Periodic Reporting for period 3 - HEcoPerMed (Healthcare- and pharma-economic models in support of the International Consortium for Personalised Medicine)
Reporting period: 2022-01-01 to 2022-06-30
The HEcoPerMed project (Healthcare and pharma economics in support of the International Consortium for Personalised Medicine – ICPerMed) is a cooperation and support action (CSA) funded by the European Commission (EC). It is part of the so-called ICPerMed “family” of projects and initiatives that supports the research and implementation of personalised medicine in Europe and beyond. HEcoPerMed responds to the demand for economic models that evaluate treatments made possible through innovations in personalised medicine (PM) and seeks to identify funding and reimbursement mechanisms that provide financial incentives for the rapid development and uptake of such innovations. PM aims to optimally match patient and treatment by assessing the characteristics of patients in which treatments yield (the most) results. HEcoPerMed explores the next generation of assessment and payment models, to support comprehensive cost-effectiveness estimates of PM as well as the sustainable affordability of health innovations.
HEcoPerMed provides a concise overview of and guidance on high-quality methodological approaches for model-based economic evaluations of PM. In three case studies, HEcoPerMed demonstrates practical and methodological issues in economic evaluation of personalised medicine and suggests possible solutions, therewith advancing the economic modelling of PM. Findings and lessons learned from HEcoPerMed are directed at a broad audience including industry, reimbursement authorities, health economic researchers and policy-makers at European, national, and regional level, with the aim of supporting their decision-making on the development and implementation of value-based PM approaches.
HEcoPerMed results indicate that there seems to be a large and probably underused potential to improve health and reduce costs by improving the targeting of existing therapies. Better stratification would increase the cost-effectiveness of current treatments and, therefore, the efficiency levels of the entire healthcare system. Also, where possible economic evaluations of PM should be conducted earlier and be shared among EU Member States to improve timely access to clinically and cost-effective new PM interventions. Furthermore, evidence requirements of European regulatory and reimbursement authorities should be better aligned.
HEcoPerMed provides a concise overview of and guidance on high-quality methodological approaches for model-based economic evaluations of PM. In three case studies, HEcoPerMed demonstrates practical and methodological issues in economic evaluation of personalised medicine and suggests possible solutions, therewith advancing the economic modelling of PM. Findings and lessons learned from HEcoPerMed are directed at a broad audience including industry, reimbursement authorities, health economic researchers and policy-makers at European, national, and regional level, with the aim of supporting their decision-making on the development and implementation of value-based PM approaches.
HEcoPerMed results indicate that there seems to be a large and probably underused potential to improve health and reduce costs by improving the targeting of existing therapies. Better stratification would increase the cost-effectiveness of current treatments and, therefore, the efficiency levels of the entire healthcare system. Also, where possible economic evaluations of PM should be conducted earlier and be shared among EU Member States to improve timely access to clinically and cost-effective new PM interventions. Furthermore, evidence requirements of European regulatory and reimbursement authorities should be better aligned.
To date, the output from HEcoPerMed includes two systematic literature reviews: one identifying current practices in the modelling of PM and investigating the net benefit of PM, and another one on financing and reimbursement models for PM.
The output also includes a good practice guideline for the economic modelling of PM interventions, as well as a demonstration of the application of these guidelines in three selected case studies:
• an extended genetic panel for DPYD testing (TOXNAV) used to identify poor metabolizers of fluoropyrimidine-based chemotherapy and to personalize the dose so as to avoid serious toxicity;
• a (combination of) immunohistochemistry (IHC) and next-generation sequencing (NGS) RNA testing used to detect the presence of rare neurotrophic receptor tyrosine kinase (NTRK) fusions in tumour tissue to identify those eligible for treatment with the histology-independent NTRK-inhibitor entrectinib
• a risk calculator, an autoantibody test and a genetic test used to screen and test for maturity-onset diabetes of the young (MODY), the most common form of mono-genetic diabetes, in which insulin treatment is not beneficial.
This was complemented by a set of hypothetical scenarios to demonstrate benefits at national and European level if different levels of innovation and uptake of personalised medicine were to be achieved. The scenarios were developed with the involvement of patient representatives, experts and stakeholders, to capture their perspectives on aspects shaping the acceptance and institutionalisation of personalised medicine.
The output also includes a good practice guideline for the economic modelling of PM interventions, as well as a demonstration of the application of these guidelines in three selected case studies:
• an extended genetic panel for DPYD testing (TOXNAV) used to identify poor metabolizers of fluoropyrimidine-based chemotherapy and to personalize the dose so as to avoid serious toxicity;
• a (combination of) immunohistochemistry (IHC) and next-generation sequencing (NGS) RNA testing used to detect the presence of rare neurotrophic receptor tyrosine kinase (NTRK) fusions in tumour tissue to identify those eligible for treatment with the histology-independent NTRK-inhibitor entrectinib
• a risk calculator, an autoantibody test and a genetic test used to screen and test for maturity-onset diabetes of the young (MODY), the most common form of mono-genetic diabetes, in which insulin treatment is not beneficial.
This was complemented by a set of hypothetical scenarios to demonstrate benefits at national and European level if different levels of innovation and uptake of personalised medicine were to be achieved. The scenarios were developed with the involvement of patient representatives, experts and stakeholders, to capture their perspectives on aspects shaping the acceptance and institutionalisation of personalised medicine.
All results produced by HEcoperMed have been developed through exchange with European health care systems, the European Commission and strategic platforms like ICPerMed and EP PerMed and are intended to be taken up and live on through these structures. There has been a close connection between HEcoPerMed and ICPerMed, as well as active exchange with other ICPerMed Family members like PERMIT, Saphire and R4PerMed. The achievements and results of HEcoPerMed are considered to be of importance for a further future initiative of Member States and the EC, the European Partnership of Personalised Medicine (EP PerMed).
• HEcoPerMed provides guidance to health economic modellers of PM and to the evaluators and reviewers of health economic models. A set of 23 modelling-recommendations was developed and published.
• HEcoPerMed produced evidence on the added value of PM versus non-PM by conducting a systematic literature review of economic evaluations of PM. It concluded that PM interventions tend to have (modest) health benefits compared to non-PM interventions but that because of the high costs associated with PM, the health gains for an individual do not always translate into added value for healthcare systems and society.
• HEcoPerMed identifies and analyses existing financing and payment models suitable for the diffusion of personalised medicine in European health systems in a large literature review of published scientific and grey literature. A set of recommendations for the implementation of appropriate financing and reimbursement models for personalised medicine was developed.
• HEcoPerMed incorporates a future-oriented perspective for the uptake of PM-based health care services across countries at the national and European level.
• HEcoPerMed involves patient representatives, economic modellers and other experts and stakeholders for input on aspects shaping the acceptance of personalised medicine and creating a wider awareness raising and diffusion of personalised medicine as part of future European health systems.
Together, these outputs provide the main basis for a Position Paper, which describes these findings and the lessons learned from HEcoPerMed for different kinds of stakeholders. Furthermore, results can be accessed via a considerable number of open access publications, visit the HEcoPerMed project website for an overview.
• HEcoPerMed provides guidance to health economic modellers of PM and to the evaluators and reviewers of health economic models. A set of 23 modelling-recommendations was developed and published.
• HEcoPerMed produced evidence on the added value of PM versus non-PM by conducting a systematic literature review of economic evaluations of PM. It concluded that PM interventions tend to have (modest) health benefits compared to non-PM interventions but that because of the high costs associated with PM, the health gains for an individual do not always translate into added value for healthcare systems and society.
• HEcoPerMed identifies and analyses existing financing and payment models suitable for the diffusion of personalised medicine in European health systems in a large literature review of published scientific and grey literature. A set of recommendations for the implementation of appropriate financing and reimbursement models for personalised medicine was developed.
• HEcoPerMed incorporates a future-oriented perspective for the uptake of PM-based health care services across countries at the national and European level.
• HEcoPerMed involves patient representatives, economic modellers and other experts and stakeholders for input on aspects shaping the acceptance of personalised medicine and creating a wider awareness raising and diffusion of personalised medicine as part of future European health systems.
Together, these outputs provide the main basis for a Position Paper, which describes these findings and the lessons learned from HEcoPerMed for different kinds of stakeholders. Furthermore, results can be accessed via a considerable number of open access publications, visit the HEcoPerMed project website for an overview.