About: rare diseases

Health 15-06-2022

Push to revamp rare disease EU framework intensifies before Czech presidency

Improving screening tools and strengthening the incentive system for orphan drugs are among the main suggestions for overhauling the European legislation on rare diseases, a topic included in the health priorities of the forthcoming Czech presidency of the EU.
Health 19-05-2022

EU cooperation key for rare diseases research, accessible treatment, says expert

France is "ahead of the curve" when it comes to treating rare diseases, but with 36 million Europeans affected, cooperation at the EU level to advance research and make treatments more accessible is essential, an expert warned. EURACTIV France reports.
Health 28-01-2022

Commission: Pharma companies need incentives to develop orphan drugs

The market will not regulate itself when it comes to finding solutions to rare diseases, said Sandra Gallina, director at the European Commission’s DG SANTE, adding there is pressure to kickstart innovation on orphan drugs.
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Health 07-01-2022

Access to treatment for SMA around Europe

Rare diseases are very serious conditions that affect around 30 million people in the European Union. Spinal Muscular Atrophy (SMA) is a rare genetic, neuromuscular disease affecting approximately 1 in 3,900–16,000 live births in Europe. In general, as rare diseases...
Health 01-07-2021

Incentives alone are not enough to cope with rare disease, report says

A new report by a multidisciplinary group of experts bringing together researchers, academia and the pharma industry, has urged policymakers to look beyond the EU's Orphan Medicinal Products (OMP) regulation to better address the unmet needs of patients with rare diseases.
Health 14-06-2021

Media Partnership – How to address the unmet needs of rare disease patients by transforming the European OMP landscape

The European Expert Group on Orphan Drug Incentives (OD Expert Group) has been established to discuss and bring forward innovative proposals to stimulate innovation and foster research into therapies for rare diseases.
Health 12-11-2020

Economist: ‘Premature’ to decide changes in Orphan Drugs law

Europe needs to give EU Orphan Drugs regulation more time to show its effectiveness before reviewing the incentives it provides and putting pharma innovation to the test, economist Adam Hutchings told EURACTIV in an interview.
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Health 24-07-2020

Europe at the crossroad of Rare Diseases innovation: lessons from the past and future outlooks

In shaping the future of rare diseases policies all stakeholders should cooperate to create an environment that actively supports research and clinical development of orphan medicinal products (OMPs). This entails building synergies all across the innovation ecosystem to address the...
Health 23-07-2020

Early stakeholder dialogues crucial for success of rare orphan medicines

Early dialogue between stakeholders is crucial for the effective development and success of rare orphan drugs, used to treat patients with rare diseases, Anna Bucsics, project adviser at the mechanism of coordinated access to orphan medicinal products (MoCA), told EURACTIV in an interview.
Health 13-07-2020

Data sharing critical to close ‘innovation blackhole’ for rare diseases

Access to data and 'real world evidence' obtained from observational data is needed to help push research and innovation on rare diseases, with support needed to encourage and harmonise this across Europe, stakeholders have said. 
Health 13-07-2020

Rare diseases: Ensuring Europe remains at the forefront of innovation

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In the past two decades, a combination of scientific breakthroughs coupled with strong incentive systems has seen considerable progress for rare disease patients, but this innovation has tapered off in recent years.
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Health 13-07-2020

The complexity of R&D for orphan medicines

There are multiple challenges inherent to drug development for rare diseases, all consequences of the small number of patients. For most of these diseases, the body of pre-existing knowledge is extremely limited with a lack of crucial information such as...
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Health 10-07-2020

From pandemic to rare disease, medical innovation is the answer

Living through a global pandemic, we can take some comfort from the enormous collaborative research and development response that is taking place to find a way out from under the shadow of the COVID-19 crisis. Scientists around the world are...
Health 09-07-2020

MEP: A common EU tool to incentivise production and availability of orphan drugs

When it comes to rare diseases and 'orphan drugs', technology is there to support an “objective” EU tool that could provide the pharma industry with the necessary incentives to research and produce orphan drugs and simultaneously avoid exhausting ailing health systems, MEP Stelios Kympouropoulos told EURACTIV in an interview.
Health 08-07-2020

EU orphan drugs law and the emergence of SMEs

The legal framework provided by the current EU orphan drugs regulation has encouraged a number of small and medium sized companies (SMEs) to develop innovative technologies in the fight against rare diseases in Europe, stakeholders told EURACTIV.com.
Health 07-07-2020

EURORDIS: Rare diseases need more centralised processes within the EU

Future improvement of the EU Orphan Medicines Regulation (OMP) could involve more centralised processes for all rare diseases at the EU level, the European Organisation for Rare Diseases  (EURORDIS) told EURACTIV in a written interview.
Health 06-07-2020

Commission’s ‘risky’ move to re-visit orphan drugs regulation

The European Commission is expected to present by the end of July an evaluation study on the pros and cons of the application of orphan and paediatric regulations, which deal with a niche segment of rare diseases that affect fewer than five in 10,000 people.
Coronavirus 05-05-2020

COVID-19 pandemic hinders access to care for patients with rare diseases

Nine out of ten people living with rare diseases across Europe have been negatively impacted by the coronavirus pandemic, including three out of ten who said that this has caused “life-threatening” disruption, according to a recent study by EURORDIS-Rare Diseases Europe.
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Health 28-02-2020

Rare diseases Day at the European Parliament

People living with SMA, ALS and other rare disease was the focus of the Rare Disease event at the EU Parliament on 18 February. The event was organised by Biogen in collaboration with EURORDIS, the patient organisation for rare diseases.
Health 04-12-2018

Romanian health minister: Prevention and innovative drugs to kill cancer

In order to face the rising cancer incidence across Europe, governments should prioritise prevention policies, as well as access to innovative medicines under an EU-wide plan, Romanian Health Minister Sorina Pintea told EURACTIV.com in an interview.
Health 02-03-2018

EU and UK patients with rare diseases in Brexit limbo

It is still unclear whether the UK will participate after Brexit in the European Reference Networks, which aim to help patients with rare diseases, potentially putting at risk millions of patients.
Health 12-10-2017

Patients: ‘Think carefully’ before questioning orphan drugs’ incentives

Organisations of patients with rare diseases have warned EU policymakers to “think carefully” before reviewing the incentives in the orphan drugs regulation, claiming that the pharma industry should not be discouraged from investing in new therapies.
Health 07-04-2017

IMI chief: ‘We need to learn how to share data in a safe and ethical manner’

Ethicists, humanists, lawyers and patients should come together to understand the risk of data in healthcare and whether patients are willing to take that risk, Pierre Meulien told EURACTIV.com.
Health 01-03-2017

Commission launches reference networks to face rare and complex diseases

In an effort to combat rare diseases and improve patient accessibility, the European Commission launched the European Reference Networks (ERNs) yesterday (28 February).