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National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Health Sciences Policy; Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union; Shore CK, Worku TL, Smith CW, et al., editors. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington (DC): National Academies Press (US); 2024 Oct 30.
Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities.
Show details| Disease/Condition Name | Meeting Type | Meeting Date |
|---|---|---|
| Narcolepsy | FDA-led PFDD meeting | 9/24/2013 |
| Sickle cell disease | FDA-led PFDD meeting | 2/7/2014 |
| Pulmonary arterial hypertension (PAH) | FDA-led PFDD meeting | 5/13/2014 |
| Hemophilia A, hemophilia B, von Willebrand disease (VWB), and other heritable bleeding disorders | FDA-led PFDD meeting | 9/22/2014 |
| Idiopathic pulmonary fibrosis (IPF) | FDA-led PFDD meeting | 9/26/2014 |
| Huntington’s disease (HD) | FDA-led PFDD meeting | 9/22/2015 |
| Alpha-1 antitrypsin deficiency | FDA-led PFDD meeting | 9/29/2015 |
| Non-tuberculous mycobacterial (NTM) lung infections | FDA-led PFDD meeting | 10/15/2015 |
| Amyloidosis | EL-PFDD meeting Host: The Amyloidosis Research Consortium | 11/16/2015 |
| Myotonic dystrophy (DM) | EL-PFDD meeting Host: Myotonic Dystrophy Foundation | 9/15/2016 |
| Spinal muscular atrophy (SMA) | EL-PFDD meeting Host: Cure SMA | 4/18/2017 |
| Friedreich’s ataxia (FA) | EL-PFDD meeting Host: Friedreich’s Ataxia Research Alliance | 6/2/2017 |
| Tuberous sclerosis complex (TSC) | EL-PFDD meeting Host: Tuberous Sclerosis Alliance | 6/21/2017 |
| Alopecia areata | FDA-led PFDD meeting | 9/11/2017 |
| Hereditary angioedema (HAE) | FDA-led PFDD meeting | 9/25/2017 |
| Pachyonychia congenita (PC) | EL-PFDD meeting Host: Pachyonychia Congenita Project | 4/6/2018 |
| Barth syndrome (BTHS) | EL-PFDD meeting Host: The Barth Syndrome Foundation | 7/18/2018 |
| Alport syndrome | EL-PFDD meeting Host: National Kidney Foundation and the Alport Syndrome Foundation | 8/3/2018 |
| Charcot-Marie-Tooth and inherited neuropathies (CMT/IN) | EL-PFDD meeting Host: Hereditary Neuropathy Foundation | 9/28/2018 |
| Fabry disease | Patient listening session | 12/4/2018 |
| Niemann-Pick type C (NPC) | 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 | 3/18/2019 |
| Fibrodysplasia ossificans progressiva (FOP) | Patient Listening session | 5/29/2019 |
| Neurofibromatosis (NF) | Patient Listening session | 6/13/2019 |
| Immune thrombocytopenia (ITP) | EL-PFDD meeting Host: Platelet Disorder Support Association | 7/26/2019 |
| IgA nephropathy (IgAN) | EL-PFDD meeting Host: National Kidney Foundation and the IgA Nephropathy Foundation of America | 8/19/2019 |
| Osteogenesis imperfecta (OI) | Patient listening session | 9/17/2019 |
| Pyruvate kinase deficiency | EL-PFDD meeting Host: National Organization for Rare Disorders (NORD) | 9/20/2019 |
| Cerebral cavernous malformation (CCM) | Patient listening session | 11/6/2019 |
| Gastroparesis | Patient listening session | 12/2/2019 |
| Ocular melanoma (OM) | Patient listening session | 1/27/2020 |
| Von Hippel Lindau (VHL) | Patient listening session | 6/11/2020 |
| Homocystinuria (HCU) | Patient listening session | 6/26/2020 |
| Pulmonary alveola proteinosis (PAP) | Patient listening session | 7/8/2020 |
| Pompe disease | EL-PFDD meeting Host: Muscular Dystrophy Association | 7/13/2020 |
| Progressive multifocal leukoencephalopathy (PML) | Patient listening session | 7/22/2020 |
| Smith-Magenis syndrome (SMS) | Patient listening session | 8/12/2020 |
| Focal segmental glomerulosclerosis (FSGS) | EL-PFDD meeting Host: National Kidney Foundation and NephCure Kidney International | 8/28/2020 |
| Guillain-Barre’ syndrome (GBS) | Patient listening session | 9/29/2020 |
| Primary hyperoxaluria (PH) | EL-PFDD meeting Host: The Oxalosis and Hyperoxaluria Foundation | 10/5/2020 |
| Systemic sclerosis | FDA-led PFDD meeting | 10/13/2020 |
| Limb-girdle muscular dystrophies (LGMD) | Patient listening session | 10/20/2020 |
| Primary sclerosing cholangitis (PSC) | EL-PFDD meeting Host: PSC Partners Seeking a Cure | 10/23/2020 |
| Gorlin syndrome (GS) | Patient listening session | 11/9/2020 |
| SYNGAP1 | EL-PFDD meeting Host: SYNGAP1 Foundation | 11/19/2020 |
| Acromegaly | EL-PFDD meeting Host: Acromegaly Community, Inc | 1/21/2021 |
| Pemphigus and pemphigoid | Patient listening session | 2/8/2021 |
| Fragile X syndrome (FXS) | EL-PFDD meeting Host: National Fragile X Foundation | 3/3/2021 |
| Frontotemporal degeneration (FTD) | EL-PFDD meeting Host: Association for Frontotemporal Degeneration (AFTD) | 3/5/2021 |
| Glycogen storage disease (GSD) type 1 – adult patients | Patient listening session | 3/18/2021 |
| Glycogen storage disease (GSD) type 1 – caregivers to pediatric patients | Patient listening session | 3/25/2021 |
| Cystic fibrosis (PM – nonsense mutations | Patient listening session | 7/15/2021 |
| Cerebrotendinous xanthomatosis (CTX) | EL-PFDD meeting Host: United Leukodystrophy Foundation of America | 9/14/2021 |
| Ichthyosis | Patient listening session | 9/17/2021 |
| Mastocytosis | Patient listening session | 9/28/2021 |
| Gorlin syndrome (GS) | EL-PFDD meeting Host: Gorlin Syndrome Alliance | 10/8/2021 |
| Hypothalamic obesity (HO) | Patient listening session | 10/22/2021 |
| Adult polyglucosan body disease | Patient listening session | 10/28/2021 |
| Thymidine kinase 2 deficiency (TK2) | Patient listening session | 1/31/2022 |
| Dravet syndrome (DS) | EL-PFDD meeting Host: The Dravet Syndrome Foundation | 2/3/2022 |
| Primary biliary cholangitis (PBC) | EL-PFDD meeting Host: Global Liver Institute | 2/4/2022 |
| Glycogen storage disease (GSD) type 1B | Patient listening session | 3/3/2022 |
| Rett syndrome | EL-PFDD meeting Host: Rett Syndrome Research Trust (RSRT) | 3/11/2022 |
| Chronic inflammatory demyelinating polyneuropathy (CIDP) | EL-PFDD meeting Host: GBS|CIDP Foundation International | 3/25/2022 |
| Congenital muscular dystrophy (CMD) | EL-PFDD meeting Host: Cure CMD | 7/1/2022 |
| Succinic semialdehyde dehydrogenase deficiency (SSADHD) | EL-PFDD meeting Host: SSADH Association | 7/8/2022 |
| Short bowel syndrome (SBS) | Patient listening session | 7/19/2022 |
| X-linked adrenoleukodystrophy (ALD) | EL-PFDD meeting Host: ALD Connect | 7/22/2022 |
| Huntington’s disease (HD) presymptomatic population | Patient listening session | 7/25/2022 |
| Spinal muscular atrophy (SMA) | Patient listening session | 8/4/2022 |
| Narcolepsy and idiopathic hypersomnia (IH) | Patient listening session | 8/8/2022 |
| Galactosemia | EL-PFDD meeting Host: National Organization for Rare Disorders (NORD) | 9/1/2022 |
| Fabry disease | EL-PFDD meeting Host: National Kidney Foundation and Fabry Support & Information Group | 9/19/2022 |
| Alström syndrome | EL-PFDD meeting Host: Alstrom International | 9/22/2022 |
| 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 | 9/23/2022 |
| Metachromatic leukodystrophy (MLD) | EL-PFDD meeting Host: Cure MLD, The Calliope Joy Foundation, MLD Foundation, The United Leukodystrophy Foundation, and the Global Leukodystrophy Initiative | 10/21/2022 |
| Phelan-McDermid syndrome (PMS) | EL-PFDD meeting Host: CureSHANK | 11/8/2022 |
| Kennedy’s disease (KD)/spinal and bulbar muscular atrophy (SBMA) | EL-PFDD meeting Host: Kennedy’s Disease Association | 11/9/2022 |
| Hypophosphatasia (HPP) | EL-PFDD meeting Host: Soft Bones, Inc., The US Hypophosphatasia Foundation | 11/15/2022 |
| Proteus syndrome | Patient listening session | 12/1/2022 |
| Cerebral creatine deficiency syndromes (CCDS) | EL-PFDD meeting Host: Association for Creatine Deficiencies | 1/24/2023 |
| Pemphigus and pemphigoid | EL-PFDD meeting Host: International Pemphigus and Pemphigoid Foundation | 1/25/2023 |
| Autoimmune hepatitis (AIH) | EL-PFDD meeting Host: Autoimmune Hepatitis Association | 1/27/2023 |
| Wiskott-Aldrich syndrome (WAS) | EL-PFDD meeting Host: Wiskott-Aldrich Foundation | 2/3/2023 |
| Bronchopulmonary dysplasia (BPD) | Patient listening session | 3/20/2023 |
| Sickle cell disease | Patient listening session | 5/5/2023 |
| Prader-Willi syndrome (PWS) | EL-PFDD meeting Host: PWSA|USA | 6/22/2023 |
| Pyruvate dehydrogenase complex deficiency (PDCD) | Patient listening session | 9/8/2023 |
| Atypical hemolytic uremic syndrome (aHUS) | Patient listening session | 9/21/2023 |
| Spinocerebellar ataxia type 3 (SCA3) | Patient listening session | 9/22/2023 |
| Amyotrophic lateral sclerosis (ALS) | N/A | N/A |
SOURCE: U.S. Food and Drug Administration. 2024. Condition-specific meeting reports and other information related to patients’ experience. https://www.fda.gov/industry/prescriptiondrug-user-fee-amendments/condition-specific-meeting-reports-and-other-information-relatedpatients-experience (accessed August 15, 2024).
Footnotes
- 1
For more information on these meetings such as summaries and agendas, see FDA (2024k): https://www
.fda.gov/industry /prescription-drug-user-fee-amendments /condition-specific-meetingreports-and-other-information-related-patients-experience (accessed July 1, 2024).
- Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Lis...Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023 - Regulatory Processes for Rare Disease Drugs in the United States and European Union
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