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February 26, 2016.
FDA needs to make major changes in how it evaluates the research and development of new combination therapies for cancer and other serious conditions, as scientific discoveries continue to revolutionize medical product discovery and patient care, according to patient advocates, cancer specialists and biopharma companies. Spearheaded by the Friends of Cancer Research (FOCR), a group is urging Congress to further encourage FDA to adopt a new “disease-oriented” approach biomedical regulation, and for FDA to formulate a pilot to test this approach.
The advocates are encouraged by a provision in the Obama administration’s “Cancer Moonshot” initiative, which would provide FDA with $75 million to form an Oncology Center of Excellence to expedite the development of novel cancer treatments. Functioning as a “virtual” entity, the Center would better coordinate FDA scientists and reviewers that reside in separate FDA Centers for drugs, biologics and medical devices to achieve a more integrated approach to regulating new cancer treatments.
This “moonshot” proposal reflects the broader campaign launched by FOCR chair Ellen Sigal to shift FDA from regulation based on product type to a disease-oriented approach. A first step would be to establish a new Center of Excellence for oncology to facilitate the testing and clinical development for all cancer therapies, including diagnostics and delivery devices. This would evolve from an FDA pilot that brings together key staffers from drugs, biologics and devices so they could collaborate more closely on the design, testing and approval of new cancer treatments.
This approach aims to address the regulatory confusion that has resulted from the emergence of more combination cancer treatments that now are regulated by different FDA Centers and are governed by different policies, procedures and statutory requirements. The result is delay in product development and approval, along with greater outlays for multiple tests and clinical studies.
At a Senate briefing Feb. 24, 2016 organized by FOCR, Steve Galson, senior vice president for global regulatory affairs and safety at Amgen, noted that the new wave of highly targeted cancer therapies come with increasingly sophisticated medical device delivery systems and with a range of diagnostics designed to ensure appropriate prescribing. Such products involve consultation on clinical development and application review with multiple FDA Centers. Galson noted that Amgen is testing a new treatment that combines a herpes simplex virus previously approved by the Center for Biologics Evaluation and Research (CBER) with an immunotherapy from another firm that is regulated by the Center for Drug Evaluation and Research (CDER) to create what hopefully will be a more effective treatment for melanoma. The process for gaining agreement on clinical trial design thus is more complicated, and there is more uncertainty about how the review process will proceed.
However, a shift to a disease-based regulatory approach would impose significant changes on FDA’s organizational structure that could stress its limited resources and disrupt long-held practices and procedures. FDA has tried to better coordinate oversight of therapies with multiple components through its central Office of Combination Products, but this has not resolved many stakeholder complaints about delay and uncertainty. Mark McClellan, former FDA commissioner and now director of the Duke-Margolis Center for Health Policy, acknowledged at the briefing that more new products don’t fit FDA’s current Center-based structure, but that any major agency overhaul requires “serious, thoughtful consideration.” To move forward, he suggested assessing the most serious problems related to developing combination products and how a new disease-oriented oversight approach would outweigh risks.
Sigal now is looking for FDA leaders to propose a pilot to test these concepts. While she believes that this disease-based oversight approach also is suitable for dealing with other serious health conditions such as neurodegenerative, cardiovascular and infectious disease, she and others accept plans to limit an initial test to oncology, where more advanced, targeted, combination products now dominate the R&D environment.