The 21st Century Cures: Requirement on Patient Experience Data and Related Information
By Aimee Lee Russell, Associate, Programs
On Friday, November 8, Annie Kennedy, Chief of Policy & Advocacy, EveryLife Foundation for Rare Diseases, and Meghana Chalasani, MHA, Senior Research Analyst, Patient-Focused Drug Development Program, Center for Drug Evaluation and Research (CDER), Food and Drug Administration (FDA), shared their perspectives on the 21st Century Cures requirements on patient experience data. This was the 12th webinar in the NHC’s Clinical Outcome Assessment (COA) series.
Ms. Kennedy gave a brief history of how patients advocated for the Statement of Patient Experience in the 21st Century Cures Act. The Food and Drug Safety and Innovation Act (FDASIA), passed in 2012, strengthened stakeholder engagement in drug development and review. Since then, many patient groups began developing tools and data to help facilitate patient focused drug development, including conducting benefit/risk preference studies, patient groups developing draft guidance on their disease, and sharing testimonies from patients and clinical experts on outcomes and measures that patients truly care about.
Ms. Kennedy described how, in her past work at Parent Project Muscular Dystrophy (PPMD) partnering with researchers and members of the health policy community, they advocated for FDA gathering information on the patient experience data submitted to them. Ms. Kennedy suggested it would be helpful for the FDA to give organizations who submit data for FDA review a better idea of how the data they submit will be used and incorporated into the report. This resulted in the language in the below table being incorporated into 21st Century Cures. Simply put, they wanted to ensure that the tools being created as part of Patient-Focused Drug Development would make the regulatory impact they were intended to.
Patient-Focused Impact Assessment Act of 2016
(Sec. 2) This bill amends the Federal Food, Drug, and Cosmetic Act to require the Food and Drug Administration (FDA), after approving an application for a new medication, to publish a brief statement on any patient experience data or related information that was part of the application. Patient experience data is information about the impact of a medical condition or a related therapy on a patient’s life and the patient’s preferences for treatment.
(Sec. 3) The FDA must issue guidance on the collection of patient experience data and the use of that data in drug development.
(Sec. 4) The Paperwork Reduction Act does not apply to voluntary collection of patient experience data.
(Sec. 5) The FDA must report on its review of patient experience data as part of approving applications for new medications.
114th Congress (2015-2016). S.1597 – Patient-Focused Impact Assessment Act of 2016. Available from: https://www.congress.gov/bill/114th-congress/senate-bill/1597
Ms. Chalasani described implementation of the Statement of Patient Experience to date. Ms. Chalasani echoed sentiment that incorporating the patient voice is needed to provide regulators with insights about what matters to patients in the clinical context. The Statement of Patient Experience seeks to ensure the FDA will describe what patient experience data were submitted and how the data were used in regulatory decision-making. Below is the table used by the FDA to capture the patient experience data submitted for review and where it can be found in the report. This is only available for approved products.
Ms. Kennedy and Ms. Chalasani agree that this is still in early stages of implementation, but the steps the FDA to include the newest Statement of Patient Experience under the 21st Century Cures Act – moves the field in the direction of increasing meaningful patient engagement.