Whitepapers

Therapies designed to target cancers with specific molecular signatures have reshaped the landscape of oncological drug development.1 As the next generation of tumor-agnostic therapies make their way through clinical trials, we should be asking ourselves what we can do to prepare for the upcoming challenge of reimbursement in this space, and what lessons can be learnt from the experience of existing therapies. Over the coming months we will provide recommendations from our own experience and a review of the experiences of the tropomyosin receptor kinase (TRK) inhibitors (larotrectinib and entrectinib) on how to overcome the common challenges in this space in five short papers. Here, we look at what to consider when developing economic models of tumor-agnostic therapies, with a particular focus on the role of genetic testing in this space.

In this piece, Renu and Matthew break down why the market has failed to tackle antimicrobial resistance and look into some of the initiatives created to deal with the problem. Focusing on England’s novel payment evaluation and purchasing frameworks, they investigate how the new ‘de-linked’ approach could impact the development of future treatments.

Drug development is changing, and guidance around the evaluation and approval of new treatments must change with it. The drive to develop medicines for conditions with the most severe unmet need – often rare and with no established or effective standard of care – frequently requires manufacturers to launch technologies based on evidence characterized by non-randomized trial designs with small sample sizes and limited follow-up. Of course, such evidence leads to regulatory and payer decision makers facing a high level of uncertainty when assessing the magnitude of the novel technology’s relative treatment benefit. If we are to accelerate access to transformative therapies, then we must acknowledge that greater flexibility in assessment and payment models is needed to address this uncertainty.