The Spanish Association for Orphan and Ultra-Orphan Drugs (AELMHU) will hold the fourth edition of the National Conference on Advanced Therapies for Rare Diseases, sponsored by CSL, Novartis, PTC, Ultragenyx, and Vertex, companies affiliated with AELMHU.
Once again this year, the event will bring together experts, researchers, patient organizations, and national and regional authorities to share and discuss the progress made over the past year in advanced therapies for rare diseases in Spain, as well as the latest developments in research, access, and funding.
During the conference, participants will discuss, among other topics, experiences and lessons learned regarding the European Regulation on Health Technology Assessment (HTA); the importance of research and clinical trials; challenges related to access, funding, and innovative procurement; and the approach to advanced therapies from the perspective of patients and the autonomous communities.
Event details:
📅 Date: Wednesday , May 6, 2026.
🕒 Time: 10 :00 a.m. to 1:00 p.m.
🏢 Hybrid format:
- In-person: Espacio Bertelsmann (10 O’Donnell Street, 28009, Madrid).
- Streamed live (the link will be sent via email toregistered participants).
📋 Program:
10:00 a.m. Opening ceremony:
- Ms. Beatriz Perales Zamorano, President of the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU)
10:15 a.m. European Regulation on Health Technology Assessment (HTA): Experiences and Lessons Learned for Patients:
- Mr. Pedro Carrascal Rueda, Director of the Platform of Patient Organizations (POP)
10:30 a.m. Discussion between researchers and clinicians:
A discussion of advanced therapies for diseases, covering research, clinical trials, and the therapeutic value of treatments.
- Dr. Cristina Avendaño Solá, president of the Federation of Spanish Scientific and Medical Associations (FACME).
- Dr. Juan Antonio Bueren Roncero, director of the Biomedical Innovation Unit at the Center for Energy, Environmental, and Technological Research (CIEMAT).
- Dr. José Luis Poveda Andrés, coordinator of the Advanced Therapies Group of the Spanish Society of Hospital Pharmacy (SEFH).
- Mr. José Valenzuela Mendoza, Head of the Clinical Trials Division at the Spanish Agency for Medicines and Health Products (AEMPS).
Moderator: José María López Alemany, B .S. in Pharmacy and specialist in healthcare and pharmaceutical policy.
11:15 a.m. Coffee break.
11:35 a.m. Discussion on: Access to and funding for advanced therapies: perspectives from the National Health System, the industry, and regulatory authorities.
- Mr. Lluís Alcover Llubià, an attorney specializing in pharmaceutical law at Faus & Moliner Abogados
- Ms. Lourdes Gil Tornero, Head of the Pharmaceutical Technologies Division within the Sub-Directorate General for Strategy and Industrial Ecosystems at the Ministry of Industry and Tourism
- Mr. César Hernández García, Director General of the Common Portfolio of Services for the National Health and Pharmacy System at the Ministry of Health
Moderator: Álvaro Hidalgo Vega, professor and director of the Research Group on Health Economics and Health Management at the University of Castilla-La Mancha (UCLM)
12:15 p.m. Discussion between regional governments and patients: a regional approach to advanced therapies.
- Laura Quintanilla Cuesta, Division Head of the Advanced Therapies Unit, Department of Health of the Community of Madrid.
- Ms. Mónica Gayoso Rey, a pharmacist specializing in pediatrics and gastroenterology at the Vigo Health District (Álvaro Cunqueiro Hospital).
- Ms. Isabel Motero Vázquez, Executive Director of the Spanish Federation for Rare Diseases (FEDER).
- Mr. Óscar Fernández Torre, Director General of Planning, Regulation, Knowledge Management, and Digital Health, Department of Health of the Government of Cantabria.
Moderator: Jorge Mestre Ferrándiz, associate professor at Carlos III University and expert in health economics.
1:00 p.m. End of the day.
*To be confirmed
