In this issue, Facey and colleagues have eloquently summarized the product of the February 2015 HTAi Policy Forum discussion—the need for health technology assessment (HTA) to shift from a historically reactive role in both evaluating current evidence and requesting additional evidence generation to a more proactive role engaging with stakeholders to ensure that evidence produced is appropriate for any given intervention at different stages of the clinical development program. This makes logical a priori sense, of course, as proactive and engaged discussion is always superior to reactive and potentially adversarial interaction.

Objectives: Health Technology Assessment (HTA) needs to address the challenges posed by high cost, effective technologies, expedited regulatory approaches, and the opportunities provided by collaborative real-world evaluation of technologies. The Health Technology Assessment International (HTAi) Policy Forum met to consider these issues and the implications for evidence production to inform HTA. This paper shares their discussion to stimulate further debate.

Methods: A background paper, presentations, group discussions, and stakeholder role play at the 2015 HTAi Policy Forum meeting informed this paper.

Results: HTA has an important role to play in helping improve evidence production and ensuring that the health service is ready to adopt effective technologies. It needs to move from simply informing health system decisions to also working actively to align stakeholder expectations about realistic evidence requirements. Processes to support dialogue over the health technology life cycle need to be developed that are mindful of limited resources, operate across jurisdictions and learn from past processes. Collaborations between health technology developers and health systems in different countries should be encouraged to develop evidence that will inform decision making. New analytical techniques emerging for real-world data should be harnessed to support modeling for HTA.

Conclusions: A paradigm shift (to “Health Innovation System 2.0”) is suggested where HTA adopts a more central, proactive role to support alignment within and amongst stakeholders over the whole life cycle of the technology. This could help ensure that evidence production is better aligned with patient and health system needs and so is more effective and efficient.

With an increasing awareness that active engagement between policy decision makers, HTA agencies, regulators and payers with industry in the premarket space is needed, a disruptive comprehensive approach is described which moves the evidentiary process exclusively into this space. Single harmonized studies pre-market to address regulatory and coverage needs and expectations are more likely to be efficient and less costly and position evidence to drive rather than test innovation. An example of such a process through the MaRS EXCITE program in Ontario, Canada, now undergoing proof of concept, is briefly discussed. Other examples of dialogue between decision makers and industry pre-market are provided though these are less robust than a comprehensive evidentiary approach.

Objectives: The AIFA (Agenzia Italiana del Farmaco—Italian Medicines Agency) Monitoring Registries track the eligibility of patients and the complete flow of treatments, guaranteeing appropriateness in use of pharmaceutical products, according to approved indications.

Methods: This study describes the Italian pharmaceutical context and the aims and functioning of AIFA Monitoring Registries, focusing on the applications to the Managed Entry Agreements (MEAs) and HTA approaches.

Results: The AIFA Monitoring Registries System has been operational in Italy since 2005. In 2012, the system became part of the NHS Information Technology system, aiming at enhancing appropriate use of pharmaceuticals and efficiency of the administrative activity. Currently, seventy-six medicines are monitored through the system, corresponding to fifty-eight therapeutic indications; individual treatments recorded are more than 515,000, for a population of approximately 505,000 patients. For each monitored product, patients eligible for treatment are registered in the specific therapeutic indication dynamic monitoring database to collect epidemiologic and clinical data, including data on the safety profile, and ex-post information missing at first evaluation stage.

Conclusions: AIFA Monitoring Registries allow the evaluation of the pharmaceuticals’ performance in clinical practice and may promote innovation and quicker access to medicines at affordable prices, for the benefit of patients.

Background: Availability of real-time electronic healthcare data provides new opportunities for rapid-cycle evaluation (RCE) of health technologies, including healthcare delivery and payment programs. We aim to align decision-making processes with stages of RCE to optimize the usefulness and impact of rapid results. Rational decisions about program adoption depend on program effect size in relation to externalities, including implementation cost, sustainability, and likelihood of broad adoption.

Methods: Drawing on case studies and experience from drug safety monitoring, we examine how decision makers have used scientific evidence on complex interventions in the past. We clarify how RCE alters the nature of policy decisions; develop the RAPID framework for synchronizing decision-maker activities with stages of RCE; and provide guidelines on evidence thresholds for incremental decision-making.

Results: In contrast to traditional evaluations, RCE provides early evidence on effectiveness and facilitates a stepped approach to decision making in expectation of future regularly updated evidence. RCE allows for identification of trends in adjusted effect size. It supports adapting a program in midstream in response to interim findings, or adapting the evaluation strategy to identify true improvements earlier. The 5-step RAPID approach that utilizes the cumulating evidence of program effectiveness over time could increase policy-makers' confidence in expediting decisions.

Conclusions: RCE enables a step-wise approach to HTA decision-making, based on gradually emerging evidence, reducing delays in decision-making processes after traditional one-time evaluations.

Health systems around the world cope with the challenge of difficult economic times, and the value of health technology assessment (HTA) is increasing. Making the right choices, with limited resources, in the face of increasingly complex technologies requires decisions informed by data and analyses that help us to manage the risks involved. Those who undertake and use HTA can play a greater role in helping decision makers meet these challenges; they need to think how to define innovation and respond to it, how to communicate their analyses, and, critically, how to align their work with the ambitions of their health systems. HTA can become a key health system enabler without compromising its objectivity or independence. It can say that it is too early to determine the value of a new technology when the data simply will not support a safe decision. However, it can also be bold and recommend the managed introduction of new technologies, even when the when the data is immature, provided that the health system understands the risks and there is a plausible case for believing that further research will support the value proposition. The goal for HTA is to be able confidently to do both.

This article gives a patient organization's perspective on health technology assessments (HTAs) and the role that such organizations can, and should, play in them.

Are we in a period of significant change? Is health technology assessment (HTA) undergoing a fundamental transformation? Or should it be? Are we in the middle of a paradigm shift? These are some of the questions the paper by Facey et al in this journal (1) raises about the future of HTA, based on discussions at the HTAi Policy Forum in February 2015. To further share the deliberations of the Forum and with a view to opening this debate among the wider HTA community, a panel within the HTAi 2015 Annual Meeting in Oslo was organized. Presentations at the panel included a summary of the HTAi Policy Forum deliberations and perspectives from a patient, a clinician, and representatives of an HTA organization, industry, and a health system. This letter presents issues and thoughts raised in the panel session.

Background: “Mapping” onto generic preference-based outcome measures is increasingly being used as a means of generating health utilities for use within health economic evaluations. Despite publication of technical guides for the conduct of mapping research, guidance for the reporting of mapping studies is currently lacking. The MAPS (MApping onto Preference-based measures reporting Standards) statement is a new checklist, which aims to promote complete and transparent reporting of mapping studies.

Methods: In the absence of previously published reporting checklists or reporting guidance documents, a de novo list of reporting items was created by a working group comprised of six health economists and one Delphi methodologist. A two-round, modified Delphi survey with representatives from academia, consultancy, health technology assessment agencies, and the biomedical journal editorial community was used to identify a list of essential reporting items from this larger list.

Results: From the initial de novo list of twenty-nine candidate items, a set of twenty-three essential reporting items was developed. The items are presented numerically and categorized within six sections, namely: (i) title and abstract, (ii) introduction, (iii) methods, (iv) results, (v) discussion, and (vi) other. The MAPS statement is best applied in conjunction with the accompanying MAPS explanation and elaboration document.

Conclusions: It is anticipated that the MAPS statement will improve the clarity, transparency. and completeness of reporting of mapping studies. To facilitate dissemination and uptake, the MAPS statement is being co-published by seven health economics and quality of life journals, and broader endorsement is encouraged. The MAPS working group plans to assess the need for an update of the reporting checklist in five years’ time.

Objectives: There is widespread commitment—at least in principle—to “evidence-informed” clinical practice and policy development in health care. The intention is that only “appropriate” care ought to be delivered at public expense. Although the rationale for an appropriateness agenda is widely endorsed, and methods have been proposed for addressing it, few published studies exist of contemporary policy initiatives which have actually led to successful disinvestment. Our objective was to explore whether the direct involvement of policy stakeholders could advance appropriateness and disinvestment.

Methods: Several collaborative engagements with policy stakeholders were undertaken to adapt and combine conceptual and empirical material related to appropriateness and disinvestment from the literature to create tools and processes for use in Canada and the province of Ontario in particular.

Results: By combining inputs from the literature with colloquial evidence from policy stakeholders, a definition of appropriateness was developed and, importantly, endorsed by all the provincial and territorial ministers of health in Canada. Second, a reassessment framework was successfully implemented for identifying priorities for selective disinvestment.

Conclusions: When scientific evidence was combined with colloquial evidence from policy stakeholders, progress was made on the design and successful implementation of policies for appropriateness and disinvestment.

Objectives: The aim of this study was to determine the role of community preference information from discrete choice studies of colorectal cancer (CRC) screening in health technology assessment (HTA) reports and subsequent policy decisions.

Methods: We undertook a systematic review of discrete choice studies of CRC screening. Included studies were reviewed to assess the policy context of the research. For those studies that cited a recent or pending review of CRC screening, further searches were undertaken to determine the extent to which community preference information was incorporated into the HTA decision-making process.

Results: Eight discrete choice studies that evaluated preferences for CRC screening were identified. Four of these studies referred to a national or local review of CRC screening in three countries: Australia, Canada, and the Netherlands. Our review of subsequently released health policy documents showed that while consideration was given to community views on CRC, policy was not informed by discrete choice evidence.

Conclusions: Preferences and values of patients are increasingly being considered “evidence” to be incorporated into HTA reports. Discrete choice methodology is a rigorous quantitative method for eliciting preferences and while as a methodology it is growing in profile, it would appear that the results of such research are not being systematically translated or integrated into HTA reports. A formalized approach is needed to incorporate preference literature into the HTA decision-making process.

Objectives: The aim of this study was to assess effect of adult diapers on health-related quality of life (HRQOL) and the independent level of performing activities of daily living (ADLs) in people with urinary or fecal incontinence. Psychological consequences of patients’ caregivers were also measured.

Methods: This quasi-experimental study was conducted at two rehabilitation centers in Thailand. People aged 15 years or greater with chronic urinary or fecal incontinence were eligible. Study participants received adult diapers for 10 weeks after recruitment. Thai EuroQol Five Dimensions (EQ-5D) and the Barthel Index were measured at baseline and weeks 2, 6, and 10 to evaluate HRQOL and the independent level of performing ADLs, respectively. The Braden Scale was used to assess the risk of having pressure ulcers. Mean differences in the Thai EQ-5D, the Barthel Index, and the Braden Scale, before and after receiving adult diapers, were estimated using a multilevel linear regression model.

Results: There were ninety patients and forty-eight caregivers who took part in this study. HRQOL and independent level of performing ADLs had improved significantly by week 10 after receiving adult diapers with mean differences of 0.102 (95% confidence interval [CI], 0.046–0.158) and 4.40 (95% CI, 1.74–7.07), respectively. The risk of having pressure ulcers had significantly decreased by 67 percent (95% CI, 16 percent–78 percent) by week 10 after receiving adult diapers.

Conclusions: The results indicate a significant improvement of HRQOL and the independent level of performing ADLs among incontinent patients after receiving adult diapers. These results were used to inform the development of the health benefits package under the Universal Health Coverage Scheme in Thailand.

Background: The intelligent bed is a medical bed with several home healthcare functions. It includes, among others, an “out of bed” detector, a moisture detector, and a catheter bag detector. The design purpose of the intelligent bed is to assist patients in their daily living, facilitate the work of clinical staff, and improves the quality of care. The aim of this sub-study of the iCare project was to explore how health professionals (HPs) experience and use the intelligent bed in patients’ homes.

Methods: The overall research design is inspired by case study methodology. A triangulation of data collection techniques has been used: log book, documentation study, participant observations (n = 45 hr), and qualitative interviews (n = 23). The data have been analyzed by means of Nvivo 9.0.

Findings: We identified several themes: HP transformation from passive technology recipient to innovator; individualized care; work flow redesign; and sensor technology intruding on patient privacy.

Conclusions: It is suggested that functions of the intelligent bed can result in more individualized care, workflow redesign, and time savings for the health professionals in caring for elderly patients. However, the technology intruded on patients’ privacy.

Objectives: Lung cancer has been the most common cancer since 1985, accounting for 12–13 percent of cancer cases worldwide. Newer targeted therapies with potential increased survival benefits may not be affordable to patients. Many countries use arbitrary thresholds to determine whether a medical intervention is cost-effective. As such, many effective, albeit expensive, therapies are not being reimbursed. To understand the value placed on effective therapies, this study evaluates the patient and public willingness to pay (WTP) for a quality-adjusted life-year (QALY) for lung cancer treatments using Thailand as an example.

Methods: A total of 300 subjects responded to hypothetical lung cancer health states, described by three levels of severity and two levels of side effects, and provided their valuation of the level of quality of life and their WTP to improve from one state to another.

Results: The patients with the lowest income and general public were willing to pay more than twice the threshold for acceptability in Thailand (US Dollar 5,123/QALY [Thai Baht 160,000/QALY]). This increased significantly by wealth category. Patients’ WTP was associated with quality of life, financial difficulties, health insurance, diarrhea, and wealth.

Conclusions: The current study highlights the value patients and general public place on effective lung cancer therapies.

Owing to an editorial error, in the article by Thongprasert et al. (1) in the August 2015 issue of International Journal of Technical Assessment in Health Care, Bruce Crawford was incorrectly listed as the corresponding author. The corresponding author is Unchalee Permsuwan.