Patient involvement in drug evaluation decision making is increasing. The aim of the current study was to develop a multi-criteria decision analysis (MCDA) framework that would enable the inclusion of the patient perspective in the selection of appropriate criteria for MCDAs being used in the value assessments of oncologic drugs.

A literature review was conducted to identify and define criteria used in drug assessments from patient perspectives. The Evidence and Value: Impact on Decision Making methodology was used to develop a MCDA framework. Identified criteria were discussed by a sample of oncology patient association representatives who decided which criteria were important from patient perspectives. Selected criteria were rated by importance. The preliminary MCDA framework was tested through the assessment of a hypothetical oncology treatment. A discussion was carried out to agree on a final pilot MCDA framework.

Twenty-two criteria were extracted from the literature review. After criteria discussion, sixteen criteria remained. The most important criteria were comparative patient reported outcomes (PRO), comparative efficacy and disease severity. After the discussion generated by the scoring of the hypothetical oncology treatment, the final pilot MCDA framework included seven quantitative criteria (“disease severity”, “unmet needs”, “comparative efficacy / effectiveness”, “comparative safety / tolerability”, “comparative PROs”, “contribution of oncological innovation”) and one contextual criterion (“population priorities and access”).

The present study developed a pilot reflective MCDA framework that could increase patient's capability to participate in the decision-making process by providing systematic drug assessments from the patient perspective.

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 recent development of value frameworks to inform healthcare resource allocation responds to a demand to make the decision-making process more inclusive and explicit. The objectives of the 2018 Latin American (LAtam) Health Technology Assessment International (HTAi) Policy Forum were to explore the current international experiences and to discuss the potential application of value frameworks in Latin America.

A background paper, presentations, and group discussions of Policy Forum members (43 participants, 12 LAtam countries represented) at the 2018 HTAi Policy Forum meeting informed this paper.

Participants agreed that HTA and decision making based on more comprehensive and inclusive value frameworks could improve health system effectiveness, efficiency, sustainability, and equity; promote transparency in the decision process; sustain a more comprehensive assessment of technologies; and facilitate stakeholder participation as well as accountability of decisions. Criteria that were identified as essential to be included in a value framework for LAtam were burden of illness and severity of the disease, effectiveness and safety of the technology, quality of the evidence, cost-effectiveness, and budget impact. Potential challenges identified for the application of value frameworks in LAtam, included scarcity of human resources and delays in the assessment process.

Forum participants agreed that the next steps should be to identify appropriate processes and methodologies, adapted to the context of each country, regarding the application of value frameworks to improve the link between HTA and decision making.

There is little evidence in China regarding the cost-effectiveness of non-invasive prenatal testing (NIPT) for Down syndrome (DS). This study aims to evaluate the cost-effectiveness of NIPT and provide evidence to inform decision-making.

To determine the cost-effectiveness of NIPT for DS, a decision-analytic model was developed using the TreeAge Pro software from a societal perspective in a simulated cohort of 10 000 pregnant women. Main indicators were based on field surveys from sampled hospitals in four locations in China and a literature review.

The conventional maternal serum screening (CMSS) strategy, contingent screening strategy (NIPT delivered to high risk pregnant women after CMSS), and universal screening strategy could prevent 3.02, 7.53, and 9.97 DS births, respectively. NIPT would decrease unnecessary invasive procedures, resulting in fewer procedure-related miscarriages. The cost-effectiveness ratio of the contingent screening strategy was the lowest. When compared with the CMSS strategy, the incremental cost per DS birth averted by the contingent screening strategy and universal screening strategy were USD 20,160 and 352,388, respectively. One-way sensitivity analysis showed that, if the cost of NIPT could be decreased to USD 76.92, the cost-effectiveness ratio of the universal screening strategy would be lower than the CMSS strategy.

Although NIPT has the merits of greater effectiveness and safety, CMSS is unlikely to be replaced by NIPT at this time because of NIPT's higher cost. Contingent screening may be an appropriate strategy to balance the effectiveness and cost factors of the new genetic testing technology.

Written evidence is submitted to the National Institute for Heath and Care Excellence (NICE) by patient organisations for all ultra-orphan evaluations. To enhance the capability of patient involvement at NICE and to further develop understanding of how patient generated evidence and input in ultra-orphan conditions can support the Health Technology Assessment (HTA) agencies beyond 2020, the Public Involvement Programme systematically reviews the impact the evidence has on committee decision making.

This study captured data from September 2017 to August 2018 for seven ultra-orphan evaluations. A paper questionnaire was given to each committee member to complete for each evaluation and entered in to an online system for analysis. Findings were used to inform the committee views which were highlighted in feedback letters to the patient groups. The questions included: how much impact and what sort of impact the patients had; both qualitative and quantitative data; and, a specific question on clarification of quality of life data

We obtained 83 responses showing the submissions: had a moderately high or high impact; gave the committee particular insight into quality of life data not provided elsewhere; provided new evidence; interpret the data from other sources; and, demonstrated consistency with other sources

Patient evidence is particularly useful for ultra-orphan conditions where other forms of evidence are limited. Patients can provide a unique insight into the burden of disease, the patient population, any updates of treatments and the impact on patient and carers. They provide real life data to the committee including information that standard Quality Adjusted Life Years measures do not. Evidence varied by condition with impact themes highlighting the effects on patient and carers including fear, stress and anxiety. The examples are recorded, updated annually and will be shared with national patient groups and offered internationally through the HTAi Interest Group on Patient and Citizen Involvement.

Uncontrolled pain in advanced cancer is a common problem and has significant impact on individuals’ quality of life and use of healthcare resources. Interventions to help manage pain at the end of life are available, but there is limited economic evidence to support their wider implementation. We conducted a case study economic evaluation of two pain self-management interventions (PainCheck and Tackling Cancer Pain Toolkit [TCPT]) compared with usual care.

We generated a decision-analytic model to facilitate the evaluation. This modelled the survival of individuals at the end of life as they moved through pain severity categories. Intervention effectiveness was based on published meta-analyses results. The evaluation was conducted from the perspective of the U.K. health service provider and reported cost per quality-adjusted life-year (QALY).

PainCheck and TCPT were cheaper (respective incremental costs -GBP148 [-EUR168.53] and -GBP474 [-EUR539.74]) and more effective (respective incremental QALYs of 0.010 and 0.013) than usual care. There was a 65 percent and 99.5 percent chance of cost-effectiveness for PainCheck and TCPT, respectively. Results were relatively robust to sensitivity analyses. The most important driver of cost-effectiveness was level of pain reduction (intervention effectiveness). Although cost savings were modest per patient, these were considerable when accounting for the number of potential intervention beneficiaries.

Educational and monitoring/feedback interventions have the potential to be cost-effective. Economic evaluations based on estimates of effectiveness from published meta-analyses and using a decision modeling approach can support commissioning decisions and implementation of pain management strategies.

This review aims to assess the cost-effectiveness of psychological interventions for schizophrenia/bipolar disorder (BD), to determine the robustness of current evidence and identify gaps in the available evidence.

Electronic searches (PsycINFO, MEDLINE, Embase) identified economic evaluations relating incremental cost to outcomes in the form of an incremental cost-effectiveness ratio published in English since 2000. Searches were concluded in November 2018. Inclusion criteria were: adults with schizophrenia/BD; any psychological/psychosocial intervention (e.g., psychological therapy and integrated/collaborative care); probability of cost-effectiveness at explicitly defined thresholds reported. Comparators could be routine practice, no intervention, or alternative psychological therapies. Screening, data extraction, and critical appraisal were performed using pre-specified criteria and forms. Results were summarized qualitatively. The protocol was registered on the PROSPERO database (CRD42017056579).

Of 3,864 studies identified, 12 met the criteria for data extraction. All were integrated clinical and economic randomized controlled trials. The most common intervention was cognitive behavioral therapy (CBT, 6/12 studies). The most common measure of health benefit was the quality-adjusted life-year (6/12). Follow-up ranged from 6 months to 5 years. Interventions were found to be cost-effective in most studies (9/12): the probability of cost-effectiveness ranged from 35-99.5 percent. All studies had limitations and demonstrated uncertainty (particularly related to incremental costs).

Most studies concluded psychological interventions for schizophrenia/BD are cost-effective, including CBT, although there was notable uncertainty. Heterogeneity across studies makes it difficult to reach strong conclusions. There is a particular need for more evidence in the population with BD and for longer-term evidence across both populations.

Advances in health care due to the development and introduction of new drugs and medical devices have brought considerable benefits to people and patients in terms of upgraded quality of life and extended years of survival. However, some are concerned that the very advancement of health care would increase further the inflation of national healthcare costs. In response to these concerns, Japan's Central Social Insurance Medical Council (“Chuikyo”) began in 2012 to examine how cost-effectiveness evaluation might be applied to the national health insurance system, and has been working toward establishing a system for its usage.

Cost-effectiveness evaluation was adopted on a trial basis in fiscal year (FY) 2016, targeting seven drugs and six medical devices. Analyses and re-analyses were performed by manufacturers and a public expert organization, respectively. Based on these analyses, a cost-effectiveness evaluation expert organization conducted an overall assessment (“appraisal”). Results of the evaluation were used to adjust the prices of the target items.

Following the trial adoption of cost-effectiveness evaluation, price adjustments were performed for three items in April 2018. Meanwhile, a decision was also made to examine seven items for which technical requirements were identified due to differences in the understanding of analysis methods between involved parties.

The Chuikyo will examine how to meet the newly identified technical requirements and discuss specific details with regard to establishing a system that incorporates cost-effectiveness evaluation. The Chuikyo plans to reach a conclusion by the end of FY 2018.

Health technology assessment (HTA) has been widely used to inform coverage decisions in high-income countries over the past few decades and has been getting increasing attention in middle-income countries as a tool for healthcare decision making in recent years. This study aims to use the case of the Maternal and Child Health Voucher Scheme (MCHVS) in Myanmar to understand how HTA can have a policy impact in a low or lower middle-income country.

The stages heuristic framework was used to describe the policy-making process. A document review was conducted and tacit knowledge of researchers involved was recorded.

The opportunity for a grant propelled maternal and child health to the policy agenda. An ex-ante HTA, which included a model-based health economic evaluation, informed the design of the scheme. The framework and key parameters from the ex-ante HTA were used for a mid-term review, which provided feedback to the policy implementation process. An ex-post HTA involved fielding a household survey to assess the impact of the scheme.

HTA can be a useful method for informing resource allocation throughout the policy process in low and lower middle-income settings where no formal mechanism for making coverage decisions exists.

The cost-effectiveness of molecular pathology testing is highly context dependent. The field is fast-moving, and national health technology assessment may not be relevant or timely for local decision makers. This study illustrates a method of context-specific economic evaluation that can be carried out in a limited timescale without extensive resources.

We established a multi-disciplinary group including an oncologist, pathologists and a health economist. We set out diagnostic and treatment pathways and costs using registry data, health technology assessments, guidelines, audit data, and estimates from the group. Sensitivity analysis varied input parameters across plausible ranges. The evaluation setting was the West of Scotland and UK NHS perspective was adopted. The evaluation was assessed against the AdHopHTA checklist for hospital-based health technology assessment.

A context-specific economic evaluation could be carried out on a timely basis using limited resources. The evaluation met all relevant criteria in the AdHopHTA checklist. Health outcomes were expected to be at least equal to the current strategy. Annual cost savings of £637,000 were estimated resulting primarily from a reduction in the proportion of patients receiving intravenous infusional chemotherapy regimens. The result was not sensitive to any parameter. The data driving the main cost saving came from a small clinical audit. We recommended this finding was confirmed in a larger population.

The method could be used to evaluate testing changes elsewhere. The results of the case study may be transferable to other jurisdictions where the organization of cancer services is fragmented.

Smart device apps for diabetes have the potential to support patients in their daily disease management. However, uncertainty exists regarding their suitability for empowering patients to improve self-management behaviors. This paper addresses a general research gap regarding theoretically based examinations of empowerment in diabetes research, by examining how diabetes app features correspond with conceptual indicators of empowerment.

We examined features of 121 apps for diabetes self-management available in Singapore, with the second highest proportion of diabetes among developed nations, for psychological empowerment (feeling of empowerment) and for behavioral empowerment (social support).

Diabetes apps studied offered a narrow range of features, with limited feature-sets corresponding to indicators of empowerment. Customization as a strategy to improve perceived relevance of diabetes self-management as an indicator of psychological empowerment was especially limited. Moreover, there was a lack of features supporting patients' communication with healthcare professionals and within their private social networks.

Mobile apps for diabetes self-management failed to provide relevant features for empowering patients. Specific practical recommendations target improved adoption, sustained usage, and effectiveness of diabetes self-management apps.

El reciente desarrollo de marcos de valor responde a una demanda por hacer más explícito e inclusivo el proceso de toma de decisiones sobre asignación de recursos sanitarios. El objetivo del Foro de Políticas en Latino América 2018 de Health Technology Assessment International fue explorar las experiencias internacionales y discutir la potencial aplicación de marcos de valor en la región.

Este manuscrito está basado en un documento base y en las presentaciones y discusiones mantenidas por los miembros del Foro (43 participantes, 12 países) durante el Foro del 2018.

Los participantes coincidieron en que un proceso de evaluación y toma de decisión basado en marcos de valor más inclusivos y abarcativos podría mejorar la efectividad, eficiencia, sustentabilidad y equidad; promover la transparencia y una evaluación más completa de las tecnologías, mejorar la rendición de cuentas de las decisiones y la participación de actores. Se identificaron como criterios esenciales para ser incluidos en marcos de valor en la región a la carga de enfermedad y severidad de la condición, la efectividad y seguridad de la tecnología, la calidad de la evidencia, la costo-efectividad e impacto presupuestario. Los potenciales desafíos para su implementación identificados incluyeron la escasez de recursos humanos y las demoras en el proceso de evaluación que se podrían producir.

Los participantes remarcaron que los próximos pasos deberían ser identificar procesos y metodologías apropiadas, adaptadas al contexto de cada país, para facilitar la aplicación de marcos de valor y mejorar el vínculo entre la evaluación y la toma de decisiones.

The Scottish Medicines Consortium (SMC) advises NHS Scotland on the clinical and cost-effectiveness of new medicines. Since 2014, evidence from patients and carers on end-of-life and orphan medicines has been gathered during Patient and Clinician Engagement (PACE) meetings. The output is a consensus statement which describes the added value of a new medicine from the perspective of the patient/carer and clinician. This study investigates the importance of factors identified through PACE to committee members and how these are used in their decision-making.

Survey methodology was used to gain an understanding of the factors from the PACE statement that are most likely to influence members (n = 26) in decision-making. The survey instrument was informed by a literature review and observation of PACE and SMC meetings. Likert scale questions were used to determine the relative importance of factors in the PACE statement, including information relating to eight prominent ‘quality of life’ themes (family/carer impact, health benefits, tolerability, psychological benefit, hope, normal life, treatment choice and convenience), that were identified by an earlier thematic analysis of these statements.

Analysis of survey responses will use mainly descriptive techniques to generate percentages and ranges. Correlation analysis will be considered to investigate relationships between members’ demographics, type of medicine (end-of-life, orphan) and the importance of different factors in the PACE statement. Preliminary results indicate that key quality of life themes highly valued by patients/carers are also important to committee members in their decision making. Challenges in assimilating qualitative patient-based evidence from PACE alongside quantitative clinical and economic data were highlighted.

Findings from this survey will provide valuable insight into how PACE evidence is used by SMC decision makers alongside traditional clinical and economic evidence and will help shape future improvements to the PACE methodology.

Neuroendocrine tumors (NETs) are rare, slow-growing malignant tumors. So far, there are no data on patient preferences regarding its therapy. This empirical study aimed to elicit patient preferences in the drug treatment of NET.

Based on qualitative patient interviews and an analytic hierarchy process, six patient-relevant attributes were analyzed and weighted using a discrete-choice experiment. Patients were recruited with the help of a NET support group. An experimental 3*3 + 6*3 –MNL design was created using NGene. The design consisted of eighty-four choices, divided into seven blocks. Participants were randomly assigned to these blocks. The analysis included random parameter logit and latent class models.

A total of 275 participants (51.6 percent female; mean age, 58.4 years) were included. The preference analysis within the random parameter logit model, taking into account the 95 percent confidence interval, showed predominance for the attribute “overall survival.” The attributes “response to treatment” and “stabilization of tumor growth” followed. The side effects “nausea/vomiting” and “diarrhea” were considered of relatively equal importance. Latent class analysis of possible subgroup differences revealed three preference patterns.

Preferences can influence therapeutic decisions. Preference analyses indicated that “overall survival” had the strongest influence, with participants clearly weighing outcome attributes higher than side effect attributes. In conclusion, mono-criterial decisions would not fully reflect patient perspectives.

We sought to assess the presence and reporting quality of peer-reviewed literature concerning the accuracy, precision, and reliability of home monitoring technologies for vital signs and glucose determinations in older adult populations.

A narrative literature review was undertaken searching the databases Medline, Embase, and Compendex. Peer-reviewed publications with keywords related to vital signs, monitoring devices and technologies, independent living, and older adults were searched. Publications between the years 2012 and 2018 were included. Two reviewers independently conducted title and abstract screening, and four reviewers independently undertook full-text screening and data extraction with all disagreements resolved through discussion and consensus.

Two hundred nine articles were included. Our review showed limited assessment and low-quality reporting of evidence concerning the accuracy, precision, and reliability of home monitoring technologies. Of 209 articles describing a relevant device, only 45 percent (n = 95) provided a citation or some evidence to support their validation claim. Of forty-eight articles that described the use of a comparator device, 65 percent (n = 31) used low-quality statistical methods, 23 percent (n = 11) used moderate-quality statistical methods, and only 12 percent (n = 6) used high-quality statistical methods.

Our review found that current validity claims were based on low-quality assessments that do not provide the necessary confidence needed by clinicians for medical decision-making purposes. This narrative review highlights the need for standardized health technology reporting to increase health practitioner confidence in these devices, support the appropriate adoption of such devices within the healthcare system, and improve health outcomes.

Some systematic review topics are context-sensitive and informed by evidence about a specific geographic setting. Smart information retrieval methods are required to identify such evidence in an efficient manner. This presentation will discuss how validated geographic search filters enable smart literature searching for context-sensitive reviews using the National Institute for Health and Care Excellence (NICE) United Kingdom (UK) filters for MEDLINE and Embase (OVID) as examples. The NICE UK filters were developed in 2016. The filters demonstrated high recall and high precision, however, further research was required to confirm these results.

In 2018, the filters’ recall of references from 100 UK-based multidisciplinary reviews was calculated. Reproducible search strategies were identified from twenty-six of the 100 reviews in MEDLINE and from nine reviews in Embase. From this, the precision and number-needed-to-read (NNR) were calculated.

The MEDLINE filter achieved 96 percent recall (1401 out of 1454 UK references), 2.1 percent precision and a NNR of forty-seven. The Embase filter achieved 97 percent recall (1520 out of 1560 UK references), 0.7 percent precision and a NNR of 146. Compared to not using a filter, the MEDLINE and Embase filters reduced the number of search results by an average of 87 percent and 80 percent, respectively.

The filters retrieve the majority of evidence for UK topics while reducing search result volumes and so enable smart literature searching for context-sensitive topics. Large literature search result volumes can increase development time-frames for systematic reviews. Using the filters can therefore save time for reviews with a UK focus. There are currently two other validated geographic search filters for Africa and Spain. It is hoped that the NICE UK filters’ successful retrieval performance will encourage the development of validated search filters for more geographic regions.