‘Parkinson’s is a man-made disease’
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.
rare disease
Poland sets sights on tackling postcode lottery in rare disease care
The EU has committed €77 million to strengthen European Reference Networks (ERNs) for rare disease patients.
Czech rare disease care needs improved coordination, patient group warns
Patients with rare diseases in the Czech Republic often face a fragmented healthcare system, with high-quality medical care available only for some diagnoses.
Bulgarian rare disease patients urge government to fund compassionate drug use
Insufficient screening, very low age limits, and problematic diagnosis continue to disproportionately affect patients with rare conditions in Bulgaria.
Ireland’s new Genomic Test Directory will boost rare disease treatment
Ireland’s new directory is designed to ensure that patients receive the most appropriate genetic tests to personalise treatment and improve early disease detection.
Turning aspirations into action for rare disease patients
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.
Bulgaria acknowledges discrimination against patients with rare diseases
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…
Doctors present first barrier for rare disease patients
Overcoming the doctor’s ego is a first hurdle for many rare disease patients in search of a diagnosis.
Rare disease realities: Patient perspectives across the EU
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
Rare disease realities – mobile
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
Ireland launches rare diseases public consultation to develop new strategy [Advocacy Lab Content]
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.
The call for an EU Action Plan for rare diseases
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.
Study reveals long diagnostic delays for rare diseases in Europe
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.
Belgian Deputy PM says a European Action Plan on Rare Diseases is ‘essential’
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.
EU pitches new pharma bonus to cure rare diseases. But will it work?
While DNA-altering treatments promise to cure many rare diseases, they might not qualify for planned bonuses.
Orphan medicines Pharma package provisions unleash uncertainties, stakeholders warn [Advocacy Lab Content]
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.
Poland struggles to accelerate efforts for a rare disease plan [Advocacy Lab Content]
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.
Slovakia trailing EU on rare disease treatment access [Advocacy Lab Content]
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.
25th Rare Disease Day – a year of opportunity for life-changing therapies [Promoted content]
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.
Right here, right now European public health policies must start giving people living with rare diseases a fair chance [Promoted content]
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.
Europe needs new 360 rare diseases strategy [Advocacy Lab Content]
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.
New rare diseases action plan on horizon, Europe at turning point [Advocacy Lab Content]
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.
New Swedish rare diseases strategy commissioned [Advocacy Lab Content]
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.
Athens launches first Greek language rare diseases list
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…
[Magazine] How genetic testing, new tech help in rare disease diagnoses
A European collaboration called Screen4Care bets on genetic testing and new tech to speed up diagnoses for rare diseases.
Rare disease patients across Spain don’t have the same access to treatments
Access to quick and accurate diagnosis and affordable drugs in Spain varies across the regions and depending on patients’ economic status.
Greek legal framework fails to address complexities of access to gene therapies
The Greek legislative framework lacks concrete solutions to advance gene therapies and improve access for patients, with several challenges – including financial support, treatment availability and stakeholder cooperation – remaining.
Italy’s advanced therapy journey
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…