug development; health policy; model test; policy development; policy implementation; regulatory framework; research work, adrenoleukodystrophy; blindness; checklist; disability; drug cost; drug efficacy; drug manufacture; drug safety; Duchenne muscular dystrophy; Food and Drug Administration; gene therapy; government; hemophilia B; human; Huntington chorea; India; patient advocacy; policy; rare disease; retina dystrophy; retinitis pigmentosa; Review; Stargardt disease; Werdnig Hoffmann disease; advocacy group; article; commercial phenomena; drug development, India, Ecosystem; Humans; India; Orphan Drug Production; Rare Diseases; United States; United States Food and Drug Administration
Department/Centre:	Division of Interdisciplinary Sciences > Centre for Society and Policy (formerly: Centre for Contemporary Studies)
Date Deposited:	22 Apr 2024 10:08
Last Modified:	22 Apr 2024 10:08
URI:	https://eprints.iisc.ac.in/id/eprint/84664

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