But does it work? Exploring the techno-scientific and socio-political dynamics of Health Technology Assessment and Pharmaceutical Regulation

适当使用药物可对公众健康作出重要贡献。然而，在国家医疗保健支出受到限制的情况下，药品价格昂贵，成本不断增加。因此，各国政府和卫生保健当局必须决定如何最好地分配有限的资源，为社会实现最大的健康效益。这包括决定新药是否应该报销。为了向这一决策过程提供信息，各国政府越来越有可能建立组织来进行不同形式的卫生技术评估。卫生技术评估机构的目的是审查和评估现有证据，并就新药与现有疗法相比的临床和成本效益向政策制定者提供建议。在英国，这一职能由国家健康和临床卓越研究所（NICE）承担。创新药品只有在经过国家监管机构的政府科学家或中央欧洲药品管理局（EMA）的评估后，才能进入英国和欧盟其他成员国的市场。这些组织根据技术科学证据（包括临床试验数据）确定药物是否安全有效。这些机构的监管要求在很大程度上决定了新药上市时可获得的证据基础的性质和质量。然后，像NICE这样的HTA机构将评估这些证据，以确定新药相对于其他治疗方法是否具有足够的治疗价值，以证明报销是合理的（即它们是否具有成本效益）。越来越多的利益相关者声称，新药上市时的科学证据基础未能提供关于临床有效性的可靠数据，因此不足以用于HTA目的。这反过来又可能导致决策失误，增加患者接受次优药物治疗的风险，稀缺资源被误导，以及错过其他治疗方法提供的最大化健康收益的机会。拟议的研究旨在解决制药和公共卫生政策面临的这一重大新挑战，并通过提供关于管理新药开发和上市许可的技术科学监管标准如何帮助和/或限制HTA组织评估临床和成本的能力的社会科学调查，为当前关于这些问题的政策辩论提供信息。这些药物的有效性及其对公共卫生的贡献。本研究通过对NICE技术评估（TAs）中治疗肥胖和II型糖尿病药物的实证研究，旨在：（1）确定这些药物TAs中证据缺口和不确定性的性质和程度;（2）调查HTA技术评估人员和其他利益相关者对科学不确定性的反应方式，并探讨这些反应的社会政治动力学;以及（3）绘制和评估英国和欧盟近期倡议的前景，以使卫生技术评估、药物开发和监管评估与患者和公共卫生需求保持一致。该研究计划将在地理上位于英国和超国家欧盟，并将利用文献和访谈研究方法。此外，在卫生经济学，临床流行病学和卫生政策决策的方法，特别是在专业的定量技术培训期间进行的培训，将使研究人员能够理解和有效地参与卫生技术评估的高度技术性方面。

Appropriate use of medicines can make an important contribution to public health. Yet pharmaceutical drugs are expensive and costs are increasing at a time when national health care spending is constrained. Consequently, national governments and health care authorities must decide how best to allocate limited resources to achieve the greatest health benefits for society. This includes decisions on whether new drugs should be reimbursed. To inform this decision-making process, national governments are increasingly likely to establish organisations to undertake different forms of health technology assessment (HTA). The purpose of HTA bodies is to review and evaluate the available evidence, and to advise policy-makers on the clinical and cost-effectiveness of new medicines compared to available therapies. In the UK this function is undertaken by the National Institute for Health and Clinical Excellence (NICE). Innovative pharmaceutical products can enter the market in the UK and other member states of the European Union only after they have been assessed by government scientists in national regulatory agencies or by the centralised European Medicines Agency (EMA). These organisations determine whether the drugs are safe and efficacious based on techno-scientific evidence, including data from clinical trials. The regulatory requirements of these agencies significantly determine the nature and quality of the evidence base for new drugs available at the time of a drug's launch onto the market. HTA bodies like NICE will then assess this evidence to determine whether new drugs offer sufficient therapeutic value relative to other treatments to justify reimbursement (that is, whether they are cost effective). Increasingly, stakeholders have claimed that the scientific evidence base for new drugs at time of market launch fails to provide robust data on clinical effectiveness and is consequently inadequate for HTA purposes. This in turn may lead to flawed decision-making, increasing the risk of patients being treated with sub-optimal drugs, scarce resources being misdirected, and missed opportunities for maximising health gains offered by other treatments. The proposed research aims to address this major emerging challenge for pharmaceutical and public health policy and to inform current policy debates on these issues by providing a social scientific investigation of how techno-scientific regulatory standards governing the development and marketing authorisation of new drugs assist and/or limit the ability of HTA organisations to evaluate the clinical and cost-effectiveness of such drugs and their contribution to public health. Through empirical investigations of NICE technology appraisals (TAs) of drugs to treat obesity and type II diabetes the research aims: 1) to determine the nature and extent of evidence gaps and uncertainty in TAs for these drugs; (2) to investigate the ways in which HTA technical assessors and other interested stakeholders respond to scientific uncertainties, and to explore the socio-political dynamics of those responses; and (3) to map and assess the prospects of recent initiatives within the UK and EU to align HTA, drug development and regulatory assessment with patient and public health needs. The research programme will be geographically located in the UK and the supranational EU, and will draw on documentary and interview research methods. In addition, training undertaken during the award in the methodologies of health economics, clinical epidemiology and health policy decision-making - and in particular training in specialist quantitative techniques - will enable the researcher to understand and engage effectively with the highly technical aspects of health technology appraisals.