Abstract
Background: Globally, rare diseases affect around 300 million people, but most of these disorders lack approved treatments. Orphan drugs - pharmaceuticals for rare diseases - represent a special category of drugs, balancing the profit disincentive with the compelling need. The COVID-19 pandemic (2019-2023) has ushered in an era of "warp-speed" regulatory science, with global adoption of rolling reviews, adaptive licensing, real-world evidence models and emergency use authorisations. This has seen significant regulatory changes in both the United Kingdom (post-Brexit Medicines and Healthcare products Regulatory Agency, MHRA) and India (Central Drugs Standard Control Organisation, CDSCO).
Aim: This article offers an in-depth comparative assessment of emerging orphan drug regulatory policies in the UK and India in the post-COVID-19 years (2020-24). It reviews specialised pathways, incentives, health technology assessment (HTA) integration, equitable access and institutionalisation of pandemic regulatory innovations to rare diseases.
Methods: We undertook a comparative policy analysis by way of a documentary review. It utilised data from MHRA and CDSCO open-access databases (2020-2024), India's National Policy for Rare Diseases 2021, WHO technical reports, UK Rare Diseases Framework action plans (2022-2024) and peer-reviewed publications identified by PubMed and Scopus.
Key Findings: Post-Brexit regulatory autonomy in the UK has enabled the development of the Innovative Licensing and Access Pathway (ILAP) and refined Orphan Medicinal Product (OMP) designation process, incorporating genomic medicine in genomics England's 100,000 Genomes Project. India's National Policy for Rare Diseases 2021 is a conceptual leap but lacks implementation with key flaws in funding (₹50 lakh cap), Centre of Excellence (CoE) infrastructure and orphan drug legislation. COVID-19 showed rolling reviews and real-world evidence can shorten development by 30-60%, but these approaches have been irregularly institutionalised for rare diseases in both countries.
Conclusion: The regulatory focus is on both countries. India needs a Rare Diseases Act, insurance mandate and CoE investment. The UK needs to resolve late NHS funding of MHRA-approved drugs. Capacity building for regulatory cooperation between the MHRA and CDSCO via a memorandum of understanding (MoU) presents a huge opportunity to minimise waste and benefit rare disease patients in both jurisdictions.