Patient-focused drug development (PFDD) is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation. The primary goal of patient-focused drug development is to better incorporate the patient’s voice in drug development and evaluation
- 2012: The Food and Drug Administration (FDA)Food and Drug Administration’s (FDA) evaluation of new medicines (United States). During clinical trials, researchers study whether new medicines are safe and effective for patients and whether the medicine’s benefits outweigh the risks.
www.fda.gov/ established the PFDD initiative under the Prescription Drug User Fee Act (PDUFA V)2012, the US President signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) to include the reauthorization of the Prescription Drug User Fee Act (PDUFA) that provides FDA with the necessary resources to maintain a predictable and efficient review process for human drug and biologic products.
- 2016: The National Psoriasis FoundationNational Psoriasis Foundation (NPF) drives efforts to cure psoriatic disease and to improve the lives of those affected.
www.psoriasis.org a public meeting to hear perspectives from psoriasis patients, caregivers, and other patient representatives on the most significant dermal or skin-related effects of the disease. Publishes Voice of the Patient report . While attendees were not asked to focus on the arthritic component, a large percentage of comments and concerns included those with Psoriatic Arthritis.
- 2017: The Arthritis Foundation
The Arthritis Foundation boldly pursues a cure for America’s #1 cause of disability while championing the fight against arthritis with life-changing resources, science, advocacy, and community connections. www.arthritis.org hosts the first externally ledExternally-led means it was not commissioned by the FDA so nonprofit organization(s) raise funds to host a meeting offsite. Patient-Focused Drug Development (PFDD) meeting for Osteoarthritis and, as a result, publishes OA Voice of the Patient ReportVisit www.arthritis.org to view videos and more information about this PFDD. You can also download the Report HERE .
- 2017: Lupus and Allied Diseases AssociationThe Lupus and Allied Diseases Association, Inc., is an all-volunteer national non-profit dedicated to enhancing quality of life for those impacted by lupus and allied diseases and other conditions of unmet needs by fostering collaboration among stakeholders, promoting unity in the community, and participating in innovative advocacy, awareness and biomedical research initiatives www.ladainc.org , the Lupus Research AllianceThe Lupus Research Alliance is the largest nongovernmental, nonprofit funder of lupus research worldwide. The organization aims to transform treatment while advancing toward a cure by funding the most innovative lupus research; fostering diverse scientific talent; stimulating collaborations; and driving discovery toward better diagnostics, improved treatments and, ultimately, a cure for lupus. www.lupusresearch.org , and the Lupus Foundation of America unite to host an externally-led PFDD meeting for lupus. Publishes Lupus: Patient Voices reportVisit www.lupuspfdd.org to learn more about this PFDD. The full report can be downloaded HERE .
- 2018 – 2019: FDA develops PFDD guidance materialsInformation and documents related to FDA’s development of the methodological PFDD guidance, including public workshops, draft guidance, and hypothetical scenarios, which are all intended to serve as a basis for dialogue.
View guidance materials at:
www.fda.gov to improve efforts to gain patient input and assessing outcomes in regulatory engagement.
- 2019: FasterCuresFasterCures, a center of the Milken Institute works to build a system that is effective, efficient, and driven by a clear vision: working with our partners to build a patient-centric system where science is accelerated, unnecessary barriers are overcome, and lifesaving and life-enhancing treatments get to those who need them as rapidly as possible.
https://milkeninstitute.org publishes a guide for patient advocacy organizations interested in hosting, or are currently planning, an externally led PFDD meeting. The guide includes links to other tools for planning PFDD meetings, including the PFDD Meeting Tracker, the PFDD Readiness Assessment, and the PFDD Community Toolbox.
CoPI: Patient-Focused Drug Development (PFDD), regulatory reviewers
Tools: Guidance Documents – patient-focused drug development (PFDD); Reports – patient-focused drug development (PFDD)
Context: Regulatory process, clinical trials, Research & Development, guideline development