External Resources and Information Related to Patients’ Experience
This webpage is intended to facilitate public discussion of patient-focused drug development and evaluation. 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.
Please note that although FDA reviews the materials at these links 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.
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. 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. For specific questions related to a report or resource, FDA recommends reaching out to the point of contact listed on this cover page.
To help expand the benefits of FDA’s Patient-Focused Drug Development (PFDD) initiative, FDA welcomes patient organizations to identify and organize patient-focused collaborations to generate public input on other disease areas. Submitted links to summary meeting reports from these externally-led PFDD meetings may be found here. FDA also welcomes submission of links to meeting reports from other stakeholder meetings collecting patient perspectives on disease burden and treatment burden.
- Acromegaly
In January 2021, Acromegaly Community, Inc. hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with acromegaly and their loved ones on the impact of acromegaly on their daily lives, and their perspectives on approaches to treating acromegaly. - Acute Porphyrias
In March 2017, the American Porphyria Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Acute Porphyrias and their loved ones on the impact of Acute Porphyrias on their daily lives, and their perspectives on approaches to treating Acute Porphyrias. - Alport Syndrome
In August 2018, the National Kidney Foundation and the Alport Syndrome Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Alport Syndrome and their loved ones on the impact of Alport Syndrome on their daily lives, and their perspectives on approaches to treating Alport Syndrome. - Amyloidosis
In November 2015, the Amyloidosis Research Consortium hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with systemic amyloidosis and their loved ones on the impact of amyloidosis on their daily lives, and their perspectives on approaches to treating amyloidosis. - Barth Syndrome
In July 2018, The Barth Syndrome Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Barth Syndrome and their loved ones on the impact of Barth Syndrome on their daily lives, and their perspectives on approaches to treating Barth Syndrome. - Cerebrotendinous Xanthomatosis (CTX)
In September of 2021, the United Leukodystrophy Foundation of America hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with CTX and their loved ones on the impact of CTX on their daily lives, and their perspectives on approaches to treating CTX. - Charcot-Marie-Tooth and Inherited Neuropathies
In September 2018, the Hereditary Neuropathy Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Charcot-Marie-Tooth and inherited neuropathies and their loved ones on the impact of Charcot-Marie-Tooth and inherited neuropathies on their daily lives, and their perspectives on approaches to treating Charcot-Marie-Tooth and inherited neuropathies. - Chemotherapy-Induced Hearing Loss
In September 2018, the Children's Cause for Cancer Advocacy, Children's Brain Tumor Foundation, Mattie Miracle Cancer Foundation, and Momcology hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with chemotherapy-induced hearing loss and their loved ones on the impact of chemotherapy-induced hearing loss on their daily lives, and their perspectives on approaches to treating chemotherapy-induced hearing loss. - Complement 3 Glomerulopathy (C3G)
In August 2017, the National Kidney Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with C3G and their loved ones on the impact of C3G on their daily lives, and their perspectives on approaches to treating C3G. - Dermatitis (Eczema)
In August 2019, the Allergy and Asthma Network, Asthma and Allergy Foundation of America, Global Parents for Eczema Research, International Topical Steroid Awareness Network, and National Eczema Association hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with dermatitis (eczema) and their loved ones on the impact of eczema on their daily lives, and their perspectives on approaches to treating eczema. - Facioscapulohumeral Muscular Dystrophy (FSHD)
In June 2020, FSHD Society hosted and Externally-led Patient-Focused Drug Development to hear directly from individuals living with FSHD and their loved ones on the impact of FSHD on their daily lives, and their perspectives on approaches to treating FSHD. - Focal Segmental Glomerulosclerosis
In August 2020, the National Kidney Foundation and NephCure Kidney International hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with focal segmental glomerulosclerosis and their loved ones on the impact of focal segmental glomerulosclerosis on their daily lives, and their perspectives on approaches to treating focal segmental glomerulosclerosis. - Fragile X Syndrome
In March 2021, the National Fragile X Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Fragile X Syndrome and their loved ones on the impact of Fragile X Syndrome on their daily lives, and their perspectives on approaches to treating Fragile X Syndrome. - Friedreich’s Ataxia
In June 2017, the Friedreich’s Ataxia Research Alliance hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Friedreich’s Ataxia and their loved ones on the impact of Freidreich’s Ataxia on their daily lives, and their perspectives on approaches to treating Friedreich’s Ataxia. - Frontotemporal Degeneration (FTD)
In March 2021, The Association for Frontotemporal Degeneration (AFTD) hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Frontotemporal Degeneration (FTD) and their loved ones on the impact FTD has on their daily lives, and their perspectives on approaches to treating FTD. - Gorlin Syndrome
In October 2021, Gorlin Syndrome Alliance hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Gorlin Syndrome and their loved ones on the impact Gorlin Syndrome has on their daily lives, and their perspectives on approaches to treating Gorlin Syndrome. - Hepatitis B
In June 2020, the Hepatitis B Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Hepatitis B and their loved ones on the impact of Hepatitis B on their daily lives, and their perspectives on approaches to treating Hepatitis B. - Hypereosinophilic Syndromes
In March 2018, the American Partnership for Eosinophilic Disorders hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Hypereosinophilic Syndromes and their loved ones on the impact of Hypereosinophilic Syndromes on their daily lives, and their perspectives on approaches to treating Hypereosinophilic Syndromes. - Hyperhidrosis
In November 2017, the International Hyperhidrosis Society hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with hyperhidrosis and their loved ones on the impact of hyperhidrosis on their daily lives, and their perspectives on approaches to treating hyperhidrosis. - Hypertrophic Cardiomyopathy
In June 2020, the Hypertrophic Cardiomyopathy Association hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with hypertrophic cardiomyopathy and their loved ones on the impact of hypertrophic cardiomyopathy on their daily lives, and their perspectives on approaches to treating hypertrophic cardiomyopathy. - Hypophosphatemia
In October 2018, XLH Network hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with hypophosphatemia and their loved ones on the impact of hypophosphatemia on their daily lives, and their perspectives on approaches to treating hypophosphatemia. - IgA Nephropathy
In August 2019, the National Kidney Foundation and the IgA Nephropathy Foundation of America hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with IgA Nephropathy and their loved ones on the impact of IgA Nephropathy on their daily lives, and their perspectives on approaches to treating IgA Nephropathy. - Immune Thrombocytopenia
In July 2019, Platelet Disorder Support Association hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with immune thrombocytopenia and their loved ones on the impact of immune thrombocytopenia on their daily lives, and their perspectives on approaches to treating immune thrombocytopenia. - Krabbe disease
In October 2020, the National Organization for Rare Disorders (NORD), together with KrabbeConnect, The Legacy of Angels Foundation, Partners for Krabbe Research, and Hunter’s Hope Foundation hosted an Externally-led Patient-Focused Drug Development to hear directly from individuals living with Krabbe disease and their loved ones on the impact of Krabbe disease on their daily lives, and their perspectives on approaches to treating Krabbe disease. - Lupus
In September 2017, the Lupus and Allied Diseases Association, the Lupus Foundation of America, and the Lupus Research Alliance hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with lupus and their loved ones on the impact of lupus on their daily lives, and their perspectives on approaches to treating lupus. - Major Depressive Disorder
In November 2018, the Depression and Bipolar Support Alliance hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with major depressive disorder and their loved ones on the impact of major depressive disorder on their daily lives, and their perspectives on approaches to treating major depressive disorder. - Mitochondrial Disease
In March 2019, the United Mitochondrial Disease Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with mitochondrial disease and their loved ones on the impact of mitochondrial disease on their daily lives, and their perspectives on approaches to treating mitochondrial disease. - Myeloproliferative Neoplasms
In September 2019, MPN Research Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with myeloproliferative neoplasms and their loved ones on the impact of myeloproliferative neoplasms on their daily lives, and their perspectives on approaches to treating myeloproliferative neoplasms. - Myotonic Dystrophy
In September 2016, the Myotonic Dystrophy Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with myotonic dystrophy and their loved ones on the impact of myotonic dystrophy on their daily lives, and their perspectives on approaches to treating myotonic dystrophy. - Niemann-Pick Type C
In March 2019, 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 hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Niemann-Pick Type C and their loved ones on the impact of Niemann-Pick Type C on their daily lives, and their perspectives on approaches to treating Niemann-Pick Type C. - Nonalcoholic Steatohepatitis
In November 2021, Global Liver Institute hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with nonalcoholic steatohepatitis and their loved ones on the impact of nonalcoholic steatohepatitis on their daily lives, and their perspectives on approaches to treating nonalcoholic steatohepatitis. - Obstructive Sleep Apnea
In June 2018, the American Sleep Apnea Association hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with sleep apnea and their loved ones on the impact of sleep apnea on their daily lives, and their perspectives on approaches to treating sleep apnea. - Osteoarthritis
In March 2017, the Arthritis Foundation hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with osteoarthritis and their loved ones on the impact of osteoarthritis on their daily lives, and their perspectives on approaches to treating osteoarthritis. - Pachyonychia Congenita
In April 2018, the Pachyonychia Congenita Project hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Pachyonychia Congenita and their loved ones on the impact of Pachyonychia Congenita on their daily lives, and their perspectives on approaches to treating Pachyonychia Congenita. - Pancreatitis
In March 2020, the National Pancreas Foundation and FCS Foundation hosted and Externally-led Patient-Focused Drug Development to hear directly from individuals living with Pancreatitis and their loved ones on the impact of Pancreatitis on their daily lives, and their perspectives on approaches to treating Pancreatitis. - Polyglutamine Spinocerebellar Ataxias (SCA) and Dentatorubal-Pallidoluysian Atrophy (DRPLA)
In September 2020, the National Ataxia Foundation and CureDRPLA hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with polyglutamine spinocerebellar ataxias and dentatorubal-pallidoluysian atrophy and their loved ones on the impact of polyglutamine spinocerebellar ataxias and dentatorubal-pallidoluysian atrophy on their daily lives, and their perspectives on approaches to treating polyglutamine spinocerebellar ataxias and dentatorubal-pallidoluysian atrophy. - Pompe Disease
In July 2020, Muscular Dystrophy Association hosted an Externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Pompe Disease and their loved ones on the impact Pompe Disease has on their daily lives, and their perspectives on approaches to treating Pompe Disease. - Primary Sclerosing Cholangitis (PSC)
In October 2020, PSC Partners Seeking a Cure hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Primary Sclerosing Cholangitis (PSC) and their loved ones on the impact PSC has on their daily lives, and their perspectives on approaches to treating PSC. - Pyruvate Kinase Deficiency
In September 2019, the National Organization for Rare Disorders (NORD) hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with pyruvate kinase deficiency and their loved ones on the impact of pyruvate kinase deficiency on their daily lives, and their perspectives on approaches to treating pyruvate kinase deficiency. - Sensorineural Hearing Loss
In May of 2021, the Hearing Loss Association of America hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with Sensorineural Hearing Loss and their loved ones on the impact of Sensorineural Hearing Loss on their daily lives, and their perspectives on approaches to treating Sensorineural Hearing Loss. - Spinal Muscular Atrophy
In April 2017, Cure SMA hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with spinal muscular atrophy and their loved ones on the impact of spinal muscular atrophy on their daily lives, and their perspectives on approaches to treating spinal muscular atrophy. - Tuberous Sclerosis Complex
In June 2017, the Tuberous Sclerosis Alliance hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with tuberous sclerosis complex and their loved ones on the impact of tuberous sclerosis complex on their daily lives, and their perspectives on approaches to treating tuberous sclerosis complex. - Xerostomia
In August 2021, the 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 hosted an externally-led Patient-Focused Drug Development meeting to hear directly from individuals living with xerostomia and their loved ones on the impact of xerostomia on their daily lives, and their perspectives on approaches to treating xerostomia.
The proposed draft guidance relating to patient experience data that are listed here have been drafted and submitted by external stakeholders. As with other resources on the webpage, the proposed draft guidance and their content are not endorsed by FDA, and posting a link does not mean FDA has decided to adopt the proposed draft guidance.
Natural history studies track the course of disease over time, identifying demographic, genetic, environmental, and other variables that correlate with its development and outcomes in the absence of treatment. Website links to other publicly-available reports or documents providing disease-specific background on the condition and unmet medical need may also be found here.
- Amyotrophic Lateral Sclerosis (ALS)
In October 2019, the ALS Association issued the ALS Voice of the Patient report based on the IMPACT ALS survey. The survey gathered information about the functional burden of ALS and unmet needs related to treating those burdens. - Hunter Syndrome (MPS II)
In July 2021, Project Alive issued the Hunter Syndrome Voice of the Patient Report based on the Toileting Ability Survey. The survey gathered cognitive and behavioral data to better understand the impact of neurocognitive decline.
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