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.

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.

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.

Trastuzumab is the most recent biological therapy incorporated by the Brazilian Health Ministry for HER-2 overexpressed metastatic breast cancer treatment (2012). The aim of this study was to investigate if access to this technology is appropriate.

We performed a web-based questionnaire, which received answers from October 2016 to April 2017. Oncologists that work in the care of patients with overexpressed HER-2 metastatic breast cancer were the focus of the survey. Forty-three professionals informed work location, sector (public, private or both) and trastuzumab access. This research was approved by Brazilian Ethics Committee (CAE 59076316300005260).

Among 43 valid answers, nine informed they work in the public sector, 10 in the private sector and 24 in both sectors. In total, 33 reported to work in public and 34 in private sector. We observed that 17 (51.5 percent) participants who work in the public sector do not have access to trastuzumab, while in private sector only one participant (2.9 percent) reported the lack of access to this technology for HER-2 overexpressed metastatic breast cancer treatment. Regarding the respondents who informed the lack of access, six (33.3 percent) work in Northeast Brazilian region, six (33.3 percent) in Southeast, two (11.1 percent) in South, one (5.6 percent) in Central-West and three (16.7 percent) did not give this information. Eleven respondents reported they do not have another treatment option for these patients, while seven informed access only to chemotherapy without biological therapy.

Trastuzumab is a biological therapy that can increase HER-2 overexpressed metastatic breast cancer patients overall survival by nine months on average. The questionnaire results indicate that its access in Brazil is still irregular, mainly in public sector, even five years after its incorporation by the Brazilian Health Ministry. Although universal access is one of Brazilian Public Health System main directives, there is evidence of a serious issue regarding its services equity.

Reimbursement decisions are usually based on evidence from randomized controlled trials (RCT) with high internal validity but lower external validity. Real-World Data (RWD) may provide complimentary evidence for relative effectiveness assessments (REA's) and cost-effectiveness assessments (CEA's) of treatments. This study explores to which extent RWD is incorporated in REA's and CEA's of drugs used to treat metastatic melanoma (MM) by five Health Technology Assessment (HTA) agencies.

Dossiers for MM drugs published between 1 January 2011 and 31 December 2016 were retrieved for HTA agencies in five countries: the United Kingdom (NICE), Scotland (SMC), France (HAS), Germany (IQWiG) and the Netherlands (ZIN). A standardized data-extraction form was used to extract data on RWD mentioned in the assessment and its impact on appraisal (for example, positive, negative, neutral or unknown) for both REA and CEA.

In total, fourty-nine dossiers were retrieved: NICE = 10, SMC = 13, IQWiG = 16, HAS = 8 and ZIN = 2. Nine dossiers (18.4 percent) included RWD in REA's for several parameters: to describe effectiveness (n = 5) and/or the safety (n = 2) of the drug, and/or the prevalence of MM (n = 4). CEA's were included in 25/49 dossiers (IQWiG and HAS did not perform CEA's). Of the twenty-five CEA's, twenty (80 percent) included RWD to extrapolate long-term effectiveness (n = 19), and/or identify costs associated with treatments (n = 7). When RWD was included in REA's (n = 9), its impact on the appraisal was negative (n = 4), neutral (n = 2), unknown (n = 1) or was not discussed in the appraisal (n = 2). When RWD was included in CEA's (n = 11), its impact on the appraisal varied between positive (n = 2), negative (n = 5) and unknown (n = 4).

Generally, RWD is more often included in CEA's than REA's (80 percent versus 18.4 percent, respectively). When included, RWD was mostly used to describe the effectiveness of the drug (REA) or to predict long-term effectiveness (CEA). The impact of RWD on the appraisal varied greatly within both REA's and CEA's.

Technology assessment in hospital traditionally involves parameters of safety, effectiveness and costs. The prosperity of medical innovations in an era of scarce resources requires more precisely refined methodologies to measure ‘added value’. Our aim was to reveal the added values of technologies by asking professionals to prioritize their adoption into hospitals.

Twelve innovative technologies that were discussed for adoption over three years were controversial regarding their actual “added value”. Fifty-two managerial health professionals ranked these technologies on two scales: hierarchic importance (league scale) and comparative score rating (CSR), reflecting willingness-to-pay (WTP). The distribution of ranking indicates the internal agreement (IA) among the participants.

There was only partial correlation between the two scales. For example, glucose-monitoring was ranked ‘highly important’ on the hierarchic (league) scale with high CSR/WTP, but with low IA. This can be interpreted as “a valuable technology but with disagreement on comprehensive adoption in the entire hospital”. The surgical robot was ranked ‘highly important’ on the hierarchic scale with low CSR/WTP, but with high IA, meaning “a valuable technology but with consensus to delay adoption in the hospital”. Overall, the participants raised thirty-two “values” that can be assorted into five clusters of significance: clinical effect (6 values), social/public dimension (8 values), patient-physician interaction (9 values), technological aspect (5 values) and policy-regulatory perception (4 values).

We identified different ‘;patterns’ for defining the ‘value’ of various technologies. Revealing these aspects can create a “set of values” of relative weights that may explain the added value considerations in prioritization of decision making. Interestingly, there were technologies that were ranked low, but achieved a high rating. This can be explained by individual personal-oriented added value perspectives. Using this innovative tool to incorporate social value-based scores can assist in understanding the determinants, beyond the current traditional rationing mechanism, that guide professionals while prioritizing medical technologies.

Recruitment and retention of participants in randomized controlled trials (RCTs) is challenging, and is why many RCTs fail or are not completed on time. Digital approaches such as social media, data mining, email or text messaging could improve recruitment and/or retention, but how well they match these purposes is unclear. We used systematic methods to map the digital approaches that have been investigated during the past 10 years.

We searched Medline, Embase, other databases and relevant web sites in July 2018 to identify comparative studies of digital approaches for recruiting and/or retaining participants in clinical or health RCTs. Two reviewers screened references against protocol-specified eligibility criteria. Studies included were coded by one reviewer (with 20 percent checked by a second reviewer) using pre-defined keywords to describe characteristics of the studies, populations and digital approaches evaluated.

We identified 9,133 potentially relevant references, of which 100, reporting 101 unique studies, met the criteria for inclusion in the map. Among these, 95 percent of studies investigated recruitment but only 11 percent investigated retention. Study areas included health promotion and public health (36 percent), cancer (17 percent), circulatory system disorders (13 percent) and mental health (10 percent). Most study designs were observational (89 percent). The most frequent digital approaches for recruitment were internet sites (53 percent of recruitment studies), social media (42 percent), television or radio (31 percent) and/or email (31 percent). For retention these were email (63 percent of retention studies) or text messaging (38 percent). Time and costs of recruitment were reported in 17 percent and 30 percent of recruitment studies respectively, whilst costs were reported in 19 percent of retention studies.

A wide range of digital approaches has been studied, in many combinations. Evidence gaps include lack of experimental studies; studies on retention; and studies for specific populations (e.g. children or older people) and outcomes (e.g. user satisfaction). More robust experimental studies, perhaps conducted as studies-within-a-trial (SWAT), are needed to address knowledge gaps and ensure that estimates of digital tool effectiveness and efficiency are reliable.

The COVID-19 pandemic led to a significant surge in clinical research activities in the search for effective and safe treatments. Attempting to disseminate early findings from clinical trials in a bid to accelerate patient access to promising treatments, a rise in the use of preprint repositories was observed. In the UK, NIHR Innovation Observatory (NIHRIO) provided primary horizon-scanning intelligence on global trials to a multi-agency initiative on COVID-19 therapeutics. This intelligence included signals from preliminary results to support the selection, prioritisation and access to promising medicines.

A semi-automated text mining tool in Python3 used trial IDs (identifiers) of ongoing and completed studies selected from major clinical trial registries according to pre-determined criteria. Two sources, BioRxiv and MedRxiv are searched using the IDs as search criteria. Weekly, the tool automatically searches, de-duplicates, excludes reviews, and extracts title, authors, publication date, URL and DOI. The output produced is verified by two reviewers that manually screen and exclude studies that do not report results.

A total of 36,771 publications were uploaded to BioRxiv and MedRxiv between March 3 and November 9 2020. Approximately 20–30 COVID-19 preprints per week were pre-selected by the tool. After manual screening and selection, a total of 123 preprints reporting clinical trial preliminary results were included. Additionally, 50 preprints that presented results of other study types on new vaccines and repurposed medicines for COVID-19 were also reported.

Using text mining for identification of clinical trial preliminary results proved an efficient approach to deal with the great volume of information. Semi-automation of searching increased efficiency allowing the reviewers to focus on relevant papers. More consistency in reporting of trial IDs would support automation. A comparison of accuracy of the tool on screening titles/abstract or full papers may help to support further refinement and increase efficiency gains.

This project is funded by the NIHR [(HSRIC-2016-10009)/Innovation Observatory]. The views expressed are those of the author(s) and not necessarily those of the NIHR or the Department of Health and Social Care.