JAWAHARLAL NEHRU TECHNOLOGICAL UNIVERSITY HYDERABAD Pharm. D V Year CLINICAL RESEARCH Previous Question Paper (June/July - 2022 - R08)

Drug discovery is a complex, multi-stage process aimed at identifying potential new medicines. The key steps include:

1. Target Identification: Scientists identify a biological target, such as a protein or gene, associated with a specific disease state.
2. Target Validation: The target is confirmed to play a critical role in the disease, usually through genetic or pharmacological studies.
3. Lead Discovery (Hit Identification): High-throughput screening of large chemical libraries is conducted to find compounds that interact with the target.
4. Lead Optimization: The identified 'hits' are chemically modified to improve their potency, selectivity, and pharmacokinetic properties.
5. Preclinical Development: The lead compound undergoes rigorous in vitro and in vivo testing to evaluate safety, toxicity, and efficacy before human trials can begin.

An Investigational New Drug (IND) application is the mechanism by which a sponsor obtains authorization from the regulatory authority (such as the FDA) to initiate clinical trials in humans. The procedure involves:

1. Preparation of the Dossier: The sponsor compiles data including animal pharmacology and toxicology studies, manufacturing information (Chemistry, Manufacturing, and Controls - CMC), and clinical protocols for the proposed human trials.
2. Submission: The complete IND application is submitted to the regulatory agency for review.
3. Review Period: The agency conducts a 30-day safety review to ensure that human subjects will not be exposed to unreasonable risk.
4. Institutional Review Board (IRB) Approval: Parallel to or following regulatory submission, the clinical protocol and informed consent forms must be approved by an IRB or Ethics Committee.
5. Clinical Hold: If the agency identifies safety concerns, they may issue a clinical hold, preventing the trial from starting until the issues are resolved. If no action is taken within 30 days, the sponsor may proceed with the clinical investigation.