NF Patients United - Global Network of Neurofibromatosis Patient Organisations
NFPU
NF Patients United is an international umbrella organization of non profit patient organizations that advocate for people affected by Neurofibromatosis (Neurofibromatosis Type 1, Neurofibromatosis Type 2 and Schwannomatosis) and their families.
ID: 370223242487-93
Lobbying Activity
26 Jun 2024
NFPU welcomes the initiative regarding the management of conflicts of interest in terms of EU rules on health technology assessment. Transparent and consistent rules are required to ensure that all stakeholders involved act impartially and independently. At the same time, it is important that conflicts of interest are defined and managed in a way that does not undermine patients' rights to self-organize, as this could adversely affect society as a whole and the structure of non-governmental support for patients. We find Annex I, the Declaration of Interests form for participation under Regulation (EU) 2021/2282, to be alarming for rare disease patient communities. The form considers it a conflict of interest if a (co)chair, president, director, or treasurer of an organization receives, or has received in the past three years, direct funding from a health technology developer or several health technology developers. In the realm of rare diseases, this measure would simply cut off the most active and knowledgeable patient representatives from involvement in HTA processes and adversely impact the community. These rules need to be revised so as embrace the reality in the rare diseases domain. Besides, involvement of patient groups and associations should be ensured. Please read our full feedback for more details and suggestions.
Read full responseResponse to Evaluation and revision of the general pharmaceutical legislation
8 Nov 2023
Neurofibromatosis Patients United (NFPU) serves as the preeminent umbrella association for patient organizations that work for neurofibromatosis patients within the European region. Neurofibromatosis patients throughout Europe confront remarkably uniform challenges, including constrained access to adequate medical and psychosocial care, limited information resources, restricted availability of clinical trials, insufficient public awareness, and a deficit in awareness among healthcare professionals. It is evident that these collective challenges can be more effectively addressed through collaborative efforts.<br> Recommendations from NFPU for a Proposal for a Regulation of the European Parliament and of the Council laying down Union can be found here as well as in the full version document attached. <br> Article 2 (7) The expression "substantial part" used in this concept is not appropriate for rare diseases, particularly neurofibromatosis, since the disease can affect each individual in a very different way. We therefore recommend removing the adjective substantial from the definition. <br> Article 162 (1), (2) Consultation with the organizations mentioned in paragraph 2 should not be a possibility, but an obligation, since they can make a significant contribution to defining procedures and concepts, particularly with regard to unmet medical needs and clinical trials. Patients are the best placed to help define UMN and high UMN for their disease (group of diseases), knowing that rare diseases are complex, heterogeneous and often lacking enough scientific knowledge. Patients are not involved in the HTA decision-making in all member states. Therefore there needs to be a way to involve patients in these decisions. The most important aim of innovative treatments has to be an improvement of the quality of life of the patients. A significant benefit without considering patient-reported outcomes and quality of life aspects, is not sufficient to assess the significant benefit of a new treatment. <br> Article 60 (1) Although we believe that this could not be otherwise, the current wording of the article is unclear as to whether the points identified are alternative and not cumulative. The wording of the article should be improved. (not sure what is meant here?) <br> Article 68 We believe that patient organizations are essential in the research and development of orphan medicinal products and should, whenever possible, be part of research consortia and co-recipients of research grants. We therefore suggest amending paragraph 2 to include patient organizations. <br> --- Furthermore we want to highlight the following topics that we feel are essential for orphan drug development in Europe: 1. Data sharing, 2. Improved Access to Novel Anticancer Medicines, 3. Improved Access to Essential Anticancer Medicines, 4. Academic Drug Development and Repositioning of Medicines, 5. Early Start for the Development of Paediatric Medicines, 6. First-in-Child Innovation . <br> In conclusion, patient organizations play a key role in defining the unmet medical need in their patient population. These organizations, made up of individuals who face specific health conditions or represent those who do, play a crucial role in promoting research, defending patients' rights and improving the quality of healthcare. In the context of medicines that address unmet medical needs, patient organizations are powerful allies in identifying, developing and approving innovative treatments. In summary, we consider that patient organizations are integral parts of the process of defining medicines that respond to unmet medical needs. Their personal experience and knowledge of health conditions, together with their ability to mobilize and advocate, make them valuable partners in the search for innovative treatments that significantly improve the quality of life of people affected by diseases with no cure or effective treatments.
Read full responseResponse to Evaluation and revision of the general pharmaceutical legislation
8 Nov 2023
Neurofibromatosis Patients United (NFPU) serves as the preeminent umbrella association for patient organizations that work for neurofibromatosis patients within the European region. Neurofibromatosis patients throughout Europe confront remarkably uniform challenges, including constrained access to adequate medical and psychosocial care, limited information resources, restricted availability of clinical trials, insufficient public awareness, and a deficit in awareness among healthcare professionals. It is evident that these collective challenges can be more effectively addressed through collaborative efforts.<br> Recommendations from NFPU for a Directive for a Regulation of the European Parliament and of the Council laying down Union can be found here as well as in the full version document attached. <br> Article 83 The concepts of high morbidity or mortality are very ambiguous, and we suggest that more concrete criteria be created. We recommend that the clarification of these concepts be analyzed together with patients, and their representative associations together with academia (health care professionals who are experts in the specific rare disease), to clearly understand the impact on the patients' quality of life of a rare disease. With regard to this article, we believe that the concept of unmet medical need does not cover situations in which there is a medicinal product but it has no effect on a specific group of patients, for example when there is a specific mutation in a rare disease and therefore the existing medicine has no effect on that patient. It is therefore suggested that these situations be taken into account. Not covering this type of situation could lead to disinvestment in R&D associated with rare diseases.<br> Article 70 The concept of exceptional therapeutic advancement should be clarified, as it is considered that this should be all-encompassing and not just imply a cure or innovation in treatment. For example, improvements in quality of life and pain reduction must be taken into account.<br> --- Furthermore we want to highlight the following topics that we feel are essential for orphan drug development in Europe: 1. Data sharing, 2. Improved Access to Novel Anticancer Medicines, 3. Improved Access to Essential Anticancer Medicines, 4. Academic Drug Development and Repositioning of Medicines, 5. Early Start for the Development of Paediatric Medicines, 6. First-in-Child Innovation .<br> In conclusion, patient organizations play a key role in defining the unmet medical need in their patient population. These organizations, made up of individuals who face specific health conditions or represent those who do, play a crucial role in promoting research, defending patients' rights and improving the quality of healthcare. In the context of medicines that address unmet medical needs, patient organizations are powerful allies in identifying, developing and approving innovative treatments. <br> In summary, we consider that patient organizations are integral parts of the process of defining medicines that respond to unmet medical needs. Their personal experience and knowledge of health conditions, together with their ability to mobilize and advocate, make them valuable partners in the search for innovative treatments that significantly improve the quality of life of people affected by diseases with no cure or effective treatments. Collaboration between patients, healthcare professionals, researchers and the pharmaceutical industry is essential to address these unmet needs and advance the field of medicine.
Read full responseResponse to Cancer Screening Recommendation
21 Feb 2022
Please find the feedback of the European umbrella organization NF Patients United in the attached pdf
Read full response