News

Baxter and Halozyme Therapeutics announced yesterday that they have received a marketing authorization from the European Commission for HyQvia as a treatment for adults with primary and secondary immunodeficiencies.  This follows a positive opinion for the product from the European Medicines Agency’s Committee for Medicinal Products for Human Use earlier this year.

At the occasion of the European Patients Rights Day, the European Commission published a memo highlighting the added value that the EU brings to patients and outlining 10 concrete rights that the EU have established for the benefit of patients.

Earlier today, Baxter International Inc. announced that its Phase III clinical study of Immunoglobulin (IG) did not meet its co-primary endpoints of reducing cognitive decline and preserving functional abilities in patients with mild to moderate Alzheimer’s disease.

The European Union has introduced a new process to label medicines that are being monitored particularly closely by regulatory authorities. These medicines are described as being under 'additional monitoring'. Medicines under additional monitoring have a black inverted triangle displayed in their package leaflet and in the information for healthcare professionals called the summary of product characteristics, together with a short sentence explaining what the triangle means. A medecines is placed on this list when for example it has been introduced recently on the market, or if it requires special measures to monitor adverse reactions, or if post-marketing authorisation safety studies were requested by the EMA.

Today marks the launch of the 3rd World Primary Immunodeficiencies Week (WPIW) which will culminate on the World Immunology Day, 29th April. The Week is a global action organised by the primary immunodeficiency (PID) community fighting for global awareness on this increasingly important group of diseases.

IPOPI is pleased to announce that its new Global Immunoglobulin Products List is now available online and can be accessed here The new design and user-friendly format of the List allow for an easier browsing experience and to search by products availability per country or by company and products name.

On April 17^th our friends from the Haemophilia community celebrate World Haemophilia Day. IPOPI supports World Haemophilia Day which provides a unique momentum in the year to raise awareness of haemophilia and the fact that most people with haemophilia and other bleeding disorders do not receive adequate diagnosis, treatment and management for their conditions.

 

The Spring 2013 edition of IPOPI's e-News was released today and can be accessed here. For information on how to subscribe to e-News, see our e-News webpage. e-News is a quarterly electronic newsletter covering the latest activities and news items from IPOPI as well as other developments from the international PID community.

Rare diseases without borders is the slogan of the 2013 International Rare Diseases Day celebrated today. The main objective of Rare Disease Day is to raise awareness about rare diseases, the challenges encountered by those affected by these disorders, and the inequalities that rare disease patients face every day.

Rare Diseases Day 2013 will be celebrated on 28 February 2013. The official Rare Disease Day 2013 video is now available. The video takes the viewer on a magical journey to demonstrate the urgent need for global cooperation in the field of rare diseases. It is inspired from the Rare Disease Day 2013 slogan “Rare Disorders without Borders” and captures the need for the international rare disease community to work together to find solutions. Check out the video here!

The European Parliament Committee on the Environment, Public Health and Food Safety (ENVI) published today an Oral Question on Newborn screening for SCID and Rare Diseases.
The Oral Question is supported by the EU Parliament committee responsible for all Health matters (ENVI) and follows IPOPI EU Advocacy campaign for SCID Newborn Screening.

The European Commission recently published a report on the contributions received for the consultation on chronic diseases in which IPOPI took part.
The aim of the Consultation was to gather stakeholders information and provide a basis for a potential EU policy on chronic diseases. Later in 2013 a Joint Action on Chronic Diseases will be carried out by the Commission in cooperation with one or more Member States or with competent authorities.

The European Commission recently adopted an Implementing Directive to establish a framework to ensure recognition of medical prescriptions in cross-border healthcare as required by Directive 2011/24/EU on the Application of patients' rights in cross-border healthcare adopted on 9 March 2011.

The Winter 2012 edition of IPOPI's eNews was released today and can be accessed here. For information on how to subscribe to eNews, see our eNews webpage. eNews is a quarterly electronic newsletter covering the latest activities and news items from IPOPI as well as other developments from the international PID community.

IPOPI is pleased to announce the publication of three new patient information leaflets comprising helpful information and guidance for patients, their families and other interested stakeholders of the Primary Immunodeficiencies (PID) community.

We are pleased to release the report of this new IPOPI survey which explores PID Patient Needs and Outlooks. The survey was carried out during the second half of 2011 and is based on completed questionnaires from 300 patients

The European Organisation for Rare Diseases (EURORDIS), the National Organization for Rare Disorders (NORD) and the Canadian Organization for Rare Disorders (CORD) have released a joint declaration on common principles regarding Rare Disease Patient Registries.

The African Society for Immunodeficiencies (ASID) will hold its third Congress in Sun City, South Africa on 5-9 June 2013. The Congress whose overall theme is "Hope for Africa" will feature an esteemed international and local faculty of speakers and offer pre-conference workshops and a range of thematic sessions including on allergology, immunodeficiency diseases, paediatric pulmonology, pulmunology and thoracic surgery. For more information on the congress click here.

 

The first IPOPI Bob LeBien Award Ceremony was held on the occasion of IPOPI’s 20th anniversary during the XIIth Biennial Meeting in Florence.

The IPOPI XIIth Biennal meeting in conjunction with the XVth ESID and Xth INGID took place on 3-6 October in Florence, Italy.

IPOPI is pleased to announce the launch of the website of the First International Primary Immunodeficiencies Congress (IPIC). The congress will offer clinicians and other stakeholders the opportunity to learn more about PIDs from diagnosis to clinical care development through a series of specific scientific sessions.

On Wednesday, 26^th September the 3rd Primary Immunodeficiency Forum organised by the International Patient Organisation for Primary Immunodefiencies (IPOPI) was held at the European Parliament in Brussels, Belgium. Nessa Childers, MEP, hosted the event. Forum participants included MEPs, industry representatives, EU commission representative, patients’ representatives and leading physicians and researchers.

 

Focus on Clinical Care and Diagnosis
IPOPI will organize its first congress on the 7th and 8th of November 2013 in Estoril (Lisbon), Portugal.

The Fall 2012 edition of IPOPI's eNews was released today and can be accessed here. For information on how to subscribe to eNews, see our eNews webpage. eNews is a quarterly electronic newsletter covering the latest activities and news items from IPOPI as well as other developments from the international PID community.

The European Medicines Agency has recently launched a consultation on their Draft guideline on core SmPC for human normal immunoglobulin for subcutaneous and intramuscular administration.

 

As 2012 marks the 20th anniversary of IPOPI, it is a great pleasure to report on the numerous achievements and activities carried out by IPOPI in the last two years. The new IPOPI Review of Activities and Achievements for 2010-2011 is now available for download here.

The Summer 2012 edition of IPOPI's eNews was released today and can be accessed here. For information on how to subscribe to eNews, see our eNews webpage. eNews is a quarterly electronic newsletter covering the latest activities and news items from IPOPI as well as other developments from the international PID community.

The European Medicines Agency (EMA) announced today the launch of the new European database of suspected adverse drug reaction reports website at www.adrreports.eu.

IPOPI is pleased to announce the launch of its NMO Website Toolkit accessible online on the IPOPI website. The toolkit was created specifically to help IPOPI’s National Member Organisations (NMOs) set up their own websites and learn how to manage it themselves through a user friendly Content Management System (CMS).

 

This new IPOPI statement is intended to provide a summary of IPOPI’s position with regards to the importance of ensuring access for patients living with a primary immunodeficiency (PID) to the best suited immunoglobulin (IG) replacement herapy

April 22 marks the launch of the second World Primary Immunodeficiencies Week (WPIW). The week is a yearly opportunity for all PID stakeholders to raise the recognition and diagnosis of Primary Immunodeficiencies as an increasingly important group of diseases. The theme of WPIW this year is "access to appropriate treatment". For more details and to participate in this important movement for our community, please download the WPIW Press Release or log on to www.worldpiweek.org

IPOPI recently submitted a position statement in the context of the European Commission

The Spring 2012 edition of IPOPI's eNews was released today and can be accessed here. For information on how to subscribe to eNews, see our eNews webpage. eNews is a quarterly electronic newsletter covering the latest activities and newsitems from IPOPI as well as other developments from the international PID community.

The European Commission published today its response to the Parliamentary Question on Health Technology Assessments (HTAs) tabled by Mrs Glenis Willmott

February 29^th marked the celebration of 5^th Rare Diseases Day. Launched in 2008 and held every year on the last day of February (‘a rare day for rare people’), the event has grown rapidly and successfully over the years.

This year for the first time, Eurordis, the European Organisation for Rare Diseases, organised a ‘Black Pearl’ Gala Dinner event in Brussels which was attended by approximately 200 participants. IPOPI’s Executive Director, Johan Prevot, was privileged to be invited to represent the PID patient community at this important event as well as PLUS, the Platform of Plasma Protein Users, along with Brian O’Mahony from the European Haemophilia Consortium.

In solidarity with the millions of people who live with a rare disease, World PI Week founders are using the occasion of Rare Disease Day to send

A short awareness-raising video clip has recently been released by Eurordis

In December 2011 IPOPI held its PID Forum on Health Technology Assessments at the European Parliament in Brussels.  As an outcome of the meeting’s discussion a Parliamentary Question was recently submitted to the European Commission by MEP Glenis Willmott

Rare but strong together is the slogan of the 2012 International Rare Disease Day to be held on the rarest day in the calendar: 29th February.

The European Commission published its work plan for 2012 for the second programme of Community action in the field of health. The work plan sets out the activities to be co-financed from the work plan. To find more information on how to apply for EU co-financing for projects, conferences, joint actions with Member States and operating grants for non-governmental organisations please visit the commission’s dedicated webpage here. The deadline for submission of proposals is 9 March 2012

PLUS, the Plaftorm of Plasma Protein Users, organised its 3rd Stakeholders consensus meeting in Dublin, Ireland on 12-13 January. The meeting was once again an excellent opportunity to discuss issues and developments on access to care for patients living with a rare plasma related disorder.

An article entitled ‘Newborn screening for primary immunodeficiencies: beyond SCID and XLA’ was recently published in the Annals of the New York Academy of Sciences and can be accessed here.

The Fall/ Winter 2011 edition of IPOPI's eNews was released today and can be accessed here. For information on how to subscribe to eNews, see our eNews webpage. eNews is a quarterly electronic newsletter covering the latest activities and newsitems from IPOPI as well as other developments from the international PID community.

On December 6, 2011, IPOPI held its second PID Forum at the European Parliament on Health Technology Assessments (HTAs) and Primary Immunodeficiencies. The event was very successful and was hosted by Member of the European Parliament (MEP) Glenis Willmott (UK, PES). It was attended by numerous stakeholders including patient, physician and academia representatives, industry experts, as well as by 9 MEPs from different political parties.

IPOPI’s recently released a co-edited issue of Pharmaceuticals Policy and Law in collaboration with Professor José-Luis Valverde (University of Granada, Spain) looking at Health Technology Assessments (HTAs) and Rare Diseases Therapies.

IPOPI was pleased to support the first national Russian PID patient meeting in Russia on November 17th in Moscow. The meeting was very successful and attended by numerous patients, doctors and other stakeholders from all regions if the country, and led to a resolution seeking to influence national policy and enhance the diagnosis and care of people living with a PID in Russia.

As part of its partnership with Member of the European Parliament (MEP) Glenis Willmott, IPOPI participated in a meeting in the East Midlands, United Kingdom, on the need for implementation of SCID newborn screening. During the meeting, which was also attended by Prof Bobby Gaspar and families from the East Midlands who were affected by SCID and followed on IPOPI's campaign to introduce SCID newborn Screening in the European Union, Mrs Willmott re-emphasized her support to the swift implementation of the screening test.

IPOPI held its second landmark Global Leaders Meeting in London on 4-5 November 2011. The meeting was attended by a range of key stakeholders including patient representatives, physicians, nurses, regulators, industry and other specialists. The meeting resulted in a set of recommendations which can be accessed here

Octapharma issued today a press release announcing that the U.S. Food and Drug Administration (FDA) approved the return of Octagam 5% in the United States. Last year, Octapharma had initiated a voluntary market withdrawal of Octagam in the U.S. in response to an observed increase in thromboembolic events (TEEs).

The European Commission recently adopted revised proposals setting out EU-wide rules on information on prescription-only medicines. In a press release Commissioner Dalli stated that "these revised proposals take forward the valid rationale of the initial proposals from 2008, but re-define them from a patients' perspective".

Brian O’Mahony and Johan Prévot met on behalf of PLUS’ Executive Committee with EU Health and Consumer Policy Commissioner John Dalli at the European Commission in Brussels on 13 October 2011. The meeting’s main items for discussion were the outcomes of the PLUS 2011 Dublin Consensus meeting and the last Blood Directive report.

The Summer 2011 edition of IPOPI's eNews was released today and can be accessed here. For information on how to subscribe to eNews, see our eNews webpage. eNews is a quarterly electronic newsletter covering the latest activities and newsitems from IPOPI as well as other developments from the international PID community.

The Immune Deficiency Foundation (IDF) recently announced that it had launched a free accredited online continuing education (CE) course for nurses. The goal of this CE course is to increase the knowledge of nurses by providing an educational update on primary immunodeficiency disease and immunoglobulin therapy. More information on this new course can be found in IDF's Press Release

IPOPI is pleased to announce that the Second Meeting of the Latin Amercian Society for Immunodeficiencies (LASID) coorganised with IPOPI will be held in Mexico City, Mexico on 13-15 October 2011. Click here to access more information about the meeting.

The European Commission has published yesterday its response to the Parliamentary Question on the need for SCID Newborn Screening tabled by Mrs Glenis Willmott, Member of the European Parliament (S&D, UK), in July. Mrs Willmott had chaired the first IPOPI Policy Forum on SCID Newborn Screening earlier in June. 

Mrs Glenis Willmott, Member of the European Parliament (S&D, UK), recently submitted a European Parliament Question on the need for SCID newborn screening in the European Union. Mrs Willmott had chaired the first IPOPI Policy Forum on SCID Newborn Screening earlier in June. You may access the full text of the Question here. IPOPI will closely monitor upcoming developments.

As part of its PID patient awareness programme, IPOPI organised the first patient awareness NMO pilot workshop in Belgium on 2 July 2011. BOPPI, the Belgian Organisation for Patients with Primary Immunodeficiencies sent five participants to the meeting which was also attended by Prof Meyts. The workshop enabled all participants to share their knowledge about PID, give feedback on the IPOPI patient awareness toolkit and plan concrete awareness raising activities in Belgium.

IPOPI organised its first EU PID Forum at the European Parliament in Brussels on Wednesday 15 June. The forum was hosted by Glenis Willmott (S&D, UK), Member of the European Parliament, and focused on SCID Newborn Screening . Participants in the workshop included IPOPI representatives and other key opinion leaders on SCID newborn screening

Grifols, S.A. (GRF.MC) and Talecris Biotherapeutics Holdings Corp. (NASDAQ: TLCR) announced on 1 June 2011 that the U.S. Federal Trade Commission (FTC) has accepted for public comment a Consent Agreement that outlines the conditions necessary for Grifols' acquisition of Talecris to proceed.

We are delighted to welcome you to IPOPI’s new website.  The new IPOPI website is a direct result of IPOPI’s determination to continue to improve its communication and the visibility of Primary Immunodeficiencies (PIDs) worldwide and reflects IPOPI’s commitment to act as the global advocate for the PID patient community.

The First World Primary Immunodeficiency Week was celebrated across the world from 22-29 April 2011. A range of initiatives and awareness raising events were held in various regions of the world creating a true global movement to increase the recognition of primary immunodeficiencies as an increasingly important group of rare disorders. IPOPI was pleased to be able to support its national member organisations in their national campaigns which were all truly successful. We very much look forward to World PI Week 2012 and invite all stakeholders to join this important campaign. So stay tuned on the IPOPI website and World PI Week website for more news on the upcoming Second World PI Week !

The European Medicines Agency (EMA)'s Committee for Human Medicinal Products (CHMP) has recommended the lifting of the suspension of the marketing authorisations for Octagam (human normal immunoglobulin 5% and 10%) and associated names, and the reintroduction of the medicine onto the market in the European Union.

IPOPI is closely monitoring the latest developments in the United States regarding the risk of Thrombotic Adverse Events with subcutaneous or Inappropriate Intravenous use of Vivaglobin, a subcutaneous therapy for the treatment of primary humoral immunodeficiency manufactured by CSL Behring. The US Food and Drugs Administration (FDA) and CSL Behring have recently published statements providing detailed information on this matter.

After more than 30 years of working on behalf of patients with a plasma related disorder, and having a huge impact on the plasma product community, first on the lives of people with haemophilia and then as a fierce advocate for people living with a PID. David has decided to stop working all those crazy hours.

Experts on rare diseases from the medical, scientific and patient advocacy communities examine critical issues and solutions in Paris and Bethesda. In conjunction with Rare Diseases Day 2011 www.rarediseaseday.org CSL Behring announced a special edition of its Key Issues Dialogue series "Making Rare Diseases a Global Public Health Priority.

This new IPOPI report summarizes some of the highlights of the XIVth ESID Meeting's scientific programme. IPOPI would like to thank Dr Peter Spaeth for his precious help with this document.

This is the first international survey carried out by IPOPI. The survey was conducted between February 2010 and August 2010 to establish a mapping of several criteria amongst IPOPI's NMO's including estimated number of patients in each country, known diagnosed PID cases, patient demographics, presence of registries, collaborations with international registries, provision of care, availability of treatment, funding of care, presence of national Medical Advisory Panels and communication tools. The survey offers a first international overview of these criteria from the patient perspective and identifies positive developments, recommendations and areas where improvement is needed. The survey report can be accessed here:

This new IPOPI publication is now available for download on our website. Within a national healthcare system, selection of immunoglobulin (IG) therapies for conditions such as Primary Immunodeficiencies (PIDs) is difficult. In well-resourced countries, regulatory agencies make key decisions regarding such therapies' quality and safety; such regulatory agencies, including the U.S. Food and Drug Administration (FDA) and the European Medicines Evaluation Agency (EMEA), are dedicated to assessing products and to granting marketing licenses. However, even in countries lacking resources to form such regulatory agencies, selection of IG therapies for IDs, within a national healthcare system, can occur. For such selection to occur, national regulatory authorities (NRAs) need to understand and to use several well-established principles; this guide offers such principles to NRAs and others who are responsible for selection of IG therapies for IDs, within their respective national healthcare systems. While this guide was written with IG therapies in mind, many of its principles apply to all plasma-derived medicinal products.