The Spanish Association of Orphan and Ultra Orphan Drug Laboratories(AELMHU) has presented its contributions to the Draft Royal Decree (RD) regulating the procedures for the financing and pricing of drugs.
AELMHU, which represents 26 pharmaceutical companies committed to the research, development and commercialization of innovative therapies for rare and ultra-rare diseases, welcomes the fact that the Draft RD proposes a review and definition of the criteria for inclusion, non-inclusion and exclusion in the financing of drugs, the clinical benefit of these drugs from a patient perspective, the economic evaluation (efficiency and budgetary impact), and the uncertainty about each of these elements.
In addition, we also see it as positive that the Pharmaceutical Industry Strategy identifies three distinct phases in the funding procedure: negotiation with industry, evaluation and funding decision, all in the interests of transparency, independence and separation of the evaluation process from the negotiation and positioning process.
RECOMMENDATIONS
Our Association has welcomed the implementation of this draft Royal Decree and has provided the following recommendations on relevant and necessary aspects to regulate the financing and pricing procedure:
- We consider it essential to implement governance mechanisms and instruments that facilitate the joint and consensual development of innovation assessment models adapted to each pathology.
- Work jointly on procedures and conditions for accelerated, conditional and interim financing authorizations.
- Due to the characteristics of orphan drugs (ODM), the evaluation of their efficiency should be governed by a flexible framework that uses evaluation methodologies that value the social contribution of the drug, as well as taking into account the specific idiosyncrasies of these treatments.
- It is essential to establish and strengthen a process of early and continuous dialogue between the Administration and the companies, both for horizon scanning and preparation for evaluation.
- We consider it necessary to implement the guarantees associated with these drugs in relation to market exclusivity, including successive authorized indications of the same orphan drug and its associated market exclusivity.
- That the effectiveness of the drugs be taken into account, as well as real-life data that allow the uncertainties identified during the early dialogue and during the price and financing process to be resolved. Therefore, we recommend that the collection of these data be adapted to the clinical practice of the Centers, Services and Reference Units of the NHS (CSUR).
- Include in the definitions of transparency and confidentiality those aspects that, by the very nature of the procedure in question, must remain confidential, in order to safeguard competition and the proper functioning of the market.
- Revision of funding criteria: inclusion of attributes in the evaluation that go beyond cost-effectiveness and efficiency criteria; clearly reflecting the clinical, economic and social value of drugs is essential.
- We welcome the willingness of the Ministry to determine the criteria for the development of guidelines, guides and evaluation procedures with clear and transparent rules for each of them, as well as their approval mechanisms. AELMHU considers that they should be worked on the basis of collaboration and consensus of all the parties involved, including the industry.
- Given the nature of HMH, and even if they lose their orphan designation, AELMHU recommends limiting the use of mechanisms to reduce the price of products that already have a positive financing decision.
- Following the jurisprudence of the Spanish High Court and in accordance with current European legislation, drugs that have been designated as orphan drugs should not be included in any set of reference price orders.
