The Value of One Patient Organisation per Country

PLUS, the Platform of Plasma Protein Users, which represents organisations of patients living with rare plasma related disorders and rare bleeding disorders, recently issued a position statement on the value of one national patient organisation per country. The statement highlights that only by having a single recognised patient organisation that channels the views of patients with decision-makers, payers and other stakeholders, can national patient groups effectively and efficiently represent their patient community in a consistent strategic approach. To access the PLUS position statement click here.

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).

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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

Council of Europe adopts Resolution on Principles of Immunoglobulin Therapies for PIDs

The Council of Europe adopted an important Resolution about Therapies for Primary Immunodeficiencies. Very exciting news that will help our members’ advocacy efforts in this year’s World Primary Immunodeficiencies Week and beyond!

The Resolution CM/Res(2015)2 on Principles regarding Immunoglobulin Therapies for Immunodeficiency and Other Diseases, adopted on the 15th of April, outlines short but key messages such as the importance of making “available to patients all recognised routes of human normal immunoglobulin administration”, “to take into account that human normal immunoglobulin therapeutic products differ from one another in terms of production processes” and “to adopt a suitable process. This resolution will provide good support to IPOPI’s advocacy efforts as well as those of our National Member Organisations.

Jose Drabwell, IPOPI President, stated: “We are delighted that with this Resolution the Council of Europe has formally recognised vital recommendations concerning immunoglobulin therapies and their use in different indications including Primary Immunodeficiencies (PIDs)”. Jose added: “The fact the resolution has come out just in time for the World Primary Immunodeficiency Week (WPIW) to be held from 22 to 29 April 2015 will further raise awareness about the essential nature of immunoglobulin therapies and their importance in the treatment of patients living with primary immunodeficiencies”.

For more details please read IPOPI’s press release

The official resolution document can be viewed here