Diagnostic target product profiles for cancer: A demand signaling tool to stimulate innovation in early cancer diagnosis

BackgroundDiagnosing cancer earlier can support timely treatment and improve patient prognosis. Innovative diagnostic tests need to appropriately respond to unmet needs. Target Product Profiles (TPPs) are specification documents that serve as demand-signalling tools to inform developing improved tests – in terms of scientific performance, fit with clinical pathways and patient needs. There is growing interest in their use. Commissioned by Cancer Research UK, RAND Europe and the Office of Health Economics conducted a study to establish a methodological process for developing robust diagnostic TPPs for cancer and identify the types of features TPPs should consider.MethodsA mixed-methods approach combined international literature review, interviews, workshops with UK experts and developing an economic modelling tool for TPP requirements. 129 individuals contributed (academics, funders, healthcare professionals, industry, policy, regulatory and HTA experts, health economists, and patient and public voice).ResultsEstablishing specifications for combinations of core features driving a test’s value is key to TPP development. Trade-offs between different requirements must be considered. Relevant features types fall into 9 categories (e.g. unmet need, analytical performance, clinical validity, clinical utility, human factors, infrastructure, cost/economic, regulation, environmental). Fit-for-purpose TPPs require input from diverse stakeholders through desk research, consultation and modelling methods. TPP development has 2 stages: inception (establishing core working group, governance, action plan) and implementation (scoping unmet need and key test requirements, TPP drafting, consensus building).ConclusionsThis internationally-relevant, tumour-site agnostic guide is an evidence-based resource for designing TPPs for specific cancer tests and use cases. It highlights multifaceted factors and practical tips for deciding which features to consider and methods to use in TPP development. Robust TPPs are an important tool for developing improved cancer diagnostic tests, but need to align with wider innovation and health system efforts.Legal entity responsible for the studyRAND Europe; Cancer Research UK.FundingCancer Research UK.DisclosureAll authors have declared no conflicts of interest.