Patient-Focused Drug Development Meeting Resources

The U.S. Food and Drug Administration (FDA) and patient organizations use patient-focused drug development (PFDD) meetings as a tool to gather patients’ perspectives on living with their conditions and current and future therapies.

In 2013, FDA launched the PFDD initiative with plans to host 20 condition-specific meetings, a goal they have since surpassed. In 2015, after two years of demonstrated success, FDA shared the mantle and gave patient organizations the chance to host PFDD meetings (often called “externally led PFDD meetings”). Since then, patient organizations have successfully amplified the voices of countless patients.

Perhaps you’ve heard about the value of these meetings, or you’re currently planning one. Below is a collection of FDA and FasterCures resources for hosting PFDD meetings based on lessons from the field and specifically designed for those who are interested in hosting externally led PFDD meetings. To learn more about PFDD meetings check out our introductory blog on the topic.

FasterCures Resources

The PFDD Readiness Assessment

How do you know when your organization and your community is ready for a PFDD meeting? This self-guided PFDD Readiness Assessment is designed to help you answer that question by asking you to reflect on the knowledge and capacity of your organization and the context of therapy development for your community. To use the Assessment, answer each question and tally your points as you go. At the end, evaluate your status using the scoring key. We’ll continue to update the Assessment with links to new resources.

PFDD Meetings: Smart Practices from Patient Community Leaders

This extensive guide details common practices, lessons learned, and considerations for planning PFDD meetings. The guide is based on interviews conducted with community leaders that have planned and hosted externally led PFDD meetings, and it has been checked for accuracy by FDA. It is organized chronologically into three planning sections: Section 1: Steps to Take Before Submitting a Letter of Intent, Section 2: Planning Your PFDD Meeting, and Section 3: Preparing for Post-Meeting Outcomes. Each section includes topics relevant to that planning period depending on where you are in your planning.

The PFDD Community Toolbox

Many organizations developed tools and resources for PFDD meetings during their planning process. The PFDD Community Toolbox is a collection of materials that were donated by organizations that have hosted meetings. You’re encouraged to reference or adapt these tools for your PFDD planning purposes.

FasterCures PFDD Tracker

The PFDD Tracker located at the bottom of this page is a collated list of past and future PFDD and PFDD-like meetings, with links to resources such as reports, agendas, and archived recordings. You can also download a PDF version (Updated March 2019).

The Tracker is a work in progress. If you can contribute any additional information, please contact Brenda Huneycutt

Webinar: FDA’s Patient-Focused Drug Development Initiative: What Have We Learned? (June 7, 2018)

Panelists from FDA, the patient community, and FasterCures share their perspectives on the impact of PFDD and how it might continue to shape drug development and regulation. The panelists also discuss their experiences working with patient communities to plan for these often intense listening sessions, what they’ve learned, and how they’re applying this information. 

A Note on These Tools

FasterCures developed these three tools based on internal expertise and interviews with patient advocacy groups that have hosted PFDD meetings. While FDA provided input on the accuracy of the content in the Smart Practices guide, all of these tools represent the perspective of FasterCures alone and do not necessarily reflect the perspectives of the organizations involved or the FDA. These tools do not set standards and will continue to evolve along with patient-focused drug development.

All questions related to externally led PFDD meetings should be directed to the FDA’s PFDD Program Staff.

FDA Resources

It’s important to note that FDA will participate in, but will not officially sponsor or endorse, externally led PFDD meetings. The FDA encourages patient organizations to take the lead and has provided resources to facilitate this. We’ve collated these resources here.

Patient-Focused Drug Development Meeting Tracker 

Meeting
Date of meeting
Host Organization(s)
Meeting Report
Agenda
Recordings 
Idiopathic Thrombocytopenic Purpura (ITP) PFDD 7/26/2019 Platelet Disorder Support Association (PDSA)      
Niemann-Pick Type C PFDD 3/18/2019 Ara Parseghian Medical Research Fund   Agenda  
Major Depressive Disorder PFDD 11/16/2018 Depression and Bipolar Support Alliance     Recordings
Charcot-Marie-Tooth PFDD 9/28/2018 Hereditary Neuropathy Foundation     Recordings
Chemotherapy-induced Hearing Loss in Pediatric Oncology Patient-Focused Drug Development Workshop 9/13/2018 Children's Case for Cancer Advocacy; Children's Brain Tumor Foundation; Mattie Miracle Cancer Foundation; Momcology     Recording 
Alport Syndrome PFDD 8/3/2018 Alport Syndrome Foundation; National Kidney Foundation   Agenda  
Juvenile Idiopathic Arthritis 8/2/2018 Arthritis Foundation; Childhood Arthritis and Rhuematology Research Alliance (CARRA)     Part 1, Part 2
Barth Syndrome PFDD 7/18/2018 Barth Syndrome Foundation Report Agenda Recording 
Chronic Pain PFDD 7/9/2018 FDA      
Sleep Apnea Patient-Focused Medical Product Development (PFMPD) 6/8/2018  American Sleep Apnea Association Report Agenda Recording
Opioid Use Disorder PFDD 4/17/2018  FDA   Agenda Part 1 Part 2Part 3
Pachyonychia Congenita (PC) & Epidermolysis Bullosa (EB) PFDD 4/6/2018 Pachyonychia Congenita Project and debra Report Agenda Recording (PC)
Hypereosinophilic Syndromes PFDD 3/23/2018 American Partnership for Eosinophilic Disorders (APFED)   Agenda Recording
Duchenne Patient-Focused Compass Meeting 3/5/2018 Parent Project Muscular Dystrophy (PPMD) Report     Recording
Hyperhidrosis PFDD 11/13/2017 International Hyperhidrosis Society Report & Agenda   Recording
Lupus PFDD  9/25/2017  Lupus and Allied Diseases Association, Inc. (LADA); Lupus Foundation of America (LFA); Lupus Research Alliance (LRA)  Report & Agenda   Recording
Hereditary Angioedema PFDD  9/25/2017 FDA   Agenda Part 1Part 2
Alopecia areata PFDD  9/11/2017  FDA  Report Agenda Part 1Part 2
C3G PFDD  8/4/2017  National Kidney Foundation  Report  Agenda Recording
Tuberous Sclerosis Complex (TSC) & Lymphangioleiomyomatosis (LAM) PFDD 6/21/2017  Tuberous Sclerosis Alliance  Report Agenda Morning 1Morning 2Afternoon 1Afternoon 2 
Friedreich's Ataxia PFDD 6/2/2017  Friedreich's Ataxia Research Alliance; Muscular Dystrophy Association; National Ataxia Foundation; Cure FA Foundation Report & Agenda    Recording 
Autism PFDD 5/4/2017 FDA Report Agenda Part 1Part 2
Spinal Muscular Atrophy PFDD  4/18/2017  cure SMA Report  Agenda Recording
Sarcopenia PFDD  4/6/2017  FDA Report Agenda  Recording
Osteoarthritis PFDD  3/8/2017  Arthritis Foundation  Report Agenda Recording 
Patients who have received an organ transplant PFDD  9/27/2016  FDA  Report Agenda  Part 1Part 2Part 3Part 4 
Myotonic Dystrophy (DM) PFDD 9/15/2016 Myotonic Dystrophy Foundation  Report & Agenda   Part 1Part 2 
Neuropathic patina associated with peripheral neuropathy PFDD 6/10/2016 FDA Report Agenda  Part 1Part 2Part 3Part 4
Psoriasis PFDD 3/17/2016 FDA Report & Agenda    Part 1Part 2 
Systemic Amyloidosis PFDD 11/16/2015 Amyloidosis Research Consortium (ARC) Report & Agenda   Part 1Part 2
Non-tuberculous mycobacterial lung infections PFDD 10/15/2015 FDA Report & Agenda   Meeting Slides
Alpha-1 Antitrypsin Deficiency PFDD 9/29/2015 FDA Report & Agenda    Transcript
Parkinson's Disease and Huntington's Disease PFDD 9/22/2015 FDA  Report & Agenda    Meeting Slides 
Functional GI Disorders PFDD  5/11/2015 FDA  Report Agenda  Transcript 
Chagas Disease PFDD 4/28/2015 FDA   Report & Agenda     
Breast Cancer PFDD 4/2/2015 FDA Report     
Female Sexual Dysfunction PFDD 10/27/2014 FDA Report     
Idiopathic Pulmonary Fibrosis PFDD  9/26/2014 FDA Report  Agenda  Transcript 
Hemophilia A, Hemophilia B, von Willebrand disease and Other Heritable Bleeding Disorders PFDD 9/22/2014  FDA  Report & Agenda     
Neurological Manifestations of Inborn Errors of Metabolism PFDD  6/10/2014  FDA  Report & Agenda     
Pulmonary Arterial Hypertension PFDD 5/13/2014 FDA  Report  Agenda  Transcript 
Fibromyalgia PFDD 3/26/2014  FDA  Report  Agenda  Transcript  
Sickle Cell Disease PFDD  2/7/2014  FDA Report  Agenda  Transcript  
Narcolepsy PFDD 9/24/2013  FDA  Report Agenda Transcript 
Lung Cancer PFDD  6/28/2013  FDA Report  Agenda Transcript 
Human Immunodeficiency Virus (HIV) PFDD  6/14/2013  FDA  Report  Agenda Transcript 
Chronic Fatigue Syndrome and Myalgic Encephalomyelitis PFDD  4/25/2013  FDA  Report