Toward clarifications, improvement and international convergence in regulatory approvals of rare disease therapies with a focus on Advanced Therapy Medicinal Products.

IRDiRC - Toward clarifications, improvement and international convergence in regulatory approvals of rare disease therapies with a focus on Advanced Therapy Medicinal Products.

Introduction

The current regulatory and manufacturing environment for innovative rare disease therapies is extremely complex. The current scheme struggles with adapting to the peculiarity of ultra rare diseases, more and more often treated with gene or cell therapies, and this lengthens their route to patients and increases costs of therapies. Moreover, the need to navigate different regulatory procedures in different jurisdictions further complicates the picture. Consequently, there is an increasing disparity in medicines available to treat rare diseases between different countries.

The overall objective of the task force is to identify barriers towards mutual recognition of the approval of therapies by different jurisdictions and propose concrete solutions.

Objectives

  • Identify barriers towards mutual recognition of the approval of therapies by different jurisdictions and propose concrete solutions
  • Propose solutions in regulation and guidelines to remove the legal and administrative barriers to regulatory convergence
  • Engage with regulatory agencies to provide research funders with specific recommendations on areas where investments are needed to enable the convergence of the systems

Timeline

Recruitment and assembly of Task Force members (Q1, 2025)

Kickoff meeting (Q2, 2025)

In-person workshop (Q4, 2025)

Members

  • Christopher McMaster – CIHR Institute of Genetics, Canada
  • Violeta Stoyanova-Beninska – European Medicines Agency, The Netherlands
  • Vinciane Pirard – Sanofi, Belgium
  • Albrecht Meichle – Ultragenyx, Switzerland
  • Ana Hidalgo-Simon – Leiden University Medical Center, The Netherlands
  • Anthony Lockett – Nevrargenics, UK
  • Babs Fabriek – Medicine Evaluation Board, The Netherlands
  • Christina Kyriakopoulou – European Commission, Belgium
  • Gopa Raychaudhuri – Food and Drugs Administration / CBER,   USA
  • Kristina Larsson – European Medicines Agency, The Netherlands
  • Kristine Pierce – Child Unlimited, University of New South Wales, Australia
  • Marco Rizzi – University of Western Australia, Australia
  • Maria Cavaller – Eurordis Rare Diseases Europe, Spain
  • Mary Wang – Rare Diseases International, Italy
  • Michael Moles – Berlin Institute of Health, Charité, Germany
  • Richa Lomash – National Institues of Health/ NCATS, USA
  • Sean Russel – Fondazione Telethon / PrimeRA Pharma Partners, Italy
  • Shekhar Natarajan – Dyne Therapeutics, USA
  • Solange Corriol Rohou – AstraZeneca, France