Regulatory Frameworks for Advanced Therapy Medicinal Products (ATMPs): A Comparative Review of FDA, EMA, and CDSCO

Abstract

Background: Advanced therapy medicinal products (ATMPs) such as gene therapy medicinal products,
somatic cell therapy medicinal products, tissue engineered products and combined advanced therapies
are one of the fastest growing classes of biological medicines. The biological complexity and individualized
manufacturing of these products do not fit well within regulatory systems designed for small-molecule
drugs, and the three regulators examined here – the United States Food and Drug Administration (FDA), the
European Medicines Agency (EMA), and India’s Central Drugs Standard Control Organization (CDSCO) – have
each developed distinct legal and procedural routes for this product class.
Aim: To compare the legal basis, institutional architecture, clinical trial oversight, expedited approval
pathways, manufacturing and quality expectations, post-approval surveillance needs, and access and
reimbursement mechanisms governing advanced therapy medicinal products under the FDA, the EMA, and
CDSCO, and examine the extent to which these systems are converging/diverging.
Data Sources: Gray literature from well-acknowledged regulatory and health policy organizations, official
publications of FDA, EMA and CDSCO, PubMed and Scopus and Web of Science indexed journals focusing on
the content published from 2019 to mid-2026.
Review Methodology: Our approach was narrative and comparative. Peer-reviewed articles, regulatory
guidance documents, and health-policy analyzes on ATMP definition, licensure pathway, clinical trial
authorization, expedited designation, manufacturing oversight, and reimbursement were identified. These
were thematically synthesized and cross-checked against primary regulatory sources where available.
Main Findings: ATMPs are biologics regulated by the FDA under section 351 of the Public Health Service Act by
the Center for Biologics Evaluation and Research, with the assistance of the Regenerative Medicine Advanced
Therapy designation. The EMA has centrally overseen ATMPs under Regulation (EC) No 1394/2007 via its
Committee for Advanced Therapies, a structure currently being reviewed as the European Union completes a
broader reform of its pharmaceutical legislation. The Central Drugs Standard Control Organization (CDSCO)
oversees cell and gene therapy products along with the Indian Council of Medical Research, the Department of
Biotechnology, and the Gene Therapy Advisory and Evaluation Committee under the New Drugs and Clinical
Trials Rules, 2019, an arrangement recently strengthened by amendments that put these products under
joint central and state oversight. India’s approval last year of NexCAR19, a homegrown and much cheaper
CAR-T cell therapy, reflects both the maturing Indian pathway and the ongoing global affordability divide.
Conclusion: The FDA and EMA frameworks are demonstrating increasing procedural convergence in quality
and comparability expectations, while CDSCO has moved rapidly toward a comprehensive, if still evolving,
national framework. Across the three jurisdictions, the principal barriers to equitable patient access to
sophisticated therapy medicinal products remain the ability to reimburse and the generation of long-term
evidence, rather than scientific review alone before the market.