Whitepapers

Therapies designed to target cancers with specific molecular signatures have reshaped the landscape of oncological drug development.¹ The advent of tumour-agnostic therapies represents both a major opportunity for patients to receive therapies targeted to their specific disease on a molecular level and an important step towards discovering biomarkers of response.

In this paper we highlight several key themes and recommendations that are essential to consider when navigating our new market environment post COVID-19.

The first chimeric antigen receptor (CAR) T-cell therapies launched 3 years ago, promising dramatic benefits. Patients now await the second cluster – including the novel technologies KTE-X19, lisocabtagene maraleucel, idecabtagene vicleucel and ciltacabtagene autoleucel – alongside the launch of KYMRIAH® and YESCARTA® into earlier lines of therapy.1 As consultants engaged with the first, we remember the vista of uncertainty: how do we robustly demonstrate to health technology assessment (HTA) bodies the expected value of these expensive interventions with only short-term, single-arm data as supportive evidence, the kind of data such bodies are typically so skeptical of? To paraphrase a team member, we were to ‘go where no one has gone before’.a As the second cluster approaches launch, we can claim no such excuse. Join us as we learn from past reviews from the Institute for Clinical and Economic Review (ICER) and the National Institute for Health and Care Excellence (NICE), draw four implications for the future, and then pose four further questions for your consideration.