Ensuring patient rights, safety, and welfare in “compassionate use” of drugs

Abstract Purpose/Aim: “Compassionate use” of drugs refers to providing access to new drugs for patients with life-threatening diseases who have exhausted all other therapeutic options or patients with diseases for which no other therapeutic modalities are currently available in the market. While the U.S. and many European countries have established structured regulatory, ethical, and pharmacovigilance pathways for compassionate drug use, India lacks a comprehensive and consistent regulatory, ethical, and pharmacovigilance framework for compassionate drug use. This paper aims to critically examine the existent regulatory, ethical, pharmacovigilance, and legal frameworks for compassionate use of drugs in India and propose actionable recommendations for a compassionate drug use system tailored to the Indian context. Methods: A comprehensive review of existing policies and legal framework governing compassionate drug use in India was employed with comparative evaluation against international compassionate drug use frameworks. Supplementary review of literature on pharmacovigilance and ethics guidelines was used to assess systematic gaps in monitoring and implementation. Results: India’s existing guidelines for compassionate use of drugs have no clearly defined legal structures or accountability mechanisms. The absence of an ethical, pharmacovigilance, and legal framework in India contributes to ambiguity in access and patient safety concerns. Conclusions: There is an urgent need to develop a formal regulatory framework for compassionate drug use in India, integrating mandatory ethics committee oversight and pharmacovigilance systems. Such a framework would ensure equitable access to potentially lifesaving drugs while safeguarding patient safety and regulatory accountability.