Waiting to know the definitive text of the Draft Law on Medicines and Health Products approved by the Council of Ministers, the Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has requested today to exclude orphan and ultra-orphan drugs from the scope of application of the sixth additional provision.
This position is not new, as it has been continuously defended since 2022 before the Ministry of Health and the various ministerial departments involved. In their opinion, excluding orphan and ultra-orphan drugs from this provision responds to a matter of health, regulatory, economic, and industrial coherence.
AELMHU considers that the application of this measure lacks a specific justification from a health perspective, is hardly compatible with the objectives of competitiveness, innovation, and attraction of investment that the Government itself has established for the pharmaceutical sector, and breaks with the differentiated treatment that European regulations and Spanish legal order recognize for this category of drugs and may generate risks for the access of patients with rare diseases to new therapies.
The Association emphasizes that these drugs are already subject to one of the most demanding evaluation and funding systems of the National Health System, through conditional funding, specific agreements, usage restrictions, pay-for-results models, and periodic price reviews. Therefore, it considers that the application of a new economic burden is hardly compatible with the existing regulatory framework and with the support policies for innovation in rare diseases.
Furthermore, the Association reminds that, according to data from the Ministry of Health, the weight of orphan drugs in hospital pharmaceutical spending has remained stable - around 10% -, while the number of funded drugs has practically doubled, from 46 in 2020 to 85 in 2024. In that same period, the average consumption per funded drug decreased by 17.3%, a figure that, according to AELMHU, reflects that the increase in spending responds to the incorporation of new therapeutic alternatives and not to a disproportionate increase in the cost of treatments.
For AELMHU, the assessment of the Provision should not be limited to its possible budgetary effect, but also incorporate the opportunity cost that it may generate on the competitiveness of the Spanish pharmaceutical sector.
The Pharmaceutical Industry Strategy 2024-2028 identifies rare diseases as a strategic area for Spain and highlights that investment in this field drives biomedical innovation, technological development, and knowledge generation.
Regulatory decisions condition the location of investments, the development of clinical trials, and the prioritization of countries for the launch of new medications. In a field like rare diseases, characterized by long research processes and high levels of investment, economic measures that reduce the attractiveness of the Spanish market can impact the availability of new therapeutic options for patients.
Currently, 68 companies market orphan drugs with National Code in Spain and 54 have one or more medications funded by the National Health System. In addition, the Horizon Scanning exercise carried out by AELMHU identifies around 35 companies with advanced therapies for rare diseases that are expected to access the Spanish market in the coming years.
Therefore, any new economic burden must be evaluated not only for its potential revenue effect but also for its impact on research, innovation, and industrial development of a strategic sector.
Orphan drugs constitute a specific legal category recognized by the Regulation (EC) No 141/2000, whose objective is to incentivize research and the development of treatments for diseases of very low prevalence and high scientific complexity.
This differentiated treatment also exists in Spain. In a text that was known a few weeks ago, the Draft Bill itself maintains specific instruments such as the 4% deduction provided for in Royal Decree-Law 8/2010 and the exclusion of these medications from the reference price system, which confirms the recognition of their particularities.
Incorporating a new economic obligation through the Provision now is, according to AELMHU, "hardly compatible with that regulatory framework and with the European and national policy of support for innovation in rare diseases".
Furthermore, the reduction planned through the PROFARMA Plan would not compensate, according to their criteria, the economic impact of the Provision through the existing incentives: of the 29 companies associated with AELMHU, only 8 participate in the PROFARMA 2025-2026 call and none have obtained the rating of "Excellent".
AELMHU trusts that the final text of the Preliminary Draft will maintain the exclusion of orphan and ultra-orphan drugs, preserving coherence with European regulations, the Spanish regulatory framework, and the objectives of the Pharmaceutical Industry Strategy 2024-2028.
If the finally approved text maintains the inclusion of orphan and ultra-orphan drugs in the Sixth Additional Provision, AELMHU announces that it will continue to work during the parliamentary processing with the different political groups and institutions to promote the necessary modifications that preserve the differentiated treatment of these drugs and favor a stable regulatory framework for the research, innovation, and access of these treatments for patients.