About: rare diseases
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...
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.
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.
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.
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...
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.
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.
Rare diseases: Ensuring Europe remains at the forefront of innovation
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.
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...
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...
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.
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.
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.
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.
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. VideoPromoted content
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.
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.
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.
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.
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.
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).
Commission must bring healthcare to the fore
Given that the current European Commission has not prioritised healthcare, it looks as though it is up to the European Parliament to push the agenda, writes Denis Horgan.
Health matters should supersede European bureaucracy
European red tape should not be an obstacle to effective healthcare. For patients with diseases like sarcoma, waiting means suffering and often avoidable death, writes Marlene Mizzi.