Instituted as part of the 21st Century Cures Act, the Oncology Center of Excellence (OCE) fosters a unified interaction between three U.S. Food and Drug Administration (FDA) centers: Center for Biologics Evaluation and Research, Center for Drug Evaluation and Research, and Center for Devices and Radiological Health. Amid the COVID-19 pandemic, its role is more vital than ever.
Richard Pazdur, MD
At JADPRO Live 2020, the annual meeting of the Advanced Practitioner Society for Hematology and Oncology held virtually this year, Richard Pazdur, MD, Director of the OCE, discussed recent changes to the oncology drug development and approval process, the importance of advanced practitioners in the execution of clinical trials, and changes that have taken place as a result of COVID-19.1
Changes to Oncology Drug Development
As Dr. Pazdur explained, the OCE’s primary role is in regulatory review, but oncology differs greatly from other therapeutic areas at the FDA. For one, there’s intense public interest in the need to expedite drugs. There’s also a different risk tolerance for side effects in oncology compared with other drugs, and the OCE deals with an active advocacy community. Finally, said Dr. Pazdur, the field is moving into “very innovative indications for drugs,” including biomarker-defined populations.
To keep pace with this innovation, the OCE has piloted multiple programs. One program, the Real-Time Oncology Review, aims to explore a more efficient review process to ensure that safe and effective treatments are available to patients as early as possible.
“In contrast to the usual submission of applications, where a completed application comes to the FDA, we start a dialogue with the sponsor and receive data before an application is actually submitted to the FDA to take earlier actions on oncology products,” said Dr. Pazdur. “This is particularly important because many of these drugs are breakthrough therapies that offer substantial improvements to patients with cancer.”
“Nearly 50% of all Breakthrough Therapy designations by the FDA are for oncology products, so there is urgency to review and approve these drugs in a timely manner.”— Richard Pazdur, MD
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According to Dr. Pazdur, nearly 50% of all Breakthrough Therapy designations by the FDA are for oncology products, so there is urgency to review and approve these drugs in a timely manner. In 2019, 13 approvals were on the basis of the Real-Time Oncology Review.
“We’re not cutting corners,” Dr. Pazdur explained. “We’re simply moving the process up and reviewing drugs before the application is submitted.”
In 2019, the FDA approved a total of 49 oncology indications, including 11 new molecular entities. Bolstered by recent scientific advances that have enabled drug companies to tailor drugs to individual patients, approximately 40% of all pharmaceutical activity in the United States is now in the area of oncology and malignant hematology.
Role of Advanced Practitioners in Oncology
Because of this high degree of activity, the OCE has grown tremendously and employs advanced practitioners in a variety of settings to shoulder the additional workload. The paperwork involved in regulating drugs requires a high degree of expertise, said Dr. Pazdur, and many advanced practitioners are involved in the project management aspect of moving applications through the agency throughout the entire review process.
Advanced practitioners are also employed as data scientists to interrogate applications with regard to drug safety and efficacy as well as special population considerations. “This is an important part of what we do at the FDA, both in the early and later phases of drug development,” said Dr. Pazdur, who noted that data scientists also examine safety issues in the postmarketing setting after drugs are approved.
In addition, advanced practitioners play a major role in the FDA’s outreach program. Project Community at OCE interacts with diverse communities that have not had traditional contacts with the agency, such as small rural areas and inner-city communities, to hear points of view outside the academic setting. This involves soliciting feedback from patient groups about the drug development process as well as earlier access to treatments, especially unapproved drugs.
“We’re particularly interested in bringing underserved and minority groups into the regulatory process at the FDA, and we have a considerable number of advanced practitioners who work with us in outreach,” continued Dr. Pazdur.
Advanced practitioners are also actively involved in global outreach with respect to international drug development. Project Orbis at OCE coordinates and reviews activities with Australia, Brazil, Canada, Singapore, Switzerland, and the United Kingdom to promote international collaboration of drug regulators.
“We’re not trying to establish one uniform opinion on a drug application but rather to have a collaborative process, where we understand potential differences,” said Dr. Pazdur. He noted that Project Orbis has allowed drug companies to submit applications where there may have been a time lag of 2 to 3 years. “This makes subsequent drug development much easier.”
Other OCE Initiatives
Another OCE initiative is Project Facilitate, which aims to provide personalized support to oncologists and health-care teams seeking unapproved drugs for patients with serious and life-threatening diseases.
“The regulatory process can be quite complicated,” Dr. Pazdur observed. “The purpose of this project was to facilitate a somewhat difficult process for people who are not familiar with it and to help patients access investigational drugs.”
Furthermore, Project Patient Voice was instituted to provide an online platform for patients and their health-care providers to look at patient-reported symptom data collected from cancer clinical trials, in addition to the adverse event data normally reported by physicians and nurses. These patient-reported data can give health-care providers additional information to discuss with patients and caregivers.
“We’re particularly interested in bringing underserved and minority groups into the regulatory process at the FDA.”— Richard Pazdur, MD
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“This is our attempt to get the patient voice into product labeling and out to the medical community rather than having a third-party description of their experience,” he said.
Finally, Project Renewal is a public health initiative that aims to update the labeling information for oncology products by evaluating relevant scientific evidence from published literature.
“We spend a lot of time at the FDA on drug labeling for new drugs before a drug comes out,” said Dr. Pazdur. “With this initiative, however, we’re paying special attention to drug labels that may not have been revised for many, many years.”
FDA Oncology and COVID-19
According to Dr. Pazdur, the OCE has made a concerted effort to address the needs of patients with cancer during the COVID-19 pandemic by remaining committed to advancing oncology therapeutic options. This means assessing and approving new drugs in a timely fashion.
Since the start of the pandemic in the United States in early March through the end of August 2020, FDA Oncology has approved 12 new molecular entities. In addition to these new drugs, a total of 33 new indications have been approved, including extensions of preexisting indications and new indications of already approved drugs.
“The FDA has invested much of its resources in addressing the COVID-19 population, and rightly so,” said Dr. Pazdur. “Here at the OCE, however, we needed to focus our attention on oncology patients to make sure they are not forgotten during this pandemic.”
In addition to oncology reviews and approvals, the OCE has been active in writing guidances and supporting efforts to decentralize clinical trials. “We realize patients often cannot go back to major cancer centers, and some of the testing and doctor visits can be conducted in communities nearer to patients’ homes, and we have allowed that,” he said.
FDA Oncology has also made a marked effort to approve longer dosing regimens. Many regimens have been extended to allow patients to visit doctors less frequently to receive drugs, to have these drugs administered at home, or to have intravenous drugs administered subcutaneously.
Finally, said Dr. Pazdur, FDA Oncology has developed research collaborations on real-world evidence to look at patient outcomes during the COVID-19 pandemic. FDA Oncology also has held many listening sessions with patients and practitioners to understand how the pandemic has affected oncology care in the United States.
DISCLOSURE: Dr. Pazdur reported no conflicts of interest.