02 December 2024
Press
- Maladies Rares
- Pédiatrie
The F-CRIN-accredited "OrphanDev" and "PedStart" networks research networks for rare diseases and pediatrics, are joining forces to accelerate access to gene and cell therapies in France. This project aims to structure a new national network to offer rapid and equitable access to innovative treatments, a crucial step for the 3 million French people concerned, the vast majority of whom are children.
France leads the way in innovative therapies for rare diseases
In France, Rare Diseases, which affect around 3 million French people, or 4.5% of the population, represent a public health priority with national plans launched since 2004, which have structured the care and research landscape. Today, France has 23 "Rare Diseases" health networks, bringing together 603 centers of reference and 1,708 regional centers of expertise. Yet 95% of rare diseases remain without curative treatment. Half of these diseases affect children under the age of five, contributing to 10% of deaths in this age group. While 80% of these diseases are genetic in origin, few patients still benefit from appropriate therapeutic solutions.
OrphanDev and PedStart, with support from F-CRIN, aim to transform this observation by participating in the structuring and labeling of a national network of Centres d'Études et d'Innovation Maladies Rares (CLEIO) for research into innovative therapies. Based on the model of CLIP² centers in oncology, this project proposes to develop a clinical research framework dedicated to rare diseases, with the aim of significantly increasing the number of clinical trials, including more patients, adults and children, and improving the patient care pathway.
As part of this initiative, OrphanDev and PedStart organized a theme day at the CHU d'Orléans bringing together experts in rare diseases, clinical research and industry around advances in gene and cell therapies. The event provided an essential platform for stimulating collaboration between researchers, industry and academia.
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"The arrival of clinical trials in innovative therapies implies new challenges in terms of logistics, regulation, financing and skills. It is vital to lay the foundations for a national structure that, building on existing structures, will enable patients to benefit from these advances and strengthen France's competitiveness", says Pr. Vincent Laugel, neuropediatrician, OrphanDev (F-CRIN) co-coordinator and head of department at Strasbourg University Hospitals.
Concrete targets for greater access to treatment
Gene and cell therapies represent a major advance in the treatment of rare diseases, especially those of genetic origin. These therapies make it possible to envisage curative treatments for hitherto incurable diseases, such as ß-Thalassemia or Duchenne muscular dystrophy. However, access to innovative therapies is hampered by a lack of coordination between the various healthcare players, and the recruitment of patients for clinical trials remains complex due to the low prevalence of rare diseases. Against this backdrop, the creation of CLEIOs aims to:
- Strengthen the competence of clinical research centers and increase the number of early-phase clinical trials of Innovative Therapy Medicines (ITMs) in France, with harmonized practices to include more patients in trials.
- Improve access to treatments by reducing administrative delays and guaranteeing equitable access across the territory.
- Attract drug manufacturers in France and abroad by offering a solid regulatory and logistical framework, thanks to the certification of centers, to boost the visibility of French clinical research.
An ambitious project with a strategic impact for France
This project is part of the campaign to label new F-CRIN networks. By aiming to create a network dedicated to advanced therapies, it aims to foster cooperation between specialized centers as well as facilitate the preparation and implementation of clinical trials on advanced therapy drugs (ITDs). Through this initiative, OrphanDev and PedStart, reinforce their commitment to rare disease patients in France, for whom these scientific advances represent new hope.
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