From a funding perspective, we understand that governments face difficult ethical decisions about how best to allocate public funds and balance the health care needs of the many and the few. For extremely rare diseases, political decisions are essential to protect funding so that people living with extremely rare diseases are not left behind.
As an industry and as a continent, we continue to push scientific boundaries in Europe. We are embracing new clinical trial designs targeting specific patient groups, allowing greater flexibility, efficient data collection and faster decision making. This approach increases the chances of success in rare disease trials. EFPIA-backed Rare Disease Moonshot initiative leveraging public-private partnerships to accelerate research(8)is a great example of this collaborative mindset in action.
However, we are still not consistent in our approach. The level of budget allocated to pharmaceuticals varies considerably across Europe. In the Nordic countries, Belgium, the Netherlands, Ireland and Austria, it is between 6 and 12% of total health care spending. In other countries, such as Greece, Poland, Hungary and Bulgaria, pharmaceuticals account for 20 to 30% of total health care spending. (9). These figures speak for themselves. There is a potential for significant disparities in how new innovations are compensated, and this risks exacerbating health inequalities among European citizens. Some Member States have early access programmes for new and innovative medicines, some do not. Some of these programmes are free, others are not. If the pharmaceutical industry continues to shoulder a significant portion of the costs of some of these early access programmes, how can we incentivise payers to address the problem in a sustainable way? Europe needs to adopt an approach based on ability to pay, not willingness to pay.
The level of budget allocated to pharmaceutical spending varies considerably across Europe. Europe needs to adopt an approach based on ability to pay rather than willingness to pay.
EU competitiveness in life sciences: double opportunities in rare diseases
The EU Action Plan on Rare Diseases is not just a policy imperative, but a moral obligation for the millions of people living with rare diseases.
By aligning with efforts already underway, such as the Rare Disease Moonshot, the European Joint Programme on Rare Diseases (ERDERA), Together4 Rare Diseases (T4RD), and Screen4Care, we can bridge the gap between potential and reality with a vibrant innovation ecosystem supported by favorable policy frameworks and committed investment. Together, we can change the flow of rare disease patients and turn challenges beyond the scientific laboratory into opportunities for groundbreaking discoveries that will resonate in the lives of individuals living with rare diseases across the EU and beyond.
