Condition-Specific Meeting Reports and Other Information Related to Patients' Experience

This webpage provides links to certain publicly available external reports and resources relating to patient experience data. The patient community, patient advocates, researchers, drug developers, and federal agencies may find these materials useful. For specific questions related to a report or resource, FDA recommends reaching out to the point of contact listed on the cover page of the report.

This webpage hosts an alphabetical listing of condition-specific meeting reports and other information related to patients’ experience. These meetings include FDA-led Patient-Focused Drug Development (PFDD) meetings, Externally-led PFDD meetings, and Patient Listening Sessions. Other information includes proposed draft guidance relating to patient experience data, natural history studies, or other condition-specific background on condition and discussion of unmet medical need.

FDA’s Office of Patient Affairs is responsible for managing the Agency’s Patient Listening Session program. While conditions for which there were Patient Listening Sessions conducted are listed individually on this page, the link will direct you to the Patient Listening Session page where you will be able to see all the Patient Listening Session Summaries posted by the Office of Patient Affairs.

For more information regarding what types of resources may be included on this webpage, how to submit a publicly available website link to FDA, and other general questions, please review our Frequently Asked Questions. For EL-PFDD meeting summary reports, we request that links include a cover page or similar opening statement as part of their report or resource to provide information about the authors, funding, and related information.

Please note that although FDA reviews the materials that link to external pages before posting them to ensure that the materials are within the scope of the webpage, FDA does not assess their scientific merit or compliance with regulatory requirements. Our decision to post links to these materials does not reflect an endorsement of their authors, sponsors, or content.

Disease or Condition (alphabetical)	Type of Meeting	Resource(s)	Meeting Date
Acromegaly	EL-PFDD Meeting Host: Acromegaly Community, Inc.	Meeting Report	January 21, 2021
Acute Porphyrias	EL-PFDD Meeting Host: American Porphyria Foundation	Meeting Report	March 1, 2017
Adrenomyeloneuropathy (AMN)	Patient Listening Session	Patient Listening Session Summary	May 7, 2021
Adult Dermatomyositis	Patient Listening Session	Patient Listening Session Summary	April 26, 2022
Adult Polyglucosan Body Disease	Patient Listening Session	Patient Listening Session Summary	October 28, 2021
Alopecia Areata	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	September 11, 2017
Alpha-1 Antitrypsin	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	September 29, 2015
Alport Syndrome	EL-PFDD Meeting Host: National Kidney Foundation and the Alport Syndrome Foundation	Meeting Report	August 3, 2018
Amyloidosis	EL-PFDD Meeting Host: The Amyloidosis Research Consortium	Meeting Report	November 16, 2015
Amyotrophic Lateral Sclerosis (ALS)	N/A	IMPACT ALS Survey Report	N/A
Autism	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	May 4, 2017
Barth Syndrome	EL-PFDD Meeting Host: The Barth Syndrome Foundation	Meeting Report	July 18, 2018
Biliary Atresia, Progressive Familial Intrahepatic Cholestasis, Wilson’s Disease	Patient Listening Session	Patient Listening Session Summary	November 5, 2018
Breast Cancer	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	April 2, 2015
Celiac Disease	Patient Listening Session	Patient Listening Session Summary	February 20, 2019
Cerebral Cavernous Malformation (CCM)	Patient Listening Session	Patient Listening Session Summary	November 6, 2019
Cerebrotendinous Xanthomatosis (CTX)	EL-PFDD Meeting Host: United Leukodystrophy Foundation of America	Meeting Report	September 14, 2021
Chagas Disease	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2, Part 3, Part 4) Transcript Summary Report	April 28, 2015
Charcot-Marie-Tooth and Inherited Neuropathies	EL-PFDD Meeting Host: Hereditary Neuropathy Foundation	Meeting Report	September 28, 2018
Chemotherapy-Induced Hearing Loss	EL-PFDD Meeting Host: Children’s Cause for Cancer Advocacy, Children’s Brain Tumor Foundation, Mattie Miracle Cancer Foundation, and Momcology	Meeting Report	September 13, 2018
Childhood Cerebral Adrenal Leukodystrophy (CCALD)	Patient Listening Session	Patient Listening Session Summary	November 13, 2019
Chronic Fatigue Syndrome/Myalgic Encephalomyelitis	FDA-led PFDD Meeting	Agenda Slides (Part 1, Part 2) Recordings (Part 1, Part 2, Part 3, Part 4, Part 5, Part 6, Part 7, Part 8) Transcript Summary Report	April 25, 2013
Chronic Pain	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	July 9, 2018
Clear Cell Sarcoma	Patient Listening Session	Summary unavailable	September 10, 2020
Complement 3 Glomerulopathy (C3G)	EL-PFDD Meeting Host: National Kidney Foundation	Meeting Report	August 4, 2017
Cystic Fibrosis – nonsense mutations	Patient Listening Session	Patient Listening Session Summary	July 15, 2021
Dermatitis (Eczema)	EL-PFDD Meeting Host: Allergy and Asthma Network, Asthma and Allergy Foundation of America, Global Parents for Eczema Research, International Topical Steroid Awareness Network, and National Eczema Association	Meeting Report	September 23, 2019
Dravet Syndrome	EL-PFDD Meeting Host: The Dravet Syndrome Foundation	Meeting Report	February 3, 2022
Fabry Disease	Patient Listening Session	Patient Listening Session Summary	December 4, 2018
Facioscapulohumeral Muscular Dystrophy (FSHD)	EL-PFDD Meeting Host: FSHD Society	Meeting Report	June 29, 2020
Female Sexual Dysfunction	FDA-led PFDD Meeting	Agenda (Day 1, Day 2) Slides (Day 1, Day 2) Recordings (Part 1, Part 2) Transcript (Day 1, Day 2) Summary Report	October 27, 2014
Fibrodysplasia Ossificans Progressiva (FOP)	Patient Listening Session	Patient Listening Session Summary	May 29, 2019
Fibromyalgia	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	March 26, 2014
Focal Segmental Glomerulosclerosis	EL-PFDD Meeting Host: National Kidney Foundation and NephCure Kidney International	Meeting Report	August 28, 2020
Fragile X Syndrome	EL-PFDD Meeting Host: National Fragile X Foundation	Meeting Report	March 3, 2021
Friedreich’s Ataxia	EL-PFDD Meeting Host: Friedreich’s Ataxia Research Alliance	Meeting Report	June 2, 2017
Frontotemporal Degeneration (FTD)	EL-PFDD Meeting Host: Association for Frontotemporal Degeneration (AFTD)	Meeting Report	March 5, 2021
Functional Gastrointestinal Disorders	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	May 11, 2015
Gastroparesis	Patient Listening Session	Patient Listening Session Summary	December 2, 2019
Gene Therapy as a Treatment Modality for Hemophilia	Patient Listening Session	Patient Listening Session Summary	October 23, 2018
Glycogen Storage Disease (GSD) Type 1 – Adult Patients	Patient Listening Session	Patient Listening Session Summary	March 18, 2021
Glycogen Storage Disease (GSD) Type 1 – Caregivers to Pediatric Patients	Patient Listening Session	Patient Listening Session Summary	March 25, 2021
Glycogen Storage Disease Type 1B	Patient Listening Session	Patient Listening Session Summary	March 3, 2022
Gorlin Syndrome	Patient Listening Session	Patient Listening Session Summary	November 9, 2020
Gorlin Syndrome	EL-PFDD Meeting Host: Gorlin Syndrome Alliance	Meeting Report	October 8, 2021
Guillain-Barre’ Syndrome (GBS)	Patient Listening Session	Patient Listening Session Summary	September 29, 2020
Healthcare Challenges and Unmet Medical Needs of Transgender Adults	Patient Listening Session	Patient Listening Session Summary	June 1, 2021
Healthcare Challenges and Unmet Medical Needs of Transgender Adolescents	Patient Listening Session	Patient Listening Session Summary	June 29, 2021
Hemophilia A, B, and Other Heritable Bleeding Disorders	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	September 22, 2014
Hepatitis B	EL-PFDD Meeting Host: Hepatitis B Foundation	Meeting Report	June 9, 2020
Hereditary Angioedema	FDA-led PFDD Meeting	Agenda Recordings (Part 1, Part 2) Transcript Summary Report	September 25, 2017
Homocystinuria (HCU)	Patient Listening Session	Patient Listening Session Summary	June 26, 2020
Human Immunodeficiency Virus (HIV)	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2, Part 3, Part 4) Transcript Summary Report	June 14, 2013
Hunter Syndrome	Patient Listening Session	Patient Listening Session Summary	February 4, 2020
Hunter Syndrome (MPS II)	N/A	Toileting Ability Survey Report	N/A
Huntington’s Disease	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2, Part 3, Part 4) Transcript Summary Report	September 22, 2015
Hypereosinophilic Syndromes	EL-PFDD Meeting Host: American Partnership for Eosinophilic Disorders	Meeting Report	March 23, 2018
Hyperhidrosis	EL-PFDD Meeting Host: International Hyperhidrosis Society	Meeting Report	November 13, 2017
Hypertrophic Cardiomyopathy	EL-PFDD Meeting Host: Hypertrophic Cardiomyopathy Association	Meeting Report	June 26, 2020
Hypomyelination with Atrophy of Basal Ganglia and Cerebellum (H-ABC)	Patient Listening Session	Patient Listening Session Summary	May 9, 2022
Hypophosphatemia	EL-PFDD Meeting Host: XLH Network	Meeting Report	October 5, 2018
Hypothalamic Obesity	Patient Listening Session	Patient Listening Session Summary	October 22, 2021
Ichthyosis	Patient Listening Session	Patient Listening Session Summary	September 17, 2021
Idiopathic Pulmonary Fibrosis	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	September 26, 2014
IgA Nephropathy	EL-PFDD Meeting Host: National Kidney Foundation and the IgA Nephropathy Foundation of America	Meeting Report	August 19, 2019
Immune Thrombocytopenia	EL-PFDD Meeting Host: Platelet Disorder Support Association	Meeting Report	July 26, 2019
Inborn Errors of Metabolism	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	June 10, 2014
Inclusion Body Myositis (IBM)	Patient Listening Session	Patient Listening Session Summary	March 5, 2020
Juvenile Dermatomyositis (JDM)	Patient Listening Session	Summary unavailable	March 16, 2021
Krabbe Disease	EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD)	Meeting Report	October 29, 2020
Late Onset GM2 Gangliosidoses (Tay-Sachs and Sandhoff Diseases)	Patient Listening Session	Patient Listening Session Summary	January 15, 2021
Limb Girdle Muscular Dystrophies (LGMD)	Patient Listening Session	Patient Listening Session Summary	October 20, 2020
Lung Cancer	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	June 28, 2013
Lupus – Diversity in Clinical Trials	Patient Listening Session	Patient Listening Session Summary – Session 1 Patient Listening Session Summary – Session 2 Patient Listening Session Summary – Session 3	March 31, 2021 – Session 1 April 2, 2021 – Session 2 April 6, 2021 – Session 3
Lupus	EL-PFDD Meeting Host: Lupus and Allied Diseases Association, Lupus Foundation of America, and the Lupus Research Alliance	Meeting Report	September 25, 2017
Major Depressive Disorder	EL-PFDD Meeting Host: Depression and Bipolar Support Alliance	Meeting Report	November 16, 2018
Mastocytosis	Patient Listening Session	Patient Listening Session Summary	September 28, 2021
Mitochondrial Disease	EL-PFDD Meeting Host: United Mitochondrial Disease Foundation	Meeting Report	March 29, 2019
Myeloproliferative Neoplasms	EL-PFDD Meeting Host: MPN Research Foundation	Meeting Report	September 16, 2019
Myotonic Dystrophy	EL-PFDD Meeting Host: Myotonic Dystrophy Foundation	Meeting Report	September 15, 2016
Narcolepsy	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	September 24, 2013
Neurofibromatosis (NF)	Patient Listening Session	Patient Listening Session Summary	June 13, 2019
Neuronopathic Gaucher Disease (Types 2 & 3)	Patient Listening Session	Patient Listening Session Summary	September 9, 2021
Neuropathic Pain Associated with Peripheral Neuropathy	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2, Part 3) Transcript Summary Report	June 10, 2016
Niemann-Pick Type C	EL-PFDD Meeting Host: Ara Parseghian Medical Research Fund at Notre Dame, Hide & Seek Foundation, Dana's Angels Research Trust, Hope for Marian, National Niemann-Pick Disease Foundation, Niemann-Pick Canada, Firefly Fund, and Johnathon's Dreams	Meeting Report	March 18, 2019
Nonalcoholic Steatohepatitis	EL-PFDD Meeting Host: Global Liver Institute	Meeting Report	November 4, 2021
Non-tuberculous Mycobacterial Lung Infections	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2, Part 3, Part 4) Transcript Summary Report	October 15, 2015
Obstructive Sleep Apnea	EL-PFDD Meeting Host: American Sleep Apnea Association	Meeting Report	June 8, 2018
Ocular Melanoma	Patient Listening Session	Patient Listening Session Summary	January 27, 2020
Opioid Use Disorder	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2, Part 3) The audio recording of this meeting is publicly available using the links below. Because of the sensitive nature of the meeting topic, and the importance of gathering candid, meaningful input from individuals who have come forward to speak about living with opioid use disorder, there is no video recording of the meeting. Transcript Summary Report	April 17, 2018
Osteoarthritis	Patient Listening Session	Patient Listening Session Summary	August 7, 2019
Osteoarthritis	EL-PFDD Meeting Host: Arthritis Foundation	Meeting Report	March 8, 2017
Osteogenesis Imperfecta (OI)	Patient Listening Session	Patient Listening Session Summary	September 17, 2019
Pachyonychia Congenita	EL-PFDD Meeting Host: Pachyonychia Congenita Project	Meeting Report	April 6, 2018
Pancreatitis	EL-PFDD Meeting Host: National Pancreas Foundation and FCS Foundation	Meeting Report	March 3, 2020
Parkinson’s Disease	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2, Part 3, Part 4) Transcript Summary Report	September 22, 2015
Patients Who Have Received an Organ Transplant	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2, Part 3, Part 4) Transcript Summary Report	September 27, 2016
Pemphigus and Pemphigoid	Patient Listening Session	Patient Listening Session Summary	February 8, 2021
Polyglutamine Spinocerebellar Ataxias (SCAs) and Dentatorubal-Pallidoluysian Atrophy (DRPLA)	EL-PFDD Meeting Host: National Ataxia Foundation and CureDRPLA	Meeting Report	September 25, 2020
Pompe Disease	EL-PFDD Meeting Host: Muscular Dystrophy Association	Meeting Report	July 13, 2020
Primary Ciliary Dyskinesia	Patient Listening Session	Summary coming soon	September 1, 2021
Primary Sclerosing Cholangitis	EL-PFDD Meeting Host: PSC Partners Seeking a Cure	Meeting Report	October 23, 2020
Progressive Multifocal Leukoencephalopathy	Patient Listening Session	Patient Listening Session Summary – Session 1 Patient Listening Session Summary – Session 2	July 22, 2020 – Session 1 September 11, 2020 – Session 2
Psoriasis	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	March 17, 2016
Pulmonary Alveolar Proteinosis	Patient Listening Session	Summary unavailable	July 8, 2020
Pulmonary Arterial Hypertension	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	May 13, 2014
Pulmonary Sarcoidosis	Patient Listening Session	Patient Listening Session Summary	April 21, 2022
Pyruvate Kinase Deficiency	EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD)	Meeting Report	September 20, 2019
Sanfilippo Syndrome	Patient Listening Session	Patient Listening Session Summary	May 13, 2019
Sanfilippo Syndrome – Pediatric	Patient Listening Session	Patient Listening Session Summary	October 17, 2019
Sarcopenia	FDA-led PFDD Meeting	Agenda Slides Recording (Full recording) Transcript Summary Report	April 6, 2017
SCN2A Related Disorders	Patient Listening Session	Patient Listening Session Summary	April 28, 2021
Sensorineural Hearing Loss	EL-PFDD Meeting Host: Hearing Loss Association of America	Meeting Report	May 25, 2021
Sickle Cell Disease	FDA-led PFDD Meeting	Agenda Slides Recordings (Part 1, Part 2) Transcript Summary Report	February 7, 2014
Smith-Magenis Syndrome	Patient Listening Session	Patient Listening Session Summary	August 12, 2020
Spinal Muscular Atrophy	EL-PFDD Meeting Host: Cure SMA	Meeting Report	April 18, 2017
Stimulant Use Disorder	FDA-led PFDD Meeting	Agenda Slides Recording Transcript Summary Report	October 6, 2020
Systemic Sclerosis	FDA-led PFDD Meeting	Agenda Slides Recording Transcript Summary Report	October 13, 2020
Thymidine Kinase 2 Deficiency (TK2)	Patient Listening Session	Patient Listening Session Summary	January 31, 2022
Tuberous Sclerosis Complex	EL-PFDD Meeting Host: Tuberous Sclerosis Alliance	Meeting Report	June 21, 2017
Valosin Containing Protein Disease (VCP)	Patient Listening Session	Patient Listening Session Summary	May 5, 2020
Vascular Ehlers-Danlos Syndrome (VEDS)	Patient Listening Session	Patient Listening Session Summary	October 7, 2020
Vitiligo	FDA-led PFDD Meeting	Agenda Slides Recording Transcript Summary Report	March 8, 2021
Von Hippel Lindau (VHL)	Patient Listening Session	Patient Listening Session Summary	June 11, 2020
Xerostomia	EL-PFDD Meeting Host: Head and Neck Cancer Alliance (HNCA), the National Foundation of Swallowing Disorders (NFOSD), Support for People with Oral and Head and Neck Cancer (SPOHNC), and the THANC (Thyroid, Head and Neck Cancer) Foundation	Meeting Report	August 19, 2021
X-Linked Myotubular Myopathy (XLMTM)	Patient Listening Session	Summary unavailable	August 3, 2020

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