Latest FDA Efforts to Expand Access Involve Complex Generic Drugs, Investigational Treatments

On consecutive days, the FDA announced 2 more strategies to help novel drugs reach more patients. One plan will encourage competition by providing more guidance to developers of complex generics, while the other will make it easier for physicians to treat patients with investigational products through the expanded access program.

The initiatives were announced in FDA Voice blog posts by Commissioner Scott Gottlieb, MD, who appears to be pressing on with his multipronged efforts to increase access and lower the costs of prescription drugs, amid rumors that he may be a top pick to succeed Tom Price, MD, who resigned from the post of HHS secretary on September 29 after controversy over the use of private jets.

The first post, published on October 2, discussed ways to expand access to and affordability of complex generic medicines by encouraging development of new generics and therefore increasing competition in the market. Currently, Gottlieb explained, the regulatory complexities of these drug products can lead to a lengthy review process that deters some drug makers from developing a new product.

First, the FDA will provide guidance to those submitting abbreviated new drug applications (ANDAs) for such products, which will help them increase the efficiency of their meetings with the FDA, boost communication, and ultimately speed up the ANDA process. Additionally, the FDA will issue clarifying guidance on when ANDAs are appropriate for certain complex peptides.

Looking forward, Gottlieb wrote, the FDA’s efforts to boost competition in the complex generic drug landscape will extend beyond guidance. The agency has started holding workshops to develop tools to allow manufacturers to more easily demonstrate the “sameness” of the active ingredient in their product compared with the reference drug.

“We intend for these efforts to speed product development, reduce development costs, and improve access to these products,” Gottlieb wrote, adding that the agency is “just getting started” on these initiatives.

A day later, he authored a post about new rules that will help patients with serious conditions more easily receive investigational drugs through the FDA’s expanded access program. Currently, physicians must gain approval from their Institutional Review Board (IRB) before treating such patients under expanded access, but under new rules initiated by Gottlieb, just 1 member of the IRB can approve the treatment.

“I believe the simplified IRB process will facilitate access while still protecting patients,” Gottlieb wrote.

The FDA will also modify the rules for reporting suspected adverse reactions with investigational drugs, making it so that events must only be reported if evidence suggests a causal relationship between the drug and the event. Previously, some companies had been hesitant to provide investigational products under the expanded access program, fearing that any adverse reactions reported by these seriously ill patients would reflect poorly on the product in development.

Finally, the FDA will step up its navigation tools for patients and physicians looking to learn more about the investigational treatments available to them. The Reagan-Udall Foundation has already partnered with the agency to create an online resource, the Expanded Access Navigator, which will now include investigational therapies in the FDA’s Rare Disease Program.

“Looking ahead, there will be more options for expanded access,” Gottlieb concluded. “FDA will continue our efforts to educate stakeholders on these opportunities and further simplify the process in order to improve the program.”