Algorithms and heuristics of health technology assessments: A retrospective analysis of factors associated with HTA outcomes for new drugs across seven OECD countries.

CONTEXT: Positive health technology assessment (HTA) outcomes can have important implications for equity, efficiency and timely patient access to novel therapies. Several outcomes and dimensions of benefit beyond utility feed into HTA processes. OBJECTIVE: We analyse a proprietary dataset of HTA outcomes in 7 countries, to (a) test whether HTA decision-making is grounded in welfarist or extra-welfarist approaches; and (b) empirically determine the factors associated with positive HTA outcomes, the time to achieve these and establish the magnitude of inter-country differences in assessment processes. METHODS: Data were extracted from publicly available HTA reports on drugs that received marketing authorisation between 2009 and 2018 (N = 1415). The outcomes of interest were the probability of positive HTA outcomes and the time-to-HTA outcome; these were examined with respect to clinical, regulatory, product- and disease-related, evidence uncertainty and contextual variables. Econometric models utilising survival analysis and multinomial logistic regression were specified. FINDINGS: Positive HTA outcomes accounted for 87.3% of the sample (n = 1235), of which 71% (n = 1004) were restricted. Drugs with positive HTA outcomes were subject to clinical restrictions (n = 652, 46%), financial risk-sharing (n = 439, 31%) or had been rejected at least once (n = 282, 20%). Significant predictors of positive HTA outcomes were orphan drugs with cancer indications, high quality of evidence linked to clinical and economic evidence uncertainties which had been overcome, and contextual considerations, particularly innovativeness and unmet need. Comparative analyses revealed systematic differences between countries in their propensity to accept the same drugs, particularly oncology and orphan drugs. CONCLUSIONS: Our results are contextual and reinforce arguments in favour of explicitly accounting for social value judgements, establishing separate assessment frameworks for highly uncertain products, adopting risk mitigation strategies for novel therapies with early phase evidence, and sharing of HTA practices across settings. Lastly, HTA agencies have adopted an extra-welfarist approach to value assessment and resource allocation.

Similarities and Differences in Health Technology Assessment Systems and Implications for Coverage Decisions: Evidence from 32 Countries.

Health technology assessment (HTA) systems across countries vary in the way they are set up, according to their role and based on how funding decisions are reached. Our objective was to study the characteristics of these systems and their likely impact on the funding of technologies undergoing HTA. Based on a literature review, we created a conceptual framework that captures key operating features of HTA systems. We used this framework to map current HTA activities across 32 countries in the European Union, the UK, Canada and Australia. Evidence was collected through a systematic search of competent authority websites and grey literature sources. Primary data collection through expert consultation validated our findings and further complemented the analysis. Sixty-three HTA bodies were identified. Most have a national scope (76%), are independent (73%), have an advisory role (52%), evaluate pharmaceuticals predominantly or exclusively (76%), assess health technologies based on their clinical and cost-effectiveness (73%) and involve various stakeholders as members of the HTA committee (94%) and/or through external consultation (76%). The majority of HTA outcomes are not legally binding (81%). Although all study countries implement HTA, the way it fits into decision-making, negotiation processes, and coverage and funding decisions differs significantly across countries. HTA is a dynamic and transformative process and there is a need for transparency to investigate whether evidence-based information influences coverage decisions.

Why do health technology assessment drug reimbursement recommendations differ between countries? A parallel convergent mixed methods study.

Using quantitative and qualitative research designs, respectively, two studies investigated why countries make different health technology assessment (HTA) drug reimbursement recommendations. Building on these, the objective of this study was to (a) develop a conceptual framework integrating the factors explaining these decisions, (b) explore their relationship and (c) assess if they are congruent, complementary or discrepant. A parallel convergent mixed methods design was used. Countries included in both previous studies were selected (England, Sweden, Scotland and France). A conceptual framework that integrated and organised the factors explaining the decisions from the two studies was developed. Relationships between factors were explored and illustrated through case studies. The framework distinguishes macro-level factors from micro-level ones. Only two of the factors common to both studies were congruent, while two others reached discrepant conclusions (stakeholder input and external review of the evidence processes). The remaining factors identified within one or both studies were complementary. Bringing together these findings contributed to generating a more complete picture of why countries make different HTA recommendations. Results were mostly complementary, explaining and enhancing each other. We conclude that differences often result from a combination of factors, with an important component relating to what occurs during the deliberative process.

Value assessment of disease-modifying therapies for Relapsing-Remitting Multiple Sclerosis: HTA evidence from seven OECD countries.

This study systematically compares HTA recommendations on a number of disease-modifying therapies for patients with Relapsing-Remitting Multiple Sclerosis. We analysed publicly available HTA reports for nine medicine-indication pairs across seven OECD countries using a methodological framework enabling systematic analysis of HTA recommendations. The analysis was conducted based on a number of value dimensions, including clinical and economic variables, as well as several other dimensions of value beyond cost-effectiveness. The material was qualitatively and quantitatively coded following the different stages of HTA decision-making process. Fifty-seven medicine-indication pairs were assessed across the study countries. Of those, eight medicine indication-pairs reported diverging HTA recommendations. Although HTA recommendations were based on the same evidence submitted in most cases, significant variations were identified in interpretation and acceptance of evidence resulting in different uncertainties raised and different ways of addressing them. Uncertainties arose both in terms of the clinical and the economic evidence, including the design of key trials or the data quality in economic models. Beyond costs and effects, additional dimensions of value had an impact in the direction of recommendations, however with different magnitude across countries. We show that there is heterogeneity across countries in HTA for evaluating DMTs for RRMS with a lack of standardised methods in evaluating clinical and economic evidence and the use of social value judgments to inform decision-making.

Using IMPrESS to guide policy change in multiple sclerosis.

The International MultiPlE Sclerosis Study (IMPrESS) studied the significant impact of multiple sclerosis (MS) on the health and well-being of both people with the disease and their caregivers, along with its broader socioeconomic impact. Results confirmed that there is an urgent need to achieve better outcomes for people with MS. This paper uses results from the IMPrESS to present new international evidence on the socioeconomic burden of MS and discuss the merits of a likely paradigm shift in the management of MS towards the use of better (and more accurate) diagnostic follow-up to monitor disease progression and the earlier use of disease-modifying treatments (DMTs) to achieve better clinical, quality-of-life and socioeconomic results for individuals.

The economic burden of diabetes in India: a review of the literature.

BACKGROUND: Diabetes and its complications are a major cause of morbidity and mortality in India, and the prevalence of type 2 diabetes is on the rise. This calls for an assessment of the economic burden of the disease. OBJECTIVE: To conduct a critical review of the literature on cost of illness studies of diabetes and its complications in India. METHODS: A comprehensive literature review addressing the study objective was conducted. An extraction table and a scoring system to assess the quality of the studies reviewed were developed. RESULTS: A total of nineteen articles from different regions of India met the study inclusion criteria. The third party payer perspective was the most common study design (17 articles) while fewer articles (n =2) reported on costs from a health system or societal perspective. All the articles included direct costs and only a few (n =4) provided estimates for indirect costs based on income loss for patients and carers. Drug costs proved to be a significant cost component in several studies (n =12). While middle and high-income groups had higher expenditure in absolute terms, costs constituted a higher proportion of income for the poor. The economic burden was highest among urban groups. The overall quality of the studies is low due to a number of methodological weaknesses. The most frequent epidemiological approach employed was the prevalence-based one (n =18) while costs were mainly estimated using a bottom up approach (n =15). CONCLUSION: The body of literature on the costs of diabetes and its complications in India provides a fragmented picture that has mostly concentrated on the direct costs borne by individuals rather than the healthcare system. There is a need to develop a robust methodology to perform methodologically rigorous and transparent cost of illness studies to inform policy decisions.