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To streamline the regulatory processes for future clinical trials using gene therapeutic products (GTPs), the Union health ministry has placed draft national guidelines for gene therapy product development and clinical trials in public domain for suggestions from concerned stakeholders like scientists, academicians and professionals in the field before August 1, 2019.
The draft national guidelines is a welcome change as gene therapy products need systematic evaluation for its safety and efficacy in view of the ethical and scientific concerns. Gene therapy is one of the exciting avenues in the field of biological therapeutics.
Globally, very few gene therapy products have been approved.
The scientists and industry involved in the field seek advice and directions regarding regulatory requirements for pre-clinical studies and clinical translation.
Taking cognisance of this gap, Indian Council of Medical Research (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 department of biotechnology (DBT) and Central Drugs Standard Control Organisation (CDSCO).
As per the New Drugs and Clinical trial Rules (2019), the GTPs falls under ‘new drug’ and shall always be deemed to be ‘new drug’. Thus, as per these rules ‘academic trials’ are not applicable to clinical trials using GTPs.
The aim of the document is to guide and enable stakeholders to comprehend and comply with the regulatory requirements for research and development of GTPs in India.
It also provides basic guidance for research involving human participants, including clinical trials, pertaining to the broad area of gene therapy covering all the technologies and processes for all mendelian, non-mendelian and other complex disorders.
Disease-specific guidelines will subsequently be developed as part of standard operating procedures (SOPs) for different conditions. All GTPS being developed with the intention of potential human applications must adhere to these guidelines and should be used only under the purview of well defined and approved clinical trials.
The aim of this document is also to ensure development of safe and effective GTPs, adhering to product quality, characterization of the components and processes involved, production process and quality control strategies for GTPs, clear chemical and biological definition of the final product.
This also includes 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 or toxicological characteristics that support safety as well as efficacy of the GTP for human use, the clinical study design to establish safety and efficacy of GTPs in the target indications, GTP dosages, route of administration and selection procedures for patients and frequency of side effects or adverse events.
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