HYBRID EVENT: You can participate in person at Rome, Italy or Virtually from your home or work.

2nd International Conference on Orphan Drugs and Rare Diseases

March 17-19, 2025

March 17 -19, 2025 | Rome, Italy
Orphan Drugs 2023

The foundation for rare disease and its role in the european rare disease research landscape

Speaker at Orphan Drugs and Rare Diseases 2023 -  Daniel Scherman
Foundation for Rare Diseases, France
Title : The foundation for rare disease and its role in the european rare disease research landscape

Abstract:

The French rare disease research and clinical field has been a pioneer by structuring itself through different action plans. Competence centres and Reference centres are organized within  Rare Disease Health Networks (Filières de Santé Maladies Rares), which have been the basis of the European Reference Networks in rare Diseases.

There are 23 FSMRs each covering a wide and coherent field of diseases, either similar in their manifestations, their consequences or their management, or responsible for damage to the same organ or system. In addition, several Rare Disease Regional Platforms offers specific methodologic and technical tools to RD clinicians and researchers.

The Foundation for Rare Diseases (FFRD) aims to promote the conduct of research projects and scientific excellence as well as the sharing and dissemination of knowledge in the field of rare diseases.

In particular, it pursues the following objectives:

  • the coordination of national players in research on rare diseases.
  • accessibility to technology platforms and calls for projects, which are financed after selection by independent scientific panels.
  • rapprochement between the public sector of research, diagnostic products and innovative therapies,
  • the representation of France in the implementation of European and international projects.
  • scientific support to Patient Advocacy Organizations.

Since its creation in 2012, FFRD has funded more than 400 research projects, including 60 projects in Social Sciences and Humanities. This expertise has been recognized by several Patient Advocacy Organizations (PAOs) and FFRD has now provided advice and guidance in building scientific strategy, policy and research calls for proposals for more than 80 different PAOs. FFRD has organized several scientific meetings each year since its inception. FFRD has also developed an active and broad communication and dissemination strategy with an European outreach.

FFRD main objectives include: accelerating the translation of research into clinical development; detecting business development opportunities; enhancing access to innovative technologies; and facilitating cross-sector partnerships. With its close contacts with academic researchers and clinicians in the field, FFRD is contributing to early identification of proofs of therapeutic concept.  Drug Repurposing is a topic of paramount interest to FFRD since 35% of the projects that FFRD supports and advises refer to it.  This is why FFRD contributes with its ideas and joins work groups involved in that topic, and organizes webinars, publications, and  meetings with various stakeholders (academia, industry) at national and European. FFRD provides and facilitates the connection with other partners in the research value chain, namely SMEs, Pharmas, and TTOs.

From its inception, FFRD has been building an EU outreach. Currently FFRD is involved in the European Joint Program for Rare Diseases (EJP RD-Horizon 2020 Grant agreement N°825575), coordinating the development of the EJP RD online academic course (as a series of MOOCs) as well as two EU innovative calls for proposals: the Rare Diseases Research Challenge-bridging academic and industrial research-and the 2021 EJP RD Joint Transnational Call focusing on Social Sciences & Humanities.

Audience Take Away Notes:

  • Knowledge of the French and European granting system for Rare Diseases
  • Knoweldge of the Foundation for Rare Diseases support action to research and training in the RD field.
  • Could help to identify funding opportunity for RD research in Biological and Humanities sciences

Biography:

Daniel Scherman is Exceptional-Class Research Director at the CNRS National Scientific Research Center at the Pharmacy Faculty of Paris University. He is Director of the Foundation for Rare Diseases.
Fields: Rare Diseases, Gene therapy, Gene and Drug delivery, Biotherapy, Bioimaging (h-index: 69; 543 quoted articles in WoS)

Additional functions:

  • Head of Medicine and Life Sciences Division of the European Academy of Sciences EURASC.
  • Corresponding Member of the Acacademie Nationale de Pharmacie.
  • Editor-in-Chief of the Rare Disease and Orphan Drug Journal (https://rdodjournal.com/).
  • Editor of the “Advanced Textbook on Gene Transfer, Gene Therapy And Genetic Pharmacology (Worlf Scientific)."

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