FDA Oncology Center of Excellence Moves Forward on Coordinated Clinical Reviews, Integrated Approach to the Clinical Evaluation of Oncology Products

In January 2017, the FDA officially established the Oncology Center of Excellence (OCE) to expedite the development of oncology and malignant hematology-related medical products and support an integrated approach to the clinical evaluation of drugs, biologics, and devices for the treatment of cancer. The FDA began work on forming the OCE more than a year ago, after President Barack Obama announced the National Cancer Moonshot Initiative in his final State of the Union address. This new center leverages the combined skills of regulatory scientists and reviewers with oncology clinical expertise across the FDA, emulating the multidisciplinary organizational model of academia and cancer care centers and building on the integrative approach to medical product development that the agency’s broader oncology community has embraced over the past decade.¹

The OCE already has begun coordinating clinical reviews of oncology products. Sponsors with product applications that have proposed medical oncology or malignant hematology indications should submit their applications to the medical product center they always have: the Center for Drug Evaluation and Research, the Center for Biologics and Evaluation Research, or the Center for Devices and Radiological Health. After the submission is received and if deemed appropriate, the OCE will form a clinical review team comprising experts from the 3 medical product centers, as necessary, based on the specific application. The review team will make its recommendations on the clinical portion of the application only. As OCE director, I review and sign off on their recommendation, after which the application is returned to the corresponding center to make a final product approval determination. Other aspects of product review, such as quality review, toxicological review, statistical review, and facilities inspection, as well as final action on the application, remain the responsibility and authority of the center that normally has jurisdiction over the oncology-related medical product.

Leveraging the Power of Collaboration

By bridging the various medical product centers of the FDA, the OCE is ideally suited to support innovation and address the recognition that multiple treatment and diagnostic options are in the best interests of patients. An important aim of the OCE is to facilitate incorporation of the patient view into regulatory decision making. The OCE’s Patient-Focused Drug Development program will foster collaboration between the FDA, patients, and advocacy groups; nurture research into measurement of the patient experience; and generate science-based recommendations for regulatory policy.² An ongoing initiative at the OCE is an effort to modernize and broaden the eligibility criteria for patients enrolling in cancer clinical trials. The OCE will continue to encourage clinical trial designs that allow enrollment of patients who reflect the real-world population likely to receive a therapy, including older adults.³

Improved patient selection made possible by early biomarker discovery has enabled drug development to shift away from the conventional phase 1, phase 2, and phase 3 paradigm to a more seamless approach, using expansion cohorts to assess efficacy in additional patient populations or to evaluate alternative dosing regimens. The OCE recently published its perspective on seamless development, which could provide earlier access to highly effective therapies and complement the FDA’s expedited regulatory programs, such as Breakthrough Therapy Designation and Accelerated Approval, to get products to patients more efficiently.⁴

In addition, the OCE encourages novel trial designs, such as:

1. Large, simple trials that use easily measurable endpoints to reduce the amount of data needed to determine benefit
2. Common control trials that share a control arm, but may involve multiple drugs for the same indication, thus decreasing the number of patients that need to be enrolled and potentially decreasing development time.⁵

Other OCE programs include Precision Oncology, Immuno-Oncology, Pediatric Oncology, the Information Exchange and Data Transformation Program (INFORMED), Oncology Devices and Diagnostics, Oncology Cell and Gene Therapy, and Oncology Regulatory Affairs.⁶

Striving for Excellence

Another important goal of the OCE is to focus on the “of Excellence” aspect and strive for excellence in drug and device regulation and emerging oncology science. Achieving that goal will require collaboration with academia, industry, patient groups, and professional societies. This work has been initiated by scheduling public meetings and small collaborative workshops to discuss key oncology-related issues across the FDA and bring in perspectives from our external stakeholders. Specific areas of emphasis include:

• Harmonizing cancer-specific regulatory approaches across the FDA’s medical product centers
• Coordinating oncology-specific regulatory science initiatives and outreach
• Implementing cross-center, oncology-focused meetings
• Engaging with external stakeholders, including government agencies, industry, academia, professional societies, and patient advocacy groups
• Communicating with international regulatory agencies regarding oncology issues

These initiatives will allow us to expedite drug development and approval of truly novel agents that will have a major impact on our patients while allowing us to make thoughtful decisions regarding the risk-benefit ratio of oncology drugs.

ABOUT THE AUTHOR

Richard Pazdur, MD, is director of the Oncology Center of Excellence, FDA, and acting director of the FDA’s Office of Hematology and Oncology Products.

Address for Correspondence

Richard Pazdur, MD

Oncology Center of Excellence,

US Food and Drug Administration, WO22 Room 2212, 10903 New Hampshire Avenue, Silver Spring, MD 20993

E-mail: richard.pazdur@fda.hhs.gov

Disclosures: The author reports no financial interests or relationships with the commercial sponsors of any products discussed in this report.

Note: This is a U.S. government work. There are no restrictions on its use.

REFERENCES

1. Pazdur R. Leveraging the power of collaboration—FDA’s New Oncology Center of Excellence. FDA website. https://blogs.fda.gov/fdavoice/index.php/2016/06/leveraging-the-power-of-collaboration-fdas-new-oncology-center-of-excellence/. Published June 29, 2016. Accessed April 6, 2017.

2. Patient-focused drug development. FDA website. https://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/OCE/ucm544143.htm. Updated March 9, 2017. Accessed April 6, 2017.

3. Singh H, Beaver JA, Kim, G, Pazdur R. Enrollment of older adults on oncology trials: an FDA perspective [published online December 22, 2016]. J Geriatr Oncol. doi: 10.1016/j.jgo.2016.11.001.

4. Prowell TM, Theoret MR, Pazdur R. Seamless oncology drug development. N Engl J Med. 2016;374(21):2001-2003. doi: 10.1056/NEJMp1603747.

5. Pazdur R. Evaluating FDA’s approach to cancer clinical trials. FDA website. https://blogs.fda.gov/fdavoice/index.php/2016/09/evaluating-fdas-approach-to-cancer-clinical-trials/. Published September 16, 2016. Accessed April 6, 2017.

6. Oncology Center of Excellence. FDA website. https://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/ucm509057.htm#OCE_Programs. Updated March 9, 2017. Accessed April 6, 2017.

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