Regulatory aspects at the drug discovery development interface

Abstract

While discovery scientists are interested in proof-of-concept data, regulators seek key safety data prior to advancing new drug candidates into first-inhuman (FIH) clinical trials. Safety as well as therapeutic activity data continues to be collected and evaluated at each subsequent clinical trial stage of a program to gain proof of concept. This requires clear research and development targets to help guide the development pathway to provide phase-appropriate investigational new drug (IND) submissions. In early discovery a customized development target product profile (dTPP) describing the key attributes of the proposed product is recommended. This dTPP defines the important aspects of the discovery target that guide the development program, and as it evolves, it becomes aligned with what regulatory reviewers have outlined as the key information for ultimate approval of the proposed product. These details provide scaffolding for a development pathway with appropriate regulatory targets and a foundation for the initial IND submission. The IND is the detailed description of the sponsor’s safety data, clinical study design, and drug product. The goal of early studies in humans is the generation of sufficient data to transition to the next critical milestone and eventually to a safe, therapeutically beneficial, and therefore marketable drug product. Ultimately, this necessitates developing a product that keeps all stakeholders in mind: the innovator, the patient or end user, and regulators. Essential to achieving that goal is a strong foundation that is described in the IND.