Dear Friends,

We hear, here and there, that things are moving faster than ever, this applies to IPOPI as well! The start of 2019 has been incredibly busy, with a special focus on plasma collection and supply of plasma derived medical products (PMDPs), on diagnosis and access to care, and on the international rare diseases day. Among other topics!

Tensions on supply or even shortages of immunoglobulins are increasingly worrying people with PIDs. Therefore IPOPI is constantly working and advocating to ensure people whose health depends on these lifesaving medicines, can have continuous, safe and sustainable access their treatment. With plasma supply struggling to meet demand and the growing clinical needs for PDMPs, especially immunoglobulins, IPOPI organised and took part in different initiatives to address this sensible and crucial issue.

Firstly, the Platform of Plasma Protein Users (PLUS) organised its annual stakeholders meeting in Estoril on 24-25 January. Participants shared their perspective and produced a statement as this yearly meeting was also meant to prepare for the 2-day Symposium on plasma supply organised by the European Directorate for the Quality of Medicines & HealthCare (EDQM) and the European Commission (EC) in Strasbourg on 29 and 30 January. Considering that European patients are dependent on USA collected plasma, which puts Europe at risk in case of any sudden supply interruptions, the symposium brought together stakeholders involved in the field of blood and plasma to discuss ways forward for improving plasma supply management. This was the first time that all stakeholders in the sector have met to exchange their views on how to increase the supply of plasma for fractionation in Europe. PLUS was invited to express its position, for the first time ever at a meeting of the EDQM, and I had the opportunity to take part in the debriefing meeting that took place the following day in a small committee. This initiative was organised in the perspective of the possible revision of the European blood directive.

Still linked to Europe, and in the perspective of the forthcoming EU elections in May, IPOPI was invited to give its perspective on How to ensure appropriate patient access to PDMP treatment during a roundtable at the Parliament that PPTA organised. This was an opportunity for IPOPI to express, among others, the importance of differentiating PDMPs from labile blood products in future EU relevant policies.

Also linked to the European commission, IPOPI continues its hard work with the international Society for Neonatal Screening (ISNS) to put newborn screening on the European agenda so we can have guidelines to support the countries in implementing life-saving early screening for rare diseases. On this note, I would like to sincerely congratulate our colleagues in Germany, as German babies will be, as of August 2019 the first EU citizens to be screened for SCID at birth! Well done!

Now let’s travel to Asia where our colleague Bruce Lim, IPOPI board member from Malaysia, attended the 4th IPFA Asia Workshop on Plasma Quality & Supply in March. Volume of plasma, quality of plasma and supply of PDMPs… were among the key topics and Bruce seized the opportunity to raise an important question: how can a national health authority state that there is an adequate supply of PDMPs when PIDs are mostly undiagnosed and when there is no registry to assess the prevalence.

To tackle under and mis-diagnosis, an issue facing every region in the world, we need to raise awareness, untiringly, because we know there is a huge unmet need and also because we can see that our efforts are increasingly successful!

Awareness goes with education, this is the reason why I am so delighted with the success we encountered with our second webinar, precisely on diagnosis, thanks to the excellent and comprehensive lecture of Dr Kate Sullivan from Children’s Hospital Philadelphia, USA. I take the opportunity to address my belated wishes of happiness to all our colleagues and friends from Asia who celebrated the Lunar New Year at that moment and tell them that they can benefit from this lecture on IPOPI TV anytime!

Still on awareness, and moving to another continent, I wanted to mention our active preparation towards the next ASID meeting to be held in Dakar, Senegal, in April and highlight how appreciative we are about the collaboration between ASID, IPOPI and INGID in Africa where patients’ needs are huge, but where the enthusiasm of our African colleagues and a promising economic growth in many countries make us look at the future with confidence.

I will also emphasize the fact that we have opened the registration for IPIC2019, our biennial clinical congress, to be held on 6-8 November 2019 in Madrid. The programme has been prepared with input from doctors, patients and key PID community stakeholders and it will focus on diagnosis and clinical care of primary immunodeficiencies. I would like to thank our supporting organisations, our sponsors and all stakeholders who are helping us making this congress a success and turn patient centeredness into reality, in the most relevant and complementary manner! The joint stakeholder approach of our PID community is a good example in the rare diseases field. So, following IPIC’s moto, let me tell you: together in Madrid!

When it comes to rare diseases, I feel very proud that our colleagues Johan Prevot, IPOPI’s Executive Director, and Roberta Anido de Pena, IPOPI Board member for Latin America, actively represented IPOPI in several rare diseases events held in New York City, among which the 2nd High-Level Event of the NGO Committee for Rare Diseases held at the United Nations (UN) headquarters. This important meeting called for integrating rare diseases into the upcoming landmark UN political declaration on universal health coverage and getting a UN resolution on rare diseases.

Last but not least, our NMOs were also very committed in the international Rare disease Day as could be seen on social media: no doubt this was an excellent way to rehearse for the forthcoming Word Primary Immunodeficiency Week to be celebrated next April. The theme of this year’s campaign is ‘Putting primary immunodeficiency patients at the centre of their care’. So now, time to act: Let’s celebrate, raise awareness and make sure our voice is heard!

Enjoy reading this edition of e-News and make sure to also check our NMO updates, our membership is more active than ever!

Martine Pergent, IPOPI President