EU takes ambitious action on rare diseases and sets plan by 2030.
The Trio of EU Council Presidencies for January 2022-June 2023 (France, Czechia, Sweden) has put rare diseases as a priority of the health agenda, driving political motivation in the area. Truth is, broad consensus is emerging on some short and long-term opportunities to improve the diagnosis, treatment and care of rare disease patients in Europe by 2030, where innovation is admittedly going to play a central role.
Advancing Europe’s leadership in the response to rare diseases: where are we, what next?
The summer edition of the European Health Summit (EHS) on June 16 was a strong reminder of the intention of the Council Presidencies that a cohesive EU response and strategic vision is needed in order to support patients, systems and scientific progress. Key stakeholders presented their concerns about the challenges and ambitions to advance the response to rare diseases in Europe, with Deputy Health Minister of Czechia Jakub Dvoracek opening the conversation and reaffirming the commitment of the country to drive a new EU pathway for rare diseases.
What outlook for European policy in rare diseases by 2030? How to support an innovative ecosystem for the benefit of patients?
Transformative therapies hold the promise for a cure for many patients with rare and serious life-threatening diseases. However, they come with scientific, regulatory, technological and ethical challenges that need to be overcome to ensure their uptake by health systems and patients’ access. They have been the subject of growing attention at EU policy level, with multiple debates covering a wide range of issues, from equal patients’ access to data gaps, healthcare systems sustainability, or cross border healthcare. These have been fueled by increasing momentum for a new EU Action Plan on rare diseases, alongside the shaping of the EU Pharmaceutical Strategy, and the revision of the EU legislation on orphan and pediatric medicines.
Prof. Cédric Hermans, who heads the Hematology Division at the Belgian Saint-Luc University Hospital, explained that in certain disease areas, patients can now be cured due to new treatments, a significant revolution when compared to early mortality rates only a couple of decades ago. “Treatments for rare diseases are not a cost, but an investment,” he noted, calling for more funding to be directed to care for rare diseases at the hospital level.
AI funding opportunities in healthcare
On April 24, 2018, the European Commission published a communication on the transformation of digital health. The Commission thus affirms its intention to accelerate the use of the digital tool to improve access to healthcare, prevention, diagnostics, and solutions thanks to high-performance computing. This project opens the door to an intensification of numerous research and industrial projects in Europe.
“The potential of artificial intelligence is huge; it can improve clinical processes, hospital workflows, diagnosis and treatments; it can also support the discovery of new medicines, and help us meet one the greatest challenges of the future, which is making our healthcare system sustainable” said Salvatore Scalzo, Policy and Legal Officer at DG CNECT.
14 Jul 2022The U.S. national economic burden of 379 rare diseases reached nearly $1 trillion in 2019, study reports.
27 May 2022gMendel announces that the Innovation Fund Denmark has invested 200,000 Euro in gMendel's novel diagnostic technology.
11 May 2022Denmark’s vision to become a front-runner in the use of artificial intelligence reflects on the country’s national strategy for AI.