Last month the President’s Council of Advisors on Science and Technology (PCAST) released “Report to the President on Propelling Innovation in Drug Discovery, Development and Evaluation.” In the accompanying press release, the council stated that
“The United States should set a goal of doubling the output of innovative new medicines that meet critical public health needs over the next 10 to 15 years, while continuing to increase drug safety.”
The council goes on to recommend a number of necessary actions to achieve this goal involving industry, academia and the Federal Government.
On October 3rd, according to remarks reported by Bloomberg, Margaret Hamburg, Food and Drug Administration (FDA) Commissioner, stated that the FDA is considering employing a “special medical use” label that would allow faster clinical trials with a smaller required number of patients. The approach, originally discussed in the PCAST report, would allow doctors to prescribe these drugs to a defined group of patients where need is most dire.
If implemented, this strategy would allow for the implementation of a risk versus benefit discussion where patients facing death or other serious injury could have access to “special medical use” drugs. A “special medical use” drug may not have as extensive a safety profile as other drugs, but could provide benefits that other approved drugs could not. This shift in the FDA’s current approval paradigm considers the risks patients are willing to take and promotes innovation in situations where less safety data exists. Hamburg stated,
“This is an issue of having the right science and data to assess risks and benefits but also a broader societal discussion about risks and benefits that individuals and communities are willing to take on and under what circumstances.”
While the specific details of the plan have not been stated, it could be a real positive for patients. “Special medical use” drugs would provide patients with faster access to new treatments for debilitating diseases where there are no good treatment options available. It would also encourage patients to become more involved in their treatment plan by recognizing that patients facing certain diseases may be willing to accept drugs with less safety data or even undesirable side effects if it means improving their condition. Patients would be empowered to think about their quality of life and the risks they are willing to accept to potentially improve it. Thus, giving patients more control over their disease and how they wish to treat it.
The label could also provide a benefit to companies with drugs in development to treat these diseases. As currently discussed, it would allow a faster approval process and the opportunity for revenue while still allowing companies to prove additional safety with these patients. After additional safety monitoring and data gathering from these groups, it is conceivable that the company may be able to win more widespread approval if safety and risk versus benefit can be proved. By providing companies another potential pathway, it eases pressure on the FDA, while still allowing the agency to increase their new drug approval numbers.