More information on the Chrysalis Task Force here: https://irdirc.org/chrysalis-project/

« It was a real pleasure working with such a talented IRDiRC Task Force. We also truly appreciate the critical input provided by the survey and interview respondents. All of them provided specific yet nuanced responses that were framed in an important general context. Their responses allowed the Task Force to compose and frame what I consider to be an extremely informative report. » says Adam L. Hartman, MD, Co-chair of the Chrysalis Task Force and Program Director at the National Institute of Neurological Disorders and Stroke (NINDS) part of U.S. National Institutes of Health (NIH)

This session will take place between December 2023 and May 2024 (written exam in June and oral in October 2024).

If you would like to apply, please send your CV and covering letter to diumyologie@institut-myologie.org by 27/10/2023.

For more information: https://www.institut-myologie.org/enseignement/diu-de-myologie/ (French)

The new RSC “brings broad expertise across different stakeholders and geographies allowing for a balanced synergy that will provide insights and guidance into the advancement of IRDiRCs mission” said David A. Pearce, Chair of IRDiRC. 

Read the full press release here: https://irdirc.org/irdirc-announces-creation-of-regulatory-science-committee/

• First therapy to treat two types of Niemann-Pick disease, a rare genetic metabolic disorder: Xenpozyme (olipudase alfa), a therapy for the treatment of non-central nervous system (CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), a rare and progressive genetic disease. Xenpozyme is indicated for use in children and adults of all ages with type A/B or type B ASMD. Historically referred to as Niemann-Pick disease types A (NPD A) and B (NPD B), ASMD is a genetic disorder.

• First treatment for children with Progeria or progeroid like syndromes (rare premature aging syndromes): Zokinvy (lonafarnib), a treatment for patients with a genetically confirmed diagnosis of Hutchinson-Gilford Progeria Syndrome or progeroid laminopathies. Zokinvy is indicated for use in children one year of age and older.Hutchinson-Gilford Progeria Syndrome is an ultra-rare multi-systemic ‘premature aging’ disease.

• First therapy to treat rare genetic nervous system disorder AADC deficiency: Upstaza (eladocagene exuparvovec), a therapy for the treatment of adult and paediatric patients with severe aromatic L-amino acid decarboxylase (AADC) deficiency with a genetically confirmed diagnosis. AADC deficiency is an ultra-rare, inherited genetic disease which typically manifests within the first year of life.

• First gene therapy to treat severe haemophilia A: Roctavian (valoctocogene roxaparvovec) for the treatment of severe haemophilia A in adults who do not have factor VIII inhibitors (auto-antibodies produced by the immune system which make factor VIII medicines less effective) and no antibodies to adeno-associated virus serotype 5 (AAV5). Patients with haemophilia A cannot produce factor VIII (an essential protein required for blood to clot and stop bleeding); they are more prone to bleeding and have prolonged bleeding, e.g. after injury or surgery. Haemophilia A is a rare debilitating disease affecting approximately 0.7 in 10,000 people in the EU.

To function properly, the EHDS will need sustainable organisational and resource arrangements at EU and national, and in some cases regional, levels. Funding is one of the most important of these arrangements. TEHDAS has called for a sustainability plan to address these questions as a prerequisite for the successful launch and running of the EHDS.

The report concludes that both EU and national and local funds will be needed to build a European data access and sharing system.

Data cannot be shared in the EU without effective systems for data collection and access in member states. While the European coordination needs to be funded from the EU budget, member states will incur expenses as governments will have to develop their national capacity to join the European data exchange and benefit from using data.

The report highlights many complex questions in reaching the economic sustainability of the EHDS. For example, often-used project-based EU funding is not predictable and needs substantial resources from the member states.

The infrastructures that will be developed as part of the EHDS need to be constantly maintained and co-ordinated. This requires permanent resource allocations and funding similar to the data sharing initiatives managed and maintained by EU agencies.

The report is based on an analysis of the funding of six existing EU health data sharing mechanisms. It gives an overview of the EU’s funding instruments relevant to the secondary use of health data and looks at the country experiences.

TEHDAS will continue to study the economic sustainability of the secondary use of health data and a final report will be issued in early 2023.

For more information and to download the report: https://tehdas.eu/results/tehdas-identifies-funding-options-for-secondary-use-of-health-data/

The deadline for providing feedback to the Draft SRIA is October 29^th, 2021.

The draft SRIA was prepared by the Working Group on SRIA and the contribution by selected experts. In particular, ERA4Health and its SRIA aim at:

• Supporting relevant medical research including clinical fields and interventions area (prevention, diagnosis and treatment).
• Conducting large, multinational non-commercial/pragmatic clinical trials within the (new) regulatory framework for clinical trials. ERA4Health will connect clinical research capacities and research infrastructures of high public health interest.

Relevant stakeholders include: decision makers, research and innovation funding bodies, relevant national and regional stakeholders and experts (e.g. Health and Care providers, Public Health experts, researchers, Health and Care Innovators, enterprises, civic and patient associations, Health and Care professionals and formal and informal carers associations).

More information: https://ec.europa.eu/eusurvey/runner/era4healthopenconsultation

The webinar will take place on September 7, 2021 from 16.00 – 17.00 CET.

The event will address whether EU cross-border access to advanced treatments is a reality or a goal for the future and the value of treatments for rare disease patients, followed by a presentation on gene therapies and their value for rare disease patients. The panel includes:

• Martin Dorazil, Deputy Head of Unit Digital Health, DG Health, European Commission
• Brieuc Van Damme, Director-General Healthcare, RIZIV-INAMI
• Dirk Vander Mijnsbrugge, Vice President Medical Affairs Lead Rare Diseases International Developed Markets, Pfizer
• Irina Grossenbacher, Region Europe Patient Access Director, Novartis Rare Diseases
• Tomislav Sokol, Menmber of the European Parliament, EPP, Croatia

More information and registration here: https://www.europabio.org/event/treatment-without-borders-the-eu-case-for-equitable-patient-access-to-advanced-therapies/