Written Comment Generation Patient

Submitted to the U.S. House of Representatives Committee on Energy and Commerce

March 17, 2022

The Future of Medicine: Encouraging Innovation while Improving Oversight

Generation Patient represents young adults with chronic and rare conditions across the United States. We are the only organization exclusively supporting the transition of this demographic cohort with lifelong health conditions into adulthood. Our organization does not accept funding from the pharmaceutical, insurance, hospital, or related healthcare industries. We appreciate this opportunity to address you about critical issues affecting young adult patients.

Generation Patient was created—and is led—entirely by young adult patients. Our work has touched regions across the United States from South Texas to Indiana to California.

Through our nonpartisan policy outreach and networking initiatives—the Health Policy Lab and the Crohn’s and Colitis Young Adults Network—we work to increase the health literacy, patient knowledge, skill, and confidence for self-management, and the public policy advocacy skills of young adult patients. We have facilitated more than 200 virtual meetings during the past two years and programming related to navigating the higher education landscape, peer support, and educating others about health policy with independent research and analysis.

As an organization, Generation Patient is concerned about the pharmaceutical industry’s failure to include significant numbers of adolescents and young adults with chronic and rare conditions in clinical trials. We support efforts to diversify clinical trials by explicitly including these patients. We know there are substantial differences among pediatric, adolescent, and adult patient populations in terms of drug disposition and response (Kapogiannis & Mattison, 2008). We also believe the inclusion of adolescents and young adults should be done in a racially diverse and inclusive manner.

Because adolescence and young adulthood are such distinct developmental periods, we believe post-market reported data from clinical trials should be stratified across narrower age ranges. For example, the U.S. Food and Drug Administration’s Adverse Event Reporting System reports 18 years old to 64 as a single demographic, and this does not allow for the understanding of how a drug products affect young adults versus older adults. More relevant insights regarding young adults could be gleaned from narrower reporting of adverse events involving 18- to 26-year-olds. Such an approach would ensure a focus on developmental differences. An 18-year-old body is physically different from that of a 60-year-old. One such approach could involve the FDA developing clinical trial enrollment targets that adequately represent the demographic spread of patient populations experiencing specific chronic conditions. To ensure enrollment of patients who are Black and brown, the FDA should require drug companies to meet more stringent enrollment criteria. Generation Patient believes such a requirement could expedite the enrollment of these underrepresented groups rather than simply relying on the drug trial sponsors.

The FDA also should ensure the prompt public release of post-approval clinical drug trial data. Patients need and deserve this information. Generation Patient supports the agency’s authority to quickly withdraw an accelerated approval drug to prevent patients from being harmed.

Our organization supports the concept of a Rare Disease and Drug Advisory Committee. Many of us at Generation Patient live with rare diseases. We cannot overstate the essential role of patient voices in these conversations. We recommend that members of such advisory groups maintain the integrity of the advisory process by disclosing potential and actual conflicts of interest, including any payments from the pharmaceutical industry. Independent patient leaders from an array of age and racial or ethnic demographic groups should serve in such advisory capacities to ensure patients’ interests are fairly represented. Most of the approximately 7,000 to 10,000 people with rare diseases are disproportionately children, adolescents, and young adults (Tisdale et al., 2021). Thus, we believe the voices of young adults will be critical to any advisory committees.

For young adults living with medical conditions that could potentially last a lifetime, developing new therapeutic options is extremely important. Many of us have been diagnosed with rare and complex medical conditions for which treatment options are limited. Our lives and quality of life depend on real innovation and the prompt development of alternative treatments. Although we need new therapeutics, we also need to ensure the integrity of the clinical trial and post-market processes. We hope that today’s discussions will prioritize patient safety and engagement in testing and ensuring the safety of new drugs.

Thank you for your time and to all parties for contributing perspectives. We applaud the work of the House Committee on Energy and Commerce in facilitating discussions that directly benefit patients. As the emerging generation of patient advocates, we hope our perspectives will be strongly considered, and we would be pleased to elaborate on our prepared testimony.