In January 2022, the Oncology Center of Excellence marked its fifth anniversary by looking back at what we accomplished and looking ahead to further progress toward achieving our vision to create a collaborative scientific environment to advance the development and regulation of oncology products for patients with cancer.
Since its formation in 2017, the OCE has established more than 30 outward-facing programs and projects to educate, inform, conduct research, and collaborate. 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 patients with cancer. This begins with rigorous medical product review for efficacy and safety, but also stepping back to look at what’s happening across the field of oncology and determining how the OCE can leverage its expertise.
This past year, OCE developed additional projects looking at various aspects of cancer drug development:
- Project Optimus – Reforming the dose optimization and dose selection paradigm in oncology
- Project Significant – Statistics in Clinical Trials: Promoting collaboration in design and analysis of cancer clinical trials
- Project Catalyst – Providing a regulatory platform to connect scientific knowledge, creative insight, and medical professionals to foster early-stage product innovation
- Rare Cancers Program – Promoting development of new drug and biological products to treat patients with rare cancers
- Project Protect: Providing consistent review of safety signals across drug classes
Two projects launched in 2020 continued to expand the work to influence cancer drug development in positive ways for the benefit of patients with cancer: Project Equity and the Oncology Real-World Evidence Program.
Regulatory Review Continued Unabated Despite COVID-19
Despite the challenges of the second year of the pandemic, CDER and CBER oncology review teams granted a total of 80 drug approvals in 2021. That includes 15 NMEs, 1 original BLA, 50 supplemental approvals for new indications, 8 supplemental approvals in new populations, and 6 505b2 approvals. Eight approvals included indications for pediatric patients.
CDRH granted marketing approval to 16 in vitro diagnostic devices, including 12 companion diagnostics.
The OCE’s Project Orbis collaborated with international regulators on 26 of the drug approvals. All of these approvals use the OCE’s Assessment Aid, a concise review template that facilitates the FDA assessment of an application. In 2021, Project Orbis added as partners the Israel Ministry of Health Pharmaceutical Administration and the United Kingdom’s Medicines and Healthcare Products Regulatory Agency.
Reevaluating Accelerated Approval
Accelerated approval provides a tradeoff of expediting approvals of drugs with increased uncertainty. Oncology has successfully applied the principles of accelerated approval over the last 29 years, making transformative oncology indications available to patients years earlier.
However, in 2021, the OCE identified certain trials, in particular for drugs targeting programmed death 1 or programmed death ligand 1 for which confirmatory trials did not verify benefit. OCE proactively contacted 10 development programs and through multiple discussions and advisory committee meetings, encouraged companies to withdraw 7 of these. One indication was converted to regular approval with additional data and 2 remain under regulatory review. The percentage of drugs that do not ultimately confirm clinical benefit should not be viewed as a failure of the program but rather an expected tradeoff to expedite drug development of promising agents for severe and life-threatening disease like cancer.
- To increase transparency around oncology Accelerated Approvals, the OCE's Project Confirm developed a database of oncology Accelerated Approvals that are ongoing, confirmed, or withdrawn.
Empowering Patients, Survivors, Advocates and Consumers
In 2021 OCE’s Project Community began the first National Black Family Cancer Awareness Week to increase cancer awareness in one of the most vulnerable segments of the US population. This effort coincided with the 50th anniversary of the National Cancer Act and the signing of Presidential Executive Order 13985, “Advancing Racial Equity and Support for Underserved Communities Through the Federal Government, Section 8.”
In addition, Project Community held eight Conversations on Cancer panel discussions on issues of health equity in racial and ethnic minority populations, challenges faced by cancer survivors, and progress in drug development for treatment of children with cancer.
Resources and Educational Opportunities for Oncology Professionals
The OCE provides educational opportunities and professional resources to oncology professionals about the role of the FDA in oncology product development. These include learning opportunities for oncologists, oncology fellows, new FDA oncology staff members, and high-school students. In addition to formal programs, the OCE offers webinars and publications. In addition, the OCE runs a unique “call center” to assist oncology care teams in filing Expanded Access requests for investigational therapies for patients with cancer who have no other available therapy.
I welcome your involvement and interest in OCE’s work and hope that our Annual Report will inspire you to consider taking part in our programs and projects to improve the lives of patients with cancer.
Richard Pazdur, M.D.
Director, Oncology Center of Excellence