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Clinical Informatics News – FDA’s Oncology Center of Excellence to Innovate Regulatory Approaches for Cancer Therapies

Clinical Informatics News – FDA’s Oncology Center of Excellence to Innovate Regulatory Approaches for Cancer Therapies

Cancer seems to touch most Americans, with an estimated 1.6 million diagnosed in 2016, and nearly 40% of Americans diagnosed at some point during their lifetime. That’s why speeding therapies to market is at the heart of the newly organized Oncology Center of Excellence (OCE), an initiative meant to support an integrated approach to evaluating products for cancer detection, diagnosis, prevention, and treatment. Committed to meeting the needs of patients, OCE, a virtual center within the Food and Drug Administration (FDA), will coordinate and review all cancer treatments coming before the agency—drugs, biologics, and diagnostics—instead of continuing with separate review in different silos. Key to this effort are OCE’s plans to revisit and possibly modify eligibility criteria in clinical trials, which could expand the number of people who qualify, providing more clinical data faster, and ultimately leading to quicker regulatory approvals for safe and transformative anticancer agents. 


Richard Pazdur, MD, is Acting Director of OCE. He is also FDA’s so-called Oncology Drug Czar, and director of the Office of Hematology and Oncology Products (OHOP) in the Center for Drug Evaluation and Research. During his tenure at OHOP, numerous oncology drugs have been approved under various expedited review pathways. In the last five years, some 60% of OHOP’s new molecular entity approvals were ahead of timeframes established by the Prescription Drug User Fee Act. Pazdur plans to work with experts to bring this type of efficiency to OCE, using multiple approaches. Most notably, there is an interest in shifting away from the conventional Phase 1, Phase 2, and Phase 3 paradigm toward a seamless approach that could expedite the regulatory pathway by addressing drug dosing, activity in various cancers, and efficacy in one trial.


This methodology may include adding cohorts to a first-in-human trial, and was described in a recent article in theNew England Journal of Medicine, on which Pazdur as a co-author. The researchers identified more than 40 active commercial investigational new drug applications for large first-in-human oncology trials using this strategy. According to Pazdur, this approach and others reflect how research progress and innovation are enhanced by regulatory expertise and clarity. To encourage development, “OCE will share its current regulatory thinking by fostering public dialogue and interacting with industry, stakeholders, and patients,” Pazdur says.


To further streamline cancer clinical trials, OCE may also explore use of a common control arm, which could involve different drug companies studying multiple drugs for the same indication. Pazdur explains, “When a common control arm is used, it decreases the overall number of patients that need to be recruited and enrolled, optimizing clinical trial resources and potentially decreasing the time it takes to get a new study off the ground.” Although common control arm studies are yet to take place, Pazdur notes that FDA, and ultimately OCE, are actively working with companies and other stakeholders to fuel discussion and workshops focused on this novel clinical trial design and others. 


OCE is part of the National Cancer Moonshot, an ambitious effort to cure cancer, first announced by President Barack Obama in January 2016 during his State of the Union Address. Its intent is to make safe and effective therapies available to more patients sooner by cutting bureaucracy and fostering collaboration among scientists, industry, patients, and the government. With input from scientific experts, Vice President Joe Biden is leading National Cancer Moonshot, which is charged with targeted incentives, private sector efforts from industry and philanthropy, and patient engagement initiatives to support and facilitate cancer research, treatment, and care in an effort to maximize this federal investment. The White House proposed $1 billion to jumpstart the Moonshot, which includes programs besides OCE, such as prevention and cancer vaccine development, enhanced data sharing, and more. But, the program is not slated to receive funding in fiscal 2017, as neither the House nor Senate appropriations bill includes monies for this initiative. 


There is a ray of hope, however. According to The Cancer Newsletter, lawmakers are expected to pass a Continuing Resolution that would last at least through December, but what that would mean for OCE is unclear. In late September, Clifford Hudis, Chief Executive Officer of American Society of Clinical Oncology moderated a panel discussion on this subject on Capitol Hill, and stated that passing a Continuing Resolution at current funding levels would not be sufficient to meet the goals of the National Cancer Moonshot—that an increase in appropriations to the tune of $2 billion, is needed. This increase would be split between the National Institutes of Health and the National Cancer Institute in support of Moonshot.

Starting (and Ending) With the Patient

Participating in that panel discussion were some members of the initiative’s Blue Ribbon Panel, including Ellen Sigal, Founder and Board Chair of Friends of Cancer Research (Friends)—the organization which proposed establishment of Centers of Excellence at FDA, starting with oncology. Friends advocates for policies and solutions that will speed cancer treatments to patients in the safest and fastest way possible. “It starts and ends with the patient, so that means that we work with all sectors: industry, patient advocacy groups, government, and academics. But, we have been focusing heavily on the regulatory environment and FDA specifically, because new treatments don’t get to a patient if they don’t get approved,” Sigal says. 


Ten years ago, Friends proposed and achieved a consolidation of oncology regulation at FDA by establishing OHOP, which focuses the agency’s review process for drugs and biologics. OCE is aiming to build on this idea by breaking down silos within the agency while taking advantage of today’s scientific advancements. “This work was the beginning of a very important relationship, as with the advent of biologics, monoclonal antibodies, and other therapies, we knew the best efforts needed an integrated approach. This recognition directed our work with FDA and led to our re-thinking how clinical trials were conducted, considering the framework hasn’t been modernized since the 1970s.” Sigal says. 


The notion of integrating offices within FDA to streamline review of investigational products reflects how they would be used in actual clinical practice. Specifically, a drug might be used based on results of a companion diagnostic test, so it makes sense for both products to be regulated in one center of excellence, allowing for more congruent timelines. Also, this approach is one more tool in the growing basket of options for accelerating much needed therapies to patients, such as breakthrough therapy designation, fast track designation, accelerated approval, and priority review. Each is different, and as Pazdur explains, “New approaches to product development, such as seamless trial design, could complement these regulatory programs meant to expedite approval and get safe and effective products to patients in the most efficient manner possible.” Also, because the framework of OCE will evolve over time, the authority to make approval decisions will remain with the center with oversight of the product, meaning that programs for expedited review of drugs will remain intact. 


The OCE model has broad application for bringing innovation to the clinical trials process, and could serve as a model for other diseases, such as neurodegenerative disease, cardiovascular health, and infectious disease.…