"Methodological Choices for the Effectiveness Assessment” published by HAS

One of the goals of the French High Authority for Health (HAS) is to provide people with long-term and equitable access to appropriate, safe, and effective care. Thus, the assessment of efficiency is a central focus of HAS. In 2011, HAS published the first version of the methodological guide, which explains the principles on which the HAS relies on conducting efficiency assessments, whether in the context of public health assessments carried out by the HAS or, since 2013, as part of the efficiency notices issued by the Committee on Evaluation and Public Health (CEESP). In June 2019, the CEESP validated a second, provisional version, updating the methodological guide for the evaluation of efficiency at the HAS.

The guide has 27 recommendations, which are described in detail. Below is the summary of all 27 recommendations:

Structural methodological choices for evaluating effectiveness:

1. The object is defined: The context is specified (clinical context, regulatory context, expected or observed impacts) and purpose of the assessment of efficiency is clearly stated and justified in this context
2. The choice of evaluation method: The benchmark analysis uses cost-utility analysis and cost-effectiveness analysis as evaluation methods. The choice of the method depends on the nature of the expected or actual health consequences of the evaluated intervention
3. The choice of perspective: The baseline analysis captures a collective perspective, referring to all affected individuals or institutions, whether in terms of health effects or in terms of cost, through the production of the intervention evaluated within the framework of comprehensive care. Otherwise, the choice of a restricted perspective to the health system in the reference analysis is incorrect
4. The choice of the analyzed population: The population of analysis is composed of all individuals whose health is affected by the intervention evaluated directly or indirectly. The impossibility of integrating into the analysis of reference some of the affected individuals is not correct
5. The choice of the comparator: The benchmark analysis identifies all available options
6. The time horizon: The time horizon of the evaluation is defined on the unlimited or on a predefined duration
7. The discounting method: The costs and future results are discounted to count their present value

Methodological choices for evaluation of results:

8. The assessment of health outcomes - general principles: The consequences considered in the assessment of efficiency are the effects of interventions on health
9. The choice of criterion in cost-utility analysis: If performing the cost-utility analysis, the recommended result criterion is the QALY, which allows weighting of the service life by the quality of life
10. The choice of criterion in cost-effectiveness analysis: If performing the cost-effectiveness analysis, the recommended outcome is the life-years. The mortality indicator should be all-cause mortality
11. Evaluation of efficiency and tolerance - general principles: The assessment of effectiveness is based on the estimation of a health outcome differential, related to a cost differential
12. Sources of clinical data: All clinical data sources for the evaluated intervention and its comparators are identified according to a systematic and reproducible methodology and presented according to international standards and HAS recommendations
13. Methods of evaluating comparative effectiveness: The same method is applied to estimate a difference in effectiveness between the intervention studied and a comparator intervention. The choice of the method is explained, and the method is presented in a clear and detailed manner
14. Assessment of safety: The same methodological rigor is expected for the identification and estimation of the tolerance data as for the effectiveness data
15. The choice of the evaluation method of a utility score: Utility scores are estimated from a multi-attribute approach, based on the collection of health status from a patient's perspective. Generic questionnaire and valuation of these states of health established on the preferences of the general population
16. The choice of data sources to estimate utility scores: Utility scores are derived from an ad hoc study or systematic review of the literature. The use of expert opinion to document utility scores is not acceptable
17. The method of estimating utility scores in specific populations: From 16 years of age, it is recommended to use the EQ-5D. Before the age of 16, the use of pediatric tools is recommended. In the absence of a valuation matrix of French preferences, foreign matrices are accepted

Methodological choices for cost evaluation:

18. Cost evaluation - general principles: The assessment of the total cost of a health intervention is based on the production costs of that intervention
19. Assessing direct costs: The evaluation of total costs is based on three stages: the identification, measurement, and evaluation of the resources associated with the intervention
20. Assessing indirect costs: When indirect costs are documented, they are subject to further analysis and are not included in the calculation of the cost-result differential ratios of the benchmark analysis

Methodological choices for modeling:

21. Modeling – general principles: The development of a model conforms to a triple imperative of justification, validation, and exploration of uncertainty
22. The choice of the type of model and its structure: The type of modeling and the structure of the model are defined to represent clinical progression and patient management, without introducing more complexity than necessary
23. An estimate of the value of the model parameters: The observed values and distributions of the parameters are preferred for documenting the model
24. Model validation: The model's ability to produce consistent and credible simulations is systematically explored through the technical verification of the model (internal validation) and a validation procedure to ensure coherent results, that results are the same as with the data not used in the model, the results are similar to the ones of the other model (external validation and cross-validation)
25. Assessing uncertainty in the model: A systematic exploration of the sources of uncertainty associated with the structuring choices of the evaluation, the modeling choices and the parameters of the model, is presented according to a suitable methodology

Presentation and interpretation of conclusions:

26. Interpretation of the assessment of effectiveness: The health interventions that overcome the efficiency frontier are identified
27. Presentation of the assessment of effectiveness: The assessment of effectiveness is presented in a structured, clear, and detailed manner. The methodology is transparent and the data, as well as the sources used, are clearly reported

The guide is open for public consultation until August 9, 2019. The interested parties can submit their comments through an online link. The whole news (with the links to the guide and the comment form) can be found in French here.

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