- Janet Woodcock, director of the Food and Drug Administration’s Center for Drug Evaluation and Research, touted the agency’s move toward precision medicine in her latest blog post.
- Two recent approvals underscore the move to green light drugs based on genetic mutations or features — Kalydeco’s (ivacaftor) approval in 23 additional rare mutations and the go-ahead of Merck & Co.’s Keytruda (pembrolizumab), based on genetic features of a tumor rather than part of the body.
- Woodcock notes that over the last three years alone, CDER has approved 25 new drugs for patients with specific genetic characteristics, as well as new indications for older drugs based on genetic qualifiers.
Precision medicine has been a buzzword in the industry for the last several years, and the FDA, as well as the industry, has been pushing to make drugs more tailored to the individual patient.
In a blog post published Tuesday, Woodcock acknowledged the leaps that the industry and the agency have made in bringing patients new treatments that could work better based on their individual genetics, but she underscored that there is still a long way to go.
“Precision medicine holds great promise, but to continue developing targeted therapies, we will need scientific advances in the use and development of ‘biomarkers,’” wrote the director, who goes on to explain that biomarkers are indicators that can be measured, such as blood pressure and even genetic variants.
“Biomarkers can enable health care professionals and researchers to identify patients at risk of disease, determine the stage of a disease, and predict the likelihood that a patient will benefit from a drug. They also play a role in drug development,” she wrote.
“A particular biomarker, for example, can be used to identify appropriate candidates for a clinical trial, such as those patients likely to respond to treatment. This can make it easier and faster to recruit patients and may result in a shorter time for drug approval.”
For the industry, this is a positive sentiment. Biomarkers are becoming increasingly prevalent in the development process and it appears the agency is more than willing to work with drugmakers to approve drugs more quickly based on these qualifiers.