OrphanDev is the national network of expertise dedicated to rare diseases, founded in 2009 to facilitate clinical research in this field. Designated an F-CRIN network of excellence since 2014, it is evolving into an Operational Think Tank that brings together researchers, clinicians, patients, industry representatives, and institutional stakeholders to advance innovation, methodological progress, and regulatory science, with the goal of providing solutions for patients with rare diseases.
OrphanDev, the national network of experts dedicated to rare diseases
OrphanDev brings together, on a voluntary and nonpartisan basis, four groups of stakeholders involved in research on rare diseases: the academic community (researchers, clinicians), the private sector (pharmaceutical and biotechnology industries, CROs, consulting firms, medtech, and e-health), patients and their representatives, as well as leaders of transformative projects.
OrphanDev’s model is based on a founding principle: to serve no private or specific interest, but rather the public interest in research on rare diseases. This neutrality forms the foundation of its credibility among all its stakeholders.
To support this model, OrphanDev operates as a nonprofit organization (under the French law of 1901), OrphanDev Support, which allows for the full involvement of private-sector actors in its governance and the establishment of structured public-private partnerships, with each stakeholder contributing according to their expertise toward a common goal.
Although their status does not allow their representatives to identify themselves as members of OrphanDev, the network regularly interacts with health authorities and public authorities whenever its work requires it — European Medicines Agency (EMA), French National Authority for Health (HAS), French National Agency for Medicines and Health Products Safety (ANSM), and the ministries responsible for Health, the Economy, and Higher Education and Research. This capacity for collaboration takes the form of formal hearings, invitations to its working groups and Strategic Committee, as well as regular bilateral exchanges.
OrphanDev’s approach is transdisciplinary, pragmatic, and operational, guided by the principles of independence, objectivity, and integrity—a philosophy summarized as follows: "Cross-fertilization among all stakeholders involved in rare diseases is the key to overcoming roadblocks and fostering innovation."
Organization
OrphanDev is currently coordinated by Prof. Olivier Blin (Clinical Pharmacology and Pharmacovigilance, Marseille University Hospital) and Prof. Vincent Laugel (Neuropediatrics, Strasbourg University Hospital), within a Coordination Unit that oversees the network’s activities and represents it before regulatory authorities. It is supported by an operational team hosted by Inserm (UMR 1106), led by Dr. Vincent Montero (Head of Science) and Mr. Florent de Gassart (Head of Strategy).
The governance structure combines two complementary bodies: an Executive Committee, composed of one representative from each major stakeholder group (academia, patients, the private sector, and key initiatives), which ensures a diversity of perspectives in strategic decision-making; and a Strategic Committee, a body open to all members and volunteer partners wishing to contribute to the network’s direction. It brings together expertise covering the full range of rare disease issues—patient advocacy, clinical research, translational and regulatory science, market access and HTA, data and methodology, pharmacovigilance, European affairs, innovation, and industry (see “Scientific Expertise”).
The active core of the network currently comprises 70 to 80 members regularly engaged in its work, within a broader ecosystem of several hundred academic, industrial, and institutional contacts in France and Europe.
OrphanDev also co-coordinates the Inter-University Diploma in Clinical Trials for Rare Diseases (DIU-ETMR), run in partnership with the universities of Dijon, Lille, Lyon, and Marseille and the AnDDI-Rares Program, which trains 15 to 20 professionals and patients each year on the methodological, regulatory, and ethical challenges of clinical research in rare diseases.
Cartographie du réseau OrphanDev
Scientific Expertise
Unlike the F-CRIN thematic networks, OrphanDev does not focus on a specific disease; its expertise centers on the methodological, regulatory, and organizational challenges common to all rare diseases. This disease-agnostic approach does not preclude ad hoc collaborations on disease-specific projects, carried out alongside the Rare Disease Health Networks or other thematic F-CRIN networks.
Expertise in Methodology and Scientific Evidence
- Small-scale clinical trials and rare populations
- Bayesian methods, trials with external controls, digital twins
- Real-world evidence (RWE) and rare disease databases
- Artificial Intelligence Applied to Clinical Research
Expertise in Regulatory Science and Market Access
- Dialogue and liaison with regulatory authorities (ANSM, HAS, EMA)
- HTA assessment of orphan drugs (SMR/ASMR, JCA)
- Pharmacovigilance for rare diseases
- Waiver Access and Market Access for Health Products
Expertise in clinical research design
- Design and coordination of research projects
- National and European collaborative research consortia (Horizon Europe)
- Advanced Therapy Medicinal Products (ATMPs)
- Establishment and operation of registries and databases
Expertise in Training and Stakeholder Engagement
- Training in Rare Disease Research (DIU-ETMR)
- Involvement of Patients and Their Representatives in Research
- European cooperation and networking of national expertise
Completed Projects and Notable Achievements
OrphanDev organizes, influences, and supports research on rare diseases at the systemic level rather than conducting clinical trials directly. Several initiatives, carried out in collaboration with its partners, have led to concrete advances:
OrphanDev initiated and co-founded CLEIO, an F-CRIN network dedicated to advanced therapy medicinal products (ATMPs), overseeing the entire design and structuring of the network. CLEIO received its F-CRIN designation in 2025.
OrphanDev actively contributed to the development of the PNMR4, alongside all stakeholders in the sector, with several of the network’s proposals included in the plan published in 2024. [Link]
OrphanDev contributed its expertise to this collaborative white paper, which brought together some 50 experts from the ecosystem and was published in March 2025. [Link]
As part of a working group involving the Health Innovation Agency (AIS) and F-CRIN, OrphanDev contributed to the drafting of this white paper on innovative methodologies in clinical research. [Link]
Between 2020 and 2023, OrphanDev contributed, alongside other industry stakeholders, to the discussions that shaped the evolution of the Haute Autorité de Santé’s evaluation guidelines, which now more broadly recognize single-arm trials with external controls and real-world data in the evaluation of orphan drugs. [Link]
Current Projects
The network is currently involved in several key initiatives, at the national and European levels, aimed at improving the conditions for clinical research on rare diseases:
Since 2025, OrphanDev has been co-leading—alongside the ANSM, the CRPV network, and the BNDMR—the rollout of a national enhanced pharmacovigilance system dedicated to rare diseases.
Since 2025, OrphanDev has been leading a working group dedicated to mapping compassionate use programs for rare diseases in Europe.
Contact OrphanDev
Would you like to learn more? You can contact the network right now by sending an email to contact@orphan-dev.org
