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ALAN asbl-Maladies Rares Luxembourg

ALAN

Fostering an environment for people living with a rare disease to realize their full potential through recognition, equal opportunities and improved quality of life.

Lobbying Activity

Response to EU’s next long-term budget (MFF) – EU funding for competitiveness

11 Nov 2025

As representatives of the rare disease community in Luxembourg, we would like to express our deep concern regarding the positioning of public health in the current MFF proposal. The discontinuation of a dedicated health programmesuch as the existing EU4Health initiativeis a significant threat to critical ongoing and future EU initiatives in Health. Under the new proposal, health is a sub-topic within the European Competitiveness Fund. The main objective of this fund is to boost competitiveness in technology and strategic sectors which are critical to EU competitiveness. This shift places public health in a purely economic context, framing it as a means to improve EU competitiveness rather than as a fundamental societal goal. It appears that the Commission is primarily interested in health-related spending when it directly supports economic activities, such as investments in the biotech and pharmaceutical sectors, which may benefit patients by reducing the EUs reliance on imported medicines. Similarly, health promotion and disease prevention are referenced mainly as tools to increase workforce productivity. Even though we do believe that investments in the biotechnology sector in Europe will serve the interests of our patient population as it could facilitate more clinical trials in Europe and support the development of advanced medical products, the sole economic viewpoint on this topic is reductive. The reduction of public health to an instrument of economic growth is deeply concerning. Previous EU health programmes have demonstrated clear added value beyond national health competencies, benefiting citizens across Europe. Many initiatives under these programmes did not serve immediate economic objectives but nonetheless had a substantial positive impact on national health systems and patient experiences as they facilitated improvements in the national health system and its services. For the rare disease community, several of these initiatives have been particularly vital, including: Financial support for Orphanet to gather and exchange knowledge on rare diseases. The creation and continued support of European Reference Networks (ERNs) through direct grants and Joint Actions (JARDIN) to facilitate equity of access to specialised care for rare disease patients and to foster research and knowledge exchange on rare diseases. The operational grants to support third sector health organisations in Europe. Activities to support the national implementation of directives and legislation such as Joint Action TEDAS 2 to facilitate the implementation of the EU Health Data Space. We strongly urge the Commission to maintain its support for health mobility, knowledge exchange and the implementation of EU directives and legislation on national level through platforms like Orphanet and Joint Actions. Furthermore, we urge the Commission to continue its support for and dialogue with EU third sector health organisation such as patient associations, through operational grants and consultation. These organisations are pivotal in voicing the needs of the patients and driving healthcare improvements in Europe. And most importantly to continue to invest and support the development and functioning of the ERNs. In conclusion, we call on the Commission to recognise the improvement of public health in Europe as a priority in its own right, not merely as a tool to serve economic interests. Public health deserves a dedicated programme that continues to strengthen national health systems and serve European patients.
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Meeting with Martine Kemp (Member of the European Parliament)

14 Mar 2025 · échange sur la politique européeenne sur les maladies rares

Meeting with Isabel Wiseler-Lima (Member of the European Parliament) and EUROPEAN ORGANISATION FOR RARE DISEASES

5 Mar 2025 · Rare Disease Day

Meeting with Isabel Wiseler-Lima (Member of the European Parliament)

19 Jan 2024 · Législation pharmaceutique européenne

Response to Interim evaluation of the EU4Health Programme 2021-2027

8 Jan 2024

ALAN Maladies Rares Luxembourg asbl, the national rare disease alliance of Luxemburg is pleased with the importance given to health and health care by the EU Commission which is illustrated by the unprecedented budget of the current EU4Health Program. Furthermore, we welcome the broad scope of objectives which covers multiple areas which are key for the rare disease community. Additionally, we appreciate the support for the ERNs and the continuation of the operational grants. However, we find that there is a need for more presence of rare diseases in the EU4Health Program. Moreover, we would like to ask the EU Commission for the implementation of a European policy framework on rare diseases, an EU Action plan on Rare Diseases. The EU4Health Program could support this implementation knowing that such a policy framework contributes to multiple objectives of the program inter alia: Health promotion and disease prevention Under this objective we would like to ask for a Joint Action on newborn screening. Newborn screening is an effective way of disease prevention, thanks to the early intervention the right care and follow up can be indicated early, which saves the patient a lot of medical visits otherwise caused by the development of more severe symptoms or comorbidities and/or possible diagnostic errors over time. Besides, a European wide/shared framework/newborn screening program creates equity in access to early diagnosis and afterwards increases the likelihood of access to adapted treatment and or care pathways. Strengthening Health Systems Under this objective we would like to request the continuation of the support for the ERNs and an action to support the development of national plans on rare diseases. National plans on rare diseases help the health systems to improve the support, care and treatment for people living with a rare disease on national level. Hence, a national plan on rare diseases can improve the access to healthcare for rare disease patients. There is currently a big discrepancy in access to adapted support, care and treatment for rare disease patients between different member states. A European action to support the national plans can help the member states to exchange best practices, learn form each other and reduce health inequality. Strengthening health data, digital tools & services The exchange of health data is crucial to improve cross-border healthcare and to create optimized data for research. Many rare disease patients must travel abroad for medical expertise, it is crucial that their health data can travel too. Furthermore, this will require a certain standardization and interoperability of the data. Not to mention that only 5% of rare diseases have an approved treatment, therefore it is crucial that we support rare disease research. Providing access to optimized data will stimulate rare disease research and the development of treatments later down the road. Finally, we support the manifesto Championing the Rare of EURORDIS, https://download2.eurordis.org/publications/Manifesto_long.pdf, in which more actions are described which will improve the daily life op people living with a rare disease in Europe and which could be implemented through the EU4Health Program.
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Meeting with Marc Angel (Member of the European Parliament)

8 Dec 2023 · Révision de la législation pharmaceutique européenne

Response to Evaluation and revision of the general pharmaceutical legislation

20 Oct 2023

ALAN asbl Maladies Rares Luxembourg, the rare disease alliance of Luxembourg supports the objectives of the legislation. However, we see three areas of improvement namely: patient engagement, the concept of unmet needs and pediatric medicine. Patient engagement Although we welcome the initiatives to improve patient engagement as described in the current proposal, we see multiple areas of improvement described below. Patient Representation at the CHMP and PRAC Even though we welcome the inclusion of four patient representatives as members of the CHMP and the increase of the number of patient representatives at the PRAC, we find this inclusion too limited. It is important to acknowledge that there is a great diversity in the patient population. To improve patient representativity, we strongly encourage EMA to continue its other patient engagement activities, including the ad hoc participation of patients with a specific disease in the committees and working groups. Patient Representation in the context of Rare Diseases Another development that raises concerns for us is the dissolution of the COMP which reduces the inclusion of the rare disease patient in the orphan drug development lifecycle, while it is well established that the development of orphan medicinal products meets with specific challenges, and that patient engagement in this process contributes significantly to positive outcomes. Therefore, we would like an assurance of the continuation of the work and level of influence of the COMP in the evaluation procedures of the EMA through its newly to be formed working group and request a more detailed description of the role of the working groups in the process. Patient Representation at the national competent authorities and HTA bodies There is a lack of patient-engagement at the national competent authorities and HTA bodies. We deem the role of the patient perspective in the decentralized and mutual recognition procedures essential and believe that this should be assured through the EU legislation. Therefore, we would like to suggest that specific obligations or recommendations on how to assure patient engagement at the national competent authorities (national medicine agencies and HTA bodies) are included in the directive. These may be the establishment of a patient advisory board and the appointment of patient engagement staff. Financial compensation for patient representatives We find that the importance of patient engagement is not sufficiently recognized in the current proposal. Therefore, we believe that the patient representatives should be compensated for their work. Consequently, we would like to propose an enlargement of the compensation for patient representatives with a financial compensation for the hours of participation in the evaluation process. Unmet Needs Furthermore, we would like to make a remark on the concept unmet needs; we are satisfied that the current proposal does not include a set definition of unmet needs. However, we believe that a multistakeholder evaluation process is required to define what an unmet need is in the context of a medicinal product. This requirement should be anchored in the text, the directive could even describe the process of the multistakeholder evaluation process. Pediatric Medicine Finally, because 70% of the rare genetic disorders have an onset in childhood, we deem it essential that the development of medicines for children is stimulated by the legislation, we welcome the obligation for a pediatric investigation plan. However, we would like the legislative text to emphasize this obligation and to underline that the PIP should be submitted at the end of Phase I clinical trials. And we would welcome the inclusion of incentives that stimulate the development of treatment in children first.
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Meeting with Monica Semedo (Member of the European Parliament)

9 Oct 2023 · General discussion about the activities of ALAN and the situation of people with disabilities. European Disability Card came up in the discussion

Meeting with Nicolas Schmit (Commissioner) and

7 Feb 2023 · La stratégie européenne en matière de soins, l'égalité en matière d'emploi et l'intégration des personnes atteintes d'une maladie rare