This episode explores how real-world evidence is changing healthcare, its value in improving patients’ outcomes, its role in supporting R&D in advanced therapy and uptake of this innovation by health systems. It will discuss issues such as how to deal with evidence gaps in HTA and the role of RWE. It will reflect on the importance of the European Health Data Space in leveraging RWE collected in Europe and its benefits for rare disease patients. It will also look at how to ensure that health data is efficiently leveraged to help the development of advanced therapy in a sustainable, patient-centric, manner, patient engagement in data collection efforts and the role of clinicians.
Guests:
Marcus Guardian (COO, EUnetHTA)
Prof. Marijke van den Berg (Director, European Paediatric Network for Haemophilia Management (PEDNET))
This episode explores the EU’s leadership in response to rare diseases and innovation in the area, with a special focus on advanced therapy research and development. What are the achievements made in Europe and where are the remaining gaps? How can Europe achieve leadership in innovation for rare diseases?
Guests:
Yann Le Cam, CEO, EURORDIS
Elisabetta Zanon, Director, EU Public Affairs & Advocacy, Alliance for Regenerative Medicine, Europe
This episode explores EU’s leadership in response to rare diseases and innovation in the area, with a special focus on advanced therapy research and development. What are the achievements made in Europe and where are the remaining gaps? How can Europe achieve leadership in innovation for rare diseases? The bonus edition provides insights about the impact of the COVID-19 pandemic on rare disease patients and innovation in the field.
Guests:
Yann Le Cam, CEO, EURORDIS
Elisabetta Zanon, Director, EU Public Affairs & Advocacy, Alliance for Regenerative Medicine, Europe
This conversation takes a deep dive into the ethical issues surrounding cell and gene therapies. What are the main questions from patients and the broader public? What are the key myths surrounding these therapies? It explores topics such as transparency, responsibility in science and research efforts, inclusivity & non-discrimination in access.
Guest:
- Hervé Chneiweiss, Chairman, INSERM Ethics Committee; Chair, UNESCO International Bioethics Committee; member, WHO Expert Advisory Committee on Developing Global Standards for Governance and Oversight of Human Genome Editing
This podcast examines patient centricity in cell and gene therapy, exploring how care must be planned and delivered to be responsive to individual patient preferences, needs and values. What exactly does patient focus mean, when talking about advanced therapies? What should patients know before embarking on the therapy and how do patient advocacy organisations support patients in this process?
Guest:
· Durhane Wong-Rieger, Chair, Rare Disease International Council
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Abbreviations:
· HTA: Health technology assessment
· ERNs: European reference networks
In this episode, we look at the unique policy challenges presented by cell and gene therapies from research & development through to patient access; and the role of multi-stakeholder collaborations, in particular those active in supporting European policymaking. How can we, together, deliver these innovative therapies to patients and society, efficiently and safely?
Guests:
• Amanda Bok, Chief Executive, European Haemophilia Consortium (EHC)
• Alexander Natz, CEO, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)
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Abbreviations:
• ATMPs: Advanced therapy medicinal products
• EBMT: European Society for Blood and Marrow Transplantation
• GBA: Gemeinsamer Bundesausschuss
• HTA: Health technology assessment
• MEPs: members of European Parliament
This episode provides an introduction to cell and gene therapies and touches upon the historical, scientific, clinical, policy and regulatory considerations surrounding their development and use, with forward-looking perspectives from our guests on the future of this new chapter of medicine.
Guests:
· Professor Luigi Naldini, Director, San Raffaele Telethon Institute for Gene Therapy
· Avril Daly, Vice-president, Rare Diseases Europe (EURORDIS); CEO, Retina International
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Abbreviations:
· CAR T cells: chimeric antigen receptor T cells
· HTA: Health technology assessment
This podcast examines patient centricity in cell and gene therapy, exploring how care must be planned and delivered to be responsive to individual patient preferences, needs and values. What exactly does patient focus mean, when talking about advanced therapies? What should patients know before embarking on this therapy and how do patient advocacy organisations support patients in this process?
Guest:
• Durhane Wong-Rieger, Chair, Rare Disease International Council
Abbreviations:
· HTA: Health Technology Assessment
· ERNs: European Reference Networks