December 15, 2025

IPOPI discusses how to secure access for PID patients in the EU through the Critical Medicines Act

On November 12, 2025, IPOPI organised a PID Forum to discuss how access could be secured for persons living with primary immunodeficiencies (PIDs) in the EU. The PID Forum specifically looked at a legislative initiative under discussion at the EU level called the Critical Medicines Act and focused on immunoglobulin replacement therapies and gene therapies. The Forum, held online, welcomed the interventions of the Members of the European Parliament Tomislav Sokol (EPP; Croatia), Peter Agius (EPP, Malta) and Ondrej Dostal (NI, Czechia).

Moderating the event, Johan Prévot, Executive Director of IPOPI, emphasised the importance of the CMA for PID patients and the opportunity it presents to strengthen Europe’s capacity to ensure equitable access to essential medicines, reinforce supply resilience and improve crisis preparedness.

Speakers in this Forum were:

  • MEP Peter Agius (EPP, Malta) highlighted the challenges faced by persons living in Malta to access therapies and the lack of equitable access, and explained his hopes for the CMA to have more flexible joint procurement mechanisms and stronger and more resilient supply chains.
  • MEP Ondrej Dostal (NI, Chechia) emphasises that pricing and reimbursement remain key barriers both in the Czech Republic and across the EU, and he hopes for the CMA to address both aspects to be effective.
  • Martine Pergent, IPOPI President, is setting the scene and presenting the opportunities the CMA legislation provides for people living with a PID, especially for Ig therapies and gene therapies.
  • Daniela Ene, a person living with a PID, explains the challenges of getting authorisation and coverage for gene therapy for her condition
  • Prof Fabio Candotti, President of the European Society for Immunodeficiency (ESID), on the rocky road of gene therapy from development and authorisation to continued access to it in spite of market changes.
  • Otilia Stanga, President of the Romanian patient organisation (ARPID) and IPOPI Board member, on the need for continued and stable access to immunoglobulin replacement therapies.
  • Rafal Ramotowski, Associate Director and Lead for EU Public Affairs and Communications at the Plasma Protein Therapeutics Association (PPTA), on the need for tailored approaches to immunoglobulin therapies supply chain vulnerability assessment.
  • Dr Leni von Bonsdorff, Executive Director of the International Plasma and Fractionation Association (IPFA), emphasised the importance of strategic partnerships and projects, particularly regarding plasma-derived medicines.
  • MEP Tomislav Sokol (EPP, Croatia), rapporteur for the CMA file at the European Parliament, summarised his main priorities for the CMA: strengthening EU strategic autonomy and ensuring equitable access to medicines across the EU.

The online format of this Forum increased the number of participants, reaching an audience of around 100 participants.

IPOPI would like to thank CSL Behring, Grifols and Takeda for their continued support.

The report from the meeting will soon be made available.