The U.S. Food and Drug Administration’s (FDA’s) Oncology Center of Excellence (OCE) Patient-Focused Drug Development Program fosters collaboration between FDA Centers and external stakeholders involved in patient outcomes research in cancer populations.
The OCE’s overarching goal is to identify rigorous methods to assess the patient experience that will complement existing survival and tumor information, to better inform a cancer therapy’s effect on a patient.
However, patient-reported outcome (PRO) assessments should be held to the same standards as other clinical trial measures, says Paul G. Kluetz, MD, the FDA Acting Associate Director of Patient Outcomes, OCE. Trials should have a clear statement of objectives, distinguish the effects of the drug from other influences, and contain well-defined and reliable assessments.
When determining the objective of a PRO, it’s important to be clear, Kluetz stresses. For example, is the broad objective to:
- Describe the patient experience regarding the treatment?
- Inform safety and tolerability assessments?
- Inform efficacy assessments?
Next, identify the U.S. regulatory goal for the PRO data. Is the core goal to:
- Collect supportive data for an overall risk/benefit assessment?
- Illuminate the patient experience data on the product label?
- Make a claim of treatment benefit on the product label?
FDA expects to see a strong combination of static questionnaires and item banks or libraries to create a balanced, flexible, and modular approach to PRO assessments that address targeted trial objectives and accommodate the needs of multiple stakeholders.
Turning to trial design and conduct, Kluetz offered several suggestions:
- Consider assessment frequency. For example, researchers might want to run assessments more frequently in the early part of a trial to capture acute drug effects.
- Train and inform site staff and patients on the importance of PRO results to the trial.
- Have a plan in place to monitor PRO completion to minimize missing data.
Author: Michael Causey