Understanding FDA’s Draft Guidance on Cancer Clinical Trial Eligibility Criteria: Performance Status

The FDA’s draft guidance marks a significant step toward more inclusive and relevant cancer clinical trials. Aimed at industry stakeholders, IRBs, and clinical investigators, this document highlights the need to reconsider eligibility criteria to include patients with varying performance statuses (PS).

Performance status (PS) is crucial in oncology trials, assessing how patients perform daily activities. Historically, trials have excluded patients with lower PS, limiting the generalizability of results. The FDA’s draft guidance aims to make eligibility criteria more inclusive, reflecting real-world populations.

Key Points

Expanding Eligibility Criteria: Patients with lower PS should be included in trials unless there are strong clinical or scientific reasons for exclusion. This will improve trial diversity and representativeness, enhancing the validity of results.

Benefits and Risks:

• Benefits: Including lower PS patients can speed up trial enrollment, better reflect patient diversity, and provide comprehensive safety data for post-approval treatment decisions.
• Risks: There may be more adverse events in lower PS patients, impacting their ability to complete treatment and affecting overall outcomes. Sponsors should design trials to address these challenges.

Recommendations for Inclusion: The FDA recommends including patients with ECOG PS2 (or KPS 60-70) unless there is a justified rationale for exclusion. This inclusion should be re-evaluated throughout drug development.

Alternative Trial Designs: For trials where including lower PS patients is concerning, the FDA suggests using pre-specified cohorts that include these patients but exempt them from primary analysis, allowing for safety data collection without compromising primary outcomes.

Accurate Characterization of Functional Status: Beyond traditional PS measures, patient-reported outcomes and wearable devices should be used to better understand patients’ functional status, especially for older adults.

Implications for Stakeholders

Sponsors and CROs: Adopt more inclusive trial designs reflecting real-world populations. Early engagement with the FDA can help navigate regulatory requirements and address challenges.

Investigators and Study Sites: Ensure trial designs are inclusive and provide adequate support for all participants. Training on new assessment tools and open communication with participants are crucial.

Participants: Broader eligibility criteria will allow more patients to participate in clinical trials, providing access to new treatments and contributing to more generalizable results.

Implementing Inclusive Trial Designs

• Inclusive Eligibility Criteria: Develop criteria that reflect the intended patient population and re-evaluate them throughout the trial.
• Robust Monitoring and Support: Implement comprehensive plans to manage adverse events and support participants, especially those with lower PS.
• Patient-Centric Approaches: Use patient-reported outcomes and wearable devices for detailed functional status data, ensuring they are easy to use and informative.
• Proactive Regulatory Engagement: Engage with the FDA early and throughout the trial process to align expectations and address regulatory concerns, facilitating successful implementation of inclusive designs.

Conclusion

The FDA’s draft guidance on performance status eligibility criteria for cancer clinical trials is a pivotal move toward greater inclusivity and representation in clinical research. By broadening eligibility criteria and adopting innovative trial designs, stakeholders can improve the diversity and relevance of trial outcomes, ultimately advancing oncology research and treatment options for all patients.

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