On November 17, Friends of Cancer Research (FOCR) released a white paper report, Enhancing Use of Patient-Centered Data in Regulatory Decision Making.1 The contents of that paper are summarized below.
Improving Patient Input
Many stakeholders agree that to ensure truly transformational therapies, patients must play a major role. They are uniquely equipped to identify critical gaps and unmet needs, can help set priorities, aid in the design of clinical trials that measure outcomes that matter, and define benefits that are clinically meaningful as well as risks that may or may not be acceptable.
To expand and improve patient input, new mechanisms are needed, said FOCR. They must go beyond the scope of traditional clinical trials that may not fully reflect the experience of “real-world” patients. Such data collection should involve patient registries, electronic medical records, information about relative benefits and risks, important patient-centered outcomes, patient diversity, outreach to patients who have been adversely affected by a treatment, education, standardization of patient-reported data, and publishing negative trials.
Because only about 3% of adult cancer patients participate in clinical trials, much of what is known about a drug under investigation may be inaccurate and incomplete. Thus, approval should not be the end of testing but rather, said FOCR, the first step in gathering data about patient outcomes in clinical practice, thus creating an opportunity for enhanced safety surveillance, particularly as it pertains to off-label use.
The white paper acknowledges the challenges of drug development and suggests corrective action:
- Increase trial participation, collect data from patients who do not meet eligibility criteria, minimize exclusion criteria, and enroll patient populations with unique genetic subsets.
- Improve the research infrastructure by encouraging institutional collaboration, reducing the number of uninterpretable safety reports in [Investigational New Drug applications], and expand the use of central institutional review boards to minimize trial delays.
- Randomize as much as possible and use observational data to identify efficacy in small subpopulations with a particular genetic characteristic. ■