Clinical Research - Drugs & Biologics

A General Overview of Medical Product Clinical Investigations

Before pharmaceutical or biologic products become available to the public, clinical studies must be conducted to ensure their safety and efficacy. These studies, also known as trials, involve human participants. The medical product development process typically includes four main types of studies: Phase I, II, III, and IV. The selection of the appropriate study type depends on factors such as the stage of development, the type of marketing application, as well as the complexity of the product and the risks they pose to consumers.

Considerations for Drug Substances/Generic Drugs Clinical Trials

• Phases I and II are typically initiated following Investigational New Drug Application (IND) approval.
• Phase III studies play an important role in supporting New Drug Applications (NDAs) and Abbreviated New Drug Applications (ANDAs) in order to market these therapeutics.
• Marketing approval studies for generic products must demonstrate bioavailability or bioequivalence to the reference drug, often a brand-name drug. These studies can include animal research or clinical trials, but they are not always required.

Considerations for Biologics/Biosimilars Clinical Trials

• In addition to requiring clinical trial data to support Biologics License Applications (BLAs), the FDA also requires information about the manufacturing process of biological products to demonstrate purity and potency.
• Clinical trials for biosimilars may be conducted after the completion of analytical and animal studies to further address any clinically meaningful differences between the proposed biosimilar and reference biologic i.e., investigating pharmacodynamics and pharmacokinetics of the biosimilar in study participants.
• The FDA permits risky gene therapy trials involving the pediatric population, provided that the IRB determines the study can be conducted under the criteria outlined in 21 CFR 50, Subpart D – Additional Safeguards for Children in Clinical Investigations.

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