When chimeric antigen receptor (CAR)-T cell therapy first arrived, Time Magazine called it ‘cancer’s newest miracle cure’. Certainly, the therapy – which uses a patient’s own genetically altered T cells to treat cancers such as lymphoma, multiple myeloma and leukaemia – heralded a medical breakthrough. Hard-to-treat conditions with no prior curative alternative and very low rates of survival, such as relapsed or refractory diffused large B-cell lymphoma (r/r DLBCL), suddenly had a highly effective treatment available, seemingly even a ‘cure’.
But ever since the first two CAR-Ts were approved in Europe in 2018 – Kymriah and Yescarta – the focus has been on price and logistics as much as therapeutic value. With their highly complex treatment procedures and high price tags, CAR-Ts posed a unique challenge for healthcare systems, exacerbated by uncertainty around their long-term, real-world value.
Industry and healthcare systems collaborated to find solutions. Novel outcomes-based reimbursement schemes were installed to address the uncertainty on long-term value. For example, in France and the UK, reimbursement was on the condition of collecting additional data and subject to future reassessments. Elsewhere, rebates (Germany) or staged payments (Italy and Spain) were linked to individual patient outcomes. Moreover, CAR-T specific processes and infrastructure to allow patient identification, referral and treatment in authorised treatment centres have been established.
Six years of progress reinforce the benefit
Since then, substantial real-world evidence has accumulated for these first CAR-T indications. For instance, outcomes in r/r DLBCL have been shown to align with clinical trial results, even in a broader patient population.
Much has been learned to allow for these real-world outcomes. Healthcare professionals involved in patient management have acquired extensive experience in the resolution of adverse reactions. Through stakeholder coordination, product manufacturing and delivery times have been highly optimised, reducing turnaround time from over a month to less than three weeks, helping to improve CAR-T effectiveness and patient outcomes.
The progress in this period has been exceptional. However, hurdles remain to broad patient access – and in a rapidly changing environment, these hurdles may be increasing.
Read the article in full here.