I had the honour and the trust of being a keynote speaker and, indeed, the only patient and consumer voice at the 30th Anniversary Conference of the European Medicines Agency (EMA) in Amsterdam, Netherlands. Here are the key messages I conveyed on this occasion.
I highlighted the primary benefits of having a European common regulatory framework built over the last 30 years across 27 Member States for 450 million people living in the European Union (EU). This includes the benefit of having just one regulatory framework rather than 27.
The body of EU legislation and policies has enhanced the collaboration between the EU Member States’ regulators, medical experts, patient representatives, and companies. All patients and consumers benefit from a more robust scientific and ethical development or assessment of medicines. We directly benefit from European common scientific advice, benefit-risk assessment, and pharmacovigilance. Collaborating at the European level rather than the national level has enabled us to work with the best experts across Europe, whatever their nationality, and to pool their expertise, creating a specific added value. And overall, a common culture of collaboration and excellence has emerged, leading to more decisions focused on the clinical evidence of medicines and the pursuit of public interest.
The European regulatory framework and the EMA are a public good of high value to its citizens, which has enhanced equality and equity. It generates more equality for all citizens in Europe. In its absence, there will be inequalities of expertise, assessments, decisions on marketing authorisation, and pharmacovigilance across the territories in the EU. The framework drives more equity for some of the most vulnerable populations in Europe, thanks to the legislation on orphan drugs for people living with rare diseases and the legislation on the paediatric use of medicine. These two legislations have successfully steered scientific advancements, attracted public and private investments in therapy development, built clinical research infrastructures, and initiated new clinical trial designs and statistical methods. Today, they are the building blocks of the European ecosystem for clinical trials and for biotech
Along the way, this European regulatory framework has not only built the EMA as a world-class respected medicines agency, but it has also built a resilient decentralised network of national agencies, which have been reinforced thanks to this science-driven and public interest-driven European collaboration.
Nevertheless, I also shared our concerns. First, the current regulatory framework lacks strategic alignment across all relevant regulations and policies. A real compass should be integrated to further the European market integration, focusing on EU attractiveness and competitiveness, particularly on clinical trials, market access, and timely patient access.
Second, regulations for rare disease/orphan products, as well as those used in paediatrics, women’s health, and ageing persons, need to be established through specific policies and actions. The EMA has led the way for 25 years of patient engagement within regulatory scientific committees and scientific procedures. Patient representatives are full members of the Management Board, of most scientific committees and many working groups. In 2024, over 1,000 patient engagements took place at the EMA in relevant and meaningful ways. Peer-reviewed scientific publications show evidence of their impact on the scientific outcomes.
There remains incredible public trust in the EMA and the EU regulatory system. While there are evolving challenges, it remains a great asset in our field today. Rather than continue to reinforce the prevention of potential competing interests, we should focus our attention on more public debates at the time of authorisation and pharmacovigilance.
Yann Le Cam, Entrepreneur in Residence at VOZ Advisors, Founder & Past CEO of EURORDIS-Rare Diseases Europe, and Past member of the Management Board of the EMA
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