Ongoing Clinical Oncology Projects 2024

The Oncology Center of Excellence supports clinical projects to improve oncology product development for the benefit of people with cancer.  

Jump to:

• Patient-Focused Drug Development Program
• Cancer in Older Adults Program
• Oncology Real-World Evidence Program  
• Precision Oncology Program

Patient-Focused Drug Development Program

OCE's Patient Focused Drug Development (PFDD) Program advanced its mission of incorporating patient experience data, including patient-reported symptoms and function into oncology drug development and regulatory decision making.  

Highlights of 2024 include multiple publications on patient-reported outcomes (PRO) in oncology drug development, including manuscripts based on in-house research as well as funded external research through the OCE Scientific Collaborative. Among many OCE PFDD specific projects is the In4M project, a Centers of Excellence in Regulatory Science and Innovation (CERSI) collaboration with Yale-Mayo. This collaboration has the goal of characterizing the longitudinal measurement characteristics of physical function assessments, including clinician-reported outcome, patient-reported outcome, performance outcome tests and wearable data.

The PFDD Program hosted the Ninth Annual Clinical Outcomes Assessments in Cancer Clinical Trials Workshop, which focused on selection of appropriate symptoms and side effects relevant to tolerability assessment of anti-cancer therapies. The program has sought to provide clarity and practical advice on measurement of side effects from anti-cancer therapy. To that end, assessment of key symptoms is feasible in all stages of drug development, including incorporation of PROs in early phase drug development. The PFDD Program participated in an FDA-AACR public workshop on this topic in early 2024.  

In October 2024, the OCE-led Core Patient-Reported Outcomes in Cancer Clinical Trials Guidance was finalized, which provides actionable advice on selecting PROs in cancer clinical trials. Finalization of this guidance is a key milestone for the OCE PFDD program, as it is a distillation of many publications and years of regulatory work.  

Cancer in Older Adults Program

The Cancer in Older Adults Program, begun in September 2024, is a focal point for enhancing engagement across FDA’s oncology divisions with external stakeholders to improve outcomes for older adults with cancer. The program also liaisons with other OCE programs and projects to coordinate science and policy related to cancer in older adults.  

The program engaged with internal and external stakeholders to address topics pertinent to improving outcomes for older adults with cancer. Since its launch, OCE Cancer in Older Adults Program:

• Initiated a research project to characterize the information available to inform safety and efficacy of oncology products in the product label.
• Participated in the inaugural International Society of Geriatric Oncology SIOG Patient Advocacy Resources and Communities (SPARC) Roundtable at the annual meeting.
• Engaged with external stakeholders in a workshop addressing topics of modernizing trial design to Improve the evidence base for older adults.

Oncology Real-World Evidence Program  

Established in 2020, the Oncology RWE Program aims to advance the appropriate use of real-world evidence in oncology product development to facilitate patient-centered regulatory decision-making. The program operates across three key areas: regulatory review, regulatory science research, and education and engagement. As our core mission, regulatory review has achieved significant progress, with over 200 completed reviews spanning drugs, biologics, and devices. The program maintains active engagement with the scientific community through publications, presentations at national and international conferences, and educational initiatives.

Strategic Research Accomplishments: In 2024, the program completed four major strategic research initiatives:

• Foster Consistent Terminology through the Modernizing Research and Evidence (MoRE) Glossary: An FDA-NIH Collaboration.  
• Enhance Source Data through cross-agency efforts to support USDCI+ and EHR data standardization (publication, ICARE).
• Facilitate Standardized Submissions through the The Oncology QCARD Initiative: Fostering efficient evaluation of initial real-world data proposals.
• Develop Real World Endpoints through collaborative efforts with Friends of Cancer Research on Evaluation of Real-World Tumor Response Derived From Electronic Health Record Data Sources: A Feasibility Analysis in Patients With Metastatic Non-Small Cell Lung Cancer Treated With Chemotherapy.

Evidence Modernization and Pragmatic Approaches: To facilitate opportunities to enhance efficiency in medical product development as an integral component of patient-centric evidence generation, OCE's Project Pragmatica expanded to include broader evidence generation efforts across HHS, interaction through public-private partnerships, and the crowdsourcing initiative Project 5 in 5 to gather clinically meaningful ideas for pragmatic trials.  

In 2024, the OCE launched an oncology-specific AI initiative with three primary objectives:

• Advancing understanding of AI applications in oncology drug development
• Providing training on current AI methodologies
• Coordinating regulatory review and guidance for AI applications

Precision Oncology

The Precision Oncology Program continues to coordinate and integrate efforts across the FDA Centers to deliver the promise of precision oncology for new and better drugs, diagnostics, and biologics to reduce the burden of cancer.  

In 2024, the program worked on FDA publication of the final guidance on Circulating Tumor DNA for Curative-Intent Solid Tumor Drug Development, a landmark document that standardizes the use of this biomarker. Our team participated in numerous high-impact discussions on biomarker utilization in oncology drug development, both within the FDA and with external stakeholders.  

The year saw 32 notable precision oncology therapeutic approvals, including the first tumor-agnostic approval of an antibody-drug conjugate for patients whose tumors overexpress HER2 with a 3+ score by immunohistochemistry.  

In 2024, OCE, in collaboration with Center for Drugs Evaluation and Research (CDER) and Center for Devices and Radiological Health (CDRH), reviewed the initial experience with the Oncology Drug Products Used with Certain In Vitro Diagnostic Tests: Pilot Program. Several challenges were identified, and the pilot was extended with plans to communicate this experience and learnings in the coming year.

Looking ahead, the Precision Oncology Program plans to expand on development of biomarker-based clinical trial designs, including advancing policy on the use of minimal performance characteristics for clinical trials intended to treat a serious or life-threatening condition for which no satisfactory alternative treatment exists.