Early Health Technology Assessment Research Articles

The role of health technology assessment bodies in shaping drug development.

The use of health technology assessment (HTA) to inform policy-making is established in most developed countries. Compared to licensing agencies, HTA agencies have different interests and, therefore, different evidence requirements. Criteria for coverage or reimbursement decisions on pharmaceutical compounds vary; however, it is common to include, as part of the HTA, a comparative effectiveness evaluation. This type of clinical data might go beyond that required for market authorization, thus creating an additional evidence gap between the regulatory and the reimbursement submission. The relevance of submissions to HTA agencies is consistently increasing in a pharmaceutical company’s perspective, as market prospects are strongly influenced by third-party payers’ coverage. In this study, we aim to describe current HTA activities with a potential impact throughout the drug development process of pharmaceuticals, with a comparative emphasis on the systems in place in Italy and in the UK. Based on an extensive literature and website review, we identified three major classes of HTA activities, beyond mainstream HTA, with the potential to influence the drug development program: 1) horizon scanning and early HTA; 2) bipartite and tripartite early dialogue between manufacturers, regulators, and HTA assessors; and 3) managed market entry agreements. From early stages of clinical research up to postauthorization studies, there is a trend toward increased collaboration between parties, anticipation of market access evidence collection, and postmarketing risk-sharing. Heterogeneity of HTA practices increases the complexity of the market access environment. Overall, there are signals that market access departments are gaining importance in the pharmaceutical companies, but there is still a lack of evidence and reporting on how the increasing relevance of HTA has reshaped the way clinical development is designed and managed.

Early economic evaluation of emerging health technologies: protocol of a systematic review.

BackgroundThe concept of early health technology assessment, discussed well over a decade, has now been collaboratively implemented by industry, government, and academia to select and expedite the development of emerging technologies that may address the needs of patients and health systems. Early economic evaluation is essential to assess the value of emerging technologies, but empirical data to inform the current practice of early evaluation is limited. We propose a systematic review of early economic evaluation studies in order to better understand the current practice.Methods/designThis protocol describes a systematic review of economic evaluation studies of regulated health technologies in which the evaluation is conducted prior to regulatory approval and when the technology effectiveness is not well established. Included studies must report an economic evaluation, defined as the comparative analysis of alternatives with respect to their associated costs and health consequences, and must evaluate some regulated health technology such as pharmaceuticals, biologics, high-risk medical devices, or biomarkers. We will conduct the literature search on multiple databases, including MEDLINE, EMBASE, the Centre for Reviews and Dissemination Databases, and EconLit. Additional citations will be identified via scanning reference lists and author searching. We suspect that many early economic evaluation studies are unpublished, especially those conducted for internal use only. Additionally, we use a chain-referral sampling approach to identify authors of unpublished studies who work in technology discovery and development, starting out with our contact lists and authors who published relevant studies. Citation screening and full-text review will be conducted by pairs of reviewers. ed data will include those related to the decision context and decision problem of the early evaluation, evaluation methods (e.g., data sources, methods, and assumptions used to identify, measure, and value the likely effectiveness and the costs and consequences of the new technology, handling of uncertainty), and whether the study results adequately address the main study question or objective. Data will be summarized overall and stratified by publication status.DiscussionThis study is timely to inform early economic evaluation practice, given the international trend in early health technology assessment initiatives.

PRM177 What is the Role of Early Health Technology Assessment of Biomarkers in the Pre-Clinical Development Phase? A Reflection on Lessons Learned With Multiple Myeloma

PRM177 What is the Role of Early Health Technology Assessment of Biomarkers in the Pre-Clinical Development Phase? A Reflection on Lessons Learned With Multiple Myeloma

A method for the early health technology assessment of novel biomarker measurement in primary prevention programs

Many promising biomarkers for stratifying individuals at risk of developing a chronic disease or subsequent complications have been identified. Research into the potential cost-effectiveness of applying these biomarkers in actual clinical settings has however been lacking. Investors and analysts may improve their venture decision making should they have indicative estimates of the potential costs and effects associated with a new biomarker technology already at the early stages of its development. To assist in obtaining such estimates, this paper presents a general method for the early health technology assessment of a novel biomarker technology. The setting considered is that of primary prevention programs where initial screening to select high-risk individuals eligible for a subsequent intervention occurs, for example, prevention of type 2 diabetes. The method is based on quantifying the health outcomes and downstream healthcare consumption of all individuals who get reclassified as a result of moving from a screening variant based on traditional risk factors to a screening variant based on traditional risk factors plus a novel biomarker. As these individuals form well-defined subpopulations, a combination of disease progression modeling and sensitivity analysis can be used to perform an initial assessment of the maximum increase in screening cost for which the use of the new biomarker technology is still likely to be cost effective.

EARLY AWARENESS AND ALERT ACTIVITIES IN LATIN AMERICA: CURRENT SITUATION IN FOUR COUNTRIES

The aim of this study was to briefly describe the current state of early awareness and alert (EAA) activities and systems in four Latin-American countries (Argentina, Brazil, Colombia, and Mexico). Key informants were selected and completed an open questionnaire that included the following domains: current state of EAA activities and systems in each country, potential role for EAA systems in the health system, and future EAA projects that are currently being considered. In all four countries, health technology assessment (HTA) processes are used to prioritize the use of health resources, albeit at varying degrees and with different mechanisms and methodologies. EAA activities are still limited and there are virtually no institutions or units with specific functions explicitly devoted to EAA activity. However, most countries have developed some initial forms of EAA systems. Being in its initial stages there is no clear differentiation between these early awareness activities and other HTA functions, and no specific methodologies or processes are used to anticipate the emergence of new technologies. Consequently, early evaluation of technologies generally occurs in a reactive manner, after they have been introduced in the market and under the pressure of different stakeholders. There is growing awareness that the early identification and assessment of emerging technologies should be an integral part of HTA and the decision-making process. Many initiatives are currently focusing on building partnerships between the various regulatory bodies involved in the incorporation of technologies at national levels. It is reasonable to foresee that EAA activities will continue to develop and expand in the region.

Early Assessment of Medical Technologies to Inform Product Development and Market Access

Worldwide, billions of dollars are invested in medical product development and there is an increasing pressure to maximize the revenues of these investments. That is, governments need to be informed about the benefits of spending public resources, companies need more information to manage their product development portfolios and even universities may need to direct their research programmes in order to maximize societal benefits. Assuming that all medical products need to be adopted by the heavily regulated healthcare market at one point in time, it is worthwhile to look at the logic behind healthcare decision making, specifically, decisions on the coverage of medical products and decisions on the use of these products under competing and uncertain conditions. With the growing tension between leveraging economic growth through R&D spending on the one hand and stricter control of healthcare budgets on the other, several attempts have been made to apply the health technology assessment (HTA) methodology to earlier stages of technology development and implementation. For instance, horizon scanning was introduced to systematically assess emerging technologies in order to inform health policy. Others have introduced iterative economic evaluation, e.g. economic evaluations in earlier stages of clinical research. However, most of these methods are primarily intended to support governments in making decisions regarding potentially expensive new medical products. They do not really inform biomedical product developers on the probability of return on investment, nor do they inform about the market needs and specific requirements of technologies in development. It is precisely this aspect that increasingly receives attention, i.e. is it possible to use HTA tools and methods to inform biomedical product development and to anticipate further development and market access. Several methods have been used in previous decades, but have never been compiled in a comprehensive review. The main objective of this article was to provide an overview of previous work and methods in the field of early HTA, and to put these approaches in perspective through a conceptual framework introduced in this paper. A particular goal of the review was to familiarize decision makers with available techniques that can be employed in early-stage decision making, and to identify opportunities for further methodological growth in this emerging field of HTA.

Measuring the validity of early health technology assessment: Bibliometrics as a tool to indicate its scientific basis

The aim of this study was to assess whether publications of importance for improving the health system and its technologies are highly cited intrascientifically. Bibliometric assessment of the 596 publications used as sources in the fifty SBU Alerts from 2001 to 2004 from the Swedish Council on Technology Assessment in Health Care was carried out using the Thomson Scientific citation indexes. Normalized citation scores were calculated for all included studies. Additional factors such as the time and place of the research, subject categories, and journal source were analyzed. On average, the sources in SBU Alert have been cited eight times more than the world average consistently during the time period and across research areas. Articles used as its scientific basis are often published in a few, high impact, general medical journals. However, many of the articles are published in field-specific journals with low impact factors. Most articles used in SBU Alert are published by authors based in the United States or the United Kingdom. However, Swedish, Danish, and Dutch publications are overrepresented in its science base, whereas Japanese, Taiwanese, Indian, and Russian publications are underrepresented. Publications used as sources in a Swedish system for identification and early assessment of new methods in health care are also highly cited within the scientific community. This finding increases the appropriateness of using bibliometric indicators in evaluations of clinical research and suggests that decision makers through SBU Alert are getting scientifically sound advice.

Health Technology Assessment: a field still maturing!

With this issue’s focus on Health Technology Assessment (HTA), the Italian Journal of Public Health has tackled an area of growing importance in today’s increasingly complex health care delivery systems. As the articles in this issue demonstrate, HTA has grown from a relatively narrow technical focus to a form of policy research underway in dozens of countries. Since its inception just over three decades ago,HTA has evolved through three distinct phases: the machine, the disease and the delivery mode, with the third of these still underway. As the focus has shifted from machines to disease conditions to service delivery approaches, HTA has drawn on research and modes of discourse from a growing variety of disciplines. Thus, despite the evolution that continues, HTA remains, at its core, both multidisciplinary and pragmatic, for the strengths of HTA arise from its integration of the efforts of actors in multiple, diverse disciplines with a view to producing knowledge that will assist decision-makers. The machine phase was marked by a focus on the technical performance of health technologies, often embodying innovative approaches to diagnosis or treatment of human illness. Given the newness and costliness of many technologies selected for assessment, a significant emphasis was placed on assessing the safety of these devices. Imaging technologies were the subject of assessment in many settings, perhaps in part because devices such as the CT scanner produced remarkable visual results that were heralded as affording breakthroughs in diagnosis and treatment. One need only look through the programs of early HTA conferences to see the emphasis on high cost, infrastructure-intensive health technologies that was the hallmark of the machine period.