Improving decision-making for drug reimbursement in Iran

Reimbursement decision making in any healthcare system is an important process to ensure patient access to various healthcare services in an affordable manner. A proper reimbursement decision making happens when it can concurrently pursue three main goals including quality of care, population of health, and affordability. However, achieving the goals requires enough knowledge and proper healthcare governance. In middle-income countries (MICs) improving the three main goals is likely to be difficult due to weakness in the knowledge production infrastructure and healthcare governance. To investigate possible solutions for improving the drug reimbursement decision-making system in MICs, this thesis focuses on the drug reimbursement decision-making system in Iran. Subsequently, we focus on use of a monoclonal antibody in breast cancer. Trastuzumab (Herceptin) is widely used in the treatment of overexpressed human epidermal growth factor receptor 2 (HER2-positive) breast cancer. Trastuzumab, as an expensive drug, has continued to be a topic of conversation in many healthcare systems since its launch into the pharmaceutical markets. The overall aim of this thesis is to investigate how an MIC (in this case, focusing on Iran) can improve its drug reimbursement decision-making system. Therefore, this thesis provided important information on how an MIC can improve the drug reimbursement decision-making system in three parts. Firstly, we discussed the current situation and subsequent consequences of the drug reimbursement decision-making system in Iran. Secondly, we provided some solutions to improve limited health economics-related evidence in Iran. And finally, some recommendations are provided to improve the system. This thesis includes three parts and each of these provides an answer to one or more research questions. In the first part (chapters 2 and 3), the current situation and subsequent consequences of the drug reimbursement decision-making system in Iran are discussed. The second part focuses on knowledge production in MICs. As already mentioned, obtaining information is not an easy task in MICs and researchers have to find solutions to overcome this shortage of information. Therefore, this part provides some solutions for this problem. In addition, it describes some economic evaluations and a scenario analysis for efficient and affordable treatment. This part has three chapters (4−6). The third part provides recommendations to improve the system. It describes how MICs can benefit from HTA studies and knowledge production in order to achieve the overall goals of a reimbursement decision-making system. This part has two chapters, which are chapter 7 and 8

Adjuvant Trastuzumab Therapy for Early HER2-Positive Breast Cancer in Iran: A Cost-Effectiveness and Scenario Analysis for an Optimal Treatment Strategy

Introduction: Clinical guidelines have recommended a 1-year trastuzumab regimen as standard care for early human epidermal growth factor receptor 2 (HER2)-positive breast cancer; however, th

The Drug Reimbursement Decision-Making System in Iran

Background:  Previous studies of health policies in Iran have not focused exclusively on the drug reimbursement process. Objective: The aim of this study was to describe the entire drug reimbursement process and the stakeholders, and discuss issues faced by policymakers. Methods: Review of documents describing the administrative rules and directives of stakeholders, supplemented by published statistics and interviews with experts and policymakers. Results: Iran has a systematic process for the assessment, appraisal, and judgment of drug reimbursements. The two most important organizations in this process are the Food and Drug Organization, which considers clinical effectiveness, safety, and economic issues, and the Supreme Council of Health Insurance, which considers various criteria, including budget impact and cost-effectiveness. Ultimately, the Iranian Cabinet approves a drug and recommends its use to all health insurance organizations. Reimbursed drugs account for about 53.5% of all available drugs and 77.3% of drug expenditures. Despite its strengths, the system faces various issues, including conflicting stakeholder aims, lengthy decision-making duration, limited access to decision-making details, and rigidity in the assessment process. Conclusions: The Iranian drug reimbursement system uses decision-making criteria and a structured approach similar to those in other countries. Important shortcomings in the system include out-of-pocket contributions due to lengthy decision making, lack of transparency, and conflicting interests among stakeholders. Iranian policymakers should consider a number of ways to remedy these problems, such as case studies of individual drugs and closer examination of experiences in other countries

Severity-Adjusted Probability of Being Cost Effective

Background In the context of priority setting, a diferential cost-efectiveness threshold can be used to refect a higher societal willingness to pay for quality-adjusted life-year gains in the worse of. However, uncertainty in the estimate of severity can lead to problems when evaluating the outcomes of cost-efectiveness analyses. Objectives This study standardizes the assessment of severity, integrates its uncertainty with the uncertainty in cost-efectiveness results and provides decision makers with a new estimate: the severity-adjusted probability of being cost efective. Methods Severity is expressed in proportional and absolute shortfall and estimated using life tables and country-specifc EQ-5D values. We use the three severity-based cost-efectiveness thresholds (€20.000, €50.000 and €80.000, per QALY) adopted in The Netherlands. We exemplify procedures of integrating uncertainty with a stylized example of a hypothetical oncology treatment. Results Applying our methods, taking into account the uncertainty in the cost-efectiveness results and in the estimation of severity identifes the likelihood of an intervention being cost efective when there is uncertainty about the appropriate severity-based cost-efectiveness threshold. Conclusions Higher willingness-to-pay thresholds for severe diseases are implemented in countries to refect societal concerns for an equitable distribution of resources. However, the estimates of severity are uncertain, patient populations are heterogeneous, and this can be accounted for with the severity-adjusted probability of being cost efective proposed in this study. The application to the Netherlands suggests that not adopting the new method could result in incorrect decisions in the reimbursement of new health technologies

Use of data-mining to support real-world cost analyses: An example using HER2-positive breast cancer in Iran

INTRODUCTION: Patient registries play an important role in obtaining real-world evidence of the cost-effectiveness of treatments. However, their implementation is costly and sometimes infeasible in many middle-income countries (MICs). We explored the combination of data-mining and a large claims database to estimate the direct healthcare costs of HER2-positive breast cancer (BC) treatment in Iran and the fraction of total costs from trastuzumab use.METHOD: We performed a retrospective analysis of claims data from the Iran Social Security Organization, a health insurer which covers approximately 50%(~40 million) of the Iranian population, in the period of 21/03/2011-20/03/2014. A data-mining algorithm using R software and validated using patient dossiers in the Cancer Research Center identified 1295 patients and divided them into the three main HER2-positive breast cancer stages (early, loco-regional and advanced). A payer perspective was used to calculate the absolute and relative direc

Adjuvant Trastuzumab Therapy for Early HER2-Positive Breast Cancer in Iran: A Cost-Effectiveness and Scenario Analysis for an Optimal Treatment Strategy

Introduction: Clinical guidelines have recommended a 1-year trastuzumab regimen as standard care for early human epidermal growth factor receptor 2 (HER2)-positive breast cancer; however, this recommendation can have a dramatic impact on total drug expenditures in middle-income countries (MICs). We performed a cost-effectiveness analysis from the Iranian healthcare perspective to find an optimum duration of trastuzumab use in Iran. Method: We compared four treatment strategies comprising chemotherapy and varying durations of trastuzumab use (no trastuzumab, 6, 9 months, and 1 year), and a Markov model and probabilistic sensitivity analysis were used to estimate the costs and effects of the strategies. We then examined the cost effectiveness of the strategies at different willingness-to-pay (WTP) thresholds and ages at onset of treatment. Results: Incremental costs (versus no trastuzumab) were €8826 (6 months), €13,808 (9 months) and €18,588 (12 months), while incremental quality-adjusted life-years (QALYs) were 0.65 (6 months), 0.87 (9 months) and 1.14 (12 months). At a threshold of 3 × gross domestic product (GDP)/capita (€21,000/QALY) and for patients younger than 59 years, the 6-month protocol was most likely to be cost effective (probability of 42%). At a threshold of 4 × GDP/capita (€28,000/QALY), the 6-month and 1-year regimens were essentially equal in cost effectiveness (37 and 35%, respectively). At this WTP threshold, the 6-month and 1-year regimens were optimal strategies only for patients up to 66 and 44 years of age, respectively. Conclusion: In contrast to clinical guidelines, 6 months of trastuzumab may be the most cost-effective option for Iran. The lower absolute WTP threshold and lower life expectancy compared with high-income countries are two crucial parameters in the cost effectiveness of interventions in MICs. It is therefore necessary to strike a balance between maximum population health and maintaining affordability in these countries