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.

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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

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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

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New SCID website page

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Publication of PIDs Principles of Care

Over the last two years, IPOPI combined its efforts with a worldwide multi-disciplinary team of specialists to develop the “Primary Immunodeficiencies Principles of Care”. The paper outlines the key necessary elements and policies that should be in place to provide a “gold standard” framework of diagnosis and care for primary immunodeficiencies.
IPOPI is delighted to announce that the PIDs principles of care were published in Frontiers in Immunology on December 15th, 2014. The publication aims at providing guidance to decision-makers and healthcare professionals throughout the world on the different aspects affecting PIDs. ThePIDs Principles of Care cover the following aspects: the role of specialised centres, importance of registries, the need for international collaborations for scientific research, the role of patients groups, management and treatment options for PIDs, and management of PID diagnosis and care throughout the world.
The document is the result of the effort of the PIDs Principles of Care Working Party led by IPOPI, which brought together IPOPI representatives and 28 medical experts representing the 5 continents and different areas of work related to PIDs. IPOPI would like to express its most sincere thanks to all the co-authors of the PIDs Principles of Care with special thanks to Professor Helen Chapel, IPOPI’s Honorary Vice President, whose coordination and input were pivotal in getting the document published. We invite all our NMOs and other PID stakeholders to use the PIDs Principles of Care to promote better access to early diagnosis and care for PID patients worldwide.
PID Principals of Care – original version
Translated versions:
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