About: 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.
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 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 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.
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 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).
Health 10-02-2017

How Italy and France take on rare sarcoma cancers

EU member states are exploring ways to effectively tackle the rising occurrence and complex nature of rare cancers known as sarcomas.
Health 09-02-2017

Complex sarcoma cancer needs an overall strategy, report says

The diverse nature of rare cancers like sarcomas poses severe challenges to the medical community and policy-makers who need to seek a holistic approach, according to a report.
Health 07-02-2017

Rare cancers: A headache for policymakers

The complexity of diagnosing and treating rare forms of cancers like sarcomas can be addressed by a multi-level approach and better coordination among member states, insist experts in the field.
Vytenis Andriukaitis
Health 26-02-2015

Health Commissioner calls for national plans to tackle rare diseases

Patients with rare diseases are more vulnerable, as they live with more ambiguity about their affliction than others, according to Health Commissioner Vytenis Andriukaitis. But with the help of patient groups and national action plans in member states, their lives can be improved.

€144 million earmarked for new research on rare diseases

The European Commission will provide €144 million of new funding for 26 research projects, with the objective to deliver 200 new therapies for rare diseases by 2020, the EU's executive announced today (28 February).

Study finds orphan drugs economically viable

Orphan drugs, pharmaceutical agents that are developed specifically to treat rare diseases, have the potential to generate as much lifetime revenue as drugs used for more common health conditions, according to a new study.

Sufferers want EU ethics code for drug testing

The European Union needs to play a stronger role in coordinating research into new drugs and should help produce a code of ethics for trials, health officials said last week.
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