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introduction
There are around 7,000 rare diseases that we know of today. Each of these conditions affects only a small number of people, but collectively they affect a large population – estimated at 30 million people in the EU. Most rare diseases are serious and life threatening. However, 95 percent of those affected still do not have an approved treatment option.
In 2000, the EU Orphan Medicines (OMP) Regulation entered into force, establishing a centralized procedure for the designation of OMPs and providing incentives for their research, development and commercialization. At the time, there were only eight products to treat rare diseases on the market, compared to 196 today. Despite the success of the regulation, much remains to be done to advance innovation. This is a critical time to reflect on possible improvements to the regulation, based on developments over the past 20 years. The planned revision of the OMP regulation should aim to address unmet patient needs and strengthen the strengths of the current framework.
To achieve this, this review must take into account the perspectives of the different actors involved. As part of this effort, three stakeholders, representing patients, policy makers and industry, share their views on this topic.
Pernille Weiss, Danish MEP from the EPP Group
As a politician and shadow rapporteur for the Pharmaceutical Strategy for Europe in the Committee on Industry, Research and Energy, one of my priorities is to ensure the advancement of research in Europe. Innovation is only possible if companies invest in research and development. To ensure European autonomy in this crucial industrial sector, we must put in place measures that facilitate these businesses.
The current COVID pandemic has demonstrated that a strong and innovative European pharmaceutical sector is crucial. Likewise, it is essential that as policy makers, together with the pharmaceutical sector, contribute to the creation of a resilient healthcare landscape, which ensures innovative medicines for EU citizens.
This is even more important for rare diseases, where innovation is urgent. Despite the existing merits of the OMP regulation, much remains to be done: its revision represents an opportunity to further improve the environment for research and innovation in Europe. It is necessary to give an adequate impetus to innovation, where the protection of intellectual property rights and the competitiveness of the market are guaranteed. These conditions are also essential for the EU to be a hub for innovation in the pharmaceutical sector and to keep in step with other regions.
Camilla Harder Hartvig, Senior Vice President, International at Alexion Pharmaceuticals
Alexion is a global biopharmaceutical company focused on developing life-changing therapies for people living with rare diseases and devastating conditions. We want to help meet the huge unmet medical need that remains in rare diseases by continuing to innovate in areas where there are great opportunities to help patients and their families live their lives to the fullest.
The EU framework for orphan drugs has certainly enabled companies like ours to commercialize therapies for these small patient populations. While we recognize the need to revise and reassess incentives, given the current rare disease landscape, we also call for a careful and balanced review of the regulation. It should serve the original purpose of enabling continued innovation in rare diseases in order to ultimately improve the care and lives of patients living with rare diseases.
In addition to legislative tools, new models must be established to achieve significant progress in rare diseases. Here, cooperation between stakeholders can play a key role and have a real impact for the benefit of patients with rare diseases. A revised framework must find a common vision that addresses unmet needs, fosters innovation, and presents a forward-looking, patient-centered and predictable ecosystem.
Yann Le Cam, Managing Director of EURORDIS
#Jobsnotdone: a new European policy framework for rare diseases
While great progress has been made over the past decade and rare diseases are recognized as a public health and human rights priority, for people living with a rare disease in Europe, huge challenges remain. COVID-19 has further exacerbated the vulnerability of people with rare diseases. He demonstrated the need for more resilient health systems, while demonstrating the possibilities that arise from international research collaborations and rapidly streamlining procedures and regulatory activity.
A new policy framework is the only way to ensure that the progress made for the 30 million people living with a rare disease in Europe is not left to chance or chance over the next decade. We have a rare opportunity to bring politics into line with scientific, technological and societal changes.
Only by implementing new national plans and policies adapted to the goals of this decade, aligned with Europe’s strategy, can we actively design our preferred future: a future that prioritizes innovations. by patients and for social justice, fills major gaps in non-needs while strengthening Europe’s role as a global innovation leader, based on a cohesive rare disease ecosystem
Join the debate with #rare sickness & #rareconversations
This content was commissioned by Alexion
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