FDA Prioritizes Patient-Reported Outcomes in Clinical Trials

FDA patient-reported outcomes

Patient-reported outcomes can help optimize clinical trial design and improve study results.

When conducting clinical trials, sponsors and CROs are often focused on the endpoint: the final results that pave the way for bringing a drug or product to market. However, this approach puts sponsors and CROs at risk of overlooking important aspects of the patient experience.

To fill in these gaps, the Food and Drug Administration (FDA) is placing an increased emphasis on patient-reported outcomes (PROs). PROs are the details patients provide about their illness or condition, their experience with treatment, and any side effects they may have. This data offers crucial insights that sponsors and CROs may not be able to access through traditional methods.

PROs can be used to inform trial improvements and guide the drug approval process. They also mark an important step toward patient-centricity in clinical research, which in turn boosts patient enrollment and engagement.

The FDA Focuses on PROs

Many sponsors and CROs are aware of the benefits of PROs, but face challenges when it comes to implementation. For instance, the lack of standardization in data collection can put the validity of results in jeopardy. Unlike more objective measurements, depending on their wording, two surveys about type 2 diabetes symptoms can prompt very different responses from patients. Further, the potential variability of PROs may make researchers less willing to rely on them, which means PROs often receive less attention than other study endpoints.

The FDA discussed these challenges — and ideas for improving PROs programs — during a panel discussion at the Drug Information Association’s annual meeting. From 2013 to 2017, the FDA’s Center for Drug Evaluation and Research (CDER) met with chronic illness patients 24 times to learn about the burden of treatment and the patients’ needs that were going unaddressed. CDER plans to use the feedback gathered during these meetings to make the PROs process more patient-centric and consistent.

The FDA has also launched a pilot program to create publicly available, standardized sets of Clinical Outcome Assessments for specific indications. The National Institutes of Health has already shown its support by offering grants for qualified participants. This initiative, while still in its early stages, is an important step toward incorporating PROs into a wide variety of clinical research.

How Digital Technology Can Help

Digital health tools like smartphone apps will likely be instrumental in expanding the role of PROs. In one study, researchers found that the artificial intelligence-driven app ePAL improved cancer pain management by conveniently collecting information from patients. The app prompts patients to monitor and report their pain symptoms, as well as utilize an accessible library of relevant resources. The app also analyzes patient data to offer patients a customized coaching message each day.

In addition to providing patients with a convenient, user-friendly platform to share information, ePAL has actually helped decrease the severity of cancer pain. The aforementioned study showed that patients' pain, as measured by the Brief Pain Inventory, was reduced significantly over an eight-week period. Further, patients using ePAL averaged just four pain-related hospital admissions over the course of the study, as compared to 20 admissions for those not using the app.

Digital health tools like ePAL have the potential to turn PROs into real, provable results. As PROs become more common in clinical trials, researchers can use the data they collect to monitor patient symptoms more accurately and identify possible adverse events. In the future, physicians may also be able to use PROs to better prepare patients for the experience of undergoing treatment.

While the FDA acknowledges there are challenges that still need to be addressed, the agency continues to highlight the importance of patient-reported outcomes. Fortunately, by incorporating patient feedback and digital health tools, sponsors and CROs can streamline the implementation of PROs programs. Plus, as clinical trials become more personalized, sponsors and CROs will likely see an increase in patient participation and engagement.

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