Earlier this week, I joined over 200 people from pharma, biotech, patient advocacy, regulatory agencies and patients at Patients as Partners 2019 in Philadelphia, PA. It was great to hear about how researchers and study sponsors are working to infuse the patient voice into clinical trial design, and to have conversations about where gaps still exist.

In PKD, I know that patients are more than just passive recipients of care: they’re active participants in their kidney disease management. With this in mind, on Monday we talked about the complex decisions that go into an individual’s (like a PKD patient’s) choice to participate in research. Discussions at the conference also made the important point to remind pharma and regulatory attendees that a large part of a patient’s experience with their disease happens outside of the doctor’s office. Because of this, it’s important to urge researchers to measure outcomes like quality of life and health economics.

Mark Scrimshire talking about the development of new tools by the Department of Health and Human Services to help patients access and control their health data.

On Tuesday, we heard from a panel of representatives from the FDA. I was especially excited to hear about their ongoing and evolving support for research measures that look at patient-reported outcomes. Since the FDA ultimately decides whether a study has properly demonstrated that a drug or intervention is effective, they have a large responsibility to instruct investigators in how trial outcomes should be defined. We saw this process in action in 2016 when PKDF worked with the FDA and others to acknowledge total kidney volume as an acceptable clinical endpoint when eGFR didn’t adequately represent the ADPKD patient experience earlier in their disease stage. The field seems to be evolving further to recognize the importance of asking patients about, for example, fatigue or pain in addition to clinical measures like kidney size and function.

Elise with Cynthia Verst (left), President of Design and Delivery Innovation at IQVIA–soon to partner with PKDF on a special project.

As research in the PKD space grows and expands, it’s encouraging to see that pharma and biotech research developers are involving patient perspectives when designing clinical research. As an advocacy community we need to pay attention to these efforts and make sure that patient perspectives are not just being heard, but also applied and addressed in clinical research. To quote a phrase I heard at the NIH during Rare Disease Week, patients aren’t guinea pigs – they’re test pilots! I’m looking forward to helping PKD patients find new ways to participate in research.

Are you interested in advancing PKD research and potential therapies? Check out our current clinical trials and get involved today!