The new Spanish framework for Health Technology Assessment (HTA) has recently been approved by Royal Decree 415/2026. What impact will it have on rare diseases and orphan drugs?
Among the possible benefits, the greater participation of patients and associations stands out, as the Royal Decree expressly mentions the need to incorporate the participation of patient organizations, including those related to rare diseases. This aspect represents a formal recognition that patient experience should be part of the assessment.
Furthermore, there is a commitment to more structured and transparent assessments with the creation of a specific Office for drug assessment within the HTA system. This body will provide more homogeneous and predictable procedures that will be very useful for orphan drug developers, as methodological clarity usually facilitates market access planning.
Another point in favor is the alignment with the European system, as Spain is fully integrated into the new European HTA framework derived from the European Regulation. This progress can avoid duplication and leverage joint clinical assessments carried out at the European level, something especially relevant for rare diseases, where the number of patients and available evidence are limited.
Also noteworthy is the possibility of managing uncertainty through additional evidence, as the Royal Decree provides that, when such uncertainties exist, registries, monitoring studies, or additional evidence generation may be proposed. This aspect is especially important for orphan drugs, where large clinical trials are rarely available at the time of authorization.
More economic evaluation
On the other hand, it should be taken into account that economic evaluations are gaining weight. Thus, in addition to clinical aspects, five non-clinical domains are incorporated: economic, ethical, organizational, social, and legal evaluation. It should be noted that orphan drugs often have high costs and limited evidence, so they could face more demanding economic scrutiny.
Another recurring concern of the rare disease community is that the traditional cost-effectiveness criteria do not adequately capture social value, disease severity, lack of therapeutic alternatives, or family impact. The outcome will depend on how the specific methodologies, which are yet to be finalized, are developed.
It should also be taken into account that, although the Royal Decree makes it clear that HTA informs the decision, it does not replace it. Thus, even if an evaluation is favorable, the final price and funding decision will still depend on the competent bodies.
There is also speculation about a possible increase in evidence requirements. Thus, although mechanisms exist to manage uncertainties, drug developers may be forced to provide more real-world data, registries, or post-marketing surveillance to justify continued funding. These requirements could delay or condition access in some cases.
Opportunity for improvement
But the overall assessment, according to different sources consulted by Demócrata, is that the Royal Decree opens an opportunity to improve the evaluation of treatments for rare diseases thanks to greater patient participation, better European coordination, and mechanisms to manage uncertainty. However, it also introduces a broader and more demanding evaluation that could increase pressure on high-cost orphan drugs.
Strengthening science
Javier Padilla, Secretary of State for Health, has highlighted advances in the Royal Decree, such as greater transparency, patient participation, and the setting of evaluation deadlines. He also argued that evaluation should provide the best available scientific evidence to guide decisions, but without replacing them.
For these reasons, he insists that evaluations will be mandatory, although not binding, as funding also depends on strategic, economic, and social factors. From his point of view, the regulation reinforces a health policy guided by science and aligned with European advances.
The health economists Jaume Puig-Junoy and Juan Oliva recall that, despite Spain being one of the largest pharmaceutical markets in Europe and the world, it lacks a public drug evaluation system comparable to those in countries like the United Kingdom, Germany, or France. "Although the regulations provide for evaluating efficiency to set prices and funding, these procedures are not clearly developed," they point out.
In their opinion, the new Royal Decree, "driven by European regulations, introduces improvements in clinical evaluation, transparency, and governance." However, they believe that further reforms are necessary, such as guaranteeing technical independence, unifying methodologies with Europe, increasing resources, and conducting periodic reevaluations. "Otherwise, opportunity costs that harm public health will persist," they warn.
For Daniel Aníbal García Diego, president of the Spanish Federation of Hemophilia (Fedhemo), the model proposed by the Royal Decree "is the way to go, increasingly separating authorization from evaluation and decision-making. And for each of the processes to be public, transparent, and for us to know what stage we are in. Now, the evaluation part will be much clearer, with defined bodies. And until now, it wasn't. The doubt is how it will be specified, but it is better to start even with doubts, learning from the process, separating drugs from health technology."