The Scottish Medicines Consortium (SMC) works in partnership with patient groups and carers to capture their experiences to help inform decisions on new medicines. To better inform their participation in the SMC assessment process, patient groups highlighted a need for information from submitting pharmaceutical companies about the new medicine under review.

We established a multi-stakeholder short life working group (SLWG) to explore how to meet these needs. The group comprised members of the SMC Public Involvement Network (PIN) Advisory Group, representatives of two pharmaceutical companies and the Association of British Pharmaceutical Industries, and the SMC public involvement team. The main outputs were the development of a new Summary Information for Submitting Patient Groups (SIP) form and supporting guidance document. The SIP form completed by the submitting pharmaceutical company is then shared by SMC's Public Involvement Team, to assist submitting patient groups.

The SIP form was implemented in June 2016, and following positive evaluation, became essential for inclusion with the pharmaceutical company's new medicine submission in June 2017. Feedback has been positive, with patient groups reporting that the form includes valuable information that they may not otherwise have been able to access including the positioning of the medicine in the treatment pathway, information on dosage, administration and side-effects. The form is also completed in plain English without overly technical or marketing information. Company representatives who have completed the form state that it provides clear information on the licensed indication, enables accessible scientific evidence for patients and families/carers, and allows them to give accurate and balanced information about the medicine.

Partnership working with key stakeholders has enabled SMC to provide improved information to submitting patient groups. A better understanding of a new medicine may in turn allow patient groups to participate more effectively in the HTA.

The National Institute of Health and Care Excellence (NICE) assesses the efficacy and safety of interventional procedures for use in the National Health Service (NHS). Since 2006, NICE's Public Involvement Programme (PIP) has obtained ‘patient commentary’ to inform committee decisions, using a questionnaire asking patients about their experience of the procedure including benefits, disadvantages and side effects. Commentary is considered by the committee alongside other evidence. The PIP has piloted a project to: capture the impact of the patient commentary on the committee's decision-making; explore patterns of impact; and identify criteria that indicate when patient commentary may not be required.

The pilot included all interventional procedures guidance started between February 2016 and February 2017. Committee members’ views were captured using a form completed whenever patient commentary was considered. Responses were anonymized, entered into an electronic system, analyzed, and correlated against ‘committee comments’ in the published guidance. After twelve months, there was an unrepresentatively narrow spread of conditions, and most topics were updating previously published guidance rather than novel topics. The pilot was therefore extended by six months.

Patient commentary commonly had an impact on decision-making; however, no discernible patterns have yet been identified, nor criteria for when it may not be required. Key findings were: (i) patient commentary is equally useful for guidance updates as novel guidance, and (ii) interpretation and assessment of ‘impact’ varied across committee members but the majority agreed it reinforced the other evidence.

Patient commentary has a measurable impact on committee decision-making. Very occasionally it provides new evidence and routinely provides reassurance that the published evidence is substantiated by real-world patient opinion. Measuring the impact of commentary seems to have raised its profile, with more committee comments about patient issues included in guidance during the pilot than in preceding years. The project needs to be extended to identify which procedures are least likely to benefit from patient commentary and why.

This session will share lessons learned from implementing a comprehensive patient and public engagement framework (developed by winners of the 2017 Egon Jonsson Award) in one government agency's health technology assessment (HTA) process. The presentation will share strategic and operational considerations for successful implementation, and the early effects of patient involvement activities on the agency's HTA recommendations.

This presentation used a case study approach to understand the application of the framework described above.

The comprehensive framework by Abelson and colleagues describes many different public and patient engagement activities that could be conducted at each stage of an HTA process. Health Quality Ontario has chosen to focus on engaging patients to: prioritize topics; develop an additional evidence stream on patient preferences and values; serve on a committee that reviews the HTA, deliberates, and makes recommendations; and provide feedback on draft recommendations. Strategic considerations for these decisions include: aligning engagement activities to an evidence-focused organizational culture, and investing in engagement activities earlier in the HTA process to allow for sufficient consideration of the patient voice in developing recommendations. These activities have impacted the agency's organizational culture, and evidence suggests they have also influenced recommendations for what should be publicly funded. Patient engagement activities have also led to increased feedback from the public and patients for some HTAs and the associated draft recommendations.

Public agencies must make strategic decisions about how and when to invest scarce resources in patient and public engagement. Investing in direct patient engagement as an additional stream of evidence and supporting the involvement of health system users in decision-making has had a significant impact on HTA deliberations and recommendations. For some HTAs, these activities have facilitated greater public engagement as well.

Evidence and guidance alone do not change practice. A multitude of factors are influential upon whether a particular health technology is adopted in practice. The adoption team at the National Institute for Health and Care Excellence (NICE) engages with healthcare professionals to develop specifically tailored support for the adoption of NICE health technology assessments (NICE medical technologies, diagnostics and technology appraisal guidance).

The NICE adoption team uses a structured process which involves engagement of healthcare professionals with experience or knowledge of the technology to identify the barriers to adoption. This information is used to populate the topic selection tool which presents the impact of adopting the technology under five headings: care pathway change; finance; difficulty to implement; education; and, patient acceptance. The result indicates which guidance would benefit from adoption support: plan and develop tailored solutions to address barriers to adoption which include a resource impact assessment and targeted communications; quality assure; and, publish tailored resources.

Examples of tailored outputs include: adoption resources sharing real world experiences of sites that have adopted the technology; and, NICE pilot projects, where the adoption team work closely with sites to support adoption of the technology at a local level. The team then share learning and results from the project to facilitate: engagement with national planning groups to coordinate wider scale adoption; resource impact assessments which help local cost impact of adoption to be estimated; engagement with general and specialist media; and, influencing national tariff.

NICE's processes have evolved to facilitate the development of a wider variety of more tailored resources, to support adoption of NICE health technology assessments guidance into practice. We will continue to engage with healthcare professionals and be responsive in our processes to ensure the packages of adoption support are tailored to need.

Immune globulin (IG) is a publicly funded blood product with high utilization rates and rapidly rising costs. Inappropriate use of IG, particularly in dose and treatment duration, is observed in about 10 percent of cases, and the national guidelines for IG treatment are outdated. To develop a utilization management policy for IG, the Alberta, Manitoba and Saskatchewan Ministries of Health collaborated with health technology assessment (HTA) researchers and clinicians to develop evidence-based guideline recommendations for IG treatment to inform an authorization policy for IG utilization in the provinces.

A multidisciplinary committee comprising HTA researchers and 22 physicians from seven medical specialties adapted recommendations from 43 “seed” guidelines into one locally contextualized IG guideline. HTA methods and rapid review products were used extensively to update gaps in the evidence base. The guideline recommendation document was used to develop a provincial IG utilization management policy. The challenges of achieving a methodologically rigorous guideline development process will be discussed.

The guideline contained over 60 recommendations for IG use in different medical specialties. The health ministries used the guideline recommendations to develop an IG authorization policy. The clinician-sanctioned review criteria were used to construct a conditional reimbursement system for generating outcome data from controlled off-label IG use for conditions where evidence gaps existed, and were built into policies for benchmarking compliance.

Three provinces successfully collaborated to develop an IG utilization management policy. The unique approach involved a credible and transparent process that incorporated key review elements for compliance benchmarking and reimbursement, promoted clinician buy-in, and created a cadre of clinical champions willing to assist in policy development and implementation. The proactive, rather than retroactive, incorporation of clinician-sanctioned benchmarking and review criteria into policy will help bridge the know-do gap and foster a stronger, more direct link between health policy and evidence.

Health technology assessment (HTA) agencies wish to ensure the impact of their HTAs. HTA impact assessment measures the influence of a HTA on decision-making and downstream to patient outcomes. Despite their potential to provide insights, the use of impact assessment frameworks by HTA agencies is limited. Understanding the underlying mechanisms of adopting HTA impact assessment frameworks is therefore important. Using a social cognitions lens, this study aims to provide insights into the enabling and hindering factors associated with the assessment of HTA impact by INAHTA members.

Using an interpretive description design, this cross-sectional study used semi-structured interviews of INAHTA members to gain insight into attitudes, social support, self-efficacy, barriers, and intentions towards HTA impact assessment. Transcriptions were analyzed using a social cognitions lens by two researchers using a constant comparative method to identify themes.

Twenty-six of forty-seven INAHTA members participated. Preliminary results showed that interviewees most often perceived support for assessing impact from their ministry of health or from agency staff. Most interviewees noted challenges to measuring impact at the right time and a lack of human resources, methods, and tools as internal barriers. A lack of transparency and a limited impact assessment culture were perceived as the main external barriers. Interviewees reported feeling fairly confident in overcoming internal barriers, but were less confident in overcoming external barriers. Providing feedback for improvement to HTA processes and making achievements visible were the most frequently reported advantages of assessing impact, whereas its time consuming nature was the biggest disadvantage.

This is the first study to use a social cognitions model to understand HTA impact assessment. Although the results of this convenience sample need to be interpreted with caution, they contribute knowledge on factors that facilitate and hinder agencies in the assessment of impact and illuminate opportunities for developing effective strategies to support HTA agencies in this area.

According to our local data, elderly patients accounted for 14 percent of the population yet, represent 58 percent of hospitalization and, they are more likely to return after discharge. These patients are more likely to return to the hospital following discharge. In order to meet ministerial target for length of stay of patient on a stretcher, the UETMIS-SS was requested to evaluate interventions aiming to improve the fluidity of patient trajectories in the acute care services. The objective of this health technology assessment is to evaluate the effectiveness of discharge planning and transitional care interventions aiming at reducing the readmission rate of the elderly.

An umbrella review was conducted following the PRISMA statement to summarize the scientific evidence. The search was conducted in five databases along with the grey literature search. Two reviewers independently performed the study selection, the quality assessment and the data extraction. To better illustrate the activities and the healthcare professionals (HCP) involved in the interventions, an analytical framework was developed. Results were summarized in a narrative synthesis. The contextual and experiential data were collected through interviews with HCP and directorates from different settings. The level of evidence was and a committee was then held to elaborate the recommendations.

In the nine systematic reviews included in the narrative synthesis, three models were identified: Post-discharge planning and follow-up by the same HCP was established to be effective in reducing the readmission rate. Discharge planning interventions with follow-up by non-specific HCP have been shown to be promising, while discharge planning without follow-up after the hospital discharge has shown to be ineffective in reducing the readmission rate.

An individualized discharge plan, coordination of services and follow-up performed by the same HCP is established to be effective in reducing readmission rate.

Within the local context in Montréal, the elderly population makes up more than 50% of patient hours in emergency department. To meet ministerial targets for length of stay, our health technology assessment unit was requested to conduct an umbrella review to evaluate interventions aimed at reducing health care services use for this population. Within that context, our unit was asked to further evaluate the efficacy of advance practice nurse (APN)-led interventions. The objective of this rapid response was to summarize the scientific literature for APN-led interventions on hospital services use.

An umbrella review using the PRISMA statement was conducted to review the scientific literature. Systematic searches were conducted in five databases, along with a grey literature search. Two reviewers performed the study selection, quality assessment using the ROBIS, and data extraction. The primary studies within the selected systematic reviews were extracted by two reviewers and a meta-analysis was conducted to analyze the efficacy of APN-involved in discharge planning and transitional care.

From the twenty-seven systematic reviews identified in the literature search, four reported data on APN-led interventions. In all, sixteen primary studies were included in the four systematic reviews. While most studies focused on transitional care, there was heterogeneity in the components of the interventions implemented. At six months post-discharge, a reduction of forty-one percent in relative risk of readmission was observed with APN-led discharge planning and transitional care with patient education, follow-up and services coordination. Studies with fewer components reported less significant results than studies with comprehensive discharge planning and transitional care. The few APN-led primary care studies identified in the systematic reviews reported inconsistent results.

APN-led comprehensive discharge planning and transitional care can reduce hospital readmission rate. Several components were identified and should be considered in the discharge planning and transitional care.

The purpose of this study is to gather evidence on safety and overall effectiveness of three alternative technologies for gait rehabilitation in diplegic children with Cerebral Palsy: robotic, conventional and joint conventional and robotic gait training.

A new methodology, decision-oriented health technology assessment (DoHTA), was applied to assess the technology on clinical, technical, organizational, economic, social and ethical, legal and safety domains. This method, conceived as a hospital-based HTA tool for supporting the introduction of innovative technologies, has been implemented merging the EUnetHTA Core Model® with the Multi-Criteria Decision Analysis. In particular, the general items of the EUnetHTA Core Model® are re-formulated as performance indicators and re-placed along a decision tree structure that, from the one hand, respects the original top-down design of the EUnetHTA model (growing level of detail from domains to issues) and, from the other hand, allows obtaining a quantitative evaluation of each identified performance indicator.

The multiple indicators, which have been identified for the seven domains, play important and different roles in the alternative technologies evaluation. DoHTA results showed that robotic system offers the possibility to control more accurately the exerted forces and movement trajectories than the traditional therapy. It gives the possibility to measure the task performances parameters and to receive the patient feedback simultaneously. To carry out robotic gait rehabilitation fewer therapists are required compared with the conventional therapy, resulting in lower therapists’ physical workload.

Despite the great perspectives that robotics offer to motor rehabilitation, it seems that robotic gait training could not provide greater benefits in terms of motor and functional recovery compared to the conventional therapy. Preliminary results, supported by most recent literature evidence, lead to the hypothesis that joint use of robotic and conventional therapy can produce better clinical outcomes than the separate use of the two rehabilitation techniques.

In 2016, a health technology assessment (HTA) was conducted to gather evidence on the safety and overall effectiveness of performing laparoscopic surgery by using 3D videolaparoscopy (3DVL) versus 2D videolaparoscopy (2DVL) display systems in a variety of pediatric surgical procedures in order to efficiently support the final investment decision on video system to be acquired. Results showed that 3DVL might be a good alternative to 2DVL. Moreover, sensitivity analysis has also confirmed that the results associated to the best technology (3DVL) are robust; this has led to a confident decision for recommending it in Bambino Gesù Children's Hospital (OPBG). The objective of this work is to evaluate the impact of 3DVL within the hospital setting after 12 months its introduction in clinical practice.

After 12 months since the technology's introduction, clinical data, identified in previous HTA study, were extracted from surgery registries; data concerning the number of surgeries, duration of intervention, blood loss and surgery complications were analyzed. Statistical analyses on these data, between pre and post 3D system implementation period were carried out.

Results confirmed the 2016 HTA results, highlighting clinical advantages identified a priori. The percentage of the number of laparoscopic procedures significantly increased from 12 percent in pre-3D system installation period to the 20 percent in post 3D system installation (p=7,35E-6). No statistical differences in length of hospital stay, operative time, incidence of perioperative blood loss and surgery complication, between pre- and post- 3D installation period were identified.

This study highlighted the importance of a HTA process before the acquisition of a technology for which the investment decision is not obvious, because benefits and drawbacks of the new technology are unclear. Preliminary results showed that 3D video laparoscopy system seems to be better than the 2D laparoscopy system. However, more data has to be examined to be able to establish the final judgement.

Patients with adolescent idiopathic scoliosis frequently receive X-ray imaging at diagnosis and subsequent follow monitoring. To achieve the ALARA concept of radiation dose, a biplanar low-dose X-ray system (BLDS) has been proposed. The aim of the study is to gather evidence on safety, accuracy and overall effectiveness of a BLDS compared with CT scanning, in a pediatric population, in order to support the final decision on possible acquisition of such innovative diagnostic system.

The new method Decision-oriented HTA (DoHTA) was applied to carefully assess the diagnostic technology. It was developed starting from the EUnetHTA Core Model® integrated with the analytic hierarchy process in order to identify all the relevant assessment aspects of the technology involved, identified from scientific literature, experts’ judgments and specific context analysis of Bambino Gesù Children's Hospital. A weight was associated to each assessment element and the alternatives’ ranking was defined.

This innovative system provides orthopedic images in standing or sitting position, being able to examine the spine and lower limbs under normal weight-bearing conditions. This system is recommended for particular clinical indications as scoliosis and other congenital deformities of the spine. It is able to acquire simultaneous posteroanterior and lateral images in a single scan without vertical distortion and with lower radiation exposure than CT scanning. 2D images acquired can be combined to obtain a 3D reconstruction scanning based on a semi-automated statistical model.

The major advantages of BLDS are the relatively low dose of radiation and the possibility of obtaining a 3D reconstruction of the bones. Our preliminary results show that data on the clinical effectiveness are limited but the technical advancements of BLDS appear promising in terms of patient management and patient health outcomes associated with its use.

Current organization of mental health services in Canada imposes a rupture during youth transition to adulthood, when severe mental health disorders start appearing. This can have a major impact on youth recovery and social integration. A health technology assessment (HTA) was initiated to evaluate the efficacy of programs that simultaneously target adolescents and young adults to support decision making.

A systematic review of systematic reviews was conducted. Four databases were searched (MEDLINE, Embase, Applied Social Sciences Index and Abstracts, and CINAHL) for articles published between 2000 and 2017. Article selection and quality assessment (ROBIS tool) were performed and inter-rater agreement was measured. To be included, the systematic review had to study specialized models or programs serving both adolescents and young adults. An analytical framework was constructed based on the categorization of performance measures for early intervention and the five dimensions of recovery. Group and individual interviews were conducted to collect contextual and experiential data.

A total of 1,054 references were identified. After applying the selection criteria, five systematic reviews were selected. The majority of programs identified were developed for early psychosis. This HTA did not identify specialized programs for other types of mental illness or at-risk youth. Evidence on early interventions for psychosis is emerging in regards to their efficacy in improving functional and clinical recovery. However, evidence has yet to be established for their impact on access. Contextual and experiential data from our organization validated and completed the scientific findings. Facilitating and constraining factors in the implementation of a person-centered care model and inter-agency collaboration were identified.

Services targeting at-risk youth should be developed as part of a continuum of care that is adapted to clinical stages so that all youths living with psychological distress can be treated, regardless of diagnosis or age. These services may draw inspiration from models of early intervention for psychosis. Recommendations from this HTA are currently being put into action in the West Island of Montreal.

An increasing number of anti-cancer medications are indicated for multiple tumors. Existing pharmacoeconomic evaluations routinely examine the cost-effectiveness (CE) and budget impact (BI) of such drugs by indication, as and when each indication is reviewed. The impact of indication-specific conclusions on the holistic value of such medications across all indicated patients is not currently evaluated, yet is important to stakeholders including health technology assessment (HTA) agencies, payers and patients. We introduce a holistic framework that considers the value of multiple indications together at a product level. Application of this approach is illustrated via an example across multiple indications for a novel, targeted anti-cancer therapy (pembrolizumab) in Canada.

Previously-HTA-evaluated indication-specific CE and BI models serve as the foundation for this multi-indication model. Comparing to standard of care (SoC) per indication, the model evaluates the potential BI, clinical outcomes and CE of pembrolizumab among the individual indications along with the overall multi-indication patient population from the perspective of a third-party payer. For the contextual model, incremental costs and quality-adjusted life years (QALYs) were weighted using indication populations derived from national incidence rates.

The indication-specific incremental cost-effectiveness ratios (ICER) from CE analyses of ipilimumab-treated advanced melanoma, ipilimumab-naïve advanced melanoma, second-line non-small cell lung cancer (NSCLC), first-line NSCLC and fourth-line classical Hodgkin lymphoma range from USD 52 K to USD 163 K per QALY. Accounting for the relative contributions of the various sizes of indication-specific patient populations results in an overall ICER for pembrolizumab vs. SoC of USD 100 K.

A holistic model can provide stakeholders with a tool to evaluate the overall value of multi-indication drugs. Results enable an understanding of the outcomes and economic consequences of treatment with pembrolizumab versus SoC by both individual indications and across all indications. Insights from this contextual approach will enable data from less-developed clinical trials to be considered when previously they might have gone unevaluated by decision-makers.

The cost-effectiveness of endovascular therapy (EVT) compared to tissue plasminogen activator (tPA) alone for acute ischemic stroke (AIS) has been established in the literature. However, decision-makers still face challenges of how to best deliver EVT in a timely manner to maximize patient outcomes while minimizing the burden to the healthcare system, given that AIS has time-dependent treatment outcomes. The objective of this presentation is to report an optimization approach for improving health system value and outcomes for patients with AIS who are eligible for EVT in Alberta.

An economic model was developed to compare combinations of “mothership” (transport directly to a comprehensive stroke center [CSC] to receive tPA and EVT) and “drip-and-ship” (transport to a primary stroke centre to receive tPA, followed by transport to a CSC to receive EVT) methods across Alberta. The model considered geographical variation and searched for the best delivery methods through a pairwise comparison of all possible strategies. The controlled variables including in the model were population densities, disease epidemiology, time/distance to hospitals, available medical services, treatment eligibility and efficacy, and costs. Patient outcomes were measured by functional independence. The model defined optimal strategies by identifying the transport methods that produced the highest probability of improved health outcomes at the lowest cost.

The analysis produced an optimization map showing optimal strategies for EVT delivery. The lifetime cost (standard deviation [SD]) per patient and likelihood (SD) of good outcomes was CAD 291,769 (CAD 11,576) [USD 226,207 (USD 8,975)] and 41.82 percent (0.013) when considering optimal clinical outcomes, and CAD 287,725 (CAD 4,141) [USD 223,097 (USD 3,211)] and 41.67 percent (0.016) when considering optimal economic efficiency.

Our model reduces the gap that exists between health technology implementation and cost-effectiveness analysis; namely, neither fully addresses relative efficiency driven by geographical variation, which may misrepresent system value in local settings. Implementation strategies generated in our model capture full values in terms of patient outcomes and costs.

Fully probabilistic analyses are now standard for economic models, with all parameters varied according to probability distributions. Using univariate sensitivity analyses to explore the influence of different parameters on the model results are also standard. Although there are several approaches available, there has been little discussion of the merits of each or justification for the method used in any given analysis. The aim of this study was to compare three approaches to univariate sensitivity analysis using a case study.

We considered three univariate sensitivity analysis approaches: (i) set one parameter at its upper and lower bounds while all others are set at their mean value; (ii) analysis of variance; and (iii) set one parameter at its mean and vary all others. We compared these approaches using an economic model of mechanical thrombectomy for the treatment of acute ischemic stroke, considering outcomes of incremental costs, incremental quality-adjusted life-years (QALYs), and net monetary benefit (NMB).

For incremental costs and QALYs the correlation between the approaches was moderate to high, with correlation coefficients between 0.46 and 0.94. For NMB the correlation between approaches was also high (range 0.89 to 0.98), but some of the most influential parameters were ranked differently. Setting one parameter at its upper and lower bounds was the only method that facilitated an analysis of direction of influence.

The three approaches addressed different but relevant questions. Setting individual parameters at their bounds is effectively a systematic scenario analysis and may be misleading to decision makers. Analysis of variance may be more easily interpreted, but it has disadvantages. Setting a parameter at its mean, while varying other parameters, is similar to value of information analysis. As with any sensitivity analysis, it is imperative that the uncertainty associated with each parameter is adequately captured in the model.

People with traumatic brain injuries (TBIs) commonly report memory impairments which are persistent, debilitating, and reduce quality of life. As part of the Rehabilitation of Memory in Brain Injury trial, a cost-effectiveness analysis was undertaken to examine the comparative costs and effects of a group memory rehabilitation program for people with TBI.

Individual-level cost and outcome data were collected. Patients were randomized to usual care (n=157) or usual care plus memory rehabilitation (n=171). The primary outcome for the economic analysis was the EuroQol-5D quality of life score at 12 months. A UK NHS costing perspective was used. Missing data was addressed by multiple imputation. One-way sensitivity analyses examined the impact of varying different parameters, and the impact of available cases, on base case findings whilst non-parametric bootstrapping examined joint uncertainty.

At 12 months, the intervention was GBP 26.89 (USD 35.76) (SE 249.15) cheaper than usual care; but this difference was statistically non-significant (p=0.914). At 12 months, a QALY loss of −0.007 was observed in the intervention group confidence interval (95% CI: −0.025–0.012) and a QALY gain seen in the usual care group 0.004 (95% CI: -0.017–0.025). This difference was not statistically significant (p=0.442). The base case analysis gave an ICER of GBP 2,445 (USD 3,252) reflecting that the intervention was less effective and less costly compared to usual care. Sensitivity analyses illustrated considerable uncertainty. When joint uncertainty was examined, the probability of the intervention being cost-effective at a willingness-to-pay threshold of GBP 20,000 per QALY gain was 29 percent and 24 percent at GBP 30,000.

Our cost-utility analysis indicates that memory rehabilitation was cheaper but less effective than usual care but these findings must be interpreted in the light of small statistically non–significant differences and considerable uncertainty was evident. The ReMemBrIn intervention is unlikely to be considered cost-effective for people with TBI.

There is growing evidence that many people attending annual screening for diabetic retinopathy in the United Kingdom (UK) are at low risk of developing the disease. This has led to new policy statements. However, the basis on which to establish a risk-based individualized variable-recall screening program has not yet been determined. We present a methodology for using information on an individual's risk factors to improve the allocation of resources within a screening program.

We developed a patient-level state-transition model to evaluate the cost-effectiveness of risk-based screening for diabetic retinopathy in the UK. The model incorporated a recently developed risk calculation engine that predicts an individual's risk of disease onset, and allocated individuals to alternative screening recall periods according to this level of risk. Using the findings, we demonstrate a means of estimating: (i) a threshold level of risk, above which individuals should be invited to screening, and (ii) the optimum screening recall period for an individual, based on the expected cost-effectiveness of screening and treatment.

The cost-effectiveness analysis demonstrated that standardized screening (current practice) is the least cost-effective program. Individualized screening can improve outcomes at a reduced cost. We found it feasible – though computationally expensive – to incorporate a risk calculation engine into a decision model in Microsoft Excel. In an optimized screening program, the majority or patients would be invited to attend screening at least two years after a negative screening result.

Individualized risk-based screening is likely to be cost-effective in the context of diabetic eye disease in the UK. It is expected that risk calculation engines will be developed in other disease areas in the future, and used to allocate screening and treatment at the individual level. It is important that researchers develop robust methods for combining risk calculation engines into decision analytic models and health technology assessment more broadly.

Recently developed direct-acting antiviral (DAA) treatments for hepatitis C virus (HCV) are groundbreaking in their high efficacy across disease genotypes and lack of severe side effects. This study used a cost-of-illness (COI) approach to estimate the net value conferred by one of these novel drug combinations, sofosbuvir and velpatasir (SOF/VEL), recently licensed for generic manufacture in India.

This study considered COI from lifetime earnings lost due to disability and premature death from HCV infection. Risk of death and disability in future years was calculated using a Markov state-transition model with parameters determined from the literature. The future earnings of sampled patients were predicted using an empirical earnings model, with coefficients determined from India Human Development Survey data. Costs to both the patient and secondarily infected individuals were considered.

Preliminary results suggested that curing individuals diagnosed with chronic HCV in India would preserve INR 3.7 million (USD 55,750) in earnings per person. For non-cirrhotic (NC) and compensated cirrhotic (CC) individuals, the expected benefits associated with preventing secondary infections were worth between one and forty-one percent of the value of benefits conferred to the diagnosed individuals (depending on sex and extent of liver damage). Treating decompensated cirrhotic (DC) individuals with DAAs alone offered minimal earnings benefits because these individuals will likely remain disabled and unable to work without a liver transplant. Expected net benefits of treatment were substantial for NC and CC patients, ranging from INR 640,349 (USD 9,531) for NC women to INR 10.7 million (USD 158,968) for CC men. The cost of treatment for DC individuals exceeded the expected earnings benefits.

For average NC and CC individuals, the cost of treatment with SOF/VEL is offset by the benefits of increased future productivity. Increased earnings are not sufficient to offset the cost of treatment for DC individuals, but treatment may still be justified on the basis of the intrinsic value of health improvements and other treatment benefits.

The high prevalence, disability, and work absenteeism associated with back pain make it the single most costly musculoskeletal health condition in developed countries. However, the majority of back pain has no identifiable pathological cause and resolves without surgery or imaging. This paradox suggests that we need to change how back pain is managed to reduce unnecessary burden to individuals and the healthcare system. This study evaluated the cost of a new model of early triage-based, interprofessional care for patients with back pain.

We evaluated the outcomes and cost of implementing a provincial care pathway for early assessment of patients with back pain at three sites: (i) adjacent to an emergency department in a community hospital; (ii) co-located with an orthopedic surgeon's clinic in a hospital; and (iii) in a primary care network (PCN) with private practice physiotherapists and chiropractors. Time-driven activity-based costing (TDABC), in combination with discrete event simulation, was used to estimate costs.

Costs were significantly lower in the models that used hospital-based physiotherapists and in the PCN model that used private practice physiotherapists and chiropractors to triage patients. These costs ranged from CAD 20 (USD 16) to manage patients identified with low severity back pain to CAD 175 to 200 (USD 137 to 156) for those with moderate to severe back pain. Models that implemented the care pathway using family physicians and surgeons to review non-surgical patients were more expensive at CAD 339 (USD 265) and CAD 514 (USD 402), respectively.

New models of care that use the skills of physiotherapists and chiropractors to assess and triage patients with back pain adjacent to emergency departments and in the primary care sector are cost effective, compared with traditional physician-led models. The overarching intent is to use these data to enable evidence-informed policy and practice changes, so that more appropriate and cost-effective care is provided to patients with back pain.

Proponents of precision oncology report that genomic testing has the potential to reduce health system costs and improve patient health. Yet, testing also involves significant expenditures that challenge the sustainability of adopting technologies into routine practice. Our study explores the availability and scope of economic evaluations of precision oncology informed by next-generation sequencing (NGS).

We searched Medline (PubMed), Embase (Ovid), and Web of Science databases for English-language full-text peer reviewed articles published between 2000 and 2016. We focused our search on articles that estimated the benefit of precision oncology in relation to its costs. We excluded studies that did not undertake full economic evaluations or did not focus on NGS. We reviewed all included studies and summarized key methodological and empirical study characteristics.

Fifty-five economic evaluations met our inclusion criteria. The first study was published in 2005 and the number of published studies increased steadily, from three studies between 2005 and 2007 to twenty-six between 2014 and 2016. Most studies evaluated multiplex panels (86 percent). Testing was frequently used to diagnose patients (24 percent) or predict prognosis (67 percent), rather than identify targeted therapies (7 percent). Methods varied considerably and cost-effectiveness differed according to test type, test strategy, and cancer type. Deterministic and probabilistic analyses were typically used to characterize uncertainty (91% percent and 75% percent).

While the availability of economic evidence examining precision oncology increased over time, methods used often did not align with current guidelines. Future evaluations should undertake extensive sensitivity analysis to address all sources of uncertainty associated with rapidly changing NGS technologies. Further, additional research is needed evaluating the cost-effectiveness of more comprehensive next-generation technologies prior to implementing these on a wider scale.