Rare Diseases International launched today

Over 60 patient representatives from 30 countries are gathering this afternoon in Madrid, Spain, for the inauguration of Rare Diseases International (RDI) and to adopt a joint declaration to advocate for rare diseases as an international public health priority.

This Eurordis initiative represents patients and families of all nationalities across all rare diseases and its aim is to be the voice of all people living with a rare disease and to give visibility to rare diseases in the global health agenda.
Johan Prevot, IPOPI’s Executive Director is also taking part in the launch meeting and will present IPOPI’s advocacy work at the international level.
The preliminary phase of RDI has been steered by EURORDIS and national rare disease alliances from the US (NORD), Canada (CORD), Japan (JPA), China (CORD), India (I-ORD), the Ibero-American pan-regional alliance (ALIBER) and the International Federation for Epidermolysis Bullosa (DEBRA International).

For more information please visit: http://www.eurordis.org/content/rare-diseases-international

Marketing application for new ADA-SCID gene therapy submitted to EMA

GSK (GlaxoSmithKline), Fondazione Telethon (Telethon) and Ospedale San Raffaele (OSR) recently announced the submission of a marketing application to the European Medicines Agency (EMA) for a gene therapy (GSK2696273) to treat patients with adenosine deaminase severe combined immunodeficiency syndrome (ADA-SCID), for whom no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available. For more information see the official press release here.

EU-wide overview of the market of blood, blood components and plasma derivatives

The European Commission (EC) published in April a report on ‘An EU-wide overview of the market of blood, blood components and plasma derivatives focusing on their availability for patients’.

The key objective of the document is to provide an economic perspective on the European blood sector. This was done against the background of understanding and improving the safety, quality and availability of therapies delivered by the sector to European patients.

This report highlights the complexity of the market due to diverging activities and interests of the stakeholders some from the private sector, others from the public or semi-public sectors. These differences often create divergent opinions on, for example, the interpretation of the principle of voluntary and unpaid donation. Another topic of divergency is the definition of self-sufficiency, a concept strongly encouraged at European level.
The definition of shortage also creates controversies. While safety and quality of blood and blood components are within the mandate of the EC, availability, which is a recurring concern for patients, healthcare authorities and healthcare professionals, is a competence of the Member States.

The EC recommends the development of common definitions for clearer interpretation, common understanding and transparency as these differences lead to different views on risks, epidemiology or processing steps, and related safety and quality measures.

Full report
Key findings