Valentine W J, Bottomley J M, Palmer A J, Brändle M, Foos V, Williams R, Dormandy J A, Yates J, Tan M H, Massi-Benedetti M
Center for Outcomes Research, A Unit of IMS Health, Allschwil, Switzerland.
Diabet Med. 2007 Sep;24(9):982-1002. doi: 10.1111/j.1464-5491.2007.02188.x. Epub 2007 Jun 25.
To determine the cost-effectiveness of adding pioglitazone to existing treatment regimens in patients with Type 2 diabetes with a history of macrovascular disease who are at high risk of further cardiovascular events.
We conducted two analyses. A within-trial cost-effectiveness analysis (CEA) based on data from the PROspective pioglitAzone Clinical Trial In macroVascular Events (PROactive) Study was performed to estimate the impact of additional pioglitazone treatment on life expectancy, quality-adjusted life expectancy (QALE) and macrovascular events. PROactive data was then used as a basis for a lifetime modelling analysis using a modified version of the validated CORE diabetes model that simulated the same outcomes over a 35-year time horizon. We accounted for direct medical costs from a health-care payer perspective and related these to the clinical outcomes from the study. Costs and benefits were discounted at 3.5% per annum and extensive sensitivity analyses were performed to account for uncertainty in input parameters.
(i) Within-trial CEA: compared with placebo, pioglitazone was associated with improved life expectancy (undiscounted 0.0109 years), increased QALE [0.0190 quality-adjusted life years (QALYs)] and slightly higher costs ( pounds 102 per patient). After a mean treatment period of 3 years, the incremental cost-effectiveness ratio (ICER) of pioglitazone vs. placebo was pounds 5396 per QALY gained. The ICERs were relatively insensitive to cost and utility values and were most sensitive to event rates in the pioglitazone arm. (ii) Long-term CEA: pioglitazone was associated with improvements in clinical outcomes based on model projections beyond the PROactive Study. Patients treated with pioglitazone could expect improved life expectancy (undiscounted 0.406 years), increased QALE (0.152 QALYs) and higher costs of care ( pounds 619 per patient) compared with those on existing treatment alone. The base case analysis indicated that the ICER of pioglitazone vs. placebo was pounds 4060 per QALY gained. The cost-effectiveness acceptability curve showed there was an 84.3% likelihood that pioglitazone would be considered cost-effective in the UK using a willingness-to-pay threshold of pounds 30 000 per QALY gained. These long-term results were most sensitive to variation in the time horizon, the duration of cardiovascular benefit of pioglitazone, and changes in mortality rates.
The addition of pioglitazone to existing therapy in patients with Type 2 diabetes at high risk of further cardiovascular events is cost-effective and represents good value for money by currently accepted standards in the UK.
确定在有大血管疾病病史且有进一步心血管事件高风险的2型糖尿病患者中，在现有治疗方案基础上加用吡格列酮的成本效益。
我们进行了两项分析。基于前瞻性吡格列酮大血管事件临床试验（PROactive）研究的数据进行了试验内成本效益分析（CEA），以评估加用吡格列酮治疗对预期寿命、质量调整预期寿命（QALE）和大血管事件的影响。然后，将PROactive数据用作使用经过验证的CORE糖尿病模型的修改版本进行终生建模分析的基础，该模型在35年的时间范围内模拟相同的结果。我们从医疗保健支付者的角度考虑直接医疗成本，并将其与研究的临床结果相关联。成本和效益按每年3.5%进行贴现，并进行了广泛的敏感性分析以考虑输入参数的不确定性。
（i）试验内CEA：与安慰剂相比，吡格列酮与预期寿命改善（未贴现0.0109年）、QALE增加[0.0190质量调整生命年（QALY）]和成本略高（每位患者102英镑）相关。在平均3年的治疗期后，吡格列酮与安慰剂相比的增量成本效益比（ICER）为每获得一个QALY 5396英镑。ICER对成本和效用值相对不敏感，对吡格列酮组的事件发生率最敏感。（ii）长期CEA：基于PROactive研究之外的模型预测，吡格列酮与临床结果改善相关。与仅接受现有治疗的患者相比，接受吡格列酮治疗的患者预期寿命改善（未贴现0.406年）、QALE增加（0.152 QALY）和护理成本更高（每位患者619英镑）。基础病例分析表明，吡格列酮与安慰剂相比的ICER为每获得一个QALY 4060英镑。成本效益可接受性曲线显示，使用每获得一个QALY支付意愿阈值为30000英镑，吡格列酮在英国被认为具有成本效益的可能性为84.3%。这些长期结果对时间范围的变化、吡格列酮心血管益处的持续时间以及死亡率的变化最为敏感。
在有进一步心血管事件高风险的2型糖尿病患者中，在现有治疗基础上加用吡格列酮具有成本效益，按照英国目前公认的标准，物有所值。
