At the heart of the European Union’s pursuit of excellence in life sciences lies a profound responsibility. A mandate not driven solely by a desire for economic growth but propelled by a human mission: To offer hope and progress for the millions across Europe living with rare conditions and their families.
Rare diseases, despite their individual rarity, collectively impact an estimated 36 million individuals within the European Union. This accounts for approximately 8 percent of the population[1]. These conditions are not as “rare” as one might think. They present unique challenges that demand our immediate attention and action — not only because of the emotional and financial strain they impose on patients and their families but also because of the intrinsic inequities in health care access and treatment opportunities they present[2].
As we look toward a global stage, particularly in sectors where China and the U.S. are making substantial investments, the recent Antwerp Declaration[3] — launched by Ursula von der Leyen and Belgian Prime Minister Alexander De Croo — as well as the European Council’s call for Competitiveness Deal including a dedicated Health and Life Sciences Strategy[4], has ignited a renewed focus on the EU’s competitiveness. This ambition to strengthen the EU’s competitiveness needs to leverage and recognize the strategic importance of the life sciences sector to the Union.
Maintaining leadership
The introduction of Orphan Medicinal Products legislation has catalyzed remarkable progress, with over 200 orphan medicines authorized by the EMA since 2000[5]. It is vital that this progress is not lost in the current legislative review of the Pharma Package.
A shared commitment to crafting and implementing an EU action plan for rare diseases, unifying strategies across member countries, and amplifying existing National Action Plans and policies, could deliver transformational value to people living with rare diseases by ensuring all parties are pulling in the same direction. EU policymakers must rise to the occasion, crafting an environment that continues to nurture innovation, accelerate research, and ensures groundbreaking therapies reach those most in need.
Whilst it is important we recognize progress made over the past decade in collaborations, public private partnerships and innovation, it is also imperative we reflect on remaining gaps, treating them as opportunities upon which the foundations of an EU action plan must be built.
With increased investment, and more targeted translational research, new treatment options for many rare and ultra-rare conditions could soon be within our reach. To truly realize the value of these innovations, it is imperative we adapt and accelerate processes, aligned across the value chain, from both regulatory and payer standpoints. We should carefully assess learnings from existing initiatives such as the EMA PRIME scheme[6] and the Mechanism of Coordinated Access to orphan medicinal products (MoCA) from EURORDIS[7] and consider how we implement this knowledge.
EU policymakers must rise to the occasion … to nurture innovation, accelerate research, and ensure groundbreaking therapies reach those most in need.
New therapies must get to patients who need them, when they need them
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.
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.
Our system needs to change. The current approach does not deliver the right solution for many rare conditions. However, there is potential waiting to be realized. Europe holds many of the necessary ingredients to be a world-leader in delivering new medicines to treat rare diseases, but lacks consistency in the systems and frameworks which could allow these innovations to truly flourish.
A renewed focus on rare diseases presents a dual opportunity: it could contribute to the EU’s competitiveness as a new frontier for life sciences innovation, and its health and prosperity could be propelled by it.
References:
[1] European Commission. Rare Diseases. https://health.ec.europa.eu/european-reference-networks/rare-diseases_en [Accessed April 2024]
[2] Chiesi. Global rare diseases report: Rare disease burden of care and the economic impact on citizens in Germany, France and Italy. https://chiesirarediseases.com/assets/pdf/rare-disease-burden-of-care-and-the-economic-impact-on-citizens.pdf. [Accessed June 2024]
[3] Europur signs the Antwerp Declaration for a European Industrial Deal. Available at: https://europur.org/europur-signs-the-antwerp-declaration-for-a-european-industrial-deal/#:~:text=On%2020%20February%202024%20in,associations%20have%20signed%20the%20declaration. [Accessed June 2024]
[4] Towards A New European Competitiveness Deal, Special Meeting of the European Council, 17-18 April 2024
[5] European Commission. Orphan medicinal products. https://health.ec.europa.eu/medicinal-products/orphan-medicinal-products_en. [Accessed June 2024]
[6] European Medicines Agency. PRIME: priority medicines. Available at: https://www.ema.europa.eu/en/human-regulatory-overview/research-development/prime-priority-medicines. [Accessed June 2024]
[7] EURODIS. Mechanism of Coordinated Access to orphan medicinal products (MoCA). Available at: https://www.eurordis.org/moca/. [Accessed June 2024]
[8] Rare Disease Moonshot. Available at: https://www.rarediseasemoonshot.eu/. [Accessed June 2024]
[9] Organisation for Economic Co-operation and Development. Pharmaceutical Spending: Available at: https://data.oecd.org/healthres/pharmaceutical-spending.htm#indicator-chart. [Accessed June 2024]