We are living on the cusp of a new era in human medicine – for the first time, scientists are developing medicines that can be tailored to an individual person based on the characteristics of their disease.
We enjoyed this video featuring U.S. Food and Drug Administration (FDA) Commissioner Scott Gottlieb, who, in an interview with Politico, shares his thoughts on what he sees as the most promising new medicines in development today, and the challenges and opportunities for evolving the regulatory system to accommodate them.
Top of Gottlieb’s list of the most exciting precision medicine approaches? Viral vector gene therapies, which deliver a new gene into the body via a genetically engineered virus – adenoviruses, retroviruses and lentiviruses are most commonly used. Then there is CAR-T, which involves removing T cells from the patient, re-programming the genetic makeup of the cells to express certain factors, and re-implanting them into the body via infusion. Finally, CRISPR technologies involving editing the human genome to have a therapeutic effect.
With these new approaches is coming a new challenge – how do you regulate a drug that has a different makeup for each patient? The key to regulatory success, Gottlieb says, is to develop a method for validating not just the product, but the process used to create it, and ensuring that process is reproducible at scale.