NEW YORK - More and more these days, the FDA’s efforts to facilitate faster drug development are starting to come to pass.
"Critical Path is happening," Scott Gottlieb, the agency’s deputy commissioner for medical and scientific affairs, told BioWorld Today. "People don’t realize that."
He outlined several current and near-term activities as part of a discussion Wednesday at the BIO CEO & Investor Conference, held here at the Waldorf Astoria Hotel.
Among the newest, the FDA on Tuesday unveiled a multi-agency effort called the Oncology Biomarker Qualification Initiative to identify, validate and standardize predictors for cancer. In collaboration with the National Cancer Institute and the Centers for Medicare and Medicaid Services, initial work will involve a clinical trial to test the combined use of positron emission tomography and flurodeoxyglucose as a predictor of tumor response in non-Hodgkin’s lymphoma patients.
Similarly, the FDA is set to announce next month its development of a consortium that includes major pharmaceutical companies to create toxico-genomic assays. In addition, there’s talk that the initiative will include an opportunities list for the drug industry. Also down the road, the agency is expected to issue guidance on an approval pathway for pairing drugs and diagnostics, and for getting a biomarker validated and approved for future use.
Gottlieb conceded that "it could take many years" before those sorts of endeavors are put into regular practice, because of the nature of scientific studies required for proof. Also, there will need to be confidence among developers that the FDA will base decisions on biomarker data, and confidence among the public that the markers are linked to disease.
Those types of projects, all part of the Critical Path Initiative, speak to the FDA’s push to improve drug development efficiency.
"It has become clear that the drug development process itself has really become a bottleneck," Gottlieb said.
He later added that the FDA does have a responsibility to help break up the drug approval logjam. Notably, Gottlieb said the agency has caused some development slowdowns - not because of protracted review times, but because of its reliance on outdated, rigid guidelines, such as those that shape clinical programs, trial designs and ultimately drug companies’ costs.
He said the FDA is looking at adaptive trial designs, which is music to the ears of the growing number of people who believe that there is no single path anymore toward drug approval.
At the same time, the agency is working to improve its management and change some of its internal culture. In particular, the FDA is aiming to work more collaboratively with drug companies on Phase IV trials to get relevant data out of post-approval studies, and also expects to get better minutes out of meetings with drug companies, such as those frequently held after Phase II, for better exchanges on issues such as approval pathways. Such meetings continue to grow in popularity, Gottlieb said, and they lead to potentially shorter and less expensive development.
Gottlieb also contested the popular notion that the speed of review at the FDA competes with safety. And while some critics charge that the FDA is cutting corners in trying to build new efficiencies into drug development, Gottlieb challenged that idea, noting that biomarkers for HIV, diabetes and cardiovascular diseases have increased efficiency without compromising safety.
The two-day conference ended Wednesday. It drew more than 2,400 people, evenly split between investors and company executives.