Asociación Española de laboratorios Huérfanos y Ultrahuérfanos
AELMHU
Aelmhu is a non-profit association which joins pharmaceutical and biotechnological companies that share the common goal of discovering, researching, developing and commercializing innovative therapies to improve the lives of patients who suffer from rare diseases.
ID: 429216742750-40
Lobbying Activity
Response to Evaluation and revision of the general pharmaceutical legislation
6 Nov 2023
AELMHU, Asociación Española de Laboratorios de Medicamentos Huérfanos y Ultrahuérfanos, a non-profit organization that brings together 25 biotechnology and pharmaceutical companies committed to the discovery, research, development and commercialization of innovative therapies capable of improving the situation of patients living with rare and ultra-rare diseases, appreciates the opportunity to participate in this consultation. We recognize the need to update the European legal framework governing orphan medicinal products in order to continue working in an incentivizing environment. In this regard, we welcome the efforts made to reduce the regulatory and administrative burden to promote scientific advances and the objectives that the reform aims to achieve. However, we are concerned about certain measures and sections of the two legal instruments that, we believe, could, in particular in a sector as complex as that of orphan medicines, harm R&D efforts, the interest in innovation and the competitiveness of the European market, and result in an effect contrary to that pursued by the reform. For those reasons, our feedback focuses on the proposals surrounding high unmet medical need (HUMN) and unmet medical need (UMN); the reduction of the market exclusivity period and link to the active substance of a medicinal product; the measures addressing equity in access and innovation between Member States; the reduction of the duration of regulatory data protection periods; and, finally, medicinal products access times. In summary, rare disease research is a long, costly and highly unpredictable process that requires a long-term vision and well-established legal certainty. Conditions such as linking incentives for innovation with definitions like the HUMN one, or market exclusivity to an active substance, lead to uncertainty within the research field and increase the risk of disincentivizing research, including the exploration of the full potential of a single active substance. We detail our concerns surrounding the changes in market exclusivity and regulatory data protection conditions, which now offer as "extra" years, the ways to, in reality, maintain the existing exclusivity, increasing the burden on laboratories, with possible negative repercussions on patients. We also highlight that, elements such as the conditions of an EU market launch, or measures to improve approval times for medicinal products must consider the particularities of the different markets and their approval times. Our comment contains a total of 8 concrete recommendations and, encouraging an active dialogue with all stakeholders, we place ourselves at the complete disposal of the European Commission and all the European institutions for further exchanges.
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