FDA decisions are pivotal for our clients and the people with diabetes they serve. At dQ&A, we’ve recently been involved in helping our clients create rigorous patient preference research packages for inclusion with FDA submissions. In this note, I’d like to give you some insight on why and how we are doing this exciting work.
The FDA first issued guidance for taking patients’ risk/benefit trade-offs into account in medical device premarket approval back in 2012. By 2015, an implantable weight-loss device was approved in large part, it’s believed, because of a strong package of patient preference research. Via clinical data, the device had established a good safety record, but it fell short of the target for efficacy. However, on the non-clinical side, the patients had spoken – they were willing to trade off the risks for the amount of weight loss that could be expected from the technology.
More recently, the FDA has started a number of initiatives to engage patients in the regulatory process, including holding two Public Workshops focused on patient preferences and establishing the Patient Engagement Advisory Committee. And just a couple weeks ago, the Agency released draft guidance on incorporating feedback from patients and caregivers in clinical trial design for medical devices.
Today, the FDA is openly saying that patient preferences are a true decision-making factor in device and drug approvals for people with diabetes. This has the potential to be a game changer. Since the FDA’s role is to make judgment calls in the best interests of public health, an understanding of the benefits patients value most, how much risk they’re willing to tolerate, and whether they would accept the burden associated with the treatment regimen, can in some cases tip the balance of the decision.
Giving patients a clearer voice in key decisions is the reason we started dQ&A. So over the last two years, dQ&A has worked with a number of major clients on patient preference research, which has been included in FDA submissions and published in two peer-reviewed papers. We’ve typically used conjoint analysis in this work, since we feel it offers the best way to quantify risk and reward and to understand the nuance of specific tradeoffs that patients make.
Given the high stakes, we have invested time to ensure that our work has credibility with the FDA. The FDA is still refining its guidance, but we’ve carefully studied their decisions and have taken into account guidance from other organizations such as ISPOR. We also took a close look at our methodologies, in particular the challenges of conceptualizing risk neutrally and realistically (the human brain isn’t good at quantifying future abstract risk), and opportunities for eliminating bias based on the structure and design of survey instruments.
We’re very grateful that our clients have trusted us with an increasingly important pillar of their regulatory efforts. My view is that the patient perspective can only become more relevant to FDA decision making. Going forward, it will be critical for manufacturers to demonstrate that the patient sample is reliable and representative, that the methodology and execution is of the highest quality, that the reports make sense to regulators, and that their chosen research partners have credibility and a track record.
We’re very proud of this work, but it represents only a small fraction of the research that we’ve done during 2019. High-quality patient input is critical at every stage, from understanding unmet needs during the discovery and ideation process, to incorporating feedback during design and testing, to assessing experiences and outcomes after launch. If you have important diabetes questions and you need a trustworthy and speedy collaborator, please don’t hesitate to reach out at email@example.com.