We need your expertise!

Dear participant,

Our Horizon Europe project, ASCERTAIN, has a mission to make innovative high-cost drugs and technologies more accessible in Europe. ASCERTAIN will develop easy to use tools including pricing models, value assessment models, and reimbursement models, adaptable to country-specific conditions and patient preferences.

As part of the ASCERTAIN Horizon Europe research project we are conducting research to Cost-Effectiveness Thresholds. To complement our recent scoping review on this topic we are now conducting a multi-stakeholder online survey. This survey aims to:

- Understand Current Practices: Assess how Cost-Effectiveness Thresholds are determined, applied, and interpreted in different healthcare systems.

- Stakeholder Perspectives: Capture insights and opinions from diverse stakeholders on the relevance, appropriateness, and future direction of Cost-Effectiveness Thresholds in healthcare decision-making.

- Broaden the Scope of Value: Identify additional factors or criteria that should be considered in the development and application of Cost-Effectiveness Thresholds to reflect broader elements of value.

The online survey will be conducted from January 20, 2025 until February 28, 2025. The estimated time for taking the survey is around 20-30 minutes. Your input will provide valuable insights to advance this research.

Background

Innovative Health Technologies (IHTs), including medicines, medical devices, and procedures, are healthcare solutions that introduce new or enhanced approaches with the potential to significantly boost positive health outcomes. The growing presence of IHTs poses financial challenges for existing healthcare systems, as their potentially high costs strain the limited budgets of healthcare systems, diverting resources from other essential healthcare services. As a result, an economic evaluation of these IHTs is needed to ensure the cost-effective innovation of healthcare systems.

The outcome of an economic evaluation is generally expressed as an incremental cost-effectiveness ratio (ICER), which reflects how much additional resources are needed for an intervention to generate one additional health gain, mostly depicted in a quality adjusted life year (QALY), in comparison to the most effective alternative (often the standard of care). This ICER serves as a valuable standalone estimate, representing an efficiency ratio.

However, the ICER can also be compared against a Cost-Effectiveness Threshold value (i.e., the maximum acceptable ICER). This comparison tells if a new intervention is deemed more (ICER<threshold) or less (ICER>threshold) efficient in creating health gains than the interventions in the current healthcare system. To ensure accessibility and affordability of IHTs, an adequate Cost-Effectiveness Threshold that represents the needs and resources of the country of interest is essential.

No universal method to determine a Cost-Effectiveness Threshold exist. Theoretically there are two main approaches, demand-side and supply-side approach, and combination of these. In practice some countries have used the GPD per capita as a benchmark to set the Cost-Effectiveness Threshold (e.g. 3x the GDP per capita).

The demand-side approach relies on population preferences, often gathered through contingent valuation surveys—where people are asked how much they would value, or would be willing to pay for, specific health outcomes. This approach reflects a societal perspective.

The supply-side approach aligns with a healthcare system perspective by focusing on the opportunity costs – the value of the benefits lost by not using the available budget to fund the best alternative intervention.

Some countries adjust the value of the Cost-Effectiveness Threshold in specific contexts to account for unique factors or societal values. These so-called modifiers are intended to ensure that the value-for-money assessments align with broader health systems goals or societal preferences. Common areas where modifiers might be applied include:

· Severity of the disease: Higher thresholds might be used for severe or life-threatening conditions.

· Vulnerable groups: Societies may prioritize treatments for vulnerable groups, like children or people with rare diseases, and modify thresholds accordingly.

· Uncertainty or risk: If evidence about an intervention's effectiveness is uncertain, the threshold might be adjusted to account for this.

Other modifiers, like an intervention’s impact on the environment and other values beyond the QALY might in principle be possible.

Who should participate?
We are seeking European participants from various stakeholder groups: patients/ patient representatives, HTA bodies, policy makers, healthcare providers/ healthcare professionals, payers/ insurers, citizens and health technology developers, to share their opinions from their respective stakeholder group perspective indicated in question 2.

What can you expect?

This questionnaire will guide you through a series of questions about the use, application, and development of Cost-Effectiveness Thresholds in healthcare decision-making. You will be asked to share your insights and opinions on current practices, potential improvements, and key factors influencing Cost-Effectiveness Thresholds. The survey is structured with a mix of multiple-choice and open-ended questions.

You decide whether to participate
Participation in this study is completely voluntary. You can stop at any time and would not need to provide any explanation. All information provided by all participants will be fully anonymized.

There will be no negative consequences if you decide not to participate or withdraw. Until you submit the survey, you can still decide not to take part in the research. If you stop, your data will not be stored. After you click ‘send’, we cannot trace what data you have shared with us anymore

Who can see your data? / What will happen to my data?
All data collected during this study will be anonymised to protect your identity. Your data will be stored securely, adhering to stringent data protection standards. Only individuals directly involved in the research will have access to the data. The findings of this study will be published in academic journals. These results will be presented in an aggregated, anonymised format and will be accessible to the public.


Your data will be retained for 10 years after completion of the research. We retain the data so that other researchers have the opportunity to verify that the research was conducted correctly.


Do you have questions about the study?
If you have any questions about the survey, the study or your privacy rights, such as accessing, changing, deleting, or updating your data, please contact:

Name: prof. Tomas Tesar

Email: tesar@fpharm.uniba.sk


Or find additional information on the project website https://www.access2meds.eu/.


In case of any technical issues, please contact:

Name: Peter Keckes

Email: keckes@fpharm.uniba.sk



Declaration of Consent
I have read the information provided and understand what the study is about and what data will be collected from me. By signing this form, I:

1. consent to participate in this research;
2. confirm that I understand participation in this research is entirely voluntary and that I may withdraw at any time. However, I acknowledge that once I click 'send,' my data could not be traced back and be deleted.

*1.Consent:(Required.)

Please, check the box if you consent to this