Articles tagged with: Rare diseases
Innovation is delivering therapies for people with rare diseases – but R&D needs support Partner Content
22 Feb Opinion
Innovation is delivering therapies for people with rare diseases – but R&D needs support
Investment in R&D in Europe is vital if we are to tackle unmet medical needs, says Nathalie Moll
Boosting orphan drugs' development and accessibility in Europe Partner Content
21 Feb Opinion
Boosting orphan drugs' development and accessibility in Europe
Just 5% of people living with a rare disease have received a transformative treatment approved for the entire EU. The upcoming revision of the Orphan Medicinal Products Regulation is the EU’s chance to change that, writes Frederique Ries MEP
Rare Diseases: Time for Innovation Partner Content
27 Jun
Rare Diseases: Time for Innovation
Pfizer's Reda Guiha and MEP Maria da Graça Carvalho want to see the EU do more to deliver on its promise for people living with a rare disease, by supporting research in new medical innovations.
Unlocking the potential for rare disease therapies through wider collaborations between European Reference Networks and industry Partner Content
27 Jun
Unlocking the potential for rare disease therapies through wider collaborations between European Reference Networks and industry
Prominent MEPs seeking quicker wins for rare disease patients are backing closer collaboration between Europe’s reference networks and pharma companies through a novel multistakeholder initiative.
Coming together for Global ALS Awareness Day Partner Content
21 Jun
Coming together for Global ALS Awareness Day
MEP Cristian-Silviu Buşoi and Biogen’s Alessandro Tempra explain why all stakeholders should work together in the fight against ALS and other rare diseases.
Rethinking the Orphan Regulation Partner Content
19 May
Rethinking the Orphan Regulation
Europe risks undercutting its best asset for treating millions of rare cancer sufferers, says Stéphane Lassignardie, VP for Region North & Oncology at AbbVie Europe.
Orphan Regulation: Time for a rethink? Partner Content
19 May
Orphan Regulation: Time for a rethink?
The upcoming review is an opportunity to not only accelerate the development of rare disease treatments, but also to provide equitable access for patients across Europe, says MEP Nicolás González Casares, S&D group shadow rapporteur on the EU’s Beating Cancer Plan report.
Orphan medicinal products: onwards and upwards
28 Feb
Orphan medicinal products: onwards and upwards
The Orphan Medicinal Products regulation delivered impressive results for rare disease patients, but the pending revision can move it up a gear, argues Stelios Kympouropoulos
Tackling rare diseases through enhanced coordination Partner Content
28 Feb
Tackling rare diseases through enhanced coordination
European Reference Networks, support for research, and the revision of EU medicines legislation, is key to the Commission’s plans for helping people suffering from rare diseases
Rare Diseases Day: How the French Presidency can change the rare disease landscape Partner Content
28 Feb
Rare Diseases Day: How the French Presidency can change the rare disease landscape
The Parliament Magazine spoke to French and European policymakers to find out.
Learning from the rare disease community Partner Content
25 Feb
Learning from the rare disease community
Europe’s pharmaceutical entrepreneurs are taking the lessons learned from this community to develop health innovation which would not have been dreamt of a few decades ago
Rare Diseases: Towards a patient-centred EU Blood Directive Partner Content
24 Feb
Rare Diseases: Towards a patient-centred EU Blood Directive
For rare disease patients who rely on plasma-derived medicinal products, supply has become the major safety issue
Rare Diseases: EU Orphan Regulation, backing innovation to address unmet needs Partner Content
23 Feb
Rare Diseases: EU Orphan Regulation, backing innovation to address unmet needs
Our capacity to keep on developing new treatments for people living with rare disease depends on Europe’s research ecosystem
Partnerships are at the Heart of Future Innovation for Rare Diseases Partner Content
22 Feb
Partnerships are at the Heart of Future Innovation for Rare Diseases
Rare disease treatment is entering a new era thanks to new partnerships unlocking innovative ways of working
Issue 548 | 21 February 2022
21 Feb
Issue 548 | 21 February 2022
The latest edition of The Parliament Magazine is now available to read online for free.
Transforming Rare Diseases Partner Content
21 Feb
Transforming Rare Diseases
A special supplement looking at how Europe can come together to find solutions for people suffering from a rare disease
Europe’s time to act on rare diseases Partner Content
21 Feb
Europe’s time to act on rare diseases
Renewed collaboration at EU level for a new joint strategy on rare diseases could transform the rare disease landscape for patients
Rare diseases: Tackling unmet needs and evolving the orphan drug environment Partner Content
05 Jul
Rare diseases: Tackling unmet needs and evolving the orphan drug environment
A ‘Rare Conversation’ with EURORDIS’ Yann le Cam, Alexion’s Camilla Harder Hartvig and MEP Pernille Weiss.
How do we solve the rare disease crisis? Partner Content
27 May
How do we solve the rare disease crisis?
Pfizer often made the headlines over the past year after mobilising their scientific expertise and manufacturing capabilities to respond to the COVID-19 pandemic. Reda Guiha, Regional President for Rare Disease at Pfizer, argues that the same sense of urgency and public-private sector collaboration that brought vaccines to patients within a year should be applied to other areas of critical unmet medical need, such as rare disease.
Tilly Metz interview: Animal Farm 2.0
26 May
Tilly Metz interview: Animal Farm 2.0
A long-time advocate for animal rights, Tilly Metz is working to create a Europe that focuses on less-but-better animal farming and one that faces up to the climate crisis while factoring in sustainability. Lorna Hutchinson reports.
Rare Diseases: Celebrating patient-centred innovation Partner Content
26 Feb
Rare Diseases: Celebrating patient-centred innovation
After a challenging year, Rare Disease Day 2021 is a wonderful opportunity to recognise the incredible efforts and achievements of patients, their associations and the scientific community, explains Alexander Natz.
Rare Diseases: How European reference networks have improved the lives of rare disease sufferers immeasurably Partner Content
26 Feb
Rare Diseases: How European reference networks have improved the lives of rare disease sufferers immeasurably
But more can, and must, be done to work together and take ERNs to the next stage, says EURORDIS HealthCare Director Inés Hernando.
Rare Diseases: How the new EU rare disease policy framework provides an opportunity to move from building infrastructures to measuring impact Partner Content
26 Feb
Rare Diseases: How the new EU rare disease policy framework provides an opportunity to move from building infrastructures to measuring impact
‘Rare 2030’ Foresight Study highlights unmet needs and continued obligation to improve outcomes, explains EURORDIS Health Policy Director Anna Kole.
Rare Diseases: Why medical innovation needs to be one step ahead Partner Content
24 Feb
Rare Diseases: Why medical innovation needs to be one step ahead
Accelerating innovation key to ensuring that no one is left behind in fight against rare diseases, says Nathalie Moll EFPIA Director General.