Amid the ongoing COVID-19 public health emergency, the US Food and Drug Administration (FDA) has emphasised the importance of caring for patients with established conditions – particularly for those requiring chronic medical care and/ or with weakened immune systems, such as cancer patients. In a July 2020 message to healthcare providers and patients with cancer, the FDA’s Oncology Center of Excellence (OCE) stated that it recognises that cancer patients represent a vulnerable population at risk of contracting COVID-19.1 The OCE acknowledged that although the nation’s emphasis is on the need to combat SARS-CoV-2, the virus that causes COVID-19, “patients with cancer and their unique needs continue to be a top priority.”
Authorised by the 21st Century Cures Act of 2016 and established in January 2017, the OCE collaborates with the three FDA product centres – the Center for Drug Evaluation and Research (CDER), the Center for Biologics Evaluation and Research (CBER), and the Center for Devices and Radiological Health (CDRH) – in an integrated regulatory approach to enhance cross-centre coordination for the clinical review of oncology products.
The OCE’s director, Richard Pazdur, MD, commented in February 2022 on the fifth anniversary of the centre that the OCE looks forward to continued progress toward achieving its “vision to create a collaborative scientific environment to advance the development and regulation of oncology products for patients with cancer.”2 Since its inception in 2017, the OCE has created more than 30 externally facing programs and projects to educate, inform, conduct research, and collaborate, Pazdur noted. He said the OCE “is committed to driving change in cancer drug development that results in more efficient and accessible clinical trials, more effective and safer medical products, and better outcomes” for cancer patients.
In 2021, the OCE developed additional research and development projects addressing various aspects of cancer drug development, as summarized in the 2021 OCE Annual Report. 3 Among these were Project Optimus, an initiative to reform the dose optimisation and dose selection paradigm in oncology drug development, and the Rare Cancers Program, which works in conjunction with CDER’s Office of Oncologic Diseases (OOD) to promote the development of safe and effective new drugs and biologics to treat patients with rare cancers.
Pazdur noted that, despite the challenges of the second year of the COVID-19 public health emergency, the CDER and CBER oncology review teams granted a total of 80 drug approvals in 2021. This included 15 new molecular entities (NMEs), one original biologics license application (BLA), 50 supplemental approvals for new indications, eight supplemental approvals in new populations, and six 505(b)(2) approvals. Eight approvals included indications for pediatric patients. The CDRH, also collaborating with the OCE, authorized 16 oncology-related in vitro diagnostic devices, including 12 companion diagnostics.
Facilitating Regulatory Reviews
Other endeavours in the OCE aim to facilitate the regulatory review process, potentially allowing patients to receive earlier access to medical products. Among these initiatives is Project Orbis, which the OCE began in May 2019 to provide a framework for concurrent submission and review of oncology products among international regulators. Another is the Real-Time Oncology Review (RTOR) program, which the OCE, in collaboration with the OOD, commenced in February 2018.
Through participation in the voluntary RTOR program, applicants can submit components of individual modules (e.g., portions of the clinical module) at separate times. This differs from the existing mechanisms for rolling review in which, generally, complete modules (e.g., the complete clinical module) are submitted prior to complete application submission. According to the FDA, by providing the agency with earlier access to key safety and efficacy data, it can identify data quality and potential review issues and provide feedback to the applicant, which can permit a more streamlined and efficient review process. In July 2022, the FDA published the draft guidance for the industry, Real-Time Oncology Review (RTOR), to provide recommendations to applicants on the process for submission of selected new drug applications (NDAs) and BLAs with oncology indications for review under the RTOR program.4