There are currently more than 50 cell and gene therapies being developed in the UK for several rare and ultra-rare diseases. In the US, one cell therapy called Descartes-08 is being investigated in clinical trials to see if it can be used to treat myasthenia gravis. This is the first type of cell therapy of its kind to be used to treat an autoimmune disease. Most cell and gene therapies offer a “one and done” approach, where one treatment is all a patient receives. This approach can be life-changing for someone with a chronic illness like myasthenia.

The potential for these treatments is clear, but unfortunately the cost is high. It is estimated that one dose of a cell or gene therapy costs between 1 and 2 million dollars per patient. The Cell and Gene Therapy Summit was held to get the perspective from experts across the healthcare economical field – investors, clinicians, patient groups, and more – to see if a solution could be formed to ensure that in a future where more cell and gene therapies became available, it is feasible for anyone in need to have access.

Myaware were invited to attend, and our research and partnerships officer Charlotte Campbell travelled to Brussels between the 23rd to the 25th of April. There, she was able to take part in these conversations and provide the perspective of patients and make sure that outcomes important to our members were heard. Other patient groups, such as the Sickle Cell Society and the Anthony Nolan charity, were also in attendance.

The summit highlighted several ways the cost of cell and gene therapies could be managed. Namely, that the existing process of regulating clinical trials and therapeutic development needs to be reassessed. The length of time taken from a drug concept to become an approved reality is long, and the COVID-19 vaccine programme proved that these processes can be amended when necessary. Another point brought up was the importance of patient data. Registries, such as the one being developed for myasthenia in the UK, provide access to data that can also improve the time it takes researchers to identify trends and avenues of exploration for therapeutic design. Finally, a discussion on government policy and improvements to the existing framework were held, with representatives from the MHRA and EMA present at the summit.

Overall, this summit was interesting and educational, and it was very important for patient groups such as myaware to be included in the conversation surrounding cell and gene therapies. The way these therapies are assessed by regulatory groups and economists should include criteria for the effect a therapeutic such this will have on a patient’s life. It is clear this is an incredibly difficult factor to relate to a price, and it is only by having discussions with all groups involved that a solution can be found.

To learn more about the project to develop a myasthenia patient registry, click here

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