Today, the Spanish Association of Orphan and Ultra Orphan Drug Laboratories (AELMHU) held the II National Conference "The Future of Advanced Therapies for Rare Diseases" in the Auditorium of the Camilo José Cela University.
This event, whose main objective was to promote dialogue and critical reflection among the different agents of the healthcare system, highlighted the development of advanced therapies for rare diseases and the need to work together to facilitate their availability and access.
The day, which had the director of AELMHU, Marian Corral, as master of ceremonies, began with welcoming remarks by Beatriz Perales, president of AELMHU, followed by the official inauguration by Raquel Yotti, commissioner of PERTE for Vanguard Health of the Ministry of Science, Innovation and Universities.
Key Topics and Featured Speakers
During the event, participants were able to discuss various relevant topics, including clinical advances in the field of advanced therapies, challenges in their evaluation, financing and public procurement, as well as possible economic models that can facilitate access to these treatments. Importance was given not only to the health and quality of life benefits for patients and their families, but also to the remarkable economic and social value of these therapies.
Sol Ruiz, head of the Division of Biological Products, Advanced Therapies and Biotechnology of the AEMPS, focused her speech on the current situation of advanced therapies, highlighting the difficulties of access to them.
Alvaro Hidalgo, health economist, professor and director of the Health Economics and Health Management Research Group at the University of Castilla La Mancha (UCLM), spoke about the economic models for facilitating access to these therapies.
Discussion Tables
The first Roundtable 'Evaluation, Financing and Public Procurement', moderated by Jorge Mestre, Health Economist, was made up of:
- José Luis Poveda, manager of the Hospital Universitari i Politècnic La Fe.
- Juan Manuel Fontanet, representative of the Medicines Area of the Catalan Health Service.
- Elena Casaus, head of the Division of the Advanced Therapies Unit of the Community of Madrid.
- Lluís Alcover, lawyer specialized in pharmaceutical law at Faus & Moliner Abogados.
This roundtable addressed relevant issues such as the obstacles and difficulties faced by evaluators when dealing with limited data or the evaluation of the economic value of advanced therapies, not only in terms of direct costs, but also in terms of long-term savings for the healthcare system.
The second Panel Discussion: Clinical Advances and Challenges, moderated by Borja Smith, CEO of BioInnova Consulting, was integrated by:
- Víctor Jiménez, head of the Hematology Department of the Hospital Universitario La Paz.
- Alessandra Magnani, head of the Platform for Advanced Therapies and Immunotherapy at the Hospital Sant Joan de Déu.
- Javier García, director of the Department of Drug Development of Advanced Therapies of the ISCIII.
Medical experts highlighted innovation as the main driver of advanced therapies. They also shared their views on the challenges in the regulatory approval process for new therapies and the current research projects and future directions in the field of advanced therapies.
Closing
By way of closing, Isabel Motero, director of the Spanish Federation of Rare Diseases (FEDER), attended on behalf of patients, stressing the importance of these therapies and their impact on the lives of those suffering from rare diseases.
Iván Silva, member of the Board of Directors of AELMHU, closed the day by thanking all the participants for their involvement and highlighting the success of the meeting and the need to establish collaboration and joint work, combining a multidisciplinary perspective to continue advancing in this field.
This event has reaffirmed AELMHU's commitment to continue promoting initiatives that facilitate collaboration between the different agents of the healthcare system, with the common goal of improving the quality of life of patients with rare diseases. It would not have been possible without the collaboration of its sponsors and partners BioMarin Pharmaceutical Inc, CSL Behring España S.A., Novartis España, PTC Therapeutics, Inc, Ultragenyx and Vertex Pharmaceuticals, or without BioInnova Consulting for its support as technical secretariat.
He also thanks the participants and attendees for their dedication and commitment, which are essential to continue advancing the development of and access to advanced therapies for rare diseases.
You can watch the complete event on our YouTube channel
