From a funding perspective, we understand that governments are faced with difficult ethical decisions on how best to allocate public funds, balancing the health care needs of the many and the few. For ultra-rare conditions, it is imperative there be a political decision to protect funding so that people living with very rare conditions 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, which allow for greater flexibility, efficient data collection and faster decision-making. These approaches enhance the chances of success in rare disease trials. The EFPIA-supported Rare Disease Moonshot initiative, which leverages public-private partnerships to accelerate research[8], is a fantastic example of this collaborative mindset in practice.
However, we are still inconsistent in our approach. The level of budget allocated to pharmaceutical spending varies significantly across Europe. In Nordic countries, Belgium, the Netherlands, Ireland and Austria, it is between 6 and 12 percent of the total health care expenditure. Whereas in other countries, such as Greece, Poland, Hungary and Bulgaria, pharmaceutical spend is between 20 and 30 percent of their total health care spend [9]. These numbers speak for themselves — creating potential for significant disparities in how new innovations are reimbursed and risking exacerbating health inequalities for European citizens. Some member countries have early access programs for new and innovative medicines, some do not. Some of these programs are free of charge, and others are not. If the pharmaceutical industry continues to significantly cover the cost for some of these early access programs, how can we incentivize payers to address the problem in a sustainable manner? Europe needs to adopt an approach based on the ability to pay, not willingness to pay.
The level of budget allocated to pharmaceutical spending varies significantly across Europe. Europe needs to adopt an approach based on the ability to pay, not willingness to pay.
EU competitiveness in life sciences: A dual opportunity for the rare disease sector
An EU Action Plan for rare diseases is more than a policy imperative; it is a moral obligation to the millions of individuals living with a rare condition.
By aligning with efforts already underway such as the Rare Disease Moonshot, 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 ecosystem of innovation, supported by a conducive policy framework and dedicated investment. Together, we can turn the tide for rare disease patients, transforming challenges into opportunities for breakthroughs that resonate beyond scientific laboratories, and into the lives of individuals living with rare diseases — in the EU and beyond.
Discussion about this post