Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities (2024)
Chapter: Appendix C: Public Meeting Agendas
Appendix C
Public Meeting Agendas
COMMITTEE ON PROCESSES TO EVALUATE THE SAFETY AND EFFICACY OF DRUGS FOR RARE DISEASES IN THE UNITED STATES AND THE EUROPEAN UNION
Meeting #1: November 6 – 7, 2023: Public Agenda
MONDAY, NOVEMBER 6, 2023 2:30 – 4:30 PM: OPEN SESSION
| 2:30–2:35 pm | Welcome and Introductions |
|
JEFFREY KAHN, Committee Chair
Andreas C. Dracopoulos Director Robert Henry Levi and Ryda Hecht Levi Professor of Bioethics and Public Policy John Hopkins Berman Institute of Bioethics |
|
| 2:35–3:30 pm | Sponsor Perspective and Charge to the Committee |
|
KERRY JO LEE
Associate Director for Rare Diseases, Division of Rare Diseases and Medicine Genetics |
|
Office of Rare Diseases, Pediatrics, Urological, and Reproductive Medicines Office of New Drugs Center for Drug Evaluation and Research U.S. Food and Drug Administration |
|
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SANDRA RETZKY
Director, Office of Orphan Product Development Office of the Commissioner U.S. Food and Drug Administration |
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MIRANDA RAGGIO
Expedited Programs Manager, Office of Program Operation Office of New Drugs Center for Drugs Evaluation and Research U.S. Food and Drug Administration |
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JULIENNE VAILLANCOURT
Policy Advisor and Rare Disease Liaison Office of the Director Center for Biologics Research and Evaluation U.S. Food and Drug Administration |
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KATHERINE TYNER
FDA Liaison to the European Medicines Agency, Europe Office Office of Global Policy and Strategy Office of the Commissioner U.S. Food and Drug Administration |
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SARAH ZAIDI
Physician Liaison for Pediatric Cluster, Pediatric International Team Office of Pediatric Therapeutics Office of Clinical Policy and Programs Office of the Commisssioner U.S. Food and Drug Administration |
| Other Sponsor Stakeholders on Standby for Q&A | |
|
JUDITH ARCIDIACONO
International Regulatory Expert, Office of Therapeutic Products Center for Biologics Research and Evaluation U.S. Food and Drug Administration |
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ROBYN BENT
Director, Patient-Focused Drug Development Program Center for Drug Evaluation and Research U.S. Food and Drug Administration |
|
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KEVIN FAIN
Senior Policy Advisor, Office of New Drug Policy Office of New Drugs Center for Drug Research and Evaluation U.S. Food and Drug Administration |
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DIONNE L. PRICE
Deputy Director, Office of Biostatistics Office of Translational Sciences Center for Drug Evaluation and Research U.S. Food and Drug Administration |
|
| 3:30–4:30 pm | Discussion with Committee |
| 4:30 pm | ADJOURN MEETING DAY 1 |
TUESDAY, NOVEMBER 7, 2023
10:00 AM – 1:00 PM EST: OPEN SESSION
| 10:00 am | Welcome and Introductions |
|
JEFFREY KAHN, Committee Chair
Andreas C. Dracopoulos Director Robert Henry Levi and Ryda Hecht Levi Professor of Bioethics and Public Policy Johns Hopkins Berman Institute of Bioethics |
| 10:05–10:30 am | Trends in Rare Disease Drug Product Approvals and Utilization of Regulatory Pathways |
|
ANNA SOMUYIWA
Head Centre for Innovation in Regulatory Science |
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MAGDA BUJAR
Senior Manager, Regulatory Programme and Strategic Partnerships Centre for Innovation in Regulatory Science |
|
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JUAN LARA
Research Analyst Centre for Innovation in Regulatory Science |
|
| 10:30–11:00 am | Discussion with Committee |
| 11:00–11:15 am | BREAK |
| 11:15 am –12:30 pm | Perspectives from Rare Disease Organizations |
|
VIRGINIE HIVERT
Therapeutic Development Director EURODIS |
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SAIRA SULTAN
Consultant Haystack Project |
|
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ANNIE KENNEDY
Chief of Policy, Advocacy, and Patient Engagement EveryLife Foundation |
|
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KARIN HOELZER
Director of Policy and Regulatory Affairs NORD |
|
| 12:30–12:45 pm | Public Comment Public comments will provide the committee with additional insight into key issues related to the study’s statement of task. These include, but are not limited to: |
| The use of regulatory flexibilities and supplementary data (e.g. natural history studies and patient registries) when evaluating the safety and efficacy of drugs for rare diseases and conditions; and FDA and EMA engagement of people with lived experience when developing guidance, policies, and programs. | |
| Public comments, alongside other materials stakeholders have shared, will be reviewed by the committee and may help inform committee deliberations on the statement of task. All comments and materials shared with the committee will be made publicly available in accordance with institutional policies. As such, please do not send confidential or HIPAA protected information and take caution when including personally identifiable information. Should a quote from your public comment be used word-for-word in the committee’s final report, you will be contacted by study staff. | |
| If you would like to provide a verbal comment, please limit remarks to 2-3 minutes. Requests to provide verbal public comments may submitted via the meeting registration page here. Public commenters will be added to the agenda based on the order in which requests are received. Please note that space is limited and not all requests may be fulfilled. You may also submit a written public comment via email: RareDiseaseregPolicyStudy@nas.edu. | |
| Public comments made at meetings and submitted in writing are subject to the same institutional disclosure requirements. | |
| 12:45pm | ADJOURN OPEN SESSION |
COMMITTEE ON PROCESSES TO EVALUATE THE SAFETY AND EFFICACY OF DRUGS FOR RARE DISEASES IN THE UNITED STATES AND THE EUROPEAN UNION
Meeting #2: December 4 – 5, 2023: Committee Agenda
MONDAY, DECEMBER 4, 2023
Open SESSION (10:00 AM – 1:30 PM ET)
| 10:00–10:10 am |
Welcome and Introduction
JEFFREY KAHN, Committee Chair Andreas C. Dracopoulos Director Robert Henry Levi and Ryda Hecht Levi Professor of Bioethics and Public Policy Johns Hopkins Berman Institute of Bioethics |
| 10:10–11:15 am |
EMA Regulatory Policies for Drugs to Treat Rare Diseases and Conditions
STEFFEN THIRSTRUP Chief Medical Officer European Medicines Agency |
Committee Discussion (30 min)
| 11:15 am–12:00 pm | Panel Discussion: Similarities and Differences Between FDA and EMA |
|
STEFFEN THIRSTRUP
Chief Medical Officer European Medicines Agency |
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JACQUELINE CORRIGAN-CURAY
Principal Deputy Center Director Center for Drug Evaluation and Research U.S. Food and Drug Administration |
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CELIA WITTEN
Deputy Director Center for Biologics Evaluation and Research U.S. Food and Drug Administration |
| 12:00–12:15 pm | BREAK |
| 12:15–1:15 pm | Industry Perspectives on the Application of Regulatory Flexibilities |
|
LUCY VERESHCHAGINA
Senior Vice President, Science and Regulatory Advocacy PhRMA |
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DIEGO ARDIGÒ
Head of R&D, Global Rare Diseases Cheisi |
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E’LISSA FLORES
Director, Science and Regulatory Affairs BIO |
|
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VICTOR MAERTENS
Government Affairs Director EUCOPE |
Committee Discussion (20 min)
| 1:15–1:30 pm | Public Comment |
| 1:30 pm | ADJOURN OPEN SESSION |
TUESDAY, DECEMBER 5, 2023
OPEN SESSION (10 AM – 12:30 PM ET)
| 10:00–10:05 am | Welcome and Introductions |
|
JEFFREY KAHN, Committee Chair
Andreas C. Dracopoulos Director Robert Henry Levi and Ryda Hecht Levi Professor of Bioethics and Public Policy Johns Hopkins Berman Institute of Bioethics |
| 10:05–11:00 am | Use of “Supplemental Data” for Regulatory Decision making |
|
Case Study: Skyclarys approval for Friedreich’s ataxia (Use of natural history data)
COLIN MEYER (Formerly at Reata Pharmaceuticals Inc.) Biogen |
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Case Study: Elevidys approval for Duchenne’s muscular dystrophy (Challenges of regulatory review without “supplemental data”)
HUONG HUYNH Director, Regulatory Science Critical Path Institute |
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Case Study: Relyvrio approval for ALS (Use of natural history and open label extension [active treatment extension] data)
SABRINA PAGANONI Physician Scientist Healey & AMG Center for ALS |
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Case Study: Oxbyrta approval for Sickle Cell Disease (Use of natural history data)
LAKIEA BAILEY Executive Director Sickle Cell Community Consortium |
Committee Discussion (20 min)
| 11:00–11:15 am | BREAK |
| 11:15 am–12:00 pm | Trends in Designation and Approvals of Drugs for Rare Diseases and Conditions |
|
SANDRA RETZKY
Director, Office of Orphan Product Development Office of the Commissioner U.S. Food and Drug Administration |
|
LEWIS FERMAGLICH
Medical Officer, Office of Orphan Product Development Office of the Commissioner U.S. Food and Drug Administration |
Committee Discussion (15 min)
| 12:00–12:30 pm | Impact of FDA and EMA Collaborative Efforts |
|
KERRY JO LEE
Associate Director for Rare Diseases, Division of Rare Diseases and Medicine Genetics Office of Rare Diseases, Pediatrics, Urological, and Reproductive Medicines Office of New Drugs Center for Drug Evaluation and Research U.S. Food and Drug Administration |
|
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KATHERINE TYNER
FDA Liaison to the European Medicines Agency, Europe Office Office of Global Policy and Strategy Office of the Commissioner U.S. Food and Drug Administration |
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STEFFEN THIRSTRUP
Chief Medical Officer European Medicines Agency |
Committee Discussion (15 min)
| 12:30 pm | ADJOURN OPEN SESSION |
CLOSED SESSION (1:30 – 4:00 PM ET) – COMMITTEE MEMBERS ONLY
| 4:00 pm | ADJOURN MEETING |
COMMITTEE ON PROCESSES TO EVALUATE THE SAFETY AND EFFICACY OF DRUGS FOR RARE DISEASES IN THE UNITED STATES AND THE EUROPEAN UNION
Meeting #3: February 6 – 7, 2024: Committee Agenda
TUESDAY, FEBRUARY 6, 2024
OPEN SESSION (10:00 AM – 2:30 PM ET)
| 10:00–10:05 am | Welcome and Introduction |
|
JEFFREY KAHN, Committee Chair
Andreas C. Dracopoulos Director Robert Henry Levi and Ryda Hecht Levi Professor of Bioethics and Public Policy Johns Hopkins Berman Institute of Bioethics |
|
| 10:05–11:00 am |
Trial Design for Rare Disease Drug Development
LONGITUDINAL TRIAL DESIGN TIINA URV Director, Rare Disease Clinical Trial Network National Center for Advancing Translational Sciences National Institutes of Health |
|
MASTER PROTOCOLS NICOLE MAYER HAMBLETT Associate Professor of Pediatrics & Adjunct Associate Professor of Biostatistics, University of Washington Co-Executive Director, Cystic Fibrosis Therapeutics Development Network Coordinating Center |
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EXTERNAL CONTROLS WILLIAM MAIER Vice President, Rare Diseases ICON plc |
Committee Discussion (30 min)
| 11:00 am – 12:00pm | Considerations for Pediatric Trials |
|
INDUSTRY PERSPECTIVE THOMAS MILLER Global Head of the Acute, Chronic, and Pediatric Disease Nucleus Bayer, Pharmaceutical Division |
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CAREGIVER PERSPECTIVE KARA BERASI CEO, Haystack Project Vice Chair of Board of Directors, CDG Care (Congenital Disorders of Glycosylation) |
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REGULATORY SCIENCE PERSPECTIVE FLORENCE BOURGEOIS Associate Professor of Pediatrics, Harvard Medical School Co-Director, Harvard-MIT Center for Regulatory Science Director, Pediatric Therapeutics and Regulatory Science Initiative, Boston Children’s Hospital |
Committee Discussion (30 min)
| 12:00–1:00 pm | LUNCH BREAK |
| 1:00-2:00 pm | Use of “Supplemental” Data |
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USE OF AGGREGATE DATA KLAUS ROMERO Chief Executive Officer & Chief Science Officer Critical Path Institute |
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Expanded Access Programs
Alison Bateman-House Assistant Professor, Department of Population Health New York University Grossman School of Medicine |
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PATIENT REGISTRIES AND NATURAL HISTORY DATA EDWARD NEILAN Chief Medical and Scientific Officer National Organization for Rare Disorders |
Committee Discussion (30 min)
| 2:00 pm | ADJOURN OPEN SESSION |
WEDNESDAY, FEBRUARY 7, 2024
OPEN SESSION (9:30 AM – 12:15 PM ET)
| 9:30–9:35 am | Welcome and Introductions |
|
JEFFREY KAHN, Committee Chair
Andreas C. Dracopoulos Director Robert Henry Levi and Ryda Hecht Levi Professor of Bioethics and Public Policy Johns Hopkins Berman Institute of Bioethics |
|
| 9:35–10:30 am |
Novel Methodologies
ANALYSIS METHODS – BAYESIAN METHODS AND SMART DESIGN KELLEY KIDWELL Professor of Biostatistics University of Michigan |
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Analysis Methods – Causal Inference
Xabier Garcia de Albinez Martinez Director of Epidemiology, RTI Health Solutions Visiting Scientist, Department of Epidemiology, Harvard T.H. Chan School of Public Health |
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MODEL-INFORMED DRUG DEVELOPMENT
Hao Zhu Division Director Division of Pharmacometrics, Office of Combination Products, Office of Translational Sciences Center for Drug Evaluation and Research U.S. Food and Drug Administration |
Committee Discussion (30 min)
| 10:30–10:45 am | BREAK |
| 10:45–12:00 pm | Flexibilities Applied at FDA |
|
EMILY FREILICH
Division Director of Division of Neurology I Center for Drug Evaluation and Research U.S. Food and Drug Administration |
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RACHAEL ANATOL
Deputy Director of Office of Therapeutic Products Center for Biologics Evaluation and Research U.S. Food and Drug Administration |
|
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MARTHA DONOGHUE
Associate Director of Pediatric Oncology and Rare Cancers Oncology Center of Excellence U.S. Food and Drug Administration |
|
| 12:00–12:15pm | Public Comment |
| 12:15 pm | ADJOURN OPEN SESSION |
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