AELMHU is calling for orphan drugs to be exempt from the new financial obligation set forth in the future Medicines Act.
The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has asked the government to exclude treatments for rare diseases from the Sixth Additional Provision of the Draft Bill on Medicines and Medical Devices. The association believes that this measure lacks a public health justification and contradicts the differentiated treatment that European regulations and the Spanish regulatory framework grant to these drugs.
AELMHU points out that orphan drugs are already subject to one of the most stringent evaluation and financing systems within the National Health System, featuring pay-for-performance models, periodic price reviews, and specific agreements. It warns that adding a new financial burden could create tensions in a field characterized by lengthy research processes and high levels of investment.
Hospital spending on orphan drugs remains stable at around 10%, even though the number of reimbursed drugs has risen from 46 in 2020 to 85 in 2024. During that same period, average consumption per drug fell by 17.3%, which, according to AELMHU, shows that the increase in spending is due to the introduction of new therapies and not to a rise in the cost of treatments.
The organization warns that the measure could affect the competitiveness of the Spanish pharmaceutical sector and reduce the country’s appeal for investment and the conduct of clinical trials. The 2024–2028 Pharmaceutical Industry Strategy identifies rare diseases as a strategic area, and AELMHU notes that 68 companies currently market orphan drugs in Spain, while another 35 could introduce new therapies in the coming years.
Furthermore, the Association notes that the incentives provided for in the PROFARMA Plan would not offset the economic impact of the Provision, since only 8 of its 29 member companies are participating in the 2025–2026 call for proposals, and none has received an “Excellent” rating.
AELMHU is confident that the final text of the draft bill will maintain the exclusion of orphan and ultra-orphan drugs, thereby ensuring consistency with European regulations, the Spanish regulatory framework, and the objectives of the 2024–2028 Pharmaceutical Industry Strategy.
If the final approved text retains the inclusion of orphan and ultra-orphan drugs in the Sixth Additional Provision, AELMHU will continue to work with the various political groups and institutions throughout the legislative process to promote the necessary amendments that preserve the differentiated treatment of these drugs and foster a stable regulatory framework for research, innovation, and patient access to these treatments.