Chris Henshall

How long does biomedical research take? Studying the time taken between biomedical and health research and its translation into products, policy and practice

BackgroundThe time taken, or ‘time lags’, between biomedical/health research and its translation into health improvements is receiving growing attention. Reducing time lags should increase rates of return to such research. However, ways to measure time lags are under-developed, with little attention on where time lags arise within overall timelines. The process marker model has been proposed as a better way forward than the current focus on an increasingly complex series of translation ‘gaps’. Starting from that model, we aimed to develop better methods to measure and understand time lags and develop ways to identify policy options and produce recommendations for future studies.MethodsFollowing reviews of the literature on time lags and of relevant policy documents, we developed a new approach to conduct case studies of time lags. We built on the process marker model, including developing a matrix with a series of overlapping tracks to allow us to present and measure elements within any overall time lag. We identified a reduced number of key markers or calibration points and tested our new approach in seven case studies of research leading to interventions in cardiovascular disease and mental health. Finally, we analysed the data to address our study’s key aims.ResultsThe literature review illustrated the lack of agreement on starting points for measuring time lags. We mapped points from policy documents onto our matrix and thus highlighted key areas of concern, for example around delays before new therapies become widely available. Our seven completed case studies demonstrate we have made considerable progress in developing methods to measure and understand time lags. The matrix of overlapping tracks of activity in the research and implementation processes facilitated analysis of time lags along each track, and at the cross-over points where the next track started. We identified some factors that speed up translation through the actions of companies, researchers, funders, policymakers, and regulators. Recommendations for further work are built on progress made, limitations identified and revised terminology.ConclusionsOur advances identify complexities, provide a firm basis for further methodological work along and between tracks, and begin to indicate potential ways of reducing lags.

Priority Setting for Health Technology Assessment: Theoretical Considerations and Practical Approaches: A paper produced by the Priority Setting Subgroup of the EUR-ASSESS Project

This report is about setting priorities for health technology assessment (HTA). HTA examines systematically the consequences of the application of health technologies (broadly defined to include any health care intervention) to support decision making in policy and practice. Only a fraction of existing health technologies have been formally evaluated, and many more appear each year. Resources for HTA are, however, limited so that priorities have to be set, whether explicitly or implicitly. The aim of setting priorities for HTA should be to identify those assessments that offer the greatest benefits in relation to their cost, and thus to maximize the benefit derived from investments in HTA.

Health technology assessment in the United Kingdom.

The National Health Service (NHS) provides universal health coverage for all British citizens. Most services are free of charge, although modest copayments are sometimes applied. About 11% of the population also has private insurance. General practitioners, generally the first point of contact for accessing the system, are independent contractors who serve as gatekeepers for specialist and hospital services and enjoy substantial clinical autonomy. Hospitals are public and are regionalized, but the 1990 reforms made them self-governing trusts that contract with local purchasers (health authorities and general practitioner fundholders). Reforms beginning in 1990 moved the NHS away from a centralized administrative structure to more pluralistic arrangements in which competition, as well as management, influences how services develop. Health technology and health technology assessment (HTA) have gained increasing attention in the NHS during this period, as part of a wider NHS Research and Development (R&D) Strategy. The strategy promotes a knowledge-based health service with a strong research infrastructure and the capacity to critically review its own needs. HTA is the largest and most developed of the programs within the strategy. It has a formal system for setting assessment priorities involving widespread consultation within the NHS, and a National Co-ordinating Centre for Health Technology Assessment. The strategy supports related centers such as the U.K. Cochrane Centre and the NHS Centre for Reviews and Dissemination. A hallmark of the HTA program is strong public participation. The United Kingdom has made a major commitment to HTA and to seeking effective means of reviewing and disseminating evidence.

Interactions between health technology assessment, coverage, and regulatory processes: Emerging issues, goals, and opportunities

BACKGROUND The relationship between regulatory approval on the one hand and health technology assessment (HTA) and coverage on the other is receiving growing attention. Those responsible for regulatory approval, HTA, and coverage have different missions and their information requirements reflect these. There is nonetheless an increasingly popular view that improved communication and coordination between these functions could allow them all to be undertaken effectively with a lower overall burden of evidence requirements, thus speeding patient access to new products and reducing unnecessary barriers to innovation. This study summarizes the main points emerging from a recent discussion of this topic at the HTAi Policy Forum. RESULTS AND CONCLUSIONS After considering the roles of the various bodies, stakeholder perspectives and some current practical initiatives, those present at the Forum meeting discussed possible goals and challenges for improved interactions-in general and at specific stages of the product development life cycle. Opportunities for progress were seen in: continuing the dialogue to promote understanding and interaction between the different bodies and stakeholders; working to align scientific advice for manufacturers on the design and data requirements of pre- and post-marketing evaluation of products (specifically phase 2/3 and phase 4 trials for drugs); and extending the current dialogue to include discussion of product development to address unmet health needs.

SUMMARY REPORT OF THE ECHTA/ECAHI PROJECT

Health technology assessment (HTA) seeks to inform health policy makers by using the best scientific evidence on the medical, social, economic, and ethical implications of investments in health care. Technology is broadly defined to include the drugs, devices, medical, and surgical procedures used in health care, as well as measures for prevention and rehabilitation of disease, and the organizational and support systems in which health care is provided.

EXECUTIVE SUMMARY OF THE ECHTA/ECAHI PROJECT

Health technology is an indispensable part of any nations healthcare system. During the past 50 years, all member states that comprise the European Union have increased their technological base for health care, both in terms of knowledge and by investments in equipment, devices, and pharmaceuticals. Generally, this process has gone well. However, several problems have emerged related to the acquisition, diffusion, and use of modern health technology. Concerns have been also raised about the effectiveness and efficiency of already established procedures in health care.

HEALTH TECHNOLOGY ASSESSMENT IN POLICY AND PRACTICE: Working Group 6 Report

According to the HTA-Europe report, health technology assessment (HTA) has the potential to influence health policy in the fields of research and development, regulation of pharmaceuticals, devices, and services, quality assurance, reimbursement, education and training of healthcare providers, and consumer education (1). To identify and bridge the gaps between HTA production and implementation, the goal of Working Group 6 was to approach the use of HTA in policy and practice on the aggregate policy level as well as in the local context, such as in hospitals. Members of Working Group 6 included both users (e.g., members of decision-making bodies) and producers (e.g., members of HTA agencies) of HTA reports. The aim of the working group was to identify three types of information on HTA implementation and impact. These included identifying successful examples of the implementation of HTA results, examples of structures that require HTA information as part of the decision-making process, and finally, identifying groups and persons who use HTA frequently and are willing to share their experiences with other HTA users and producers.

INTERACTION INITIATIVES BETWEEN REGULATORY, HEALTH TECHNOLOGY ASSESSMENT AND COVERAGE BODIES, AND INDUSTRY

There has been an increased focus on the relationship between health technology assessment (HTA) and regulatory assessments and how regulatory, HTA and coverage bodies, and industry can work better together to improve efficiency and alignment of processes. There is increasingly agreement across sectors that improved communication and coordination could contribute to facilitating timely patient access to effective, affordable treatments that offer value to the health system. Discussions on aspects of this relationship are being held in different forums and various forms of coordination and collaboration are being developed or piloted within several jurisdictions. It is therefore both timely and of value to stakeholders to describe and reflect on current initiatives intended to improve interactions between regulatory, HTA and coverage bodies, and industry. Drawing on 2011 meetings of the HTAi Policy Forum and the Center for Innovation in Regulatory Science (CIRS), this study aims to describe and compare initiatives, and point to success factors and challenges that are likely to inform future work and collaboration.

Adaptive approaches to licensing, health technology assessment, and introduction of drugs and devices.

BACKGROUND Adaptive approaches to the introduction of drugs and medical devices involve the use of an evolving evidence base rather than conventional single-point-in-time evaluations as a proposed means to promote patient access to innovation, reduce clinical uncertainty, ensure effectiveness, and improve the health technology development process. METHODS This report summarizes a Health Technology Assessment International (HTAi) Policy Forum discussion, drawing on presentations from invited experts, discussions among attendees about real-world case examples, and background paper. RESULTS For adaptive approaches to be understood, accepted, and implemented, the Forum identified several key issues that must be addressed. These include the need to define the goals of and to set priorities for adaptive approaches; to examine evidence collection approaches; to clarify the roles and responsibilities of stakeholders; to understand the implications of adaptive approaches on current legal and ethical standards; to determine costs of such approaches and how they will be met; and to identify differences in applying adaptive approaches to drugs versus medical devices. The Forum also explored the different implications of adaptive approaches for various stakeholders, including patients, regulators, HTA/coverage bodies, health systems, clinicians, and industry. CONCLUSIONS A key outcome of the meeting was a clearer understanding of the opportunities and challenges adaptive approaches present. Furthermore, the Forum brought to light the critical importance of recognizing and including a full range of stakeholders as contributors to a shared decision-making model implicit in adaptive pathways in future discussions on, and implementation of, adaptive approaches.

Public participation in decision-making on the coverage of new antivirals for hepatitis C

Purpose - New hepatitis C medicines such as sofosbuvir underline the need to balance considerations of innovation, clinical evidence, budget impact and equity in health priority-setting. The purpose of this paper is to examine the role of public participation in addressing these considerations. Design/methodology/approach - The paper employs a comparative case study approach. It explores the experience of four countries - Brazil, England, South Korea and the USA - in making coverage decisions about the antiviral sofosbuvir and involving the public and patients in these decision-making processes. Findings - Issues emerging from public participation ac tivities include the role of the universal right to health in Brazil, the balance between innovation and budget impact in England, the effect of unethical medical practices on public perception in South Korea and the legitimacy of priority-setting processes in the USA. Providing policymakers are receptive to these issues, public participation activities may be re-conceptualized as processes that illuminate policy problems relevant to a particular context, thereby promoting an agenda-setting role for the public. Originality/value - The paper offers an empirical analysis of public involvement in the case of sofosbuvir, where the relevant considerations that bear on priority-setting decisions have been particularly stark. The perspectives that emerge suggest that public participation contributes to raising attention to issues that need to be addressed by policymakers. Public participation activities can thus contribute to setting policy agendas, even if that is not their explicit purpose. However, the actualization of this contribution is contingent on the receptiveness of policymakers.