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 Other information includes proposed draft guidance relating to patient experience data, natural history studies, or other condition-specific background on condition and discussion of unmet medical need.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 |
|---|---|---|---|
| Cyclin-dependent kinase-like 5 deficiency disorder (CDKL5/CDD) | EL-PFDD Meeting Host: Loulou Foundation and the International Foundation for CDKL5 Research (IFCR) |
Meeting Report | November 1, 2019 |
| Autosomal Recessive Polycystic Kidney Disease (ARPKD) |
EL-PFDD Meeting |
Report pending | April 28, 2023 |
| Carcinoid Syndrome |
Patient Listening Session |
Patient Listening Session Summary | April 27, 2023 |
| Canavan Disease |
Patient Listening Session |
Patient Listening Session Summary | March 21, 2023 |
| Post-finasteride Syndrome (PFS) |
Patient Listening Session |
Patient Listening Session Summary | June 2, 2023 |
| Prader-Willi Syndrome (PWS) |
EL-PFDD Meeting |
Report pending | June 22, 2023 |
| Arrhythmogenic Right Ventricular Cardiomyopathy/Dysplasia (ARVC/D) |
EL-PFDD Meeting |
Report pending | June 20, 2023 |
| Recurrent Clostridium difficile Infections (rCDI) |
Patient Listening Session |
Patient Listening Session Summary | March 10, 2023 |
| Sickle Cell Disease |
Patient Listening Session |
Patient Listening Session Summary | May 5, 2023 |
| Pulmonary Alveola Proteinosis (PAP) |
Patient Listening Session |
Patient Listening Session Summary | July 8, 2020 |
| Bronchopulmonary Dysplasia (BPD) |
Patient Listening Session |
Patient Listening Session Summary | March 20, 2023 |
| Juvenile Idiopathic Arthritis (JIA) |
EL-PFDD Meeting Host: The Arthritis Foundation and the Childhood Arthritis and Rheumatology Research Alliance (CARRA) |
Meeting Report | April 2, 2018 |
| Long COVID | FDA-led PFDD Meeting | April 25, 2023 | |
|
Congenital Disorders of Glycosylation (CDG) |
Patient Listening Session |
January 30, 2023 |
|
|
Juvenile Huntington’s Disease (JHD) |
Patient Listening Session |
February 10, 2023 |
|
|
Pouchitis after a total colectomy and ideal pouch anal anastomosis (IPAA) surgery |
Patient Listening Session |
January 14, 2023 |
|
|
SYNGAP1 |
EL-PFDD Meeting Host: SYNGAP1 Foundation |
November 19, 2020 |
|
|
Pre-Diagnosis Genetic Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Degeneration (FTD) |
Patient Listening Session |
January 12, 2023 |
|
|
Proteus Syndrome |
Patient Listening Session |
December 1, 2022 |
|
|
Stargardt Disease (STGD1) |
Patient Listening Session |
June 24, 2022 |
|
|
Recurrent Respiratory Papillomatosis (RRP) |
Patient Listening Session |
October 27, 2022 |
|
|
Wiskott-Aldrich Syndrome (WAS) |
EL-PFDD Meeting |
Report pending |
February 3, 2023 |
|
Autoimmune Hepatitis (AIH) |
EL-PFDD Meeting |
Report pending |
January 27, 2023 |
|
Pemphigus and Pemphigoid |
EL-PFDD Meeting |
Report pending |
January 25, 2023 |
|
Cerebral Creatine Deficiency Syndromes (CCDS) |
EL-PFDD Meeting |
Report pending |
January 24, 2023 |
|
Narcolepsy and Idiopathic Hypersomnia (IH) |
Patient Listening Session |
August 8, 2022 |
|
|
Hypophosphatasia (HPP) |
EL-PFDD Meeting |
November 15, 2022 |
|
|
GM1 gangliosidosis |
EL-PFDD Meeting |
October 14, 2022 |
|
|
Metachromatic Leukodystrophy (MLD) |
EL-PFDD Meeting |
October 21, 2022 |
|
|
Schizophrenia |
EL-PFDD Meeting |
November 2, 2022 |
|
|
Phelan-McDermid Syndrome (PMS) |
EL-PFDD Meeting |
November 8, 2022 |
|
|
Kennedy’s Disease (KD)/ Spinal and bulbar muscular atrophy (SBMA) |
EL-PFDD Meeting |
November 9, 2022 |
|
|
Short Bowel Syndrome (SBS) |
Patient Listening Session |
July 19, 2022 |
|
|
Huntington’s Disease (HD) Pre-Symptomatic Population |
Patient Listening Session |
July 25, 2022 |
|
|
Spinal Muscular Atrophy (SMA) |
Patient Listening Session |
August 4, 2022 |
|
|
Alstrom Syndrome (AS) |
EL-PFDD Meeting Host: Alstrom International |
September 22, 2022 |
|
|
Reducing Cardiac Late Effects in Pediatric Cancer Survivors |
EL-PFDD Meeting Host: Children’s Cancer Cause |
September 15, 2022 |
|
|
Fabry Disease |
EL-PFDD Meeting Host: National Kidney Foundation and Fabry Support & Information Group |
Report pending |
September 19, 2022 |
|
Galactosemia |
EL-PFDD Meeting Host: National Organization for Rare Disorders (NORD) |
September 1, 2022 |
|
|
Limb-Girdle Muscular Dystrophies (LGMD) |
EL-PFDD Meeting Host: Coalition to Cure Calpain 3, CureLGMD2i, the Kurt+Peter Foundation, the LGMD2D Foundation, the McColl-Lockwood Laboratory for Muscular Dystrophy Research, and the Speak Foundation |
September 23, 2022 |
|
|
Primary Biliary Cholangitis (PBC) |
EL-PFDD Meeting |
Report pending |
February 4, 2022 |
|
Food Allergies |
EL-PFDD Meeting |
September 9, 2021 |
|
|
Asthma |
EL-PFDD Meeting |
September 20, 2021 |
|
|
Myotubular and Centronuclear Myopathy (MTM/CNM) |
EL-PFDD Meeting |
Report pending |
August 3, 2021 |
|
Rett Syndrome |
EL-PFDD Meeting |
March 11, 2022 |
|
|
Membranous Nephropathy (MN) |
EL-PFDD Meeting |
August 27, 2021 |
|
|
Cancer Cachexia |
EL-PFDD Meeting |
November 2, 2021 |
|
|
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) |
EL-PFDD Meeting |
March 25, 2022 |
|
|
Primary Hyperoxaluria (PH) |
EL-PFDD Meeting |
October 5, 2020 |
|
|
Post-Transplant Lymphoproliferative Disorder (PTLD) |
EL-PFDD Meeting |
May 4, 2022 |
|
|
Bronchiolitis Obliterans Syndrome (BOS) |
EL-PFDD Meeting |
June 22, 2022 |
|
|
Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD) |
EL-PFDD Meeting |
July 8, 2022 |
|
|
X-linked Retinitis Pigmentosa (XLRP) |
EL-PFDD Meeting |
June 7, 2022 |
|
|
Congenital Muscular Dystrophy (CMD) |
EL-PFDD Meeting |
Report pending |
July 1, 2022 |
|
Hermansky-Pudlak Syndrome (HPS) |
EL-PFDD Meeting |
June 10, 2022 |
|
|
X-linked Adrenoleukodystrophy (ALD) |
EL-PFDD Meeting |
July 22, 2022 |
|
|
Acromegaly
|
EL-PFDD Meeting |
January 21, 2021 |
|
|
Acute Porphyrias |
EL-PFDD Meeting |
March 1, 2017 |
|
|
Adrenomyeloneuropathy (AMN) |
Patient Listening Session |
May 7, 2021 |
|
|
Adult Dermatomyositis |
Patient Listening Session |
April 26, 2022 |
|
|
Adult Polyglucosan Body Disease (APBD) |
Patient Listening Session |
October 28, 2021 |
|
|
Alopecia Areata |
FDA-led PFDD Meeting |
|
September 11, 2017 |
|
Alpha-1 Antitrypsin Deficiency (AATD) |
FDA-led PFDD Meeting |
|
September 29, 2015 |
|
Alport Syndrome (AS) |
EL-PFDD Meeting |
August 3, 2018 |
|
|
Amyloidosis |
EL-PFDD Meeting |
November 16, 2015 |
|
|
Amyotrophic Lateral Sclerosis (ALS) |
N/A |
N/A |
|
|
Autism (ASD) |
FDA-led PFDD Meeting |
|
May 4, 2017 |
|
Barth Syndrome (BTHS) |
EL-PFDD Meeting |
July 18, 2018 |
|
|
Breast Cancer |
FDA-led PFDD Meeting |
|
April 2, 2015 |
|
Celiac Disease |
Patient Listening Session |
February 20, 2019 |
|
|
Cerebral Cavernous Malformation (CCM) |
Patient Listening Session |
November 6, 2019 |
|
|
Cerebrotendinous Xanthomatosis (CTX) |
EL-PFDD Meeting |
September 14, 2021 |
|
|
Chagas Disease |
FDA-led PFDD Meeting |
|
April 28, 2015 |
|
Charcot-Marie-Tooth and Inherited Neuropathies (CMT/IN) |
EL-PFDD Meeting |
September 28, 2018 |
|
|
Chemotherapy-Induced Hearing Loss |
EL-PFDD Meeting |
September 13, 2018 |
|
|
Childhood Cerebral Adrenal Leukodystrophy (CCALD) |
Patient Listening Session |
November 13, 2019 |
|
|
Chronic Fatigue Syndrome/Myalgic Encephalomyelitis (CFS & ME) |
FDA-led PFDD Meeting |
|
April 25, 2013 |
|
Chronic Pain |
FDA-led PFDD Meeting |
|
July 9, 2018 |
|
Complement 3 Glomerulopathy (C3G) |
EL-PFDD Meeting |
August 4, 2017 |
|
|
Cystic Fibrosis (CF) – nonsense mutations |
Patient Listening Session |
July 15, 2021 |
|
|
Dermatitis (Eczema) |
EL-PFDD Meeting |
September 23, 2019 |
|
|
Dravet Syndrome (DS) |
EL-PFDD Meeting |
February 3, 2022 |
|
|
Fabry Disease |
Patient Listening Session |
December 4, 2018 |
|
|
Facioscapulohumeral Muscular Dystrophy (FSHD) |
EL-PFDD Meeting |
June 29, 2020 |
|
|
Female Sexual Dysfunction (FSD) |
FDA-led PFDD Meeting |
|
October 27, 2014 |
|
Fibrodysplasia Ossificans Progressiva (FOP) |
Patient Listening Session |
May 29, 2019 |
|
|
Fibromyalgia |
FDA-led PFDD Meeting |
|
March 26, 2014 |
|
Focal Segmental Glomerulosclerosis (FSGS) |
EL-PFDD Meeting |
August 28, 2020 |
|
|
Fragile X Syndrome (FXS) |
EL-PFDD Meeting |
March 3, 2021 |
|
|
Friedreich’s Ataxia (FA) |
EL-PFDD Meeting |
June 2, 2017 |
|
|
Frontotemporal Degeneration (FTD) |
EL-PFDD Meeting |
March 5, 2021 |
|
|
Functional Gastrointestinal (GI) Disorders |
FDA-led PFDD Meeting |
|
May 11, 2015 |
|
Gastroparesis |
Patient Listening Session |
December 2, 2019 |
|
|
Gene Therapy as a Treatment Modality for Hemophilia |
Patient Listening Session |
October 23, 2018 |
|
|
Glycogen Storage Disease (GSD) Type 1 – Adult Patients |
Patient Listening Session |
March 18, 2021 |
|
|
Glycogen Storage Disease (GSD) Type 1 – Caregivers to Pediatric Patients |
Patient Listening Session |
March 25, 2021 |
|
|
Glycogen Storage Disease (GSD) Type 1B |
Patient Listening Session |
March 3, 2022 |
|
|
Gorlin Syndrome (GS) |
Patient Listening Session |
November 9, 2020 |
|
|
Gorlin Syndrome (GS) |
EL-PFDD Meeting |
October 8, 2021 |
|
|
Guillain-Barre’ Syndrome (GBS) |
Patient Listening Session |
September 29, 2020 |
|
|
Healthcare Challenges and Unmet Medical Needs of Transgender Adults |
Patient Listening Session |
June 1, 2021 |
|
|
Healthcare Challenges and Unmet Medical Needs of Transgender Adolescents |
Patient Listening Session |
June 29, 2021 |
|
|
Hemophilia A, Hemophilia B, von Willebrand Disease (VWB), and Other Heritable Bleeding Disorders |
FDA-led PFDD Meeting |
|
September 22, 2014 |
|
Hepatitis B |
EL-PFDD Meeting |
June 9, 2020 |
|
|
Hereditary Angioedema (HAE) |
FDA-led PFDD Meeting |
|
September 25, 2017 |
|
Homocystinuria (HCU) |
Patient Listening Session |
June 26, 2020 |
|
|
Human Immunodeficiency Virus (HIV)
|
FDA-led PFDD Meeting |
|
June 14, 2013 |
|
Hunter Syndrome (MPS II) |
Patient Listening Session |
February 4, 2020 |
|
|
Hunter Syndrome (MPS II) |
N/A |
N/A |
|
|
Huntington’s Disease (HD) |
FDA-led PFDD Meeting |
|
September 22, 2015 |
|
Hypereosinophilic Syndromes (HES) |
EL-PFDD Meeting |
March 23, 2018 |
|
|
Hyperhidrosis (Hh) |
EL-PFDD Meeting |
November 13, 2017 |
|
|
Hypertrophic Cardiomyopathy (HCM) |
EL-PFDD Meeting |
June 26, 2020 |
|
|
Hypomyelination with Atrophy of Basal Ganglia and Cerebellum (H-ABC) |
Patient Listening Session |
May 9, 2022 |
|
|
Hypophosphatemia |
EL-PFDD Meeting |
October 5, 2018 |
|
|
Hypothalamic Obesity (HO) |
Patient Listening Session |
October 22, 2021 |
|
|
Ichthyosis |
Patient Listening Session |
September 17, 2021 |
|
|
Idiopathic Pulmonary Fibrosis (IPF) |
FDA-led PFDD Meeting |
|
September 26, 2014 |
|
IgA Nephropathy (IgAN) |
EL-PFDD Meeting |
August 19, 2019 |
|
|
Immune Thrombocytopenia (ITP) |
EL-PFDD Meeting |
July 26, 2019 |
|
|
Inborn Errors of Metabolism (IEM) |
FDA-led PFDD Meeting |
|
June 10, 2014 |
|
Inclusion Body Myositis (IBM) |
Patient Listening Session |
March 5, 2020 |
|
|
Krabbe Disease |
EL-PFDD Meeting |
October 29, 2020 |
|
|
Late Onset GM2 gangliosidoses (Tay-Sachs and Sandhoff Diseases) |
Patient Listening Session |
January 15, 2021 |
|
|
Limb-Girdle Muscular Dystrophies (LGMD) |
Patient Listening Session |
October 20, 2020 |
|
|
Lung Cancer |
FDA-led PFDD Meeting |
|
June 28, 2013 |
|
Lupus – Diversity in Clinical Trials |
Patient Listening Session |
March 31, 2021 – Session 1 April 2, 2021 – Session 2 April 6, 2021 – Session 3 |
|
|
Lupus |
EL-PFDD Meeting |
September 25, 2017 |
|
|
Major Depressive Disorder (MDD) |
EL-PFDD Meeting |
November 16, 2018 |
|
|
Mastocytosis |
Patient Listening Session |
September 28, 2021 |
|
|
Mitochondrial Disease |
EL-PFDD Meeting |
March 29, 2019 |
|
|
Myeloproliferative Neoplasms (MPNs) |
EL-PFDD Meeting |
September 16, 2019 |
|
|
Myotonic Dystrophy (DM) |
EL-PFDD Meeting |
September 15, 2016 |
|
|
Narcolepsy |
FDA-led PFDD Meeting |
|
September 24, 2013 |
|
Neurofibromatosis (NF) |
Patient Listening Session |
June 13, 2019 |
|
|
Neuronopathic Gaucher Disease (nGD) (Types 2 & 3) |
Patient Listening Session |
September 9, 2021 |
|
|
Neuropathic Pain Associated with Peripheral Neuropathy |
FDA-led PFDD Meeting |
|
June 10, 2016 |
|
Niemann-Pick Type C (NPC) |
EL-PFDD Meeting |
March 18, 2019 |
|
|
Nonalcoholic Steatohepatitis (NASH) |
EL-PFDD Meeting |
November 4, 2021 |
|
|
Non-Tuberculous Mycobacterial (NTM) Lung Infections |
FDA-led PFDD Meeting |
|
October 15, 2015 |
|
Obstructive Sleep Apnea (OSA) |
EL-PFDD Meeting |
June 8, 2018 |
|
|
Ocular Melanoma (OM) |
Patient Listening Session |
January 27, 2020 |
|
|
Opioid Use Disorder (OUD) |
FDA-led PFDD Meeting |
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.
|
April 17, 2018 |
|
Osteoarthritis (OA) |
Patient Listening Session |
August 7, 2019 |
|
|
Osteoarthritis (OA) |
EL-PFDD Meeting |
March 8, 2017 |
|
|
Osteogenesis Imperfecta (OI) |
Patient Listening Session |
September 17, 2019 |
|
|
Pachyonychia Congenita (PC) |
EL-PFDD Meeting |
April 6, 2018 |
|
|
Pancreatitis |
EL-PFDD Meeting |
March 3, 2020 |
|
|
Parkinson’s Disease |
FDA-led PFDD Meeting |
|
September 22, 2015 |
|
Patients Who Have Received an Organ Transplant |
FDA-led PFDD Meeting |
|
September 27, 2016 |
|
Pemphigus and Pemphigoid |
Patient Listening Session |
February 8, 2021 |
|
|
Polyglutamine Spinocerebellar Ataxias (SCAs) and Dentatorubal-Pallidoluysian Atrophy (DRPLA) |
EL-PFDD Meeting |
September 25, 2020 |
|
|
Pompe Disease |
EL-PFDD Meeting |
July 13, 2020 |
|
|
Primary Sclerosing Cholangitis (PSC) |
EL-PFDD Meeting |
October 23, 2020 |
|
|
Progressive Multifocal Leukoencephalopathy (PML) |
Patient Listening Session |
July 22, 2020 – Session 1 September 11, 2020 – Session 2 |
|
|
Psoriasis |
FDA-led PFDD Meeting |
|
March 17, 2016 |
|
Pulmonary Arterial Hypertension (PAH) |
FDA-led PFDD Meeting |
|
May 13, 2014 |
|
Pulmonary Sarcoidosis |
Patient Listening Session |
April 21, 2022 |
|
|
Pyruvate Kinase Deficiency |
EL-PFDD Meeting |
September 20, 2019 |
|
|
Sanfilippo Syndrome |
Patient Listening Session |
May 13, 2019 |
|
|
Sanfilippo Syndrome – Pediatric |
Patient Listening Session |
October 17, 2019 |
|
|
Sarcopenia |
FDA-led PFDD Meeting |
|
April 6, 2017 |
|
SCN2A Related Disorders |
Patient Listening Session |
April 28, 2021 |
|
|
Sensorineural Hearing Loss (SNHL) |
EL-PFDD Meeting |
May 25, 2021 |
|
|
Sickle Cell Disease |
FDA-led PFDD Meeting |
|
February 7, 2014 |
|
Smith-Magenis Syndrome (SMS) |
Patient Listening Session |
August 12, 2020 |
|
|
Spinal Muscular Atrophy (SMA) |
EL-PFDD Meeting |
April 18, 2017 |
|
|
Stimulant Use Disorder |
FDA-led PFDD Meeting |
|
October 6, 2020 |
|
Systemic Sclerosis |
FDA-led PFDD Meeting |
|
October 13, 2020 |
|
Thymidine Kinase 2 Deficiency (TK2) |
Patient Listening Session |
January 31, 2022 |
|
|
Tuberous Sclerosis Complex (TSC) |
EL-PFDD Meeting |
June 21, 2017 |
|
|
Valosin Containing Protein Disease (VCP) |
Patient Listening Session |
May 5, 2020 |
|
|
Vascular Ehlers-Danlos Syndrome (VEDS) |
Patient Listening Session |
October 7, 2020 |
|
|
Vitiligo |
FDA-led PFDD Meeting |
|
March 8, 2021 |
|
Von Hippel Lindau (VHL) |
Patient Listening Session |
June 11, 2020 |
|
|
Xerostomia |
EL-PFDD Meeting |
August 19, 2021 |
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