Multi-analyte assays with algorithmic analyses (MAAAs) use combinations of circulating and clinical markers including omics-based sources for diagnostic and/or prognostic purposes. Assessing MAAAs is challenging under existing health technology assessment (HTA) methods or practices. We undertook a scoping review to explore the HTA methods used for MAAAs to identify the criteria used for clinical research and reimbursement purposes.

This review included only non-companion (stand-alone) tests that are actionable and that have been evaluated by leading HTA or insurer/reimbursement bodies up to September 2017.

Twenty-five reports and articles evaluating seventeen MAAAs were examined, most of which have been developed in oncology. The two main models used were the EUnetHTA Core model and the Evaluation of Genomic Applications in Practice and Prevention ACCE framework. Clinical validity and utility criteria were used, as were economic, ethical, legal, and social aspects. Economic evidence on MAAAs was scarce, and there is no consensus on whether the perspectives used are sufficiently broad to include all relevant stakeholders.

Clinical utility and efficiency were the most used criteria, with stronger evidence needed linking the use of the algorithm with the clinical outcomes in real-life practice. HTA bodies must as well consider questions related to the analytical validity of MAAAs or with organizational aspects. The two main models, the EUnetHTA Core model and the ACCE framework, could be adapted to the assessment of MAAAs.

Patients and families play an important role in preventing adverse events. The quality council at our hospital produced a communication tool in considering the main causes of adverse events and requested the health technology assessment (HTA) unit to validate it.

Assess the validity of the content of a tablemat sticker as an information tool for hospitalized patients.

A qualitative validation was first performed with individual interviews and focus groups to evaluate the understanding of the content. The tool was modified and as a second step, a survey was conducted on patients and their families from a surgical care unit to validate their understanding and relevance of the content.

From the survey, patients and families found the tablemat attractive and stimulating (97 percent). It encouraged them to communicate with staff about the safety of their care (84 percent). They understood well the objective (79 percent) and text (90 percent), but less for the pictograms (30 percent to 62 percent). The communication and recommendations to avoid falling were good and 99 percent were wearing the medical identification. However, it was not clear that these indicators represented the real concerns of the patients and healthcare staff because no user evaluation was done when developing the tool.

The tool was well understood, but some improvements are needed considering that pictograms were not always well understood and so need careful consideration from patient perspective. The HTA unit recommended conducting an unbiased survey to assess the concerns of patients and professionals to identify the most relevant indicators.

Chronic wounds are frequent, affect quality of life, and increase care costs. Telemedicine provides potential for effective wound care management, especially for the monitoring of complex wounds at home.

The objective of the present study was to determine the clinical effects and costs of telemedicine for the follow-up of complex chronic wounds from the perspective of the public health insurance. The study ran over a period of 9 months.

We conducted a prospective, pragmatic, open-label, observational study and carried out a cost-effectiveness analysis. A total of 116 patients with chronic wounds were assigned to their choice of two groups: telemedicine (N = 77) and traditional follow-up (control; N = 39). The primary outcome was the time to healing. Secondary outcomes included percentage of wounds reaching target objective, percentage of wounds healed completely, outpatient care costs, travel costs, and hospitalizations.

Time to healing was shorter in the telemedicine group than in the control group (137 versus 174 days; p < .05). The percentage of wounds completely healed was not statistically different between the telemedicine and control group (66 percent versus 61 percent; p > .05). Outpatient care and hospitalization costs were not significantly different. The main results in terms of economic savings were medical transport costs reimbursed by the French public health insurance, which were significantly lower in the telemedicine group. Telemedicine costs were found to be €4,583 less per patient compared with standard practice over 9 months.

This trial suggests that telemedicine saves travel costs and results in a shorter healing time than traditional follow-up.

The HTA Core Model® was developed to improve the transferability of health technology assessment (HTA) between settings. The model has been used by HTA agencies but is also of interest to manufacturers, for improving internal evidence generation and communicating with other HTA stakeholders. To establish if the model is fit for purpose from an industry perspective, the pharmaceutical company Roche, collaborating with the European Network for HTA (EUnetHTA), conducted an assessment of the model.

A questionnaire was developed to evaluate all assessment elements in the HTA Core Model v2.0 for their usefulness in meeting payers’ evidence needs and demonstrating value. The questionnaire was completed by country affiliate teams working in evidence generation and reimbursement submissions for pharmaceuticals. Survey results were discussed in workshops to ensure consistency and alignment between teams.

The questionnaire was completed by six teams. An additional team from global pricing and market access participated in workshops. Model domains pertaining to the health problem and current technology use, technology description, clinical effectiveness, and economic value were considered most important because they meet payers’ evidence needs. Overall, the model was considered useful to improve the efficiency of HTA evidence generation, share evidence internally, and communicate value to payers and HTA agencies.

From an industry perspective, the HTA Core Model provides a useful framework and common terminology for efficient generation of transferable HTA evidence. The timeliness, efficiency, and transparency of HTA processes could be improved by a more standardized approach to HTA across settings.

For many years, several health technology assessment (HTA) agencies scanned the horizon to identify health technologies that were safe, effective and offer value for money. However, there is limited evidence regarding its impact. The role of horizon scanning in preparing health systems for the uptake of new and emerging health technologies was discussed during the 2018 HTA International (HTAi) Global Policy Forum Meeting.

Reflection of the discussion between seventy-two senior representatives from for-profit, not-for-profit organizations, and HTAi leadership. It was informed by a background paper, and presentations from four invited experts and seventeen Policy Forum members.

Current horizon scanning systems (HSS) mainly identify health technologies in the late stage of development, aiming to inform topic selection for HTA. Areas for improvement included the need for a clearer definition of the end user(s), purpose, scope, and focus of HSS, the long-term full health system effects, including all relevant stakeholders as early as possible, and considering smart data systems and international collaboration to improve HSS's efficiency. The way in which HSS could be further optimized and better shaped to prepare health systems was also discussed and good practice examples were presented.

HSS have not yet reached their full potential in preparing health systems. To improve the current situation, the HTA community could act as convenors, bringing together all relevant stakeholders and providing the information that decision makers need. This would require a new, more integrative approach to define and use HSS and HTA, and requires new skills.

The importance of Cardiac Implant Registry (CIR) for ensuring a long-term follow-up in post-marked surveillance has been recognized and approved, but there is a lack of consensus standards on how to establish a CIR. The aim of this study is to investigate the structure and key elements of CIRs in the past decade (2006–2016) and to provide recommendations on “best practice” approaches.

A systematic search on CIR was employed in line with the PRISMA guidelines. The following databases were searched: the PubMed (Medline), ScienceDirect, EMBASE and the Scopus database. After identifying the existing CIR, an inductive approach was used to explore key elements emerging in the identified registries.

The following eighty-two registries were identified: eighteen ICD registries, seven CRT registries, five pacemaker registries, and six Cardiovascular Implantable Electronic Device (CIED) registries which combined ICD, pacemaker and CRT implantation data; as well as twenty-two coronary stent registries and twenty-four TAVI registries. While seventy-one national or local registries are from a single country, forty-four are from European countries, and nine are located in USA. The following criteria have been summarized from the identified registries, including: registry working group, ethic issues, transparency, research objective, inclusion criteria, compulsory participation, endpoint, sample size, data collection basement, data collection methods, data entry, data validation and statistical analysis.

For HTA as well as regulatory decision making, medical device registries provide a “real-world” picture for patients, physicians, manufacturers, payers, decision-makers and other stakeholders. CIRs are important for regulatory decisions concerning the safety and approval issues of medical devices; for payers CIRs provide evidence on the medical device benefits and drive the decision as to whether the product should be reimbursed or not; for hospitals data from CIRs are important for sound procurement decisions, and CIRs also help patients and their physicians to reach a joint decision on which of the products is the most appropriate. However, many current CIRs are still lacking standards to inform on patient safety and ensure transparency.

Health Technology Assessment (HTA) considers the question of whether evaluated technologies are cost-effective in real world settings. As observed in HTA conducted by the Australian Medical Services Advisory Committee (MSAC), questions regarding the validity of data inputs to economic analyses that reflect real-world practice is a common reason for uncertainty on the cost effectiveness of new technologies. In addition to resource use and costs, there may be other uncertainties regarding the eligible patient population, patient management pathways and comparator selection. Our objective in this study was to present case studies from Australia where real world linked datasets could be better utilized to inform HTA conducted by the MSAC.

For selected therapy areas, assessment reports and public summary documents of HTA conducted by the MSAC published between January 2015 and November 2017 were reviewed. Our analysis identified HTAs where uncertainties around the inputs for health economic evaluations, as well as uncertainties in defining eligible patient numbers or current patient pathways of care were shown to exist. We then explored whether these uncertainties could have been addressed through real world linked datasets.

Our preliminary investigations identified two assessments: MSAC assessment of capsule endoscopy and transcatheter aortic valve implantation - where availability of real world linked data could have addressed uncertainties around the inputs required for the health economic evaluations.

Australia has a range of real world datasets with the potential to be used to inform HTA conducted by the MSAC. This can only be achieved if the datasets could be better linked and accessible for use by key stakeholders in the MSAC HTA process (e.g. industry, clinician, patient societies). Use of these data sets in HTA will enable timelier patient access to cost-effective technologies and more effective implementation and review of technologies after adoption into clinical practice.

Determine the relationship between quality of an accountable care organization (ACO) and its long-term reduction in healthcare costs.

We conducted a cost minimization analysis. Using Centers for Medicare and Medicaid cost and quality data, we calculated weighted composite quality scores for each ACO and organization-level cost savings. We used Markov modeling to compute the probability that an ACO transitioned between different quality levels in successive years. Considering a health-systems perspective with costs discounted at 3 percent, we conducted 10,000 Monte Carlo simulations to project long-term cost savings by quality level over a 10-year period. We compared the change in per-member expenditures of Pioneer (early-adopters) ACOs versus Medicare Shared Savings Program (MSSP) ACOs to assess the impact of coordination of care, the main mechanism for cost savings.

Overall, Pioneer ACOs saved USD 641.24 per beneficiary and MSSP ACOs saved USD 535.59 per beneficiary. By quality level: (a) high quality organizations saved the most money (Pioneer: USD 459; MSSP: USD 816); (b) medium quality saved some money (Pioneer: USD 222; MSSP: USD 105); and (c) low quality suffered financial losses (Pioneer: USD -40; MSSP: USD -386).

Within the existing fee-for-service healthcare model, ACOs are a mechanism for decreasing costs by improving quality of care. Higher quality organizations incorporate greater levels of coordination of care, which is associated with greater cost savings. Pioneer ACOs have the highest level of integration of services; hence, they save the most money.

This study aimed to assess the cost-effectiveness of ivabradine plus standard of care (SoC) in comparison with current SoC alone from the Iranian payer perspective.

A cohort-based Markov model was developed to assess the incremental cost-effectiveness ratio (ICER) over a 10-year time horizon in a cohort of 1,000 patients. The baseline transition probabilities between New York Heart Association (NYHA), mortality rate, and hospitalization rate were extracted from the literature. The effect of ivabradine on mortality, hospitalization, and NYHA improvement or worsening were retrieved from the SHIFT study. The effectiveness was measured as quality-adjusted life-years (QALYs) using the utility values derived from Iranian Heart Failure Quality of Life study. Direct medical costs were obtained from hospital records and national tariffs. Deterministic and probabilistic sensitivity analyses were conducted to show the robustness of the model.

Ivabradine therapy was associated with an incremental cost per QALY of USD $5,437 (incremental cost of USD $2,207 and QALYs gained 0.41) versus SoC. The probabilistic sensitivity analysis showed that ivabradine is expected to have a 60 percent chance of being cost-effective accepting a threshold of USD $6,550 per QALY. Furthermore, deterministic sensitivity analysis indicated that the model is sensitive to the ivabradine drug acquisition cost.

The cost-effectiveness model suggested that the addition of ivabradine to SoC therapy was associated with improved clinical outcomes along with increased costs. The analysis indicates that the clinical benefit of ivabradine can be achieved at a reasonable cost in eligible heart failure patients with sinus rhythm and a baseline heart rate ≥ 75 beats per minute (bpm).

To identify and appraise existing evaluation frameworks for mobile medical applications (MMA) and determine their suitability for use in health technology assessment (HTA) of these technologies.

Systematic searches were conducted of seven bibliographic databases to identify literature published between 2008 and 2016 on MMA evaluation frameworks. Frameworks were eligible if they were used to evaluate at least one of the HTA domains of effectiveness, safety, and/or cost and cost-effectiveness of an MMA. After inclusion, the frameworks were reviewed to determine the number and extent to which other elements of an HTA were addressed by the framework.

A total of forty-five frameworks were identified that assessed MMAs. All frameworks assessed whether the app was effective. Of the thirty-four frameworks that examined safety, only seven overtly evaluated potential harms from the MMA (e.g., the impact of inaccurate information). Only one framework explicitly considered a comparator. Technology specific domains were sporadically addressed.

None of the evaluation frameworks could be used, unaltered, to guide the HTA of MMAs. To use these frameworks in HTA they would need to identify relevant comparators, improve assessments of harms and consider the ongoing effect of software updates on the safety and effectiveness of MMAs. Attention should also be paid to ethical issues, such as data privacy, and technology specific characteristics. Implications: Existing MMA evaluation frameworks are not suitable for use in HTA. Further research is needed before an MMA evaluation framework can be developed that will adequately inform policy makers.

The aim of this study was to describe the historical development of the HTAsiaLink network, draw lessons for other similar initiatives globally, and to analyze key determinants of its success and challenges for its future development.

This study is based on the collective and direct experiences of the founding members of the HTAsiaLink Network. Data were collected from presentations they made at various international forums and additional information was reviewed. Data analysis was done using the framework developed by San Martin-Rodriguez et al.

HTAsiaLink is a network of health technology assessment (HTA) agencies in Asia established in 2011 with the aim of strengthening individual and institutional HTA capacity, reducing duplication and optimizing resources, transfer and sharing of HTA-related lessons among members, and beyond. During its 6 years, the network has expanded, initiating several capacity building activities and joint-research projects, raising awareness of the importance of HTA within the region and beyond, and gaining global recognition while establishing relationships with other global networks. The study identifies the determinants of success of the collaboration. The systemic factors include the favorable outlook toward HTA as an approach for healthcare priority setting in countries with UHC mandates. On organizational factors, the number of newly established HTA agencies in the region with similar needs for capacity building and peer-to-peer support was catalytic for the network development. The interactional aspects include ownership, trust, and team spirit among network members. The network, however, faces challenges notably, financial sustainability and management of the expanded network.

Although interferon beta-1a (IFNß−1a), 1b (IFNß−1b), and fingolimod have been approved as multiple sclerosis (MS) treatments, they have not yet been included on the National List of Essential Medicines (NLEM) formulary in Thailand. This study aimed to evaluate the cost-utility of MS treatments compared with best supportive care (BSC) based on a societal perspective in Thailand.

A Markov model with cost and health outcomes over a lifetime horizon with a 1-month cycle length was conducted for relapsing–remitting MS (RRMS) patients. Cost and outcome data were obtained from published studies, collected from major MS clinics in Thailand and a discount rate of 3 percent was applied. The incremental cost-effectiveness ratio (ICER) was calculated and univariate and probabilistic sensitivity analyses were performed.

When compared with BSC, the ICERs for patients with RRMS aged 35 years receiving fingolimod, IFNβ−1b, and IFNβ−1a were 33,000, 12,000, and 42,000 US dollars (USD) per quality-adjusted life-year (QALY) gained, respectively. At the Thai societal willingness to pay (WTP) threshold of USD 4,500 per QALY gained, BSC had the highest probability of being cost-effective (49 percent), whereas IFNβ−1b and fingolimod treatments showed lower chance being cost-effective at 25 percent and 18 percent, respectively.

Compared with fingolimod and interferon treatments, BSC remains to be the most cost-effective treatment for RRMS in Thailand based on a WTP threshold of USD 4,500 per QALY gained. The results do not support the inclusion of fingolimod or interferon in the NLEM for the treatment of RRMS unless their prices are decreased or special schema arranged.

While medicines and medical tests are developed in a controlled clinical trial environment, postmarketing surveillance in the real world can be challenging. MedicineInsight—a database of longitudinal patient-level clinical information from primary care practices in Australia—is a novel program that collects primary care data to improve postmarketing surveillance at a national level.

MedicineInsight collects de-identified clinical information from primary care practice information systems using data extraction tools. MedicineInsight currently includes 3.6 million regular patients of 3,300 family physicians (general practitioners) from 650 primary care practices across Australia. MedicineInsight data include longitudinal clinical information on diagnosis and medicines (dose, strength, route of administration, medication switches over time, adverse events, and allergies), and pathology testing data. A series of observational studies was developed for postmarketing surveillance of management of a range of health priorities including type 2 diabetes mellitus (T2DM), chronic obstructive pulmonary disease (COPD), depression, and antibiotics use.

Forty-four percent of patients with T2DM in the MedicineInsight database did not have a recorded hemoglobin A1c result and thirty-one percent did not have a recorded blood pressure reading in the previous 6 months. While guidelines recommend a stepwise approach to the initiation of COPD therapy, forty-nine percent of patients with COPD (with or without asthma) were prescribed dual therapy at initiation and a small number (4.5 percent) were prescribed triple therapy. Between 2011 and 2015, the annual rate of antidepressant prescribing per 1,000 family physician encounters increased by eight percent. High volumes of antibiotics were prescribed for respiratory tract infections in Australian primary care, notwithstanding guideline recommendations that antibiotics are not recommended in most cases.

Large scale, real-world clinical data from primary care practices can play an important role in postmarketing surveillance at a national level.

Severe hypercholesterolemia is a major cause of death in coronary heart disease. New adjunctive drug therapies have passed authorization processes and been launched recently. So far it is not known which properties of the new treatment options generate the highest benefit for patients. The aim was to evaluate patient priorities in the field of adjunctive drug therapy with apheresis. Therapy characteristics were examined as to their relevance to hypercholesterolemia patients.

To identify all potential patient-relevant treatment characteristics, a systematic literature review and ten interviews with patients were conducted. Seven key characteristics were identified from the patient perspective. Patients’ priorities were elicited using an analytic hierarchy process (AHP).

In total, N = 61 patients diagnosed with severe hypercholesterolemia and undergoing apheresis participated in the study. The analysis showed predominance for the attribute “reduction of LDL-C level in blood” (Wglobal:0.362). The “risk of myopathy” (Wglobal:0.164), “risk of neurocognitive impairment” (Wglobal:0.161) and “frequency of apheresis” (Wglobal:0.119) were ranked second, third and fourth. Subgroup analyses revealed that “frequency of apheresis” is of greater importance to younger patients, men and/or patients who indicated a reduction in quality of life due to apheresis.

The essential decision criteria for optimal therapy from the patients’ perspective were obtained. “Reduction of lipoprotein in blood” was ranked highest compared with the “mode of administration” and “side effects” characteristics. The study offers a transparent approach for the identification of patient priorities for adjunctive PCSK9-inhibitor therapy in apheresis-treated hypercholesterolemia. The project can be used by healthcare decision makers to understand the importance of each patient-relevant endpoint.