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FDA
Playing catch-up: FDA wants “patient’s voice” ePRO in your oncology trial
For years now, the FDA has been making one point crystal clear to sponsors and CROs across our industry; they want the patient voice incorporated whenever possible in oncology trials.
The FDA's initiative is driven by the recognition that a patient's personal experience with a disease and its treatment is a unique and essential measure of a medical product's benefit and risk.
- Rationale: The FDA explicitly states that "patients provide a unique perspective on treatment effectiveness" and "some treatment effects are known only to the patient." Outcomes that truly matter to patients, such as functioning, quality of life, and the burden of side effects, are often best measured directly by the patient.
- Mandate: The Patient-Focused Drug Development (PFDD) effort, codified in part by the 21st Century Cures Act, requires the inclusion of such patient experience data in clinical research.
- Guidance series: To formalize this approach, the FDA has released a series of methodological guidance documents (the PFDD Guidance Series) that outline how stakeholders should collect, submit, and use patient input to inform medical product development.
Back to basics: How the FDA's diversity requirements impact clinical trial treatments
The FDA is taking steps to ensure that clinical trials are more representative of the diverse populations they aim to serve. This comes after the industry and the FDA's long-term recognition that clinical trials have often excluded underrepresented groups, leading to health disparities across the United States and the world.
Key Insights from the FDA's DHT Guidance
On December 23, 2023, the FDA released guidance for the industry on how digital health technologies (DHTs) should be used in clinical trials.
DHTs, which the FDA defined in their “Framework for the Use of DHTs in Clinical Trials,” are “technologies such as wearable, implantable, ingestible, and environmental sensors and software applications on mobile phones, among others.”
DHTs can be used to collect data from clinical trial participants more efficiently and objectively than traditional methods. For example, DHTs can collect data on participants' activity levels, sleep patterns, and medication adherence. This data can be collected electronically, which can reduce the burden on participants and improve the accuracy of the data
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Medable perspective: Three points on FDA eCOA guidance
At the beginning of April 2023, the FDA published a draft of the fourth in a series of Guidance Documents entitled “Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments (COA) Into Endpoints For Regulatory Decision-Making”. Once finalized these four documents will be combined and will replace the 2009 Guidance, “Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims”. Part 3, which was released in draft in June 2022, provides advice around the development and validation of new COA (including the modification of existing COA).
Leverage novel options in oncology clinical trial design to reduce burden for patients and sites
Flo Mowlem, Senior Director, eCOA Science & Solutions shares insights on how electronic solutions, with a focus on patient-reported outcome (ePROs) can reduce burden on participants and sites to maximize the chance of success in oncology clinical trials.
J.P. Morgan Week 2023: Trends, highlights, and themes from this year’s conference
See what key trends and takeaways were at the 2023 JPM conference and how they'll drive the landscape of pharma.