The new partnership was made public yesterday in a briefing on the NIH campus in Bethesda, Maryland.
“We’re here today because we know we can deliver new treatments faster and safer if these agencies strengthen their partnership,” Kathleen Sebelius, Secretary of the Department of Health and Human Services, said during the briefing. “Communicating throughout the process will help researchers navigate the regulatory process and give regulators the scientific tools they need to quickly assess a treatment’s risks and benefits. For Americans,” she said, “this is going to mean that new treatments are available safer and sooner.”
“The new scientific opportunities we see in front of us pose the possibilities of truly novel therapeutic developments,” said Francis S. Collins, MD, PhD, director of the NIH,” but there are challenges to the way in which those are implemented that we are going to have to work closely together on to make this come true.”
Dr. Collins briefly outlined the steps in regulating medical therapies: “After identifying a promising compound, then the challenging steps called the ‘valley of death’ appear, where one has to take that promising compound and apply it in a variety of very stringent tests for specificity to be sure that it is not toxic. That is oftentimes where failure is likely to occur.”
Once a new compound goes through that process, the FDA is asked for approval for the compound to go through phase 1, 2, and 3 clinical trials. After data are collected, the FDA passes judgment on whether the compound can be safely approved for general use. It is important that the science of developing a compound is connected to the science of how FDA does its reviews, but “I think it’s fair to say that has not always been the case,” Dr. Collins observed.
Among difficult challenges for the FDA are rare diseases, for which it may be difficult to find a large number of patients, especially some childhood diseases that are often rapidly fatal. In such cases, how large does a trial have to be? And what should be the standards for testing and approving therapies?
Personalized medicine is another special case, in which a DNA test can predict which individuals are likely to respond to a new therapy. However, once a target population is identified in whom the therapy is expected to have a greater effect, it may be possible to shrink the size of trials, thereby saving both time and money.
Gateway to the Marketplace
“Many people think of FDA as the judge, a bureaucratic agency that basically gives thumbs up or thumbs down to any given application,” observed Margaret A. Hamburg, MD, commissioner of the FDA, during the briefing. “This is not the case. What we’re working on now is trying to ensure that as we go forward in this critical role as gateway to the marketplace, we can bring the best science to bear.”
The joint NIH-FDA announcement described 3 new initiatives to kick off the partnership:
Creation of a joint NIH-FDA leadership council cochaired by Dr. Collins and Dr. Hamburg that will bring together scientists from both agencies. The council will include 6 senior scientists from each agency, to be identified relatively soon.
Grants (requests for applications) involving $6.75 million available over the next 3 years for research on regulatory science, asking the scientific community to come forward with their best ideas for how science can inform the regulatory process. The program is funded jointly by the NIH ($2 million per year) and the FDA ($250,000 per year), both of which will oversee grant review and funding.
FDA and NIH outreach to the American public, including private and nonprofit organizations with key roles in bringing new medicines to market. A public meeting will be held this spring to obtain input from stakeholders and solicit suggestions for how the NIH and the FDA can work more effectively together.
“Regulatory science is the critical bridge between biomedical research and new medical product,” Dr. Hamburg said. “Today’s announcement will lead to unprecedented cooperative efforts between NIH and FDA to identify key questions in regulatory science, and then to answer them.”