FDA Wants to Ease Way for Innovation
October 10th, 2011 // 3:21 pm @ jmpickett
The FDA has announced a new “blueprint” to spur biomedical innovation, which contains a plan to make it easier for drugs and devices that are considered “personalized medicine” to come to market.
FDA Commissioner Margaret Hamburg told reporters on Wednesday that the overall plan — released as part of a sweeping report on improving innovation — addresses concerns about the long-term sustainability of the medical product development pipeline.
Despite more money than ever being spent on research and development, the medical product pipeline is slowing. In 2010, the FDA had the lowest number of new product applications in its history, Hamburg said.
The report’s proposals stem from meetings between the FDA and leaders in the biotech, pharmaceutical and medical device, academic, and patient communities about how to improve innovation.
One proposal outlines how the agency will better support personalized medicine — the movement, launched after the sequencing of the human genome, that focuses on tailoring treatments toward the individual characteristics of each patient. The idea is to use genetic profiles to determine which patients are more likely to benefit from a drug and less likely to have serious side effects.
One example of personalized medicine is a drug called vemurafenib, which was recently approved by the FDA to treat late-stage melanoma specifically in patients whose tumors express a BRAF V600E mutation. The FDA also approved a genetic mutation test to be used with vemurafenib to determine whether the patient has that specific mutation.
The agency hopes to approve more such targeted therapies, Hamburg told reporters in a press call, but the guidelines on how personalized medicine is evaluated and approved by the FDA are underdeveloped, she said.
For instance, there aren’t specific guidelines for how personalized medicine clinical trials should be carried out, and there is no list of qualified clinical biomarkers that the agency will accept.
Another question that remains unanswered is whether a drug that contained a companion device would be under the purview of the FDA’s drug center or the device center.
The agency is developing a draft guidance outlining strategies for clinical trial design and regulatory considerations developing a novel companion diagnostic and therapy together, and it will also include how to use biomarkers in such trials, according to the report.
The agency will also hold meetings and reach out to stakeholders on how to spur more personalized medicine products, the report said.
In addition, the FDA is proposing to provide better outreach to small businesses; better use existing data to share information; and better train the “next generation of innovators,” in part through a new fellowship program already launched at the FDA.
