The Food and Drug Administration (FDA) is drafting guidelines related to patient engagement in the clinical trials process and the Pulmonary Hypertension Association (PHA) is weighing in.
Recently, the FDA announced that it will produce a four-part document, “Patient-Focused Drug Development: Collecting Comprehensive and Representative Input; Guidance for Industry, Food and Drug Administration Staff, and Other Stakeholders.” This announcement follows a five-year period during which the FDA focused on soliciting feedback directly from patient-constituents about how the diseases they live with impact their lives and what they would like the FDA to focus on in drug development.
The document will provide guidance to drug manufacturers and others about topics such as how and when to engage patients in the trial design process, deciding which types of data to collect, deciding which clinical endpoints to measure in a trial and identifying effective, unbiased ways to collect data. The FDA has drafted part one of the document and has made it available for public comment. PHA views this as a positive step forward for patient involvement in clinical trials and is currently developing a response to the FDA.
PHA has been engaged in the FDA’s Patient Focused Drug Development Initiative since 2012, when PHA members advocated for pulmonary hypertension (PH) to be chosen from among hundreds of interested disease groups for a meeting with FDA staff. In 2014, PHA’s advocacy paid off and the PH community was selected for one of only 20 patient conversations with the FDA; PHA mobilized more than 100 patients to participate both in person and by phone. The FDA website provides more information about what was shared during that meeting.
PHA continues to advocate for drug development and approval processes that prioritize the patient perspective from the beginning. For more information about PHA’s advocacy initiatives, visit www.PHAssociation.org/advocate or contact gro.noitaicossAHP@ycacovdA.