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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 Other information includes proposed draft guidance relating to patient experience data, natural history studies, or other condition-specific background on condition and discussion of unmet medical need.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

GM1 Gangliosidosis

EEL-PFDD Meeting
Host: Cure GM1 Foundation

Report pending

October 14, 2022

Metachromatic Leukodystrophy

EL-PFDD Meeting
Host: The Calliope Joy Foundation

Report pending

October 21, 2022

Schizophrenia

EL-PFDD Meeting
Host: The American Foundation for Suicide Prevention, Mental Health America, the National Alliance on Mental Illness (NAMI), the National Council for Mental Wellbeing, Schizophrenia and Psychosis Action Alliance (S&PAA)

Report pending

November 2, 2022

Phelan-MeDermid Syndrome

EL-PFDD Meeting
Host: Cure Shank

Report pending

November 8, 2022

Kennedy’s Disease

EL-PFDD Meeting
Host: Kennedy’s Disease Association

Report pending

November 9, 2022

Short Bowel Syndrome

Patient Listening Session

Patient Listening Session Summary

July 19, 2022

Huntington’s Disease (Pre-Symptomatic)

Patient Listening Session

Patient Listening Session Summary

July 25, 2022

Spinal Muscular Atrophy (SMA)

Patient Listening Session

Patient Listening Session Summary

August 4, 2022

Alstrom Syndrome

EL-PFDD Meeting

Host: Alstrom International

Report pending

September 22, 2022

Reducing Cardiac Late Effects in Pediatric Cancer Survivors

EL-PFDD Meeting

Host: Children’s Cancer Cause

Report pending

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 Rear Disorders (NORD)

Report pending

September 1, 2022

Limb-Girdle Muscular Dystrophy

EL-PFDD Meeting

Host: The Speak Foundation

Report pending

September 23, 2022

Primary Biliary Cholangitis (PBC)

EL-PFDD Meeting
Host: Global Liver Institute

Report pending

February 4, 2022

Food Allergies

EL-PFDD Meeting
Host: Food Allergy Research and Education (FARE)

Meeting Report

September 9, 2021

Asthma

EL-PFDD Meeting
Host: Asthma and Allergy Foundation of America (AAFA)

Meeting Report

September 20, 2021

Myotubular and Centro Nuclear Myopathy (MTM/CNM)

EL-PFDD Meeting
Host: MTM-CNM Family Connection and Where There’s A Will There’s A Cure

Report pending

August 3, 2021

Rett Syndrome

EL-PFDD Meeting
Host: Rett Syndrome Research Trust (RSRT)

Meeting Report

March 11, 2022

Membranous Nephropathy

EL-PFDD Meeting
Host: National Kidney Foundation and NephCure Kidney International

Report pending

August 27, 2021

Cancer Cachexia

EL-PFDD Meeting
Host: Cancer Support Community

Meeting Report

November 2, 2021

Chronic inflammatory Demyelinating Polyneuropathy (CIDP)

EL-PFDD Meeting
Host: GBS|CIDP Foundation International

Meeting Report

March 25, 2022

Primary Hyperoxaluria

EL-PFDD Meeting
Host: The Oxalosis and Hyperoxaluria Foundation

Meeting Report

October 5, 2020

Post-Transplant Lymphoproliferative Disease (PTLD)

EL-PFDD Meeting
Host: NORD

Report pending

May 4, 2022

Bronchiolitis Obliterans Syndrome (BOS)

EL-PFDD Meeting
Host: Lung Transplant Foundation with NORD

Report pending

June 22, 2022

Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD)

EL-PFDD Meeting
Host: SSADH Association

Report pending

July 8, 2022

X-Linked Retinitis Pigmentosa

EL-PFDD Meeting
Host: Fighting Blindness Foundation

Report pending

June 7, 2022

Congenital Muscular Dystrophy

EL-PFDD Meeting
Host: Cure CMD

Report pending

July 1, 2022

Hermansky-Pudlak Syndrome

EL-PFDD Meeting
Host: Hermansky-Pudlak Syndrome Network

Report pending

June 10, 2022

X-Linked Adrenoleukodystrophy (ALD)

EL-PFDD Meeting
Host: ALD Connect

Report pending

July 22, 2022

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

September 11, 2017

Alpha-1 Antitrypsin

FDA-led PFDD Meeting

 

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

 

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

 

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

 

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

 

April 25, 2013

Chronic Pain

FDA-led PFDD Meeting

 

July 9, 2018

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

 

October 27, 2014

Fibrodysplasia Ossificans Progressiva (FOP)

Patient Listening Session

Patient Listening Session Summary

May 29, 2019

Fibromyalgia

FDA-led PFDD Meeting

 

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

 

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

 

September 22, 2014

Hepatitis B

EL-PFDD Meeting
Host: Hepatitis B Foundation

Meeting Report

June 9, 2020

Hereditary Angioedema

FDA-led PFDD Meeting

 

September 25, 2017

Homocystinuria (HCU)

Patient Listening Session

Patient Listening Session Summary

June 26, 2020

Human Immunodeficiency Virus (HIV)

 

 

 

 

 

 

 

 

 

 

FDA-led PFDD Meeting

 

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

 

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

 

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

 

June 10, 2014

Inclusion Body Myositis (IBM)

Patient Listening Session

Patient Listening Session Summary

March 5, 2020

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

 

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
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

 

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

 

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

 

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

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

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

 

September 22, 2015

Patients Who Have Received an Organ Transplant

FDA-led PFDD Meeting

 

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 Sclerosing Cholangitis

EL-PFDD Meeting
Host: PSC Partners Seeking a Cure

Meeting Report

October 23, 2020

Progressive Multifocal Leukoencephalopathy

Patient Listening Session

July 22, 2020 – Session 1

September 11, 2020 – Session 2

Psoriasis

FDA-led PFDD Meeting

 

March 17, 2016

Pulmonary Arterial Hypertension

FDA-led PFDD Meeting

 

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

 

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

 

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

 

October 6, 2020

Systemic Sclerosis

FDA-led PFDD Meeting

 

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

 

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

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