This initiative aims to provide advice and directions regarding regulatory requirements for preclinical studies and clinical translation.
Name in original language
National Guidelines for Gene Therapy Product Development and Clinical Trials
Initiative overview
Gene Therapy is one of the exciting avenues in the field of biological therapeutics. Globally, a very few gene therapy products have been approved. The Gene Therapy Products need systematic evaluation for its safety and efficacy in view of the underline ethical and scientific concerns. The scientists and industry involved in the field seek advice and directions regarding regulatory requirements for preclinical studies and clinical translation. Taking cognisance of this gap, ICMR proactively initiated the process of drafting the National Guidelines for Gene Therapy Product Development and Clinical Trials in consultation with the experts in the field and government agencies including DBT and CDSCO.Objectives:To ensure development of safe and effective GTPs, adhering to the following:a. product quality characterization of the components and processes involved, the production process and quality control strategies for GTPs, clear chemical and biological definition of the final product;b. the pre-clinical evaluation of the GTP to establish, with reference to its dosage and route of administration, its safety profile, bio-distribution, to identify the pharmacological/toxicological characteristics that support safety as well as efficacy of the GTP for human use;c. the clinical study design to establish safety and efficacy of GTPs in the target indication(s), GTP dosage(s), route of administration and selection procedures for patients and frequency of side effects or adverse events associated with any therapeutic strategy; and the process of regulatory approval for clinical trials.d. Long-term patient follow-up to monitor its therapeutic benefit(s) and immune response or any adverse effect(s), if any, due to the GTPs.
Name of responsible organisation (in English)
Central Drug Standards Control Organisation (CDSCO)|Ministry of Health and Family Welfare |Ministry of Science and Technology |Indian Council of Medical Research (ICMR)
