About: Orphan drugs
Revision of orphan and paediatric drug framework needed, say reports
Both the European Parliament and the World Health Organisation (WHO) have reiterated the need to update the regulations on medicines for children and orphan drugs, in order to better address unmet needs of children and adolescents in cancer care.
EU calls for stronger cooperation to fight rare diseases
Marking the World Rare Disease Day, Health Commissioner Stella Kyriakides and health ministers called for stronger coordination around rare diseases with high hopes on European Reference Networks (ERNs).
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.
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.
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.
Report: Without orphan drugs law, 2mn patients deprived access to therapies
In the absence of the EU orphan drugs regulation, which focuses on rare diseases, approximately two million patients would not get access to therapies, a new industry-funded report has found.
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.
Czech drug policy shifts toward patients with rare diseases
The Czech government has launched a legislative initiative to improve access to most modern medications and rare disease drugs, Health Minister Adam Vojtěch told EURACTIV in an interview, adding that patients are involved in the decision-making process at a very early stage, including in drafting the law.
New drugs: how much are governments paying for innovation?
The European healthcare sector is divided over the “actual value” EU governments pay for the authorised and partly public-funded novel drugs, compared to medicines already on the market.
EFPIA President: Next EU Commission should prioritise competitiveness in pharma innovation
The next European Commission should focus on the bloc’s role in the global context of innovation, as China is “decisive” about creating a reliable framework for intellectual property and connecting biotech start-ups and digital companies, EFPIA’s President Stefan Oschmann told EURACTIV.com in an interview.
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.
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.
Lawmakers warn EU pharma industry about drug pricing
Pressure is mounting on pharmaceutical companies to reconsider the way they do business by refocusing their efforts on meeting patient needs and health outcomes rather than their own profits.
Health minister: Drugs pricing will top Malta’s EU presidency
The Maltese EU presidency will seek more transparency in the way pharmaceutical companies negotiate with member states on medicines’ pricing, Health Minister Chris Fearne said in an interview with EURACTIV.