“Knowing there’s a place where someone will listen, understand, and guide you towards what’s best for your child is invaluable.”
My name is Dorica Dan. I’m from Romania, and I serve as the President of the Romanian Prader-Willi Association (RPWA)—my daughter has Prader-Willi Syndrome. I’m also the President of the Romanian National Alliance for Rare Diseases (RONARD) and the Romanian Rare Cancers Association. In addition, I am currently Vice-President of EURORDIS – Rare Diseases Europe. My main area of expertise is integrated care for people living with rare diseases. I believe patients need a holistic approach—encompassing medical care, social care, and special education services. I’ve been deeply involved in promoting and developing structures that provide this kind of comprehensive support.
Irene Norstedt: “The EU supports collaboration because no country alone, and no stakeholder alone, has the answer for such huge unmet needs in rare disease research”
We met Irene Norstedt, Director for the People Directorate at DG Research and Innovation in the European Commission, at the RE(ACT) Congress in Brussels, where she delivered a compelling presentation on diverse funding models targeting different research needs.
EMA Calls for Registrations in Real-World Data Catalogues
The European Medicines Agency (EMA), together with the European Medicines Regulatory Network, has issued a renewed call for registrations in their Real-World Data (RWD) Catalogues, marking one year since the initiative’s launch. This publicly accessible online resource replaced the previous ENCePP Resources Database and the EU PAS Register, consolidating metadata on RWD sources and studies to aid regulators, researchers, and pharmaceutical companies in assessing the real-world use, safety, and effectiveness of medicines. EMA aims to build a comprehensive resource by encouraging stakeholders who manage data sources or who have conducted RWD studies to register their metadata. Participation not only increases READ MORE
RE(ACT) Congress 2025 Concludes with Calls for Stronger Collaboration on Rare Diseases
The 8th RE(ACT) Congress and the 6th IRDiRC (International Rare Diseases Research Consortium) Conference concluded today, marking the end of a three-day summit where scientists, patient advocates, funders, and policymakers tackled the rare disease field’s most pressing challenges.
Rare Disease Day: ERDERA Charts the Path Forward in Collaborative Research
As Rare Disease Day 2025 comes to a close, its impact continues to resonate across Europe and beyond. Held annually on the last day of February, this year’s event drew critical attention to the 30 million people in Europe living with a rare disease, underscoring the urgent need for faster diagnoses, increased research, improved treatments, and stronger policies.
SANT Launches Public Consultation on Rare Diseases
On 28 February, coinciding with the 2025 Rare Disease Day, Parliament’s public health committee, SANT, launched an online survey to gather insights on rare diseases. The initiative aims to build a detailed understanding of the challenges faced by individuals affected by rare diseases, as well as to collect views from stakeholders—including patients, caregivers, industry representatives, and NGOs—to inform future policy and legislative reforms.
“Knowing there’s a place where someone will listen, understand, and guide you towards what’s best for your child is invaluable.”
My name is Dorica Dan. I’m from Romania, and I serve as the President of the Romanian Prader-Willi Association (RPWA)—my daughter has Prader-Willi Syndrome. I’m also the President of the Romanian National Alliance for Rare Diseases (RONARD) and the Romanian Rare Cancers Association. In addition, I am currently Vice-President of EURORDIS – Rare Diseases Europe. READ MORE