Health state utilities measured by the generic multi-attribute utility instruments (MAUIs) differ. Empirical evidence suggests that some MAUIs are more sensitive than others in reflecting the quality of life (QoL) of patients in particular disease areas. Additionally, in order to estimate utilities based on cancer-specific health-related quality of life instruments (CSQoLs), a number of mapping functions have emerged. Although it is common practice to apply a CSQoL instead of a MAUI in clinical trials, CSQoL cannot be used to estimate utility values for economic evaluations. Mappings based on MAUIs that are not sensitive to changes in cancer patients’ QoL may result in misleading approximations of utilities that could affect allocation of resources. The study objective is to explore the validity and sensitivity of the major MAUIs to variation in the QoL measured by cancer-specific instruments. We aimed to investigate (i) the sensitivity of the general MAUIs scores to changes in the CSQoL, and (ii) whether particular dimensions of the general instrument are more sensitive.

A two stage systematic literature review is conducted. First, an update of the review done by McTaggart-Cowan et al. (2013) on the mapping methods used to determine utilities from cancer-specific instrument. Second, an analysis of studies that measure the relationship between CSQoLs and general MAUIs.

The literature suggests that differences exist between MAUIs in their capacity to capture the QoL dimensions of the CSQoLs. Additionally, the main challenge to build an appropriate mapping function for deriving utilities values from CSQoL is the definition of an appropriate methodology that (i) responds to the distribution of the selected sample and (ii) can successfully be validated in additional samples.

In the context of health technology assessment and cost effectiveness analysis, it is crucial to carefully select and report the CSQoL and MAUI involved in the estimation of the additional benefits. Policy makers need to be awarded of the sensitivity of the instruments to changes in QoL in relation to the CSQoL dimensions QoL.

Focal-onset-seizures (FOS) are commonly experienced by individuals with epilepsy and have a significant impact on quality of life (QoL). This study aimed to develop a mapping algorithm to predict the 6 dimension short form questionnaire (SF-6D) values in adults with FOS for use in economic evaluations of a new treatment, cenobamate.

An online survey, including questions on sociodemographic, disease history, the short form (SF) 36, and an epilepsy-specific measure (quality of life in epilepsy problems questionnaire, QOLIE-31-P) was administered to individuals with drug-resistant FOS in the top 5 EU countries (UK, Spain, Germany, Italy and France). A range of regression models were fitted to SF-6D scores including direct and response mapping approaches.

The analysis included 361 people. In the previous 28 days, the mean number of FOS experienced was three, (range: 0–43) and longest seizure-free period was 14 days (range: 1–28). Mean responses on all SF-36 dimensions were lower than general population norms. Mean SF-6D and QOLIE-31-P scores were 0.584 and 45.72, respectively. The best performing model was the ordinary least squares (OLS), with root mean squared error (RMSE) and mean absolute error (MAE) values of 0.0977 and 0.0742, respectively. Explanatory variables which best predicted SF-6D included seizure frequency, seizure severity, seizure freedom, and age.

People with drug-resistant FOS have poor QoL. The mapping algorithm enables the prediction of SF-6D values from clinical outcomes in individuals with drug-resistant FOS. It can be applied to outcome data from clinical trials to facilitate cost-utility analysis.

We sought to examine patients’ perceptions of disease burden and treatment impact, and what patients value over the course of the asthma experience.

Patient focus groups were conducted in three countries, the USA (n = 8 patients), the United Kingdom (n = 7 patients), and Germany (n = 7 patients), to examine aspects of disease burden and patient experience (physical, emotional, clinical, economic, and social). Cause and effect were also explored. Global Initiative for Asthma guidelines were used to screen patients by severity, based on age, sex, time since diagnosis, number of attacks, oral corticosteroid use, and number of therapies needed to control symptoms. Patients classified and ranked aspects of disease burden, including whether it was continuous or episodic in nature, and discussed the interventions used to manage their asthma.

The overall burden of illness was driven by the combined effect of the disease and treatment burdens. All patients highlighted the negative impact of oral corticosteroids. Patients believed that they were the key actors in their asthma management (not healthcare professionals), and reported the physical and emotional burdens as being the most dominant. Understanding of the terms “attack” or “exacerbation” differed significantly between patients, and did not necessarily match the clinical definitions. Patients considered asthma to be an individualized condition that drives lifestyle changes; disease management drives burden and vice versa. Patients perceived that burden was continuous over time, with specific phases of variable duration—before, during, and after an attack—whereas other stakeholders had a more episodic focus. Patients expected more holistic and personalized approaches for managing their asthma.

The research indicated a misalignment between what patients value and what clinicians, payers, and regulators consider in their assessments and decisions. Greater alignment among the different stakeholders, and more inclusion of patients’ values in decision making, will improve outcomes.

Lack of evidence regarding safety and effectiveness at market entry is driving the need to consider adopting a lifecycle approach to evaluating medical devices, but it is unclear what lifecycle evaluation means. This research sought to explore the tacit meanings of “lifecycle” and “lifecycle evaluation” as embodied within evaluation models/frameworks used for medical devices.

Drawing on qualitative evidence synthesis methods and using an inductive approach, novel methods were developed to identify, appraise, analyze, and synthesize lifecycle evaluation models used for medical devices. Data was extracted (including purpose; audience; characterization; outputs; timing; and type of model) from key texts for coding, categorization, and comparison, exploring embodied meaning across four broad perspectives.

Fifty-two models were included in the synthesis. They demonstrated significant heterogeneity of meaning, form, scope, timing, and purpose. The “lifecycle” may represent a single stage, a series of stages, a cycle of innovation, or a system. “Lifecycle evaluation” focuses on the overarching goal of the stakeholder group, and may use a single or repeated evaluation to inform decision-making regarding the adoption of health technologies (Healthcare), resource allocation (Policymaking), investment in new product development or marketing (Trade and Industry), or market regulation (Regulation). The adoption of a lifecycle approach by regulators has resulted in the deferral of evidence generation to the post-market phase.

Using a “lifecycle evaluation” approach to inform reimbursement decision-making must not be allowed to further jeopardize evidence generation and patient safety by accepting inadequate evidence of safety and effectiveness for reimbursement decisions.

Malignant tumors have become a major public health problem and their treatment cost is increasing rapidly in China, but treatment aimed at healing diseases or extending patients’ life. There is little empirical research on utilization of healthcare resources of terminally ill cancer patients. In order to explore the optimal treatment decision for patients and provide information for relevant decision makers, this study analyzed the consumption status of medical resources in patients with cancer during the whole treatment period, and the current medical resource utilization efficiency in different levels of hospital for end-stage cancer patients.

This study was based on the clinical treatment and payment data of 2,536 cases of patients with lung cancer from the medical insurance database during the period of 2007 to 2014 in Hubei province. We retrospectively analyzed patients’ medical expenditure and utilization of different medical resources during their whole treatment period as well as at the end stage.

The per capita inpatient expenditures of patients under 50 years old was 193,000 CNY (27,451 USD), while that of the patients over 70 years were 80,000–90,000 CNY (11,379–12,802 USD). Secondly, the medical expenditures spent during the last 6 months of life accounted for 66.1 percent of the total expenditures. Lastly, the medical expenditure spent in tertiary hospitals accounted for 95.3 percent of the total expenditure, and the expenditure was 14,200 ± 17,030 CNY (2,019.82 ± 2,422.36 USD) per visit.

Population aging is not the only factor causing the rise of medical expenditure. The unclear objectives of treatment and the reverse of medical resource allocation are also important factors to boost the growth of medical expenditure. It is necessary to improve the healthcare insurance payment system, strengthen the capacity of primary medical institutions, and develop the palliative care system in China.

To facilitate implementation of new health technology assessment (HTA) methods, it is crucial to engage stakeholders. A technically good system may be brought to its knees if the intended users are not willing or able to use it. Therefore, based on these considerations, we aimed to identify relevant aspects of change models and investigated four potentially useful change models in the context of HTA. The four models were: adaptive space; midstream modulation; developmental evaluation; and knowledge brokering.

A narrative literature review was conducted to gather information into a readable and usable format. PubMed and Google Scholar were searched for relevant literature on change management and stakeholder engagement within HTA. Additionally, grey literature was selected after consulting an implementation specialist to gather more information on the background of the change management models.

Several enabling factors for successful stakeholder engagement were found, including attention to branding of the coproduction, facilitation/personal safety, and data or indicators to inform activities. Four change methods were described from the enabling factors identified. There was no “perfect model” for our aim, but all models involved relevant aspects to engage stakeholders. Notably, all models paid attention to the project management factor, whereas none of the models paid explicit attention to the branding of the coproduction factor.

Change management is a complex and elaborate field in which many factors play a role. Stakeholder engagement is a factor that might be influenced by project leaders within international projects such as the European Union's Next Generation Health Technology Assessment (HTx). To improve stakeholder engagement in modernizing HTA, it is important to keep paying attention to project management, relationships, and how to facilitate fora and meetings to improve mutual understanding. Two factors to pay more attention to are branding of the coproduction and consideration of formal structures.

The Hospital de Clínicas de Porto Alegre (HCPA), a public teaching hospital, has a Hospital-based Health Technology Assessment (HB-HTA) unit to support the decision-making process on technology incorporation, rationalization or disinvestment. In 2017, the plastic adhesive drape was standardized at HCPA for use in cardiovascular, digestive, orthopedic, and neurological surgery for the purpose of preventing surgical site infection (SSI). This study evaluated whether the plastic adhesive drape technology is more effective than the no adhesive drapes in the surgical procedures in which it is used in the HCPA, so as to support the medical board's decision regarding the rationalization of use.

The primary outcome was the surgical site infection rate (SSI). Searches were performed in PubMed, Cochrane and national and international health agencies: World Health Organization (WHO), National Institute for Health and Care Excellence (NICE), Centers for Disease Control and Prevention (CDC), Society for Healthcare Epidemiology of America (SHEA), Brazilian National Commission for the Incorporation of Technologies (CONITEC) and Brazilian National Health Surveillance Agency (ANVISA) databases. The search strategy combined terms related to the technology and types of surgery in which it is used in the HCPA. The quality of the included studies was assessed. Additionally, data on technology utilization and costs in the hospital were analyzed.

Technology assessment followed AdHopHTA project recommendations. Data from the hospital showed that the technology has been used in fifteen surgical specialties, different from the proposed incorporation, with a progressive increase in consumption from 2017 to 2018. The literature review included a systematic review with seven clinical trials, which concluded that the plastic adhesive drape lacks benefits, with potential for increased risk of SSI. The evidence was of moderate quality.

The expenses associated with the use of the technology were considered unjustified as it is not reimbursed by the Brazilian Ministry of Health and its disinvestment was recommended. The Medical Board approved the disinvestment of the technology based on the evidence found by the HB-HTA unit, and the medical staff complied with the decision.

The aim of this initiative was to examine collaboratively, in a multi-stakeholder team (health technology assessment (HTA) practitioners with patient involvement expertise, health technology industry, patient advocates, health policy experts, patient engagement experts), whether evidence generated through social media research (SMR) fills current information gaps relating to insights on specific aspects of patient experiences, preferences, or patient needs and delivers additional value to HTA.

The framing of the project was done in a co-creative, deliberative multi-stakeholder process. Challenge and refinement happened through discussions with 25 independent stakeholders from HTA bodies, industry, academia, and patient advocacy. For critical themes identified during the framing phase, scoping literature reviews were performed including the state of methods and examples for the use of SMR in HTA.

The framing and stakeholder discussions specified a set of expectations and requirements, and the scoping reviews revealed the current state of methods and usage of SMR in health-policy decision making.

The project concluded that SMR can contribute new, relevant evidence to HTA. It is however recommended to evolve the science through defining best practices when planning, conducting, and using SMR and to conduct multi-stakeholder pilot SMR projects to address questions relevant to current HTAs and to validate and improve the proposed practices.

Inhibitors of Vascular Endothelial Growth Factor (VEGF) have made possible the treatment of the Retinal Vascular Diseases (RVD) for which there were limited therapeutic resources. The aim of this work was to estimate annual direct costs of major macular edema diseases in Italy in particular Choroidal Neovascularization (CNV), Diabetic Macular Edema (DME) and Retinal Vein Occlusions (RVO) from the hospital perspectives.

This descriptive study was aimed at quantifying direct costs incurred by five hospitals in Italy. Administrative and clinical databases of Policlinico Tor Vergata in Rome were analyzed for a 6-year period. In this context, it was possible to stratify patients depending on the disease and number of eyes treated. From these results, a survey with structured questionnaires was developed involving four other hospitals in Italy. Thanks to that, direct costs (drugs and specialist) were estimated from the hospitals perspective in 2016.

Interviews included 7,356 individuals of which 1,860 were treated in both eyes. Within the considered five hospitals, 64 percent of treated patients had CNV, 21 percent DME, and 15 percent RVOs. The average annual administration rate of anti-VEGF treatment resulted in 4.03 (Standard Deviation, SD 3.46) per patient eye: 4.69 (SD 1.75) for cases enrolled for less than one year (naïve) and 3.38 (SD 0.82) per patients treated for more than one year (experienced). Naïve patients had a mean per capita annual cost of EUR2,368 per eye (EUR2,536 for CNV; EUR2,280 RVO; EUR1,986 DME) of which EUR2,952 was related to the administration of on-label drugs mainly Eylea, Lucentis, Macugen, Ozurdex and EUR49 due to off-labels such as Avastin. Experienced patients average annual cost per eye was EUR1,689: EUR2.179 for the on-label drugs, EUR34 due to off-labels (EUR1,839 for CNV; EUR1,327 RVO; EUR1,399 DME). The average rate of the specialist annual visit was four times; the most frequent types were Optical Coherence Tomography (OCT), Angiography, and Fundus Photography (FP).

This is a first attempt to study direct costs incurred from the hospital perspective associated with RVD with overexpression of VEGF in Italy. This might represent a first step for further analysis assessing the burden of RVD diseases from the Italian National Health System perspective globally.