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37th Workshop of the EURORDIS Round Table of Companies (ERTC)

January 2024

Transforming rare disease foresight into action

How can additional measures support the development of available, accessible and affordable treatments for rare disease patients?

Wednesday, 21st February 2024, Doubletree by Hilton Brussels City in Brussels


Public programme

Overarching goal and scope

As we are all looking to the details of the revision of the EU general pharmaceutical legislation, we know that no change will happen without the leadership from academics, researchers, patient groups and developers to make the most of opportunities arising from an ever-evolving healthcare environment.  

This workshop will invite participants to think beyond the legislation, reflecting on rare disease patients’ expectations of the proposed reform and their understanding of its shortcomings, taking stock of key milestones along our advocacy journey for patient engagement, and discussing how additional instruments can further support the development of and access to orphan and paediatric medicines. 

Drawing on the Rare 2030 recommendations (February 2021), which set out the roadmap towards a new, future-proof policy framework for rare diseases in Europe, this workshop will bring forward and discuss the added value of three disruptive instruments, designed to help streamline the medicines development path and better integrate patients in the decision-making processes that affect their lives:

  • Orphan Drug Development Plan: 
    This plan is both a concept and a tool derived from the Orphan Drug Development Guide book, which the International Rare Diseases Research Consortium (IRDiRC) originally created to help developers navigate the rare disease ecosystem. It includes a series of guiding principles on how to start and conduct efficient (and sustainable) drug development in rare diseases until initially identified unmet medical needs are addressed. It also ensures that patient input is captured all along the medicines lifecycle. 
  • PRIority MEdicines (PRIME) scheme: 
    To enhance early dialogue with developers, the European Medicines Agency (EMA) established PRIME, which is based on a combination of iterative scientific advice and provisions for the accelerated assessment of promising medicines that address unmet medical needs. Whilst this voluntary scheme could allow for better patient involvement, it has proven to be of crucial benefit for rare diseases at the time of orphan drug designation and market authorisation.
  • Mechanism of Coordinated Access to Orphan Medicinal Products initiative: 
    Another example of early dialogue involving developers, patients and payers, the Mechanism of Coordinated Access (MoCA) to Orphan Medicinal Products aims to support a more equitable access to rare disease therapies. 10 years after this voluntary framework was established, it is time to learn from experience and expand this pilot to a more structured and sustained platform, and to explore ways in which it can interplay with the two previous instruments.

Throughout the day, sessions will demonstrate how our community has positioned these instruments in rare disease advocacy, how these have evolved in light of the revision of the legislation and how the underlying concepts within these instruments can be adapted to bring us closer to the goal of developing 1000 new therapies by 2030 for the benefit of rare disease patients and society at large.

This workshop will conclude by placing these three tools into broader context, reflecting on perspectives from across the rare disease community (including paediatric onset, rare cancers), and against the backdrop of the EU Regulation on Health Technology Assessment and the ongoing reflections around improved collaboration between Member States in negotiations on pricing and reimbursement and single procurement.


Please send any questions concerning registration to Martina Bergna at: