Kalipso Chalkidou

How to formulate research recommendations

“More research is needed” is a conclusion that fits most systematic reviews. But authors need to be more specific about what exactly is required

The role for pragmatic randomized controlled trials (pRCTs) in comparative effectiveness research

There is a growing appreciation that our current approach to clinical research leaves important gaps in evidence from the perspective of patients, clinicians, and payers wishing to make evidence-based clinical and health policy decisions. This has been a major driver in the rapid increase in interest in comparative effectiveness research (CER), which aims to compare the benefits, risks, and sometimes costs of alternative health-care interventions in ‘the real world’. While a broad range of experimental and nonexperimental methods will be used in conducting CER studies, many important questions are likely to require experimental approaches – that is, randomized controlled trials (RCTs). Concerns about the generalizability, feasibility, and cost of RCTs have been frequently articulated in CER method discussions. Pragmatic RCTs (or ‘pRCTs’) are intended to maintain the internal validity of RCTs while being designed and implemented in ways that would better address the demand for evidence about real-world risks and benefits for informing clinical and health policy decisions. While the level of interest and activity in conducting pRCTs is increasing, many challenges remain for their routine use. This article discusses those challenges and offers some potential ways forward.

Evidence for overuse of medical services around the world

Overuse, which is defined as the provision of medical services that are more likely to cause harm than good, is a pervasive problem. Direct measurement of overuse through documentation of delivery of inappropriate services is challenging given the difficulty of defining appropriate care for patients with individual preferences and needs; overuse can also be measured indirectly through examination of unwarranted geographical variations in prevalence of procedures and care intensity. Despite the challenges, the high prevalence of overuse is well documented in high-income countries across a wide range of services and is increasingly recognised in low-income countries. Overuse of unneeded services can harm patients physically and psychologically, and can harm health systems by wasting resources and deflecting investments in both public health and social spending, which is known to contribute to health. Although harms from overuse have not been well quantified and trends have not been well described, overuse is likely to be increasing worldwide.

Evidence-informed frameworks for cost-effective cancer care and prevention in low, middle, and high-income countries

Evidence-informed frameworks for cost-effective cancer prevention and management are essential for delivering equitable outcomes and tackling the growing burden of cancer in all resource settings. Evidence can help address the demand side pressures (ie, pressures exerted by people who need care) faced by economies with high, middle, and low incomes, particularly in the context of transitioning towards (or sustaining) universal health-care coverage. Strong systems, as opposed to technology-based solutions, can drive the development and implementation of evidence-informed frameworks for prevention and management of cancer in an equitable and affordable way. For this to succeed, different stakeholders-including national governments, global donors, the commercial sector, and service delivery institutions-must work together to address the growing burden of cancer across economies of low, middle, and high income.

Priority-Setting Institutions in Health: Recommendations from a Center for Global Development Working Group

The rationing problem is common to all health systems-the challenge of managing finite resources to address unlimited demand for services. In most low- and middle-income countries, rationing occurs as an ad hoc, haphazard series of nontransparent choices that reflect the competing interests of governments, donors, and other stakeholders. Yet in a growing number of countries, more explicit processes, with strengths and limitations, are under development that merit better support. Against this background, the purpose of the Center for Global Development Working Group, which is to examine how priorities are set currently, and to propose institutional arrangements that promote country ownership and improve health outcomes by more systematically managing this complex process of politics and economics, is discussed. Current global and national priority-setting practices in low- and middle-income countries, the potential for strengthened national institutions, and increased global support are reviewed. Recommendations for action are provided.

Access with evidence development in the UK: past experience, current initiatives and future potential.

Access with evidence development (AED) describes the general approach of linking some form of access to the healthcare market with the generation of additional evidence relating to the value of the healthcare intervention under evaluation, with an explicit aim of aiding future decision making. A number of health systems around the world are interested in the potential for such schemes. This article looks in detail at the potential for some form of AED in the UK, focusing on the two major decision-making bodies: the Scottish Medicines Consortium in Scotland and the National Institute for Health and Clinical Excellence in England and Wales. We consider past experience with these approaches and current initiatives that are exploring their potential, and speculate as to how these schemes might develop in the future.

Helping poorer countries make locally informed health decisions

Health spending in low income countries is too often driven by outside pressure rather than local evidence. Kalipso Chalkidou, Ruth Levine, and Andrew Dillon report on efforts to help them match spending to local needs

Comparative effectiveness research priorities: identifying critical gaps in evidence for clinical and health policy decision making.

BACKGROUND In the debate on improving the quality and efficiency of the United States healthcare system, comparative effectiveness research is increasingly seen as a tool for reducing costs without compromising outcomes. Furthermore, the recent American Recovery and Reinvestment Act explicitly describes a prioritization function for establishing a comparative effectiveness research agenda. However, how such a function, in terms of methods and process, would go about identifying the most important priorities warranting further research has received little attention. OBJECTIVES This study describes an Agency for Healthcare Research and Quality-funded pilot project to translate one current comparative effectiveness review into a prioritized list of evidence gaps and research questions reflecting the views of the healthcare decision makers involved in the pilot. METHODS To create a prioritized research agenda, we developed an interactive nominal group process that relied on a multistakeholder workgroup scoring a list of research questions on the management of coronary artery disease. RESULTS According to the group, the areas of greatest uncertainty regarding the management of coronary artery disease are the comparative effectiveness of medical therapy versus percutaneous coronary interventions versus coronary artery bypass grafting for different patient subgroups; the impact of diagnostic testing; and the most effective method of developing performance measures for providers. CONCLUSIONS By applying our nominal group process, we were able to create a list of research priorities for healthcare decision makers. Future research should focus on refining this process because determining research priorities is essential to the success of developing an infrastructure for comparative effectiveness research.

Social values and health policy: a new international research programme.

PURPOSE This editorial aims to outline the context of healthcare priority-setting, and summarise each of the other ten papers in this special edition. It introduces a new multidisciplinary research programme drawing on ethics, philosophy, health economics, political science and health technology assessment, out of which the papers in this edition have arisen. DESIGN/METHODOLOGY/APPROACH Key normative concepts are introduced and policy and research context provided to frame subsequent papers in the edition. FINDINGS Common challenges of health priority-setting are faced by many countries across the world, and a range of social value judgments is in play as resource allocation decisions are made. Although the challenges faced by different countries are in many ways similar, the way in which social values affect the processes and content of priority-setting decisions means that those challenges are resolved very differently in a variety of social, political, cultural and institutional settings, as subsequent papers in this edition demonstrate. How social values affect decision making in this way is the subject of a new multi-disciplinary research programme. ORIGINALITY/VALUE Technical analyses of health priority setting are commonplace, but approaching the issues from the perspective of social values and conducting comparative analyses across countries with very different cultural, social and institutional contexts provides the content for a new research agenda.

Reflections on the evolution of health technology assessment in Europe

Health technology assessment (HTA) has assumed an increasing role in health systems in recent years, with many countries establishing agencies or programmes to evaluate health technology and other interventions to inform policy decisions and clinical practice. This paper reflects upon its development and evolution in Europe over the last decade, with a focus on England, France, Germany and Sweden. In particular, we explore how HTA has evolved over time as well as its impact on policy and practice. While countries share many of the same objectives, there are differences in the way HTA agencies and programmes are organised, operate, and influence decision making. Despite these differences, all systems are faced with opportunities and challenges related to stakeholder involvement and acceptance, the suitability and transparency of assessment requirements and methods, balancing evidence and values in decision making, and demonstrating impact.