R&D policy, agency costs and innovation in personalized medicine

The Orphan Drug Act (ODA) was designed to spur the development of drugs for rare diseases. In principle, its design also incentivizes pharmaceutical firms to develop drugs for “rare” subdivisions of more prevalent diseases. I find that in response to this incentive, firms develop drugs for ODA-qualifying subdivisions of non-rare diseases. The impact in these tailored drug markets represents half of the total R&D response to the ODA. I also find that 10-percent of the innovation in subdivided disease drugs induced by the ODA would have been conducted without the policy. While modest in size, this inefficiency suggests that agency problems should be considered when designing innovation policy.     

Market incentives and pharmaceutical innovation

I study the impact of the Orphan Drug Act (ODA), which established tax incentives for rare disease drug development. I examine the flow of new clinical drug trials for a large set of rare diseases. Among more prevalent rare diseases, the ODA led to a significant and sustained increase in new trials. The impact for less prevalent rare diseases was limited to an increase in the stock of drugs. Tax credits can stimulate R & D; yet because they leave revenue margins unaffected, tax credits appear to have a more limited impact on private innovation in markets with smaller revenue potential.     

三级医院创新发展的探讨与实践

创新型管理必将取代常规型管理。南京市第一医院不断加强管理创新:一是创新发展思路,形成特色办院模式;二是创新管理理念,提升医疗服务水平;三是创新激励机制,推进人才学科建设;四是创新内涵建设,强化医疗安全管理。通过以上措施,提升了医院资源的整体运行功效,取得了良好的经济和社会效益。     

新型农村合作医疗中医药补偿政策实施效果制约因素分析

目的：了解新农合中医药补偿政策对中医药服务利用的影响,分析其制约因素。方法：采取定性与定量相结合的方法,分析政策实施对中医药服务利用及参合患者受益的影响等情况。结果：中医药补偿政策在一定程度上提高了中医药服务的利用,减轻患者负担,但受到多重因素制约,主要包括：中医药服务项目少、补偿比例提高幅度有限、缺乏对医疗机构的激励机制等。结论与建议：要发挥中医药补偿政策实施效果,既要提高中医药服务的政策补偿比例,又要提高中医药服务的可及性和可得性,如推广符合中医药使用特点的补偿政策,探索对医疗机构应用中医药的激励机制等。     

A three-stage clinical trial design for rare disorders

Many clinical trials of uncommon diseases are underpowered because of the difficulty of recruiting adequate numbers of subjects. We propose a clinical trial design with improved statistical power compared to the traditional randomized trial for use in clinical trials of rare diseases. The three-stage clinical trial design consists of an initial randomized placebo-controlled stage, a randomized withdrawal stage for subjects who responded, and a third randomized stage for placebo non-responders who subsequently respond to treatment. Test level and power were assessed by computer-intensive exact calculations. The three-stage clinical trial design was found to be consistently superior to the traditional randomized trial design in all cases examined, with sample sizes typically reduced by 20 per cent to 30 per cent while maintaining comparable power. When a treatment clearly superior to placebo was considered, our design reached a power of 75 per cent with a sample of 21 patients compared with the 52 needed to attain this power when only a randomized controlled trial was used. In situations where patient numbers are limited, a three-stage clinical trial design may be a more powerful design than the traditional randomized trial for detecting clinical benefits.     

Mitigating Nonattendance Using Clinic-Resourced Incentives Can Be Mutually Beneficial: A Contingency Management-Inspired Partially Observable Markov Decision Process Model

ObjectivesNonattendance of appointments in outpatient clinics results in many adverse effects including inefficient use of valuable resources, wasted capacity, increased delays, and gaps in patient care. This research presents a modeling framework for designing positive incentives aimed at decreasing patient nonattendance.MethodsWe develop a partially observable Markov decision process (POMDP) model to identify optimal adaptive reinforcement schedules with which financial incentives are disbursed. The POMDP model is conceptually motivated based on contingency management evidence and practices. We compare the expected net profit and trade-offs for a clinic using data from the literature for a base case and the optimal positive incentive design resulting from the POMDP model. To accommodate a less technical audience, we summarize guidelines for reinforcement schedules from a simplified Markov decision process model.ResultsThe results of the POMDP model show that a clinic can increase its net profit per recurrent patient while simultaneously increasing patient attendance. An increase in net profit of 6.10% was observed compared with a policy with no positive incentive implemented. Underlying this net profit increase is a favorable trade-off for a clinic in investing in a targeted contingency management-based positive incentive structure and an increase in patient attendance rates.ConclusionsThrough a strategic positive incentive design, the POMDP model results show that principles from contingency management can support decreasing nonattendance rates and improving outpatient clinic efficiency of its appointment capacity, and improved clinic efficiency can offset the costs of contingency management.     

罕见病政策国际经验及对我国的启示

本研究系统梳理了近年来有关罕见病政策和服务的文献及文件,归纳了美国、日本、欧盟等国家和地区罕见病方面的立法及相关法规、管理制度、激励措施、保障政策等方面的政策设计和实践经验。目前,发达国家和地区对罕见病制定了较为系统的政策和法规,罕见病药品的研发和医疗保障等在法律层面得到了不同程度的保障。我国尚未形成针对罕见病的系统性的政策体系,结合中国实际,我国应从逐步建立系统的罕见病政策法规、拓宽筹资渠道、鼓励孤儿药研发,扩大新生儿筛查疾病的范围等方面着手,推动我国罕见病政策发展。     

Competition and the impact of online hospital report cards

Information on the quality of healthcare gives providers an incentive to improve care, and this incentive should be stronger in more competitive markets. We examine this hypothesis by studying Pennsylvanian hospitals during the years 1995–2004 to see whether those hospitals located in more competitive markets increased the quality of the care provided to Medicare patients after report cards rating the quality of their Coronary Artery Bypass Graft programs went online in 1998. We find that after the report cards went online, hospitals in more competitive markets used more resources per patient, and achieved lower mortality among more severely ill patients.     

Drug insurance and the strategic behavior of drug manufacturers: Evergreening and generic entry after Medicare Part D

For branded drug manufacturers, maintaining market power by managing product lifecycle – evergreening – is an important tool to navigate pharmaceutical markets. For generic manufacturers, the key decision is to enter the market at all. Expansion of drug insurance may lead firms to change their behavior because of increased demand-side market power – due to consolidation of buyers into a small number of insurers – and because of increased drug utilization. We analyze manufacturer responses to such changes through the introduction of Medicare Part D, which expanded drug coverage among seniors, in a difference-in-differences design comparing drugs used frequently by those over age 65 to those used infrequently by this population. The results show that Part D reduced generic entry, and suggest that it increased evergreening. Furthermore, while these effects are associated with higher drug prices, they are more than offset by the consolidation of demand, reducing prices overall.     

Encouraging the development of new vaccines

Investment in the development of new vaccines is suboptimal. Changing this situation requires a creative blend of "push" and "pull" strategies. One successful policy model is the Orphan Drug Act, whose key features include large research and development (R and D) tax credits as well as Food and Drug Administration (FDA) counseling and priority review. Such supply-side R and D incentive provisions can be combined with demand-side mandates and vouchers to encourage development of new vaccines. Guaranteed-purchase funds and other pull mechanisms are useful supplementary incentives in the cases of vaccines for bioterrorism and neglected diseases of poverty.     

长期护理保险制度政策评价:基于PMC指数模型

目的:以首批9个试点城市的长期护理保险政策作为研究对象进行政策分析,探索上海等9个城市的政策特色,为完善长期护理保险政策提供建议。方法:基于PMC指数模型对9个试点城市的政策进行评价,结合气泡图分析政策制定的影响因素。结果:9个城市的PMC得分均值为6.922分,政策制定总体尚可,但政策时效及激励约束方面略显不足;政策制定与千人床位数、人均医疗保健支出构成比成正相关。结论:长期护理保险政策规划有待完善,可从政策体系完善、激励机制建立、医护资源配置等方面着手。     

美国腹膜透析激励政策的经验及对我国的启示

目的：梳理美国腹膜透析激励政策（peritoneal dialysis favored policy,PD-Favored）的实施背景和发展历程,为我国居家腹膜透析治疗模式的推广提供经验借鉴。方法：基于文献分析法,系统总结美国实施PD-Favored政策的主要措施和特点。结果：美国PD-Favored政策主要通过以下四类措施影响供需双方行为：建立前瞻性捆绑支付模式以引导医疗机构提供腹膜透析;通过按人头付费提高医生提供腹膜透析的积极性;通过设立等待期、限制频繁透析和实施治疗选择教育计划引导患者采用腹膜透析;实施质量激励计划以及推动腹膜透析治疗技术创新。结论：美国PD-Favored政策通过医保支付、政府调控等手段影响供需双方的行为,有效促进腹膜透析的普及。未来我国在推广居家腹膜透析模式的过程中应重在完善医保支付政策,采用多学科方法对患者进行透析前教育,并加强医护人员培训,在确定技术规范基础上确定中国腹膜透析质量管理目标。     

The effects of a national policy to reduce c-sections in Brazil

In this paper, we assess the effects of a national policy implemented in Brazil to avoid unnecessary cesareans. The policy has a supply-side component that prohibits elective c-sections before the 39th gestational week and a demand-side awareness component. Since the policy is not binding for cases with a strong medical c-section indication, we use births of breech- and transverse-positioned babies as a counterfactual for births of cephalic-positioned babies in a difference-in-differences framework. Our results reveal that the policy decreases the rate of c-sections by 1.6 percentage point, and slightly increases gestational time, birthweight, and first-minute APGAR scores. There is evidence that policy effectiveness is driven by its demand-side component.     

The effect of competition on nursing home expenditures under prospective reimbursement.

The for-profit nursing home's incentive to minimize costs has been maligned as a major cause of the quality problems that have traditionally plagued the nursing home care industry. Yet, profit-maximizing firms in other industries are able to produce products of adequate quality. In most other industries, however, firms are constrained from reducing costs to the point where quality suffers by the threat of losing business to competing firms. In the nursing home industry, competition for patients often does not exist because of the shortage of nursing home beds. As a result, one would expect that nursing homes located in areas where there is excess demand would spend less on patient care than homes located where the bed supply is relatively abundant. This hypothesis is tested using Wisconsin data from 1983. It is found that, in counties with relatively tight bed supplies, an additional empty bed in all the homes in the county will force each home to increase expenditures by $.62 per day for each patient in the home. Overall, the average nursing home located in underbedded markets would spend $5.12 more per patient day or about $240,000 more annually (in 1983 dollars) if it were located in a market where it was forced to compete for patients. The implications for public policy are discussed.     

Don't overlook disease management programs for low-incidence, high-cost diseases to improve your bottom line.

Comprehensive coordinated care management for low-incident, high-cost diseases, like chronic renal failure, can provide a great opportunity for health plans to add immediate and significant profit to their bottom line. The resultant benefits of improved operations, improved clinical outcomes and increased patient satisfaction add further incentive for health plans to take action to implement outsourced disease management for this condition.     

Motivated health risk denial and preventative health care investments

People deny health risks, invest too little in disease prevention, and are highly sensitive to the price of preventative health care, especially in developing countries. Moreover, private sector R&D spending on developing-country diseases is almost non-existent. To explain these empirical observations, I propose a model of motivated belief formation, in which an agent's decision to engage in health risk denial balances the psychological benefits of reduced anxiety with the physical cost of underprevention. I use the model to study firms’ price-setting behavior and incentive to innovate. I also show that tax-funded prevention subsidies are welfare enhancing.     

社区卫生服务改革的模式演进与版本升级研究——以上海市长宁区为例

任何改革在基层的实施都需要转换成一系列的制度安排或微观行为激励机制,不仅确保基层行动者之间的行为策略激励相容,还要确保基层行动者的行为模式与改革政策目标方向相一致。从上海市长宁区社区卫生服务改革的实践来看,其大致经过了四个阶段:标准化建设与组织确立、服务模式与机制改革、激励设计与内涵建设、平台打造与效能提升。长宁医改在不断深化政策试验与制度创新的过程中逐步化解新医改政策实施过程所产生的各类新问题及其与环境的相容性问题,以实现医改政策的制度化并为利益相关者的福利改进提供稳定的行为预期,从而不断降低基层行动者对政策实施的潜在抵制行为,逐步地将各类行动者的行为激励引导到与政策目标相一致的方向。     

An empirical analysis of the multimarket contact theory in pharmaceutical markets

Multimarket contact theory predicts that firms will optimally reduce prices in markets where collusive prices are sustainable and allocate the slack of the corresponding incentive compatibility to increase prices in markets where collusion is not sustainable. Binding price caps in collusive markets will have different effects over the multimarket contact mechanism depending on the severity of the cap. Setting a price cap close to the unregulated case will increase the size of the redistribution of market power whereas stronger regulation will even reduce prices in unregulated markets. Therefore, price regulations aiming at capping prices in a specific market will also affect markets that are not subject to specific mandatory price regulations. We find evidence of the theory predictions using information for nine OECD countries for pharmaceutical markets. Unregulated US markets are shown to respond to the redistribution effect; Canadian markets, known to be subject to soft price regulations, with respect to the former, are shown to be consistent with a stronger redistribution effect. EU markets and Japan are either consistent with the effect of a medium regulation or strong regulation. In this last case multimarket contact cannot explain prices, and these are expected to be lower compared to the unregulated benchmark.     

基于结构变动度的药品零差价门诊医疗费用变化实证研究

[目的]分析2009-2016年宁夏12家县级综合性公立医院实施药品零差价政策门诊费用的结构变化情况,为完善药品零差价政策提供参考。[方法]收集12家县级医院2009-2016年政策实施前后门诊医疗费用资料,采用结构变动度法分析费用的结构变化情况。[结果]次均药品费占比由政策实施前的47.10%上升至政策实施后的48.41%;政策实施后,西药费、检查费比重有所下降,化验费、中成药和中草药比重上升,药品费用比重出现上升趋势。[结论]政策实施后,医疗费用增速变缓,药占比下降,但门诊次均费用呈逐年上升趋势,提示药品零差价政策对门诊患者医疗费用的控制作用不明显。建议进一步扩大医疗服务项目调整范围,提升医疗服务项目价格调整的精准度。