(online conference – CEST Time Zone)
| Gene and Cell Therapies: Hope and Realities of Advances in Regenerative Medicine |
| A Community Partnership to Expand Access to Gene and Cell Therapies |
| Day 1: February 6, 2023: Experiences of Patients and Health Care Providers |
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| 13:45 – 14:00 |
Welcome and Introduction to the Conference |
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Domenica Taruscio. President of ICORD and National Centre for Rare Diseases, Istituto Superiore di Sanità, Italy |
Session 1
14:00 – 15:00
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Managing Expectations and Education on Gene Therapy Treatments
Session Chairs: Cynthia Tifft and Manuel Posada |
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| 14:00-14:15 |
The Clinical Researcher Investigator – Fears, Feelings, and Concerns Prior to Initiating Therapy – Cynthia Tifft. Deputy Clinical Director. National Human Genome Research Institute. Director, Pediatric Undiagnosed Diseases Program. National Institutes of Health
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| 14:15-14:30 |
Patients and Family Perspectives and Experiences with Before, During and After Receiving Gene Therapy. Niclas Flysjo and Jessica Lindqvist. Family |
| 14:30-14:45 |
Clinical Nurse Experiences Before and After Treatment. Precilla D’Souza. National Institutes of Health Undiagnosed Diseases Program, Common Fund, National Institutes of Health |
| 14:45-15:00 |
Panel Questions and Answers |
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Session 2
15:00 – 16:00
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Novel Approaches to Gene Therapy and Scalability to Entire Population
Session Chairs: Emilio Roldan and Stephen Groft |
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| 15:00-15:15 |
Platform Vector Gene Therapy Pilot Project (PaVe GT). PJ Brooks, Acting Director, Division of Rare Diseases Research Innovation, NCATS NIH |
| 15:15-15:30 |
Bespoke Gene Therapies and Shared Molecular Etiologies. PJ Brooks, Acting Director, Division of Rare Diseases Research Innovation, NCATS NIH |
| 15:30-15:45 |
Strategies for advancing the story for families with n=1 disorders. David Adams, Co-Director NIH Undiagnosed Diseases Program, NHGRI, NIH |
| 15:45-16:00 |
Panel Questions and Answers |
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| 16:00 – 16:15 |
Short Break |
Session 3
16:15 – 17:15 |
Ethic issues and advances therapies.
Session Chairs: Mireia del Toro and Domenica Taruscio |
|
| 16:15-16:30 |
South American vision of access and ethics on gene therapies. Graciela Sara Moya Catholic University of Buenos Aires |
| 16:30-16:45 |
Ethical Issues Surrounding Expanded Access to Cell & Gene Therapy. Mats G. Hansson. Senior Professor of Biomedical Ethics. Centre for Research Ethics and Bioethics at Uppsala University, Sweden
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| 16:45-17:00 |
Access to Therapies: A Family and Non-profit Perspective—Andrew McFadyen, Executive Director. The Isaac Foundation |
| 17:00-17:15 |
Panel Questions and Answers |
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Session 4
17:15 – 18:15 |
Education and Training to Meet Community Needs and Managing Outcome Expectations.
Session Chair: Helene Cederroth and UDNI and Domenica Taruscio |
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| 17:15-17:30 |
Staff who Treat Patients and Monitor Outcomes Some from Japan – Yukiko Nishimura. President, NPO ASrid, Japan |
| 17:30-17:45 |
Perspectives and Needs of Patients/Families and Patient Advocacy Groups – Dr. Jesús Navarro Torres. Vicepresidente de la Asociación Iberoamericana de Enfermedades Raras o poco Frecuentes (ALIBER)
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| 17:45-18:00 |
Perspectives and Needs of undiagnosed Patients/Families. Helene Cederroth Wilhelm Foundation and UDNI |
| 18:00-18:15 |
Panel Questions and Answers |
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| End of the day |
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| Scientific and Regulatory Issues to Increase Community Access to Gene and Cell Therapies |
| Day 2: February 7, 2023: Regulatory Issues and Requirements |
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Session 5
14:00 – 15:00
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Perspectives on Meeting Patient and Medical Needs for Gene and Cell Therapies
Session Chairs: Simon Day and Wendy White |
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| 14:00-14:15 |
Global Access to Orphan Products for Rare Diseases focused on AT – W. Gahl. Medical Genetics Branch, National Human Genome Research Institute, National Institutes of Health, USA. |
| 14:15-14:30 |
Research Advances and Successful Efforts; Future Research and Development Activities – Samantha Parker. Vice-chair of IRDiRC |
| 14:30-14:45 |
Industry Perspectives on Regulatory Issues and Requirements – Kinnari Patel. President and COO of Rocket Pharmaceuticals |
| 14:45-15:00 |
Panel Questions and Answers |
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Session 6
15:00 – 16:00 |
Increasing Global Access to Interventions
Session Chairs: David Pearce and Luis Alejandro Barrera |
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| 15:00-15:15 |
Moving from Clinical Trials to Treatment and Global Access – Marc Dooms. Senior orphan drug pharmacist, University Hospitals Leuven |
| 15:15-15:30 |
Country/Regional Approaches to Manufacturing – Ramaiah Muthyala. President, Indian Organization for Rare Diseases, Hyderabad, Telangana INDIA. Associate Professor. Experimental Clinical Pharmacology, University of Minnesota |
| 15:30-15:45 |
Global Distribution, Supply, and Storage Systems – David Pearce. President of Innovation, Research & World Clinics, Sanford Health Scientist, Pediatrics and Rare Diseases Group. Professor, Department of Pediatrics, Sanford School of Medicine of the University of South Dakota |
| 15:45-16:00 |
Panel Questions and Answers |
|
| 16:00 – 16:15 |
Short Break |
| 16:15 – 16:30 |
Summary of the IX International HTA Symposium “IMPACT OF EU HTA REGULATION ON RARE DISEASES AND ORPHAN DRUGS”
Rumen Stefanov. President-Elect of ICORD, Director of Institute for Rare Diseases, Bulgaria |
| 16:30 – 16:40 |
Summary of the ICORD webinar “HOW CAN ONE HEALTH APPROACHES HELP RARE DISEASES?”.
Domenica Taruscio, President of ICORD and National Centre for Rare Diseases, Istituto Superiore di Sanità, Italy
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| 16:40 |
Wrap up and summary
Stephen Groft, Cynthia Tifft, and Simon Day |
| 17:00 |
End of the conference |
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