Brussels Event Celebrates the Establishment of Rare Disease Platform Trials in Europe
It was quite a week for the NF community and the Children’s Tumor Foundation Europe, from celebrating innovators who are helping advance clinical trials for quicker and better medical treatments, to engaging on the hopes and challenges of pending pharmaceutical legislation which may impact rare disease patients, to participating in a spectacular philanthropy conference which drives awareness, hope, and support of important causes that are changing the world. And topped off with a unique opportunity to see Queen Mathilde of Belgium!
These high-profile activities took place in Brussels in the days leading up to NF Awareness Month, which takes place each May and brings attention to those living with NF, which is a group of genetic conditions known as neurofibromatosis or schwannomatosis.
The Children’s Tumor Foundation (CTF) Europe opened the week with a marvelous celebration at the beautiful Maison Grand Place in the historic center square of Brussels. On Monday, 24 April, CTF Europe honored three extraordinary individuals with the inaugural Cloud Carrier Award for their extraordinary efforts to End NF, to Make NF Visible in Europe, and support the mission of the Foundation. The cloud carrier awards are unique pieces of art created and donated by the Italian sculptor and artist Giacomo del Giudice. Through his art, he envisions the CTF honorees as cloud carriers, because they are “carrying clouds full of dreams that will make the world a better place”. The honorees are changemakers in every sense of the word and are changing the landscape of rare disease and NF, both in Europe and globally.
The three cloud carriers this year are Magda Chlebus, Executive Director, Science Policy and Regulatory Affairs, EFPIA and Board Member of CTF Europe; Dr. Francesco Patalano, Head of Pediatric and Patient Reported Outcomes, Novartis; and Ludwig Forrest, Head of International Philanthropy, King Baudouin Foundation. These three trailblazers are the first to be honored by CTF Europe since its inception in November 2018.
Dr. Nathalie Moll, the Director General of EFPIA, was present to address the audience of 75 guests, and to present the Cloud Carrier Award to her dear friend and colleague Magda Chlebus. Dr. Annette Bakker, CTF Europe Board Chair, opened the evening and presented Dr. Francesco Patalano and Ludwig Forrest with their awards. CTF Europe Board members Sissy Windisch and Samia Arslane, as well as CTF staff members, were present. A brief recap video of the event can be viewed here.
Following this warm, optimistic, and friendly evening, CTF actively participated in the closing event of EU-PEARL (25-26 April). EU-PEARL is a public private partnership funded by the Innovative Medicines Initiative, aimed at designing platform trials for four diseases: NF, NASH, MDD, and TB. For NF, it has been a culmination of 3.5 years of constructive collaboration between industry, academia, CTF, and patients. The lasting impact of this work is the successful design of a platform trial for NF, and new partnerships/ friendships for the future. CTF Europe and partners will actively start implementing this blueprint to accelerate clinical trials for the complexities of the NF condition.
Coincidentally, on 26 April, the European Commission published the revision of a nearly 20-year long pharmaceutical legislation aimed at improving access, affordability, and availability of treatments for patients. While applauding the overall worthy intent, it is noted that careful negotiations with all stakeholders and special care are needed to avoid any unintended reduction in innovation. CTF sees the revised legislation as a clear invitation to be more efficient and effective in all the important steps that bring the right treatments to the right patients at the right time. CTF’s efforts and investments in platform trials, biomarkers, and an efficient preclinical hub for NF are all activities that will definitely accelerate the path from discovery to clinical benefit.
As the week drew to an end, the endeavors on behalf of NF patients were capped at the incredible ‘Be Philanthropy’ event hosted by friends and CTF partners at the King Baudouin Foundation (KBF), and organized by CTF’s newly named cloud carrier Ludwig Forrest and his team. The annual meeting, which had not been held in person in three years, brought together 1,100 philanthropists and non-profit executives to hear and learn from each other, further their collective belief in a future of collaboration and fulfilling shared dreams of making today’s world a better place to live for all. In these stormy times that are affecting world and local events, it was the perfectly inspired gathering, filled with hope, goodwill and the optimism of 1,100 people driven by ‘good’.
CTF Europe extends its warmest congratulations to KBF and to all those working to improve patients’ lives in Europe. There can never be enough moments of hope, progress, and celebrations of cloud carriers!
(l-r):
Francesco Patalano (Novartis)
Michele Przypyszny (Children’s Tumor Foundation)
Marco Nievo (Children’s Tumor Foundation)
Magda Chlebus (EFPIA)
Annette Bakker (Children’s Tumor Foundation)
Ludwig Forrest (King Baudouin Foundation)
Christina Economides (Squire Patton Boggs)
