Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities (2024)
Chapter: Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023
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Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Appendix F
Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 20231
| 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 |
___________________
1For 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).
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Disease/Condition Name | Meeting Type | Meeting Date |
|---|---|---|
| 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 |
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Disease/Condition Name | Meeting Type | Meeting Date |
|---|---|---|
| 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 |
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Disease/Condition Name | Meeting Type | Meeting Date |
|---|---|---|
| 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 |
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Disease/Condition Name | Meeting Type | Meeting Date |
|---|---|---|
| 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 |
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
| Disease/Condition Name | Meeting Type | Meeting Date |
|---|---|---|
| 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).
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
Suggested Citation:
"Appendix F: Non-Exhaustive List of Patient Focused Drug Development Meetings and Patient Listening Sessions for Rare Diseases Between 2013 and 2023." National Academies of Sciences, Engineering, and Medicine. 2024. Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities. Washington, DC: The National Academies Press.
doi: 10.17226/27968.
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