New publication: Guidance for the Harmonisation and Improvement of Economic Evaluations of Personalised Medicine
The objective of this study was to develop guidance contributing to improved consistency and quality in economic evaluations of personalised medicine (PM), given current ambiguity about how to measure the value of PM as well as considerable variation in the methodology and reporting in economic evaluations of PM.
Given current ambiguity about how to measure the value of personalised medicine as well as considerable variation in the methodology and reporting in economic evaluations of PM, one objective of HEcoPerMed was to develop guidance that can contribute to improved consistency and quality in economic evaluations of personalised medicine.
One objective of HEcoPerMed was to identify existing financing and reimbursement models suitable to provide incentives for rapid development, translation and uptake of PM. To fulfil this objective, we performed a systematic literature review and identified 114 publications and reports that develop and describe financing and reimbursement models used for personalised medicine approaches.
Our 1st case study addresses MODY (Maturity Onset Diabetes of the Young). MODY differs from known type 1 and type 2 diabetes and can be successfully treated with sulfonylureas or even without pharmacological treatment. Screening of high risk diabetic patients younger than 35 (determined by MODY calculator) treated with insulin could lead to a better quality of life (QoL) and decreased treatment costs. Patients’ progression is modelled by using an individual level Markov simulation model with 20 years’ time horizon to consider the costs and benefits of screening for MODY patients.
Our 2nd case study tackles adverse reactions to cancer treatments. Fluorpyrimidine-based chemotherapy drugs have long been used for the treatment of primary and advanced solid tumours such as colorectal, esophageal and breast. Usually cancer patients that are treated with such drugs tolerate the therapy well. However, up to 30% of these patients might develop severe or mild adverse reactions. ToxNav, a CE marked test, is designed to identify patients most at risk from severe drug reactions before treatment with 5FU/capecitabine. This allows to personalise their treatment with chemotherapy on the basis of individual patient characteristics. Our model evaluates the impact of adverse reactions on patients’ quality of life and survival, as well as on healthcare costs, and follows the cohort of patients through their potential progression to advanced disease, or death from metastatic breast cancer or other causes.
Our 3rd case study focuses on developing a model for the use of a specific class of drugs: Recently, the European Medicine Agency (EMA) approved entrectinib and larotrectinib for tumours driven by neurotrophin receptors (NTRK) gene fusions. Our NTRK case study focuses on entrectinib, including larotrectinib as a comparator. The structure of the health economic model consists of a decision-tree, reflecting the testing phase, combined with a partitioned survival model.
The macro-economic perspective on Personalised Medicine and what it can do for European Health Systems is still an unsolved matter. The HEcoPerMed project is dedicated to this challenge in the context of health economics and policy making. The official kick-off event of this EU Horizon 2020 project took place from 28 to 29 January in Brussels.