March 25, 2021
From the Chairperson’s desk
From the Chairperson’s desk
This year, Spring is placed under the aegis of a race. The race for an anti-COVID-19 vaccine. In every country, patients are waiting in hope for this vaccine. The hope of protection for them or their relatives. To those who are fighting on the pandemic front, working tirelessly month after month, we cannot thank you enough. Hoping for herd immunity… When will this happen? Nobody can really tell. In addition, discrepancies between countries are huge. Nevertheless, we all comply, we adapt to this situation, we accept to learn and at times we have daily updates with something different that is either denied or clarified the following day. As PIDs specific data remains pretty scarce; I’d like to address a heartfelt thank you to the IPOPI Medical Advisory Panel who established recommendations on the vaccination against SARS-COV-2 for patients with PID.
Speaking of adaptation. IPOPI made a significant one. In moving our International Clinical Congress (IPIC) to April 2022, and with a new venue: Vilamoura, Portugal. A change for the best! There is no doubt we all miss meeting in-person. The networking and having friendly catch up moments together, all these factors that spice up life during an in-person congress!
This said, COVID-19 hasn´t prevented us in having intensive activity. Educationally speaking, we are very proud of our recent released mini documentaries that touch upon cross overs between PIDs and autoinflammation. We are equally proud of our six new leaflets that have been published. Particularly addressing patients or health care professionals
Additionally, we ran a successful webinar on RECOMB clinical trial on gene therapy for RAG1-SCID (available on the RECOMB website).
In terms of advocacy, I am so pleased to mention that WHO has added several new PID diagnostic tests in its Second WHO Model List of Essential In Vitro Diagnostic. PIDs are the first Rare diseases to enter this list. This is to the benefit of the PID community, as the list is used as a reference by health decision makers when they intend to prioritise diagnostic tests in terms of availability, and possibly, cost coverage.
IPOPI is also very happy with the collaboration within the Screen4Rare platform. Together, with our partners ISNS and ESID, along with the support of ERN RITA and MetabERN, we are successfully capitalizing on months of advocacy for newborn screening for rare diseases. Both on clinical and political sides at EU level.
On the treatments front. I must mention the worry we have due to the global supply tensions on immunoglobulins, heightened by COVID-19, but whose roots are structural, going far beyond the pandemic episode. In order to enhance our response towards such treatment access threats, we have set up SAFE (Safe Access for Everyone). This is a task force that monitors the situation and has already taken action in publishing two global statements on the need for IG for patients who have no alternative treatment. So, the causes of these tensions are addressed where the only goal is to meet patients´ vital needs. Here we work hand in hand as a community, made of different stakeholders, which we believe is the only way to address complex issues in a patient centered way.
Mentioning our community, let us speak of the IPOPI family. Our NMOs who are so active, as you’ll read about in this newsletter, the US, Germany, Australia, France, Russia, Argentina, The Netherlands… who share their inspiring initiatives. Presently many of our NMOs are focused on preparing for the PID momentum towards all stakeholders, including the general public: WorldPIWeek.
On that initiative, we will be able to tell you more in the next issue of the Summer newsletter. Hopefully the first results from the vaccination will reinforce the energy and optimism which we have in our DNA. Despite everything! Happy reading, and please take care and keep safe.