Health technology assessment (HTA) is a multidisciplinary process that aims to determine the value of a health technology and informs decision-making throughout the innovation lifecycle. 1 As such, may represent a major asset for mechanism-based drug repurposing. HTA employs various methods, such as qualitative stakeholder involvement methods, health economic modelling and cost and patient-reported outcome measurement in clinical research. 2 Throughout the drug repurposing lifecycle, these methods are employed iteratively and inform each other. Health economic modelling, for example, can aid to identify scenarios in which the repurposing candidate could add value, determine the (potential) cost-effectiveness of a repurposed drug and inform further research. However, mechanism-based drug repurposing comes with challenges, such as the need for biomarkers, specific disease subgroups with limited data available, and determining a fair price when the patient population is small, which may complicate HTA. The aim is to demonstrate how HTA methods can inform the research and development of a mechanism-based repurposed drug and validate the HTA toolbox for the REPO4EU platform.
We apply HTA methods in the REPO-HYPER study, in which combinations of repurposed drugs will be investigated as new potential treatment of resistant hypertension in patients with abnormal plasma NOX5 protein levels. Patient reported outcomes, e.g. health-related quality of life (assessed by the EQ-5D) are included in the HYPER phase IIa study to enable the evaluation of patient relevant outcomes in this specific subgroup of patients. An early health economic model is developed alongside the HYPER study, which explores the potential cost-effectiveness of a biomarker plus repurposed combination drug to the standard of care. The model informs decisions throughout the drug repurposing lifecycle from a societal perspective and adopts a lifetime horizon. Relevant stakeholders, like clinicians, patient and industry representatives, are involved throughout the model development and validation process and, via semi-structured interviews, provide input on model structure, inputs, analyses and future development. Scenario and sensitivity analyses are conducted to identify scenarios in which the ‘biomarker plus combination drug’ strategy could add value and inform further research. The model is updated at different stages, when new evidence becomes available, to further evaluate the cost-effectiveness of the repurposed drug and inform pricing and reimbursement decisions.
To support (early) HTA in mechanism-based drug repurposing, the REPO4EU platform will contain a toolbox of HTA methods that provides methodological guidance to researchers during the drug repurposing lifecycle. It includes modelling templates, guidance on patient-reported outcome measurement and advice on how to overcome specific challenges. By incorporating HTA methods throughout the drug repurposing lifecycle, value-based drug repurposing for the patient, payers, and society is supported.
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