Padmasawitri T I Armina, Saragih Sarah Maria, Frederix Gerardus W, Klungel Olaf, Hövels Anke M
Division of Pharmacoepidemiology and Clinical Pharmacology, Utrecht Institute of Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
Pharmacology and Clinical Pharmacy Research Group, School of Pharmacy, Institut Teknologi Bandung, Bandung, Indonesia.
Pharmacoecon Open. 2020 Jun;4(2):223-233. doi: 10.1007/s41669-019-0162-z.
Limited evidence for the implementation of new health technologies in low- and middle-income countries (LMICs) may lead to uncertainties in economic evaluations and cause the evaluations to produce inaccurate information for decision making. We performed a systematic review of economic evaluations on implementing new short-course regimens (SCR) for drug-sensitive and drug-resistant tuberculosis (TB), to explore how uncertainties due to the limited evidence in the studies were dealt with and to identify useful information for decision making from these studies.
We searched in electronic databases PubMed, EMBASE, NHSEED, and CEA registry for economic evaluations addressing the implementation of new anti-TB SCRs in LMICs published until September 2018. We included studies addressing both the cost and outcomes of implementing a new regimen for drug-sensitive and drug-resistant TB with a shorter treatment duration than the currently used regimens. The quality of the included studies was assessed using The Consensus Health Economic Criteria checklist. We extracted information from the included studies on uncertainties and how they were managed. The management of uncertainties was compared with approaches used in early health technology assessments (HTAs), including sensitivity analyses and pragmatic scenario analyses. We extracted information that could be useful for decision making such as cost-effectiveness conclusions, and barriers to implementing the intervention.
Four of the 322 studies found in the search met the eligibility criteria. Three studies were model-based studies that investigated the cost effectiveness of a new first-line SCR. One study was an empirical study investigating the cost effectiveness of new regimens for drug-resistant TB. The model-based studies addressed uncertainties due to limited evidence through various sensitivity analyses as in early HTAs. They performed a deterministic sensitivity analysis and found the main drivers of the cost-effectiveness outcomes, that is, the rate of treatment default and treatment delivery costs. Additionally, two of the model-based studies performed a pragmatic scenario analysis and found a potential barrier to implementing the new first-line SCR, that is, a weak health system with a low TB care utilization rate. The empirical study only performed a few scenario analyses with different regimen prices and volumes of TB care utilization. Therefore, the study could only provide information on the main cost drivers.
Using an approach similar to that used in early HTAs, where uncertainties due to the limited evidence are rigorously explored upfront, the economic evaluations could inform not only the decision to implement the intervention but also how to manage risks and implementation barriers.
在低收入和中等收入国家（LMICs）实施新卫生技术的证据有限，这可能导致经济评估存在不确定性，并使评估产生不准确的决策信息。我们对实施药物敏感和耐药结核病新短程治疗方案（SCR）的经济评估进行了系统综述，以探讨如何处理研究中证据有限所导致的不确定性，并从这些研究中识别出对决策有用的信息。
我们在电子数据库PubMed、EMBASE、NHSEED和CEA注册库中检索了截至2018年9月发表的关于在LMICs实施新抗结核SCR的经济评估。我们纳入了涉及实施药物敏感和耐药结核病新治疗方案的成本和结果且治疗持续时间比当前使用方案短的研究。使用《共识卫生经济标准清单》评估纳入研究的质量。我们从纳入研究中提取关于不确定性及其管理方式的信息。将不确定性的管理与早期卫生技术评估（HTA）中使用的方法进行比较，包括敏感性分析和务实情景分析。我们提取了可能对决策有用的信息，如成本效益结论以及实施干预的障碍。
在检索到的322项研究中，有4项符合纳入标准。3项研究是基于模型的研究，调查了新一线SCR的成本效益。1项研究是实证研究，调查了耐药结核病新治疗方案的成本效益。基于模型的研究通过与早期HTA中类似的各种敏感性分析来处理证据有限导致的不确定性。他们进行了确定性敏感性分析，找出了成本效益结果的主要驱动因素，即治疗违约率和治疗提供成本。此外，两项基于模型的研究进行了务实情景分析，发现实施新一线SCR的一个潜在障碍，即卫生系统薄弱且结核病护理利用率低。实证研究仅对不同治疗方案价格和结核病护理利用量进行了几次情景分析。因此，该研究只能提供关于主要成本驱动因素的信息。
采用与早期HTA类似的方法，即预先严格探索证据有限所导致的不确定性，经济评估不仅可以为实施干预的决策提供信息，还可以为如何管理风险和实施障碍提供信息。
