Cancer drug prices are high on the policy agenda worldwide. Previous research found no association between cancer drug benefits and prices at the time of regulatory approval. Drugs approved in the US with uncertain benefits may have spill-over effects in other settings. Our objective was to compare the evidence supporting cancer drug approvals in the US and Brazil, and to examine the association between cancer drug prices and availability of added therapeutic benefit.

We matched all novel cancer drugs approved in the US from 2010–2019 to approvals in Brazil. We extracted data on pivotal study design characteristics and outcomes in the US and Brazil, and evidence supporting price approval in Brazil, including availability of added therapeutic benefit.

From 2010–2019, fifty-six cancer drugs with matching indications were approved in US and Brazil and had their prices authorized in Brazil by December 2020. Drug were available in Brazil following a median 522 days after US approval (IQR: 351–932). In the US, thirty-four (60.7 percent) of the drugs had pivotal randomized controlled trials (RCTs) and Twelve (21.4 percent) had overall survival benefit. By the time of Brazilian approval, forty-one (73.2 percent) drugs had pivotal RCTs and twenty-two (39.3 percent) had overall survival benefit. A total of twenty-eight (50 percent) drugs did not demonstrate added therapeutic benefit over other authorized drugs for the same indication and had a median reduction from requested to approved price of 6.1 percent (IQR: 0–27.8 percent) in Brazil. The twenty-seven (48.2 percent) drugs with added therapeutic benefit had a median price reduction of 2.0 percent (IQR: 0–9.2 percent).

Half of new cancer drugs approved in Brazil failed to demonstrate added therapeutic benefit. The Brazilian pricing system secured considerable price reductions, ensuring that prices for medicines with no added therapeutic benefit were not higher than existing treatments for the same approved indication. Although evidence was more mature by the time of Brazilian review, pivotal studies often lacked randomization and overall survival endpoints.

An early economic evaluation to inform the translation into clinical practice of a spectroscopic liquid biopsy for the detection of brain cancer. Two specific aims are (1) to update an existing economic model with results from a prospective study of diagnostic accuracy and (2) to explore the potential of brain tumor-type predictions to affect patient outcomes and healthcare costs.

A cost-effectiveness analysis from a UK NHS perspective of the use of spectroscopic liquid biopsy in primary and secondary care settings, as well as a cost–consequence analysis of the addition of tumor-type predictions was conducted. Decision tree models were constructed to represent simplified diagnostic pathways. Test diagnostic accuracy parameters were based on a prospective validation study. Four price points (GBP 50-200, EUR 57-228) for the test were considered.

In both settings, the use of liquid biopsy produced QALY gains. In primary care, at test costs below GBP 100 (EUR 114), testing was cost saving. At GBP 100 (EUR 114) per test, the ICER was GBP 13,279 (EUR 15,145), whereas at GBP 200 (EUR 228), the ICER was GBP 78,300 (EUR 89,301). In secondary care, the ICER ranged from GBP 11,360 (EUR 12,956) to GBP 43,870 (EUR 50,034) across the range of test costs.

The results demonstrate the potential for the technology to be cost-effective in both primary and secondary care settings. Additional studies of test use in routine primary care practice are needed to resolve the remaining issues of uncertainty—prevalence in this patient population and referral behavior.

The use of most recent Transcatheter aortic valve implants (TAVI) in the treatment of symptomatic severe aortic stenosis (SAS) is evolving with expanded indications from inoperable/high-risk to intermediate and low risk patients. Consequently, TAVI outcomes must be monitored to highlight its value under real-world conditions. Our aim was to prospectively evaluate TAVI (SAPIEN 3) outcomes in terms of patient's health-related quality of life (HRQoL), clinical outcomes, and healthcare resource utilization (HRU).

An observational prospective study including all consecutive patients with SAS undergoing a transcatheter valve implantation with Edwards SAPIEN 3 valve (transfemoral access) was conducted in full accordance with clinical guidelines from the European Society of Cardiology. Patients were evaluated before the intervention (baseline), at discharge, and after one, six and twelve months from the implant. A thoughtful and systematic evaluation of patients’ HRQoL (EQ-5D 5L, the Short Form-36 Health Survey -SF-36- and the Kansas City Cardiomyopathy Questionnaire -KCCQ-), clinical endpoints (that is, cardiovascular mortality, and rates of stroke, major bleeding, myocardial infarction, and re-hospitalization), echocardiographic measurements, and HRU (that is, Length of stay-LOS- in ward/intensive care unit -ICU-) was implemented. Multivariate regression models were applied to test outcomes while controlling key risk factors (that is, patient’ severity at baseline).

A total of seventy-six patients (fifty percent female, fifty-five percent of intermediate-high risk) with a mean age of 82.1 ± 4.78 years were included. Implant success was 97.34% and cardiovascular death was 2.6% at one year. Significant reductions in mean and maximum gradients were achieved and maintained during follow-up. Mean LOS in ward (5.2 ± 4.0days) and ICU (0.22 ± 0.64 days) were low. Statistically significant improvements were detected in the KCCQ overall summary scores, EQ-5D, and SF-36 (Physical component summary) - all adjusted - p < 0.05 - after the intervention.

TAVI represents a safe and effective innovation for SAS with clinical benefits translated into significant improvements in terms of HRQoL. Besides, the low HRU provides new insights for health-economic modelling and the optimization of limited resources of special importance under current pandemic situation.

The implementation of the electronic prescribing system follows certain objectives, and users’ perspectives can contribute to understanding the efficiency and effectiveness of this system. This study aimed to evaluate physicians’ perspectives on the efficiency and effectiveness of the electronic prescribing system.

This study was conducted on all physicians using the electronic prescribing system in clinics and hospitals affiliated with the treatment deputy of the Social Security Organization (SSO) in Sistan and Baluchistan Province in Iran. Data were collected using a self-administered questionnaire containing three sections: (i) Six items related to demographic data and clinical experience, (ii) Specific questions based on a five-point Likert scale-related physicians’ perspectives about efficiency (19 questions) and effectiveness (13 questions), and (iii) Open-ended questions about the positive and negative aspects of using the electronic prescribing system.

The mean and standard deviation of the efficiency and effectiveness of the electronic prescribing system were 3.68 ± 0.67 and 3.84 ± 0.65, respectively. Patient safety had the highest mean score among all dimensions (4.0 ± 0.64). Most participants (n = 55, 79%) considered the efficiency and effectiveness of this system high. More than 90 percent of the physicians (n = 63) believed that the electronic prescribing system enables a better medication prescription by providing alerts and access to patients’ medication history.

The findings showed that most physicians believed that the electronic prescribing system of Iran's SSO has high efficiency and effectiveness. In particular, physicians believed that using this system improves patient safety and reduces costs.

An important reason for receiving non-beneficial treatment at end-of life is the lack of timely discussions on goals of care and end-of-life preferences. A recent randomized clinical trial demonstrated that patients primed with a questionnaire on their end-of-life preferences were more likely to initiate such conversations with their doctors. Our objective is to integrate the questionnaire into a smartphone application to facilitate early goals of care discussions. To achieve this goal, we first plan to undertake a feasibility study to understand stakeholder preferences.

As part of a quality improvement initiative at our Canadian quaternary-care hospital, we conducted focus groups with oncology and palliative care physicians and patients to understand barriers to early conversations on end-of-life preferences, and to assess feasibility of using smartphone technology in facilitating these conversations. The app would integrate a questionnaire to patients and send prompts to physicians on patient readiness and timing of conversations.

We conducted separate focus groups with lung cancer patients (n = 6) and clinicians in oncology (n = 6) and palliative care (n = 6). Clinical teams expressed enthusiasm about early conversations but raised several barriers including system (lack of electronic documentation and access to data; multiple physicians), clinician (lack of time) and patient (stigma associated with end-of-life) barriers. Clinicians agreed that an app could overcome some of these barriers such as access to patient and electronic data by making patients the repository of all their data and empowering them to initiate discussions. However, they raised concerns about universal accessibility of such technology, especially among the elderly. Patient focus groups will take place in March 2021 and inform us on feasibility in this population.

There is a consensus among physicians at our hospital that early end-of-life conversations have the potential to mitigate adverse events and that use of a smart phone app could facilitate such conversations.

The New Treatment Fund (NTF), launched in January 2017, aims to support the faster introduction of new medicines recommended by the National Institute for Health and Care Excellence (NICE) and the All Wales Medicines Strategy Group (AWMSG). The NTF requires seven health boards and one trust to make recommended medicines available within 60 days of any positive recommendation decision. The project goal was to develop a system for demonstrating how monitoring the NTF improves medicines access for the people of Wales.

The process was derived via a series of task and finish group meetings with relevant stakeholders. The monitoring criteria were agreed through a collaborative expert approach using a nominal group technique. This determined a minimal dataset of formulary status, which included time to formulary addition. Pre-NTF medicines data (n = 59) were available for a six-month period.

By the three-year milestone of the NTF, the average time taken for newly recommended medicines (n = 219) to become available to patients across Wales had decreased by eighty-five percent from 90 to 13 days (p < 0.01).

An innovative and robust system has been created for accurately monitoring the formulary addition of medicines within the NTF, supporting the rapid and comprehensive uptake of medicines deemed clinically and cost effective by NICE and the AWMSG.

Music therapy (MT) is a complementary creative arts treatment aimed at maintaining, restoring, and furthering physical, emotional, and mental health. This systematic review aimed to assess the effectiveness of MT for the treatment of autism spectrum disorder, dementia, depression, insomnia, and schizophrenia. In addition, the MT methods used for these indications were analyzed.

For this update of five Cochrane reviews, four databases (Medline, Embase, The Cochrane Library, and PsycINFO) were systematically searched for studies published from 2013 to 2020. Two review authors independently performed the study selection and data extraction. The methodological quality of the included trials was assessed using the Risk of Bias in Non-randomised Studies - of Interventions (ROBINS-I) tool and the Cochrane Risk of Bias tool for randomized controlled trials.

Ten RCTs (1,248 patients) met the inclusion criteria. For schizophrenia, no study could be included. MT improved the following: behavior, social communication, and the parent-child relationship in patients with autism; mood for patients with depression; and sleep quality for patients with insomnia. In patients with dementia, MT enhanced mood, behavior (severe disease stage), and cognitive function, whereas cognition was unchanged. Memory was improved only in the mild disease stage. None of the studies observed any significant long-term effects of MT in these patient groups. Both active (playing music) and receptive (listening to music) methods were used for dementia, whereas active methods were applied for autism spectrum disorder and depression. For insomnia, only receptive methods were used.

The findings of this update of reviews provides evidence that MT may help patients diagnosed with an autism spectrum disorder, dementia, depression, insomnia, or schizophrenia. It is crucial to focus on patient-related evidence-based health care. MT improves physical, psychological, and social aspects, but more research investigating the long-term effects of MT in these patient groups is needed as it is crucial to know how long the effects of MT last.

Potential therapies and interventions for COVID-19 are emerging and developing rapidly. In a response to this public health emergency, the European Network for Health Technology Assessment (EUnetHTA) aims to support health policy in preparation for evidence-based purchasing. To monitor the emerging evidence, a new EUnetHTA product was created: Rolling Collaborative Reviews (RCRs).

RCRs are living documents that are descriptive in nature, updated monthly, and centrally coordinated. They are based on the following three sources of information: (i) published randomized controlled trials (RCTs) presented as a summary of efficacy and safety data (synthesized for a network meta-analysis conducted by the Department of Epidemiology Lazio Regional Health Service, Italy); (ii) published prospective observational studies for safety results, provided by the Map of COVID-19 Evidence conducted by the Norwegian Institute of Public Health, Norway; and (iii) RCTs registered in clinical trial registries (ClinicalTrials.gov, EudraCT Register, and the ISRCTN registry). Additionally, detailed stopping and starting rules were defined.

As of November 2020, 14 RCRs were ongoing. From the initial list of RCRs, one was suspended due to lacking effectiveness and two moved on to rapid collaborative reviews due to European Medicines Agency approvals. Four RCRs are updated on a bimonthly basis due to a lack of high-quality evidence, and five new RCRs will be started because of promising clinical studies.

RCRs can be a means of providing timely and continuous policy support, but they require a high level of coordinated effort.

Health technology reassessment (HTR) is a structured evidence-based assessment of an existing technology in comparison to its alternatives. The process results in the following four outputs: (i) increased use; (ii) decreased use; (iii) no change; or (iv) de-adoption. However, implementing these outputs remains a challenge. Knowledge translation (KT) can be applied to implement findings from the HTR process. This study sought to identify which characteristics of KT theories, models, and frameworks (TMFs) could be useful, specifically for decreasing the use of or de-adopting a technology.

A qualitative descriptive approach was used to ascertain the perspectives of international KT and HTR experts on the characteristics of KT TMFs for decreasing the use of or de-adopting a technology. One-to-one semi-structured interviews were conducted. Interviews were audio recorded and transcribed verbatim. Themes and sub-themes were deduced from the data through framework analysis using the following five distinctive steps: familiarization; identifying an analytic framework; indexing; charting; and mapping and interpretation. Themes and sub-themes were also mapped to existing KT TMFs.

Thirteen experts participated. The following three themes emerged as ideal characteristics of a KT TMF: (i) principles foundational for HTR: evidence-based, high usability, patient-centered, and ability to apply to micro, meso, and macro levels; (ii) levers of change: characterized as positive, neutral, or negative influences for changing behavior; and (iii) steps for knowledge to action: build the case for HTR, adapt research knowledge, assess context, select, tailor, and implement interventions, and assess impact. The Consolidated Framework for Implementation Research had the greatest number of ideal characteristics.

Application of KT TMFs to the HTR process has not been clearly established. This is the first study to provide an understanding of characteristics within KT TMFs that could be considered by users undertaking projects to decrease or de-adopt technologies. Characteristics to be considered within a KT TMF for implementing HTR outputs were identified. Consideration of these characteristics may guide users in choosing which KT TMF(s) to use when undertaking HTR projects.

In 2017, The French National Authority for Health (HAS) created an open, online, systematic contribution process to enable patient and consumer groups (PCGs) to contribute to health technology assessment (HTA) carried out to aid public authorities in reimbursement and pricing decision making.

This retrospective study analyzes how French PCGs contributed to the HTA process within the HAS for the first 2 years of this new mechanism.

PCG contributions received between 01 January 2017 and 31 December 2018 and the recording of deliberations leading to reports of the corresponding HTAs were included. Analysis grids were designed by the investigators with 5 rounds of refinement tests on 10 random PCG contributions and the reports. Systematic data extraction was then performed separately by two investigators. PCG answers to the open-question templates and the related final HTA report published by the HAS were analyzed.

Seventy-nine contributions from 44 PCGs were received and analyzed by the HAS for 78 out of the 592 HTAs performed for drugs or medical devices during the 2-year period. Twenty-five percent of the HTAs performed for drugs received at least one contribution. The contributions covered quality-of-life aspects, access to care, and personal and family impact. Membership and budget of the contributing PCGs varied greatly.

The experience gained in the first 2 years demonstrates the feasibility of the process and the fact that PCG contribution actually provides relevant input on the patient perspective for HTAs used for reimbursement decisions. The challenges identified on the side of PCGs were time constraints and human resources.

The European Union regulation for orphan medicinal products (OMPs) was introduced to improve the quality of treatments for patients with rare conditions. To mark 20 years of European Union OMP regulation, this study compared access to OMPs and the length of their reimbursement process in a set of European countries and Canadian provinces. Access refers to their full or partial reimbursement by the public health service.

Data were collated on European Medicines Agency orphan designation and marketing authorizations, health technology assessment (HTA) decisions and reimbursement decisions, and the respective dates of these events for all the OMPs centrally authorized in 14 European countries (Belgium, England, France, Germany, Hungary, Italy, the Netherlands, Norway, Poland, Scotland, Slovakia, Spain, Sweden, and Switzerland) and four Canadian provinces (Alberta, British Columbia, Ontario, and Quebec).

Since the implementation of the OMPs Regulation in 2000, 215 OMPs obtained marketing authorization. We found that Germany had the highest level of coverage, with 91 percent of OMPs being reimbursed. The three countries with the lowest reimbursement rates were Poland, Hungary, and Norway (below 30%). We observed that Germany had the quickest time to reimbursement following marketing authorization, followed by Switzerland and Scotland. We observed that Poland, Hungary, and Slovakia consistently had the longest time to reimbursement.

We observed substantial variation in the levels and speed of national reimbursement of OMPs, particularly when comparing countries in Eastern and Western Europe, which suggests that an equity gap between the regions may be present. The data also indicated a trend toward faster times to reimbursement over the past 10 years.

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.

To develop an international template to support patient submissions in Health Technology Assessments (HTAs). This was to be based on the experience and feedback from the implementation and use of the Scottish Medicines Consortium's (SMC) Summary Information for Patient Groups (SIP).

To gather feedback on the SMC experience, web-based surveys were conducted with pharmaceutical companies and patient groups familiar with the SMC SIP. Semistructured interviews with representatives from HTA bodies were undertaken, along with patient group discussions with those less familiar with the SIP, to explore issues around the approach. These qualitative data informed the development of an international SIP template.

Survey data indicated that 82 percent (18 of 22 respondents) of pharmaceutical company representatives felt that the SIP was worthwhile; 88 percent (15/17) of patient group respondents found the SIP helpful. Both groups highlighted the need for additional support and guidance around plain language summaries. Further suggestions included provision of a glossary of terms and cost-effectiveness information. Patient group interviews supported the survey findings and led to the development of a new template. HTA bodies raised potential challenges around buy-in, timing, and bias connected to the SIP approach.

The international SIP template is another approach to support deliberative processes in HTA. Although challenges remain around writing summaries for lay audiences, along with feasibility considerations for HTA bodies, the SIP approach should support more meaningful patient involvement in HTAs.

The use of inconsistent and outdated information may significantly compromise healthcare decision-making. We aimed to assess the extent of lag times in the publication and indexing of network meta-analyses (NMAs).

Searches for NMAs on drug interventions were performed in PubMed (May 2020). Lag times were measured as the time between the last systematic search and the date of the article's submission, acceptance, online publication, indexing, and Medical Subject Heading (MeSH) allocation. Correlations between lag times and time trends were calculated by means of Spearman's rank correlation coefficient. Time-to-event analyses were performed considering independent variables such as geographical origin, journal impact factor, Scopus CiteScore, and open access status.

We included 1,245 NMAs. The median time from last search to article submission and publication was 6.8 months and 11.6 months, respectively. Only five percent of authors updated their literature searches after submission. There was a very slight decreasing historical trend for acceptance (r =−0.087; p = 0.01), online publication (r =−0.08; p = 0.008), and indexing lag times (r =−0.080; p = 0.007). Journal impact factor influenced the MeSH allocation process (log-rank p = 0.02). Slight differences were observed for acceptance, online publication, and indexing lag times when comparing open access and subscription journals.

Authors need to update their literature searches before submission to reduce evidence production time. Peer reviewers and editors should ensure that authors comply with NMA standards and encourage the development of living meta-analyses.