IRDiRC Announces Creation of Regulatory Scientific Committee to Tackle Regulatory Challenges in Rare Diseases Research – Press Release

22 September 2022Paris, France – IRDiRC, a global collaborative initiative with the vision to enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention, today announced the formation of the Regulatory Science Committee (RSC).

“IRDiRC recognizes that the fast-moving pace of rare disease innovation and therapeutics creates many considerations in the regulatory space. This committee will be pivotal to translate progress into effective, high quality and accessible therapies,”

said Samantha Parker, Vice-Chair of IRDiRC and Chief Patient Access Officer at Innoskel.

The RSC includes 11 experts from around the world, representing multiple geographies and stakeholders from regulatory bodies, patient groups, biotech and pharmaceutical industry, public and non-for-profit organizations, clinicians and scientists.

“The new RSC brings broad expertise across different stakeholders and geographies allowing for a balanced synergy that will provide insights and guidance into the advancement of IRDiRCs mission,”

said David A. Pearce, Chair of IRDiRC, President of Innovation, Research and World Clinics at Sanford Health based in Sioux Falls, South Dakota, and a Professor of Pediatrics, Sanford School of Medicine, University of South Dakota.

The mandate of the RSC will be divided into three key actions:

  1. Contribution to IRDiRC Task Forces and Working Groups to better address regulatory questions and increase the impact of IRDiRC activities​ 
  2. Multi-stakeholder interaction to identify pathways for regulatory harmonization and foster clinical research and therapeutic development​ 
  3. Identify new regulatory questions   

Members of the RSC

  • Ana Hidalgo-Simon, EMA – head of advanced therapies
  • Anne Pariser, VP, Medical and Regulatory Affairs at Alltrna
  • Cesar Hernandez, Director General of Common National Health System Portfolio and Pharmacy
  • Hanako Morikawa, Principal Reviewer, Office of Medical Devices II, PMDA
  • Julie Vaillancourt, Policy Advisor and Rare Disease Liaison in CBER/FDA
  • Kerry Jo Lee, Associate Director for Rare Diseases, CDER/FDA
  • Marie Christine Ouillade, AFM Telethon
  • Michela Gabaldo, VP ATMP Global Regulatory Affairs, Evotec
  • Oxana Iliach, Senior Director Regulatory Strategy, Certara
  • Reda Jundi, Pfizer Saudi Arabia cluster lead Regulatory
  • Violeta Stoyanova-Beninska, Chair of COMP at EMA

About IRDiRC
The International Rare Diseases Research Consortium (IRDiRC) is a global collaborative initiative launched in 2011 by the European Commission and the US National Institutes of Health to tackle rare diseases through research and accomplish the vision to enable all people living with a rare disease to receive an accurate diagnosis, care, and available therapy within one year of coming to medical attention.

For more information about IRDiRC and the Regulatory Science Committee, visit
IRDiRC Scientific Secretariat