The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU), through its executive director, Marian Corral, has participated in the event Towards a Long-term Strategy for "One-shot" Therapies in Spain, organized by the Gaspar Casal Foundation and CSL Behring at the Australian Embassy in Madrid.
The inauguration of the event was attended by Daniel-Aníbal García (FEDHEMO), Rosemary Morris-Castico (Australian Ambassador), Markus Staempfli (CSL Behring) and Alicia del Llano (Gaspar Casal Foundation), who highlighted the need to promote innovation and strengthen international collaboration to improve access to advanced therapies.
The development of the event was based on several discussion centers:
The round table "Is the NHS ready for the "One-shot" therapies revolution?" brought together Raquel San José (Community of Madrid), Marcos Timón (EMA), María Teresa Álvarez Román (SETH) and José Luis Poveda (Hospital La Fe), under the moderation of Rubén Moreno, former Secretary General of Health of the Ministry of Health. The speakers addressed the regulatory, clinical and organizational challenges posed by the incorporation of these single-dose therapies into the National Health System.
At the round table "One-shot regulation for the drugs of the future?", the need to adapt the regulatory framework to the new advanced therapies was debated. Participants María Sainz (PSOE), Raquel Barrero Alba (VOX) and Antonio Cavacasillas (PP), moderated by health journalist Belén Gómez del Pino , were able to express their positions on how legislation should evolve to respond to scientific advances.
Under the title "Theory and practice: balance between access and sustainability for one-shot therapies", a panel moderated by Susana Álvarez, former Deputy Director General of Contracting of the Madrid Health Service, AELMHU shared its intervention with Gerardo García-Álvarez, Professor of Administrative Law at the University of Zaragoza; Lourdes Gil, Head of the Pharmaceutical Technologies Area, Ministry of Industry and Tourism and Jorge Mestre, Researcher and consultant in health economics .
During the debate, Marian Corral stressed that these types of therapies represent an advance for rare and ultra-rare diseases, but warned that their incorporation into the National Health System requires new financing models to be adapted.
Public-private collaboration as a strategic axis
At the same table, the director stressed that public-private collaboration is essential to face current challenges, especially in a context of important regulatory changes at both national and European level.
Among the measures proposed to ensure equitable access to new therapies, he has highlighted:
- Promote joint development agreements between public and private entities.
- Improve transparency in collaborative processes and share real-life data to assess the impact of therapies.
- Encourage innovation through tax incentives and collaboration programs.
In relation to regulatory barriers, Corral has pointed out the need to overcome the current focus limited to CAR-T therapies and move towards a broader national strategy, which also includes gene and cell therapies.
Finally, the executive director of AELMHU addressed the importance of improving social and healthcare coordination for patients receiving advanced therapies. Among her proposals she highlighted:
- The creation of multidisciplinary teams for clinical follow-up.
- The establishment of standardized protocols and improved inter-institutional communication.
- Specialized training of health and social professionals.
- The creation of patient registries and the strengthening of the role of patient associations in the design and evaluation of services.
From AELMHU, we thank the organizers, CSL Behring and Gaspar Casal Foundation for the opportunity to be present in such a necessary forum.
Also, to the other speakers who facilitated the debate and with whom we have reached very interesting conclusions.
