A holistic, realistic approach to OMP reform and action

The fate of the rare diseases ecosystem hangs in the balance. Our actions going forwards could have significant implications for the future of the patient community

By Dr Alexander Natz

Secretary-general at EUCOPE

28 Feb 2024

As was the case in 1999, the European Union currently finds itself at a pivotal juncture. The fate of the pharmaceutical sector and the rare disease ecosystem hangs in the balance, with significant implications for the future.  

Contributing to the development of over 230 orphan medicinal products (OMPs), the introduction of the OMP Regulation in 2000 brought stability and predictability for developers. At the time, we borrowed from experiences in other jurisdictions, crafting a system that would work for Europe. Two decades later, we have a clear understanding of its successes and where improvements are necessary. It is imperative to examine and refine our approach, ensuring that the OMP ecosystem continues to evolve and meet emerging challenges. 

Policymakers and stakeholders must be realistic about what reforms are needed, and what shortcomings can viably be addressed through the revision of the pharmaceutical legislation. While important regulatory updates are being proposed, regrettably, not all challenges, such as access equity, can be achieved through the pharmaceutical package. Careful consideration and vigilance must prevail to mitigate any unintended consequences that could harm industry, patients and healthcare systems. Some impacts would be felt immediately, and others would take years for the real impact to be seen. Rolling back the undesired changes would also come with major time lags, illustrating the severity of the revision. 

Policymakers and stakeholders must be realistic about what reforms are needed, and what shortcomings can viably be addressed through the revision of the pharmaceutical legislation

Across the product lifecycle and access pathways, creative thinking is needed at both national and EU level. Several initiatives are underway that can bring needed reform. Project JARDIN looks at embedding the European Research Networks (ERNs) in national healthcare systems. The EU Heath Technology Assessment (HTA) Regulation and the European Health Data Space Regulation bring the promise of accelerated access to transformative therapies and accelerating drug discovery through an exchange of data. At national level, initiatives are bringing key reforms, such as the rare disease action plan in Italy, which reinforces a holistic approach from early diagnosis to treatment and healthcare professional training, and in France, which reviewed its early access and reimbursement systems to accelerate access to novel OMPs. 

Legislative reforms alone will not overcome the development and access challenges in the rare disease ecosystem, especially for the 95 per cent of rare diseases with no authorised treatments. Progress lies in fostering innovation and promoting access through a collaborative journey. To drive effective change, the EU must adopt a European rare disease action plan, a strategic blueprint for cohesive policy and action in the rare disease sector. Such a plan must integrate diverse policy solutions under a unified vision, leveraging existing successes and addressing critical challenges. 

Simultaneously, the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) and our members, working in partnerships, look to address barriers in the ecosystem today, through implementable solutions.Together4RareDisease aims to bring together ERNs and developers in a pre-commercial context to leverage their different expertise to unlock research for people living with a rare disease. The European Expert Group on Orphan Drug Incentives (OD Expert Group), a multi-stakeholder partnership, outlined a series of recommendations across the product lifecycle to explore groundbreaking ideas and solutions for the rare disease ecosystem. This partnership-driven approach seeks to transform the landscape of rare disease treatment and research across Europe, aiming for a significant and tangible impact on the lives of millions affected by rare conditions. 

As we forge ahead, it is crucial to remember that each partner in this journey brings unique strengths. By working in concert, we can strive towards a future where all people living with a rare disease have access to effective treatment and the hope for a better quality of life. 

Read more about Rare Disease Day on our Rare Disease Hub or in our Rare Disease supplement.