Economist Impact Cell and Gene Therapy Summit, Brussels 15-16 April, 2024

Ljubljana, April 17, 2024 – Ljubljana, April 17, 2024 – We attended the Economist Impact Cell and Gene Therapy Summit in Brussels over the past two days. 300 delegates and 60 speakers discussed on and off stage the current opportunities and challenges in the field of CGT, especially from an economic perspective. The big unanswered question that dominated almost all discussions was how we as a society will pay for new cures.
This was perhaps best illustrated in one of the first of the many panel discussions, when Dr Flora Peyvandi from the University of Milan, also an expert in the field of heamophilia, addressed the panellists by stating clearly that the bottleneck isn’t the science, nor the regulators, but the payers. The tension in the air because cures are available but no one knows how they’ll be paid for was palpable.
Another indication of the inadequacy of the old model of market reality was given by Dr Momir Radulovic, Managing Director of the Slovenian regulatory authority JAZMP. He told us that it was unbelievable that after 4500 applications of the drug, Zolgensma never received a revised dossier for its marketing authorization. The drug was originally approved on data from about 35 patients, and although subsequently there is a wealth of new knowledge about its efficacy and safety from further patient use, it appears that no one is incentivised to update the dossier and perhaps revise the approval or pricing.
The emotional highlights of the conference, as well as perhaps the potential key moments that could change the state of reimbursement, came from the patients. Patients and patient organisations were mentioned in virtually every panel, everyone kept coming back to them. Two patient stories in particular cut through the thicket of unanswerable questions. They came from two parents, Terry Pirovolakis and Julia Vitarello. Both had to watch their child being diagnosed with a fatal genetic disorder, both decided to fight, both started campaigns to raise funds and get support from doctors and researchers, and both are still committed to helping other parents like them. With the drive and passion of people like them, it’s easy to find answers because they demonstrate that it can be done. The question that remains for society to answer is how we can achieve this on a large scale for more patients.
Representing the colours of our CTGCT project consortium was Dr Jane Kinghorn from UCL, who moderated the panel discussion on how to bring CGTs into the mainstream, which included researchers, regulators and patients. Several points were raised including the need for regulators to standardise approaches, manufacturers to share best practice on QC assays and use of standard reference material and the importance of registries and collection of real world data that speaks to the needs of regulators and payors. Dr Momir Radulović also made it clear that hospitals as centres of excellence for the provision of CGTs are necessary and can drive efficiencies, and as Dr Virginie Bros-Facer from Eurordis, the rare disease organisation, further noted, this makes well maintained registries essential to allow for follow-up care close to the patient’s home.