With the aim of highlighting the benefits of orphan therapies, not only for patients and their families, but also in economic terms for the healthcare system, the document "Differential aspects of orphan drugs and their value from a social perspective", prepared by the Weber Foundation with the support of the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU), was presented.
The report points out that treatments for rare diseases can significantly improve labor productivity and reduce personal care burdens, which translates into a reduction of direct and indirect costs. In particular, it highlights that in metabolic diseases, direct costs can be reduced by more than 80%, while in hematological and immunological diseases, indirect costs can be reduced by up to 88%.
To maximize these benefits, the report recommends increasing research funding, fostering public-private collaboration and improving the translation of research into clinical practice. In addition, it suggests the implementation of innovative access models to ensure a more equitable distribution of these drugs, regardless of region.
Despite the advances, the report stresses that 63% of orphan drugs with a national code in Spain are funded by the National Health System (NHS), and the waiting time for funding can vary between 23 and 33 months. It also highlights the need for specific regulation and adaptation of European regulations on health technology assessment to meet the challenges of artificial intelligence and data management.
The report concludes that an equitable and well-resourced national strategy is crucial to ensure greater transparency and flexibility in health technology assessment processes, thus benefiting people with rare diseases and society at large.
