Biopharmaceuticals come in many modalities with varied shapes and sizes, and the manufacturing processes can vary considerably in complexity as a result. They can range from antibodies to antibody drug conjugates, cell and gene therapies, vaccines, live biotherapeutic products, enzymes and many more. Despite this, the overarching regulatory requirements remain the same and therefore, so do the concepts of the control strategy put in place.

A regulatory starting material is a chemical compound that marks the point in the synthesis of an active pharmaceutical ingredient (API) where current Good Manufacturing Practices (cGMP) begin. It is formally designated in regulatory submissions and must be scientifically justified based on its impact on the quality and safety of the final drug product. 

FDA’s Rare Pediatric Disease (RPD) Priority Review Voucher (PRV) program was established in 2012 as a financial incentive to encourage the development of therapeutics for rare pediatric diseases. The program officially ended as of September 30, 2024 due to a sunset clause. 

On December 15, 2025, the FDA published the final guidance on the “Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices” Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices  Dr. Makary said that the FDA is, “removing unnecessary barriers that have prevented us from using powerful real-world evidence to get life-changing treatments to patients faster.”