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  1. 2023 OCE Annual Report

Ongoing Clinical Oncology Projects

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AR 2023 Ongoing Projects 2023

 

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

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Patient-Focused Drug Development Program

The OCE Patient-Focused Drug Development (PFDD) Program continued its mission of incorporating patient experience data, including patient-reported symptoms and function, into oncology drug development and regulatory decision making. Key accomplishments across main areas of work in 2023 included:

  • Engagement and Education – On June 27, 2023, the program held the eighth annual Clinical Outcomes Assessments in Cancer Clinical Trials workshop. This workshop, with nearly 1500 registrants, highlighted how a patient-reported overall side effect impact measure can characterize the totality of side effects experienced by patients. Representatives from the PFDD program also served as speakers or panelists at international conferences, communicating the importance of including the patient voice in oncology drug development. In all these events, the PFDD program engaged directly with patients and patient advocates. 
  • Regulatory and Policy – The PFDD program works closely with the FDA oncology review centers and divisions to provide a consistent and actionable approach to rigorous measurement of patient-reported outcomes. The program interacts directly with sponsors, and over the past few years its workload has increased substantially. As industry sponsors and researchers are facing increasingly complex issues, the OCE PFDD program works collaboratively with review divisions to advise sponsors on best approaches to collect and analyze this important source of information. In 2023, the program has worked on over 100 unique oncology drugs, biologics, and devices.
  • Research and Science – The PFDD program maintains a robust research portfolio, including FDA funded collaborations with Yale-Mayo Clinic, Duke University, and Northwestern University-University of Tasmania. By funding research in this area, OCE can mutually share knowledge and expertise with PRO researchers and find new ways for patient-generated data to contribute meaningfully to cancer therapy evaluation. These collaborations led to three publications in high impact journals and two conference abstracts in 2023. The PFDD program also generated some in-house research with two publications and one conference abstract published this year. 

Oncology Real World Evidence Program 

In 2023, the Oncology Real World Evidence (RWE) Program continued to advance the use of RWE in oncology drug development through involvement in several Oncologic Drugs Advisory Committee meetings, various FDA guidance working groups, and RWE initiatives. The program also established the Oncology Quality, Characterization and Assessment of Real-World Data (QCARD) initiative to facilitate communication between sponsors and FDA Oncology reviewers to improve review efficiency for early real-world data (RWD) study proposals in collaboration with the FDA Foundation. 

The program named a new cohort of FDA oncology experts to the multidisciplinary team that serves as a forum for scientific discussion and evaluation of opportunities for oncology real world data (RWD), known as TEAM FoRWD. The program has focused on practical research goals that could enhance source data, standardize regulatory submissions, and develop real world endpoints.

As part of the OCE Scientific Collaborative, the Oncology RWE Program has nearly 30 ongoing research projects with various collaborators thorough a variety of funded and nonfunded mechanisms. Examples focused on building RWD sources include a Centers of Excellence in Regulatory Science and Innovation (CERSI) collaboration with the University of Maryland focused on older adult and underrepresented racial and ethnic populations, and a research project with the University of Oklahoma focused on the Native American population. 

In pursuit of the development of real-world endpoints, Friends of Cancer Research and several data collaborators evaluated the ability to capture imaging and response endpoints in RWD. A separate collaboration with Cardinal Health is extracting Lugano Criteria from RWD to assess response to cancer treatment. 

An integral component of the Oncology RWE program is education and engagement. The program engages with the scientific community in a variety of forums through publications, presentations at national and international conferences, and by providing educational outreach and training in collaboration with OCE's Project Socrates. Notably, the program led a Global Regulatory Town Hall to discuss RWD at the International Conference on Pharmacoepidemiology and Therapeutic Risk Management alongside FDA Commissioner Califf and panel of international regulators.  

Precision Oncology Program

The Precision Oncology Program is coordinating and integrating efforts across the FDA centers to catalyze regulatory science research and development to apply new methodologies to deliver the promise of precision oncology for new and better drugs, diagnostics, and biologics to reduce the burden of cancer.

Highlights of 2023 included OCE involvement in multiple meetings on the use of biomarkers in oncology drug development, use of circulating tumor DNA in clinical trials and publication of an article on the regulatory implications of ctDNA in immuno-oncology for solid tumors in The Journal of Immunotherapy of Cancer. 

The program took part in 13 notable precision oncology approvals in 2023, including the first gene therapy for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5, and a tissue agnostic approval for patients with solid tumors with BRAF V600E mutation. Precision Oncology also participates in the education program across the centers on omics-driven oncology and biomarkers. 

In June 2023, OCE, in collaboration with Center for Drugs Evaluation and Research (CDER) and Center for Devices and Radiological Health (CDRH), issued a final guidance, Oncology Drug Products Used with Certain In Vitro Diagnostic Tests: Pilot Program, to announce and describe the FDA’s voluntary pilot program for certain CDER regulated oncology drug products used with certain in vitro diagnostic tests. OCE, CDER, and CDRH are continuing their efforts to evaluate eligible applicants under this pilot.

Project Endpoint

Project Endpoint, begun in 2023, aims to enhance the development of endpoints in oncology drug development and foster engagement with the broader community to advance our understanding of and explore potential uses for early, novel endpoints. Considering the complementary nature of early endpoints and late endpoints, such as overall survival, Project Endpoint also aims to advance the use of more established late endpoints. 

In its first year, Project Endpoint facilitated a public workshop between the FDA, the American Association of Cancer Research, and the American Statistical Association on use of overall survival in oncology clinical trials. Workshop slides and recordings are available. 

Additional workshops are planned for 2024 to discuss topics surrounding early endpoints including minimal residual disease and circulating tumor DNA, among others.

Project NextGen

The Oncology Translational Research Laboratory has continued to collaborate with scientists in government, academic centers, and industry on regulatory science research this year. The lab published an article, Trends in the approval of cancer therapies by the FDA in the Twenty-First Century, providing insights into how available cancer drugs have changed in the last two decades.

Ongoing projects are focused on multiple research areas in oncology. The lab is using machine learning to predict response to targeted therapies in acute myeloid leukemia and to predict side effects of immunotherapies in several types of cancer. The lab is also investigating changes in the immune system over time in patients with multiple myeloma or precursors to this disease.

A new project began in 2023 to examine assays that look at certain immune cells, which could be useful for drug development in hematologic malignancies. This project is testing the performance of these assays and developing standards that can be used by the research community to improve the assays in the future.

Project Optimus

In 2023, Project Optimus continued efforts to reform the dosing paradigm in oncology by publishing a Draft Guidance for Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases. 

Project Optimus also posted an Oncology Dosing Tool Kit, which assists stakeholders in evaluating data to identify the dosage that maximizes the benefit-risk profile of a drug. 

Project Optimus collaborated with the Pediatric Oncology Program to address the importance of dosage optimization for pediatric indications by holding an ODAC Pediatric Subcommittee meeting on dosage optimization for pediatric drugs. 

Project Optimus continued efforts to engage key stakeholders regarding strategies for dosage optimization by participating in sessions at ESMO, SITC, and the Friends of Cancer Research Annual Meeting. The project also collaborated with the FDA Office of Clinical Pharmacology and ISOP to plan and execute the FDA-ISOP Workshop Series on Quantitative Methods for Dosage Optimization. Each workshop was attended by approximately 1000 participants. Project Optimus also partnered with ASCO to host the 2nd Annual FDA-ASCO Workshop on Getting the Dosage Right: Optimizing Dosage Selection Strategies in Combination Cancer Therapies, which was attended by approximately 2,000 participants.

Project Pragmatica 

The OCE began Project Pragmatica in 2022 to explore the appropriate use of pragmatic design elements in trials for approved oncology medical products. Bringing together patients, investigators, government agencies, non-profit organizations, and pharmaceutical companies, Project Pragmatica is exploring the feasibility of integrating pragmatic elements into cancer clinical trials. 

Pragmatic trial elements can incorporate broadened eligibility criteria flexibilities in design, and broadened setting selection, potentially easing the burden on patients and clinicians. The degree of flexibility is tailored to the trial context, keeping the safety of patients and the integrity of trial data at the forefront.

Project Pragmatica provided feedback and interaction with the National Cancer Institute to develop the Pragmatica-Lung Study, a randomized phase 3 trial of a two-drug combination in lung cancer. The trial opened in April 2023. The trial encourages streamlined eligibility criteria and safety data collection and specific community recruitment efforts aimed at inclusive populations, while retaining randomization and prioritizing overall survival as the primary endpoint to efficiently address the key clinical question. 

Project Site Selector

Project Site Selector aims to identify trends and rationale for clinical trial site selection across all phases of oncology drug development. In 2023, the Project Site Selector team expanded engagement with a global audience of key stakeholders, facilitated by a series of workshops convened by NYU Division of Medical Ethics. This collaborative effort provided detailed qualitative assessments on the ethical considerations for global oncology drug development and the applicability of data generated to the US population. Site Selector staff additionally conducted a panel discussion at the AACR Annual Meeting and presented quantitative trends in US oncology trial participation at the ASCO Annual Meeting.

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