The Economics of Gene Therapy and of Pharmacogenetics

This paper focuses on the economic issues arising from two uses of genomics: 1) the development of gene therapy; 2) and use of pharmacogenetics to identify a patient's genotype before treatment to exclude those who will not benefit or who may be harmed. We conclude that private-sector investment aimed at developing gene therapy for monogenic diseases is likely to be socially suboptimal. Short-term administration regimens yielding long-term therapeutic benefits are likely to meet payer resistance to large "one-off" costs because of budget constraints or, in competitive systems, concerns that the savings would accrue to future insurers or would attract high-cost patients. For some monogenic diseases, patient numbers may be too small to support commercial development without changes to orphan drug legislation or payer willingness to accept higher cost-effectiveness thresholds. In the case of pharmacogenetics, we conclude that it can often be socially optimal to test before treatment, particularly if the proportion of nonresponders is high, if there is a potential for serious adverse reactions, or if the test is inexpensive. Genetic testing that fragments the patient population could reduce incentives for R&D unless prices are adjusted to reflect the higher expected benefits of targeted treatment per patient. Even in situations where prices are adjusted, patient populations may be too small to make commercial development viable. This problem with small numbers is analogous to that associated with gene therapy for monogenic diseases and may require similar remedies if society values developing treatments for these diseases.     

宫腔镜检查50例功血患者热球仪治疗后的效果分析

目的:探讨宫腔镜检查功血患者热球仪治疗后的效果。方法:对我院2007年至2008年2月以来采用热球子宫内膜治疗仪治疗功血的50例患者的诊治情况进行回顾性研究,分析术后6个月及12个月的宫腔镜检查情况。结果:50例患者术后随访有30例治愈(闭经6个月以上);20例月经量较前明显减少,总有效率达100%,治疗前后效果差异具有显著性(P<0.05)。且50例患者均无出现明显的并发症。术后6个月宫腔镜检查:宫腔粘连15例,边分离边进镜。镜下见:白色伴有膜状物的内膜10例,粉色伴有膜状物内膜13例,淡黄色伴有膜状物内膜12例。术后12个月宫腔镜检查:宫腔粘连8例,边分离边进镜。镜下:白色伴有膜状物的内膜25例,粉色伴有膜状物内膜10例,淡黄色伴有膜状物内膜15例,行诊断性刮宫2例,病理回报:子宫内膜轻度不典型增生1例,单纯性子宫内膜增生1例,所有患者宫腔镜检查双侧宫角可见正常子宫内膜。结论:热球子宫内膜剥离治疗对功血引起的月经过多具有良好的临床疗效,且安全、方便,但术后需定期行宫腔镜检查预防残余内膜病变是非常重要的,本研究提示术后一年行宫腔镜检查是十分必要的。     

特发性椎管内硬膜外脂肪增多症的诊断与治疗

目的：探讨特发性椎管内硬膜外脂肪增多症的诊断和治疗。方法：对5例特发性椎管内硬膜外脂肪增多症的病因、临床特点及影像学资料进行分析。经后正中切口入路,手术切除脂肪肿物。结果：5例中,术后症状完全缓解2例,好转2例,1例无改善。结论：MRI和CT有助于确诊本病,有脊髓压迫症状者应行手术治疗。     

阻塞性睡眠呼吸暂停低通气综合征患者主观评估与客观检查的相关性研究

目的 探讨阻塞性睡眠呼吸暂停低通气综合征(obstructive sleep apnea hypopnea syndrome, OSAHS)患者主观评估和客观检查的相关性.方法 分别采用视觉模拟量表(visual analogue scale, VAS)、Epworth嗜睡量表(Epworth sleepiness scale, ESS)和Calgary睡眠呼吸暂停生活质量指数量表(sleep apnea quality of life index, SAQLI)评估200例OSAHS患者的打鼾、嗜睡症状和生活质量,分析它们与多导睡眠仪(polysomno-graphy,PSG)检查间的相关性.结果 VAS、ESS与PSG间有一定的相关性,但不同呼吸暂停低通气指数(apnea hypopnea index, AHI)和最低血氧饱和度(lowest oxygen saturation, LSaO2)组间只有重度组VAS和ESS值与其他各组间差异有统计学意义.SAQLI同PSG间没有良好的相关性,而且不同AHI和LSaO2组间只有重度低血氧组的症状维度分值与其他各组间差异有统计学意义.结论 评估OSAHS患者须结合主观评估和客观检查.VAS和ESS在评估病情严重程度上的价值不大.(中国眼耳鼻喉科杂志,2009,9:145-147)     

阻塞性睡眠呼吸暂停低通气综合征患者健康相关生命质量与多导睡眠监测结果相关性

目的 评估阻塞性睡眠呼吸暂停低通气综合征(OSAHS)患者健康相关生命质量（HRQoL）,并研究其与多导睡眠监测（PSG）结果、过度白天嗜睡的相关性,寻找影响OSAHS患者HRQoL的因素。方法 对64例OSAHS患者和50例正常对照使用健康结局问卷（SF-36）和Epworth嗜睡问卷（ESS）评估并比较其HRQoL。研究OSAHS患者SF-36评分与PSG监测结果、ESS评分和生理指标之间的相关性,分析造成其HRQoL变化的因素。结果 OSAHS患者与正常对照相比, SF-36中4个维度及总分显著下降,整体处于白天过度嗜睡状态;OSAHS患者ESS评分与 SF-36中6个维度及总分呈显著负相关;SF-36总分、ESS评分与觉醒指数呈显著负相关;SF-36总分与血氧低于90％占睡眠比例（SaO2　90％）呈显著负相关;ESS是OSAHS患者HRQoL的独立预测因素,能解释SF-36总分变异的26.9％。结论 OSAHS患者健康相关生命质量有明显的损害。过度白天嗜睡、夜间严重持续的低氧血症和睡眠结构的破坏可能是引起HRQoL损害的主要原因。     

The role of economic evaluation in the pricing and reimbursement of medicines

In most countries, governments or health insurers have taken initiatives to influence the price and utilization of medicines. One stated objective of these schemes is to encourage efficiency, or cost-effectiveness. In principle, economic evaluation should to be relevant to decisions about the pricing and reimbursement of health technologies, since it offers a way of estimating the additional value to society of a new intervention (e.g. medicine) relative to current therapy. However, the application of economic evaluation in drug pricing and reimbursement schemes is variable. Therefore, this paper reviews the actual and potential role of economic evaluation in different drug pricing and reimbursement schemes, such as 'free pricing' systems (United Kingdom, United States), two-stage administered systems (France), reference pricing systems (Germany, Netherlands, Sweden) and economic evaluation systems (Australia, Canada). It is concluded that, other than in the case of Australia and Canada, the potential role of economic evaluation could be greatly developed, especially in the case of new medicines, for which there is no close substitute. Comments are also given on the practical problems of using this approach. However, it is noted that economic evaluation alone cannot set a price for a medicine, since a decision has to be made about the proportion of added value going to society and the proportion going to the pharmaceutical company as a reward for innovation.     

探索科学合理的医用耗材定价机制简

介绍了我国医用耗材市场主要存在的问题和发达国家的定价机制,阐述了新形势下建立科学合理的医用耗材定价机制的必要性,同时探讨我国该如何建立科学有效的医用耗材定价机制,为决策者下一步制定定价机制提供参考依据。     

基于价值定价的多适应证药品医保支付国际经验及启示

目的:为完善我国多适应证药品基于价值定价的医保支付提供参考.方法:梳理多适应证药品价值定价的理论基础和实现流程,分析法国、德国、英国、意大利和瑞典的价值评估指标和基于价值定价的医保支付策略,为我国多适应证药品医保支付提出建议.结果与结论:价值定价的实现首先需要制定一个价值框架以定义、测量、整合价值,再通过建立模型将总价...