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Rare diseases Q3 2021

Making informed choices with advanced therapy medicinal products

iStock / Getty Images Plus / Lordn

Nick Meade

Director of Policy, Genetic Alliance UK

Advanced Therapy Medicinal Products (ATMPs) are innovative treatments that use cells, genes and tissues to treat conditions by targeting their underlying causes to provide long-lasting benefits.

ATMPs are unique in many ways — manufacturing is complex, affordability is a different challenge to conventional medicines, some need complex procedures to administer and, crucially, they are unique in their relationship to the person who might be treated with ATMP.
Sometimes called cell or gene therapies, they have the potential to revolutionise the way we treat and manage many diseases and conditions.

Irreversible choice in ATMPs

You cannot choose to stop using an ATMP. Once you have had the treatment, the effects should stay for a long time. The context of the choice to take an ATMP can make this easier or less straightforward.

If I had a rapidly progressive serious condition, with no other option than an ATMP, the choice might be straightforward — I might say I have nothing to lose.

Once you have had the treatment, the
effects should stay for a long time.

Weighing the conditions with ATMPs

ATMPs are arriving in the UK health sector with a new challenge. They are becoming available, either in research or as medicines, for slower progressing conditions — chronic rare conditions.

The unmet need is not always there, either; we have daily or weekly medicines or treatments to care for people with these conditions, but these are not fully effective. They do not stop the progression of the condition.

How do you make that decision? You have a life with a chronic rare disease, and you have a treatment. The condition is lowering your quality of life, but it is not life-threatening. You can have a part-time job, but need a day off a week for treatment and sometimes need to take a day after to recover. A different option arrives: a single treatment that would stop the effects of your condition.

Making informed decisions

What are the risks of ATMPs? Is there follow-on treatment? How does that weigh against the potential benefit in quality of life? The answer will depend on individual circumstances, and that choice needs to be informed and supported.

Alongside the Cell and Gene Therapy Catapult, and with the support of EuroCGT, Genetic Alliance UK co-chairs ATMP Engage, which brings together UK-based stakeholders with an interest in ATMPs to collaborate on the crucial engagement to create the resources and environment necessary to make sure that people lucky enough to get this choice can make it comfortably, with all the information they need.

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