With the increase in technologies to support an aging population, health technology assessment (HTA) of aging-related technologies warrants special consideration. At Health Technology Assessment international (HTAi) 2016 and HTAi 2017, an international panel explored interests in HTA focused on aging.

Panelists from five countries shared the state of aging and HTA in their countries. Opportunities were provided for participants to discuss and rate the themes identified by the panelists.

In 2016, the highest ranked themes were: (i) identifying unmet needs of older adults that could be met by technology—how can HTA help?; (ii) differences in assessment of aging-related technologies—what is the scope?; and (iii) involvement of older adults and caregivers. These themes became the starting point for discussion in 2017, for which the highest ranked themes were: (i) identification of challenges in HTA and aging; and (ii) approaches to advancing effectiveness of HTA for aging.

These discussions allowed for examination of future directions for HTA and aging: engagement of older adults to inform the agenda of HTA and the broader public policy enterprise; a systems approach to thinking about needs of older persons should support the type and level of care desired by the individual rather than the health institutions, and HTA should reflect these desires when evaluating technological aides; and there is potential for health information systems and “big data” to support HTA activities that assess usability of technologies for older adults. We hope to build on the momentum of this community to continue exploring opportunities for aging and HTA.

Due to the nature of Alzheimer's disease (AD), health technology assessment (HTA) agencies might face considerable challenges in choosing appropriate outcomes and outcome measures for drugs that treat the condition. This study sought to understand which outcomes informed previous HTAs, to explore possible reasons for prioritizations, and derive potential implications for future assessments of AD drugs.

We conducted a literature review of studies that analyzed decisions made in HTAs (across disease areas) in three European countries: England, Germany, and The Netherlands. We then conducted case studies of technology assessments conducted for AD drugs in these countries.

Overall, outcomes measured using clinical scales dominated decisions or recommendations about whether to fund AD drugs, or price negotiations. HTA processes did not always allow the inclusion of outcomes relevant to people with AD, their carers, and families. Processes did not include early discussion and agreement on what would constitute appropriate outcome measures and cut-off points for effects.

We conclude that in order to ensure that future AD drugs are valued appropriately and timely, early agreement with various stakeholders about outcomes, outcome measures, and cut-offs is important.

The Belgian Health Care Knowledge Centre (KCE) formally involves stakeholders in HTA since 2012. Patients are treated as one stakeholder amongst others, but it is recognized that patient involvement (PI) requires a different approach. The success of implementing PI depends, however, on the organizational culture toward PI.

The objective of this study was to map the PI culture at KCE in the context of the development of organization-wide supported position statements about PI.

A nominal group technique was used to measure the PI culture at KCE. Arguments for and against PI and conditions for PI in different phases of the HTA process were collected. A literature review and interviews fed the draft position statements, for which support was assessed by means of a two-round Delphi process.

Arguments in favor of PI in HTA related to the relevance of the scope, expertise with data collection, bringing in fresh ideas for study design, access to survey participants, validation of data analyses, adherence to recommendations. Disadvantages and risks included the lack of scientific knowledge of involved patients, resources requirements, conflicts of interest, and heterogeneity within patient populations. Conditions for meaningful PI referred to measures mitigating the identified disadvantages. Eighteen position statements supported by KCE could be formulated.

The KCE culture seems predominantly positive toward PI, although attitudes vary between HTA researchers. KCE recognizes the potential value of PI in HTA, but considers the level of involvement to be contingent on the topic and phase in the HTA process.

Timely access to innovative medical technologies driven by accelerated patient access pathways can substantially improve the health outcomes of patients who often have few therapeutic alternatives. We analyzed lead-times for the medical procedure reimbursement coverage process undertaken in South Korea from 2014 to 2017, which is considered one of the most important factors contributing to delays in patient access to new medical technologies.

This analysis was performed using the open datasets source of “Medical Procedure Expert Evaluation Committee (MPEEC)” meeting results and medical procedure coverage application information published on the Health Insurance Review and Assessment Service Web site.

From 2014 to 2017, 90 percent of all new coverage determinations took on average >250 days with almost 20 percent taking more than 2 years (>750 days), The average lead-time from the medical procedure coverage application to MPEEC meeting in 2015 was 435.0 ± 214.7 days (n = 26), which was significantly shorter than the average lead-time in 2014 (624.9 ± 290.3 days, n = 16) (p < .05). The average lead-time from application to official enforcement in 2015 was significantly shorter than that of 2014 (540.8 ± 217.4; n = 16 versus 734.1 ± 299.7 days; n = 26, respectively) (p < .05).

While this analysis showed a general trend of a reduction in the time taken to receive a positive coverage determination for a new medical technology, the average lead-time remains well over the government mandated 100 days. To continue this trend and further enhance the patient access pathway for medical procedure coverage determinations, some measures can be applied. In particular, the extended “One-Stop Service” program encompassing coverage determinations is one such recommendation that could be considered.

The latest international guideline recommended the add-on therapy of ezetimibe and PCSK9 inhibitors in selected people for the secondary prevention of cardiovascular diseases (CVDs). However, it remains unclear whether these regimens fit the Chinese healthcare system economically.

Based on the Chinese context, this simulation study evaluated four therapeutic strategies including the high-dose statin-only group, ezetimibe plus statin group, PCSK9 inhibitors plus statin group, and PCSK9 inhibitors plus ezetimibe plus statin group. The team developed a Markov model to estimate the incremental cost-effectiveness ratio (ICER). With each 1-yr cycle, the simulation subjects could have nonfatal cardiovascular events (stroke and/or myocardial infarction) or death (vascular or nonvascular death event) with a follow-up duration of 20 yr. Cardiovascular risk reduction was gathered from a network meta-analysis, and cost and utility data were gathered from hospital databases and published research.

For Chinese adults receiving high-dose statins for secondary prevention of CVDs, the ICER was US$68,910 per quality-adjusted life year (QALY) for adding PCSK9 inhibitors, US$20,242 per QALY for adding ezetimibe, US$51,552 per QALY for adding both drugs. Given a threshold of US$37,655 (three times of Chinese GDP), the probability of cost-effectiveness is 2.9 percent for adding PCSK9 inhibitors, 53.1 percent for adding ezetimibe, and 16.8 percent for adding both drugs. To meet the cost-effectiveness, an acquisition price reduction of PCSK9 inhibitors of 33.6 percent is necessary.

In Chinese adults receiving high-dose statins for the secondary prevention of CVDs, adding ezetimibe is cost-effective compared to adding PCSK9 inhibitors and adding both drugs.

Clinical practice is shifting toward an era of precision medicine. The use of comprehensive genomic profiling (CGP) in oncology has broad potential as a universal companion diagnostic for targeted therapies which may significantly improve health outcomes while using healthcare resources more efficiently. Given the nature of this technology, assessing the value of CGP presents unique challenges.

This paper draws on evidence from the academic and policy literature in oncology, as well as stakeholder interviews (health economists, payers, clinicians, and public policy officials) in countries using incremental cost-effectiveness ratios (ICER) as part of health technology assessment (HTA).

The degree to which CGP is subject to a value assessment varies significantly across healthcare systems. Current HTA processes focus on evaluating diagnostic testing through co-dependent assessment of diagnostic testing and associated therapeutic interventions. Diagnostic tests with multiple associated therapeutic interventions are rapidly evolving and poorly unsuited to current HTA approaches. Moreover, HTA approaches are limited in their ability to consider broader systemic benefits of the expanded diagnostic capabilities and enhanced opportunities for clinical trial participation offered by CGP.

The assessment of the overall value of CGP is limited by the current models of HTA. This paper suggests policy proposals for value assessment and funding reforms to help broaden patient access to CGP. These include investing in genomic testing infrastructure; decoupling the assessment of the value of CGP testing to identifying predetermined therapeutic interventions; tailoring evaluation methodology; and developing approaches to collecting evidence of clinical, healthcare system and societal benefit.

The Patient-Centered Outcomes Research Institute (PCORI) horizon scanning system is an early warning system for healthcare interventions in development that could disrupt standard care. We report preliminary findings from the patient engagement process.

The system involves broadly scanning many resources to identify and monitor interventions up to 3 years before anticipated entry into U.S. health care. Topic profiles are written on included interventions with late-phase trial data and circulated with a structured review form for stakeholder comment to determine disruption potential. Stakeholders include patients and caregivers recruited from credible community sources. They view an orientation video, comment on topic profiles, and take a survey about their experience.

As of March 2020, 312 monitored topics (some of which were archived) were derived from 3,500 information leads; 121 met the criteria for topic profile development and stakeholder comment. We invited fifty-four patients and caregivers to participate; thirty-nine reviewed at least one report. Their perspectives informed analyst nominations for fourteen topics in two 2019 High Potential Disruption Reports. Thirty-four patient stakeholders completed the user-experience survey. Most agreed (68 percent) or somewhat agreed (26 percent) that they were confident they could provide useful comments. Ninety-four percent would recommend others to participate.

The system has successfully engaged patients and caregivers, who contributed unique and important perspectives that informed the selection of topics deemed to have high potential to disrupt clinical care. Most participants would recommend others to participate in this process. More research is needed to inform optimal patient and caregiver stakeholder recruitment and engagement methods and reduce barriers to participation.

This review intends to provide an overview of revealed preferences of decision-makers for recommendations of cancer drugs in health technology assessment (HTA) among the different agencies.

A systematic literature search was performed in MEDLINE and EMBASE databases from inception to July 2020. The studies were eligible for inclusion if they conducted a quantitative analysis of HTA’s previous decisions for cancer drugs. The factors with p-values below the significance level of .05 were considered as the statistically significant factors for HTA decisions.

A total of nine studies for six agencies in Australia, Belgium, France, South Korea, the UK, and Canada were eligible to be included. From the univariable analysis, improvements in clinical outcomes and cost-effectiveness were found as significant factors for the agencies in Belgium, South Korea, and Canada. From the multivariable analysis, cost-effectiveness was found as a positive factor for the agencies in the UK, South Korea, and Canada. Few factors related to characteristics of disease and technology were found to be significant among the included agencies.

Despite the different drug reimbursement systems and the socioeconomic situations, cost-effectiveness and/or improvement on clinical outcomes seemed to be the most important factors for recommendations of cancer drugs among the agencies.