Six key takeaways from the FDA’s 10th Annual COA in Cancer Clinical Trials Workshop

1. PROs are now essential, not optional

Cultural shift

Over the past decade, there has been a significant transformation in how PROs are viewed. Once viewed as an “after thought,” PROs are now recognized as a necessary component of oncology clinical trials and drug development programs.

Regulatory

The FDA has finalized its guidance on core PROs in oncology trials. Panelists emphasized that PROs should not be relegated to exploratory endpoints. Instead, they should be integral to regulatory decision-making and can support both efficacy and safety objectives. We’re seeing more examples of PROs being included on labels (for efficacy, tolerability, or preference). In the past 5 years, 11 new oncology drug approvals have included PRO data in product labeling.

2. The importance of PROs in early phase trials

Early phase focus and dose optimization

Multiple panelists highlighted the growing readiness and need to incorporate PROs in early phase trials. These trials are crucial for dose selection and understanding tolerability, and patient input at this stage can guide better decision-making.

Expert consensus

One panelist highlighted results of an expert roundtable that found strong endorsement for using FDA core concept PROs in early phase trials, signaling a shift toward more patient-centered early development.

3. Measuring tolerability from the patient perspective

Beyond safety

Tolerability is not just about clinician-reported adverse events or safety data. It is a multidimensional concept that must include patient-reported symptoms and experiences.

Labeling and decision-making

Recent examples show tolerability data, including PROs, being incorporated into product labels. This helps differentiate drugs with similar efficacy but different patient experiences.

Tools and measures

Instruments like PRO-CTCAE and FACT-GP5 are increasingly used to capture the overall impact of symptomatic adverse events from the patient’s viewpoint.

4. Effective communication and data visualization

Audience matters

The way PRO data is visualized and communicated should be tailored to its audience, clinicians, patients, or regulators. Clear, actionable graphics and understandable labels are essential.

5. Reducing burden and increasing relevance

Not just about length

The burden of PROs is less about the number of questions and more about their relevance and clarity. It’s more about making sure concepts matter, and not that the assessment is “short.”

Electronic capture

Digital tools that leverage reminders are becoming more frequent. We are moving to a world where much of PRO collection will be electronic. Leveraging ePRO can reduce burden by allowing patients to complete PROs at home before clinic visits. Digital tools can help in keeping the high standard for data quality.

Patient engagement

Patients are more willing to complete PROs when they understand their purpose. Providing training to patients that includes why PROs are important and what they are being used for can enhance their relevance and patient motivation.

6. Looking ahead: The future of PROs in oncology trials

Routine use and harmonization

Panelists hope for a future where PROs are a routine part of cancer clinical trial reporting, with international harmonization and standardized, yet flexible, analysis and visualization methods.

Early FDA engagement

Sponsors are encouraged to engage with the FDA early to develop a PRO strategy aligned with guidance, ensuring measures are meaningful and fit for purpose, especially if planning to use PROs in their product label.

Patient-centered outcomes

The ultimate goal is to measure what truly matters to patients, supporting both regulatory decisions and shared decision-making in clinical care.