The European Parliament’s SANT Committee has revealed it will launch hearings and commission studies on mental health, antimicrobial resistance, and digital health.
Perspectives from Martin Nielsen, Denmark, who lives with hemophilia B, and Wolfgang Miesbach, professor of medicine at Frankfurt University Hospital, Germany, on the psychological impacts of hemophilia.
Europe’s flawed oversight of pesticides may be fueling a silent epidemic, warns Dutch neurologist Bas Bloem. His fight for reform pits him against industry, regulators — and time.
The EU has committed €77 million to strengthen European Reference Networks (ERNs) for rare disease patients.
“We need a unified approach to close the gaps and eliminate inequalities,” argued Vytenis Andriukaitis (S&D, Lithuania).
Patients with rare diseases in the Czech Republic often face a fragmented healthcare system, with high-quality medical care available only for some diagnoses.
Insufficient screening, very low age limits, and problematic diagnosis continue to disproportionately affect patients with rare conditions in Bulgaria.
Belgium first called for a law to tackle drug shortages in May 2023.
A coherent strategic framework could be set by 2030, with strong political support and patient advocates underlined.
Inspired by the rare disease community’s passionate drive to advance policies that provide equitable access to care, UCB spearheaded Aspire4Rare: a global initiative supporting the development of rare disease policies.
A new binding decision in Bulgaria could add millions of leva to the health budget of one of the poorest EU countries, as rare disease patients can now seek compensation for discrimination.
The post Bulgaria acknowledges discrimination against patients…
Overcoming the doctor’s ego is a first hurdle for many rare disease patients in search of a diagnosis.
Europe’s pharmaceutical sector has grown slowly for 25 years and its share of research is shrinking. It’s time to turn things around
An opportunity for the EU and its member states to meet growing patient need for essential medicines
References [1] Fabry Disease. Cleveland Clinic. https://my.clevelandclinic.org/health/diseases/16235-fabry-disease [2] Familial Hemophagocytic Lymphohistiocytosis. National Library of Medicine. https://www.ncbi.nlm.nih.gov/books/NBK1444/ [3] Autosomal recessive congenital ichthyosis 4B. National Library of Medicine. https://www.ncbi.nlm.nih.gov/medgen/108615 [4] European Reference Networks. European Commission. https://health.ec.europa.eu/rare-diseases-and-european-reference-networks/european-reference-networks_en [5] CRA Insights. A Landscape Assessment of Newborn Screening (NBS) in Europe. Charles River Associates. https://media.crai.com/wp-content/uploads/2021/11/28135510/CRA-Insights-NBS-Policy-Updated-28-February-2024-vSTCCR.pdf
Greater collaboration between industry, government, policymakers and the NHS will be critical for treatment options in the UK to match the pace of scientific innovation. What is our route to achieving timely patient access across the country like our European neighbours?
Warsaw’s Rare Diseases Plan for 2024-2025, recently approved by the Council of Ministers, envisions comprehensive and coordinated healthcare services, with approximately €22 million allocated to achieve its implementation.
France’s Health Innovation Agency (Agence de l’innovation en santé, AIS) plans to invest €170 million in innovations to improve preventative healthcare in eight fields, including children’s health, mental health, chronic and rare diseases.
Pharma groups and patients say guidance prioritising mass trials threaten development of drugs for rare diseases
Ireland has launched a public consultation to inform the development of a new National Rare Disease Strategy. The results of the consultation will be considered by a steering group tasked with developing the strategy for 300,000 patients.
Driving excellence: A new EU Action Plan for rare diseases that drives innovation, creativity and competitiveness can position the EU as a leader on the global stage.
Genetic diagnostic technology advances for rare diseases offer hope, but gene-tech comes wrapped in complex ethical concerns. Will Europe accelerate modern diagnostics, or will ethical concerns slow and limit innovation?
A recent European-wide patient survey study published in the European Journal of Human Genetics on Tuesday has uncovered significant delays in diagnosing rare diseases across the continent.
The Bulgarian parliament has adopted a decision requiring the state to pay for the treatment of some patients with rare diseases. The decision follows decades of legal battles and accusations of discrimination.
We can meet the needs of EU patients who rely on plasma therapies — and the EU’s goals for strategic autonomy — by ensuring a vibrant plasma-based biotech sector.
Belgium is supporting the creation of a European action plan for rare diseases and is joining other EU member states adopting a national plan, Belgium’s Deputy Prime Minister and Minister of Health and Social Affairs, Frank Vandenbroucke, said.
Patients with rare diseases are clear beneficiaries of the European Parliament’s vote on the EU Pharma Package, says Belgian MEP Frédérique Ries. The revised package will also embed a European solidarity mechanism.
Patients suffering from rare diseases across the EU, and their families, are struggling on the road to lifesaving and life-altering healthcare. The difficult patient Odyssey is warped by a postcode lottery.
While DNA-altering treatments promise to cure many rare diseases, they might not qualify for planned bonuses.
A committee recommended assisted dying for people with an incurable disease and just six months to live, or those with 12 months if they have a neurodegenerative condition.
There are more than 6,000 rare diseases, with 27 different healthcare systems governed by conflicting rules and principles, with no harmonisation for orphan medicines payments. Rare disease clinicians hope the Pharma Package will bridge regulatory gaps.
Bulgaria’s lack of a comprehensive strategy for rare diseases now poses a serious risk to patient health. Insufficient screening has left Bulgarians with rare diseases diagnosed late, or not at all, while fewer orphan drugs are available on average.
Poland adopted a National Rare Diseases Plan in 2021 – by December 2023, it had lapsed. New efforts are underway to push for a sustainable plan, but an exact date for resuming the Polish Rare Diseases Plan remains undisclosed.
People living with rare diseases in Slovakia are facing a high risk of impoverishment and discrimination, while Slovak access to medicines continues to rank among the worst in the EU.
Innovative therapies are key to early diagnosis of rare diseases, but soaring costs are pricing quality treatment beyond the reach of many patients, a Czech patient organisation has warned.
On the 25th Rare Disease Day, three representatives of the rare disease community consider how to maximise the potential of the EU’s Joint Clinical Assessment to accelerate patient access to ATMPs.
This year’s Rare Disease Day (29 February) comes at a critical moment for European rare disease policy. Now more than ever, it’s essential that EU policies enable health equity for people living with rare diseases, says Alexion’s Soraya Bekkali.
Rare Disease Day, 29 February, marks a moment when an estimated 30 million people living with rare diseases across Europe gain a louder voice. Europe now has a generational opportunity to scale up programmes capable of improving millions of lives.
Rare Disease Day, 29 February, marks a moment in which Europe is facing a generational opportunity to scale up programmes capable of improving millions of lives. A new data-led, 360-comprehensive strategy is called for by rare disease experts.
Despite Belgium’s prominent position as Europe’s second-largest biotech industry and its significant achievements in medical innovation, the pursuit of fast and sustainable access to life-saving therapies remains problematic, for Belgium and Europe.
Sweden is preparing a national strategy to improve healthcare for people with a rare disease. The long-called-for plan will tackle major care inequalities amongst the estimated half a million people in Sweden living with a rare disease.
Greece has launched its own Rare Disease list, in Greek. Until now, no detailed classification existed for new rare diseases in the Greek language. The list is an analytical archive of every rare disease, and its sub-categories, including genetic mutat…
A European collaboration called Screen4Care bets on genetic testing and new tech to speed up diagnoses for rare diseases.
National and EU-level support goes a long way towards supporting research into gene therapies against rare diseases, according to German researchers, who said however that Europe still lags behind North American countries on innovation in this area.
Advanced Medicinal Therapies (ATMPs), including gene and cell therapies, offer treatment options for patients suffering from rare or ultra-rare diseases. Italy is learning to see expenditure on advanced therapy drugs as an investment but the EU’s pharm…
While gene therapies have the potential to transform the approach to rare diseases and improve the quality of life for patients, stakeholders and patients complain that they are very expensive to develop and difficult to access.
Gene therapies can transform the approach to rare diseases and improve the quality of life for patients. But developing and accessing them is hampered by regulatory burdens, cost and reimbursment schemes. In this special report, Euractiv takes a look a…
Ricardo Marek, President Europe & Canada, Takeda Pharmaceuticals, shares these insights with POLITICO Studio.
National healthcare systems must be integrated into the EU’s approach to rare diseases, EU lawmakers and stakeholders urged on Wednesday (11 October), underlining the importance of the European Reference Networks (ERN).
The EU wants to steer drug development to cover research gaps, but industry hates the idea.
Members of European Parliament welcomed the proposed revamp of the bloc’s regulatory framework for pharmaceuticals during a discussion this week but did not shy away from criticising certain aspects. The Parliament’s Public Health Committe…
Due to their complex nature, rare diseases such as amyotrophic lateral sclerosis (ALS) demand a comprehensive approach, a need that a series of EU initiatives are trying to meet.
Stringent new requirements have forced livesaving devices off the market.
She was a prominent voice on health issues in the European Parliament.
A major drug pricing crackdown in the US is threatening drug companies’ bottom lines.
The OD Expert Group and policymakers discussed the challenges ahead for the rare disease community, expressing hopes and doubts about the revision of the regulatory framework. The Group presented its multi-stakeholders and consensus-based proposals for…
