Today, the Biotechnology Innovation Organization (BIO) and Parent Project Muscular Dystrophy (PPMD) released a new report, “Key Considerations for Developing and Integrating Patient Perspectives in Drug Development: Examination of the Duchenne Case Study”.
The report draws upon the pioneering work of PPMD and the Duchenne muscular dystrophy community in launching a milestone patient preference study and a community-led draft Guidance for integrating patient and caregiver perspectives into drug development and regulatory decision making. It outlines key considerations and decision-making processes for obtaining data on patient perspectives, and more specifically on patients’ stated preferences, that can be integrated into the drug development process.
“Patients today play an increasingly important and proactive role in developing drugs that will best meet their needs. This report provides much-needed guidance for companies seeking to incorporate the patient perspective at every stage of developing and delivering innovative new treatments.”
- Jim Greenwood, BIO President and CEO
“Every patient has a story and every story has incredible value. Translating these perspectives into rigorous data is a way to give voice to perspectives and integrate what matters most to patients into the drug development process."
- Pat Furlong, PPMD Founding President and CEO
The report was guided by an Expert Review Committee (ERC) of thought leaders representing the patient community, academia, and the biopharmaceutical industry.
Members of the ERC include: Marc Boutin, JD, Chief Executive Officer, National Health Council; John Bridges, PhD, Associate Professor, Johns Hopkins University; Paul Hastings, President and CEO, OncoMed Pharmaceuticals, Inc.; Stacy Holdsworth, PharmD, Senior Advisor, US Regulatory Policy and Strategy, Eli Lilly and Company; Reed Johnson, PhD, Senior Research Scholar, Duke University; Bennett Levitan, MD, PhD, Senior Director, Benefit-Risk Assessment, Janssen Research & Development, LLC, Johnson & Johnson; Kimberly McCleary, Managing Director, FasterCures; Bray Patrick-Lake, Director of Stakeholder Engagement, Clinical Trials Transformation Initiative; and Holly Peay, PhD, Research Analyst, RTI International; Co-PI, DuchenneConnect Registry (a program of PPMD).
“This project would not have been possible without the generous assistance of these distinguished experts. We greatly appreciate their time, enthusiasm, and passionate commitment to the science of patient input,” said BIO’s Jim Greenwood and PPMD’s Pat Furlong.
The project was led by a team of BIO and PPMD staff, working with WSCollaborative:
A panel discussion of the report’s findings featuring members of the ERC, BIO, and PPMD will be held later today at the BIO International Convention, from 3:30-4:30 pm PT in room West 3000 of the Moscone Center.