The Spanish Association of Orphan and Ultra-Orphan Drug Laboratories (AELMHU) has presented ten proposals to improve access to orphan and ultra-orphan drugs, promote research, and strengthen innovation and competitiveness in the pharmaceutical industry specializing in rare diseases.
In light of the upcoming elections and the process of drafting the platforms of the major political parties, AELMHU has presented the document “AELMHU’s 10 Priorities for the Next Legislative Term,” in which it calls for a cross-cutting commitment to rare diseases and their treatments—one that encompasses not only health policies but also industrial, scientific, economic, and competitiveness policies.
The proposals address the main challenges related to clinical research, evaluation, funding, and access to orphan and ultra-orphan drugs, while also incorporating measures aimed at strengthening research and development of new treatments from an industrial and competitiveness perspective.
Ten Priorities for the Next Legislative Session
AELMHU puts forward the following proposals:
- Explicitly incorporate access to orphan and ultra-orphan drugs into the national policy on rare diseases, with objectives and indicators that allow for the evaluation of progress in terms of access times, regional equity, and health outcomes.
- Recognize orphan and ultra-orphan drugs as a strategic area of pharmaceutical industrial policy and competitiveness, while preserving the necessary incentives for their research and development and assessing the impact of economic measures and cost-containment measures on innovation and patient access.
- Shorten the time it takes to launch clinical trials for rare diseases, strengthen networks of specialized centers, and establish pathways to facilitate the identification and referral of patients.
- Promote the generation and use of data and encourage the development of registries among autonomous communities that are connected to the European Health Data Space, thereby enabling decision-making and improving health outcomes.
- Establish a stable early-access mechanism that allows for the provisional or conditional funding of drugs for rare diseases with unmet medical needs and no therapeutic alternatives.
- Develop evaluation methods tailored to the specific characteristics of orphan drugs that recognize the value of real-world data and evidence, incorporate the societal value of treatments, and encourage the participation of patients and clinical experts.
- Promote more flexible financing models for orphan and ultra-orphan drugs, taking into account their specific characteristics and evolving evidence, and encouraging the use of outcomes-based agreements and other mechanisms for managing uncertainty, within a framework of clear and predictable criteria.
- Ensure that funding decisions made by the National Health System result in effective and equitable access across all autonomous communities, avoiding unjustified delays and additional requirements that could slow down access, and improving transparency regarding actual wait times.
- Develop specific funding and coordination mechanisms for advanced therapies for rare diseases that ensure equitable access, facilitate care at referral centers, and prevent a patient’s place of residence from determining their access to treatment.
- Incorporate the principle of regulatory proportionality into the regulatory framework applicable to orphan and ultra-orphan drugs, ensuring that general measures do not have a disproportionate impact on their research, development, and access.
An opportunity to incorporate specific commitments
AELMHU hopes that these proposals will be taken into account in the drafting of electoral platforms and will lead to concrete measures during the next legislative term. The Association believes that Spain possesses scientific, clinical, and industrial capabilities that must continue to be strengthened through public policies tailored to the specific characteristics of orphan and ultra-orphan drugs. Their development requires an environment that fosters the continuity of research and facilitates the introduction of new therapeutic alternatives.