Written patient evidence is submitted to the National Institute for Health and Care Excellence (NICE) by patient organizations and their nominated patient experts. We reviewed the impact that patient evidence had on the committee's decision making. This local learning can help generate global opportunities for Health Technology Assessment (HTA) bodies and patient groups to further develop their understanding and methodology about how patient evidence can support HTA decision making for ultra-orphan conditions.

There were two phases.

Phase one was an online questionnaire about the impact of patient evidence on the committee's decision making for ultra-orphan HTA evaluations. It was sent to the committee chair, lay committee members and selected other committee members.

Phase 2 developed the initial questionnaire findings using in-depth interviews with the committee chair and the lay members. These gained further understanding of the impact of patient evidence and the themes raised.

Key findings showed patient evidence was helpful to understand the:

• • Burden of disease

• • Patient population

• • Likely uptake of new medicines

• • Impact on carers.

For ultra-orphan conditions, where other forms of evidence are scarcer, patient evidence is fundamental to understanding patient needs, the impact of the disease, patient population and preferences.

Patient evidence was useful for the committee in different ways; it provided the committee with new evidence and it helped the committee understand and interpret the evidence submitted by others. Both are key to committee decision making. It was clear that due to the very small patient population, patient groups knew the patient population, their stage of disease, and their preferences in detail.

The findings will be used to inform an updated patient submission template for ultra-orphan HTAs, and supporting guide. These will be available on the NICE website and offered to the HTAi Interest Group on Patient and Citizen Involvement so they can be shared globally.

Value frameworks, analogous to Health Technology Assessment (HTA) internationally, have emerged in the United States to aid stakeholders in assessing the value of new treatments. Since patient perspectives on value may differ significantly from other stakeholders, formalized procedures to involve patients in their work have been created. Despite these efforts, concerns persist that patient involvement is insufficient or “rhetoric.” To assist in this effort, the National Health Council (NHC) created a rubric to aid decision makers in improving the patient centricity of their value assessments.

A convenience sample of twenty-eight organizations was invited to participate in a roundtable discussion. Participants discussed experiences with value frameworks; debated and thematically grouped hallmark patient-centeredness characteristics; and developed illustrative examples of the characteristics. These materials were organized into the rubric, and subsequently vetted via multi-stakeholder peer review.

Participants agreed upon six key domains of patient centeredness: partnership (patients are involved in every step of development/dissemination processes), transparency (assumptions/inputs are disclosed in an understandable, timely way), inclusiveness (perspectives drawn from broad range of stakeholders), diversity (differences in subpopulations, trajectory of disease, and stage of a life should be accounted for), outcomes (includes those that patients have identified as important), and data (variety of credible data sources are used allowing for timely incorporation of new information and account for the diversity of patient populations and patient-centered outcomes). The Rubric describes each domain and includes illustrative examples of how patient engagement/centeredness can be operationalized through direct and indirect pathways.

The NHC Rubric is a first step toward creating patient-centered value assessments that patients and their families can rely on. It is intended to assist all stakeholders, especially the patient community, in assessing the level of patient centeredness and engagement in a given framework or model. It can be a guide to support developers in conceptualizing plans for meaningfully engaging patients.

Beta-interferons are used as first-line therapy for relapsing-remitting multiple sclerosis in Brazil. In order to evaluate the possible inferiority of one of the beta-interferons available and support a guideline update, we conducted an eleven-year (January 2000 to December 2010) nationwide real-world performance assessment using the Unified Health System (SUS) databases.

We assessed whether patients using subcutaneous beta-interferon switched treatment, relapsed or died (composite event) earlier than patients using intramuscular beta-interferons. Patients without a dispensing registry longer than three months were censored. We used the Kaplan-Meier method to estimate the cumulative probability of persistence on initial treatment, and compared groups with the Log-rank test. The influence of the drug on the occurrence of event was assessed with Cox proportional hazards analysis.

The number of patients included was 12,154, and the majority started treatment with subcutaneous beta-interferon-1a (45.7 percent), followed by subcutaneous beta-interferon-1b (27.7 percent) and by intramuscular beta-interferon (26.6 percent). Women represented 73.1 percent and the mean age was 38.93±11.34 years old. The group of patients who used intramuscular beta-interferon switched treatment, relapsed or died earlier (median 47 months; 95 percent Confidence Interval, CI 44–52) than patients using the subcutaneous beta-interferons, (69 months (95 percent CI 64–76) for beta- interferon 1a and 73 (95 percent CI 66–84) months for beta-interferon 1b) (p< .0001 for both comparisons). Accordingly, the use of intramuscular beta-interferon was associated with a higher probability of event (Hazard ratio, HR 1.38; 95 percent CI 1.29-1.48), while the use of the other beta-interferons had a protective effect (1a: HR .86; 95 percent CI .81-.92; 1b: HR .89; 95 percent CI .83-.95).

The inferiority of intramuscular beta-interferon found in the real-world corroborates findings from head-to-head studies and systematic reviews conducted by Cochrane and the National Commission for Technology Incorporation in SUS (CONITEC/Brazil). This result led to disinvestment in intramuscular beta-interferon and was the first case of clinical guideline update using real-world evidence in Brazil.

With the promotion of a tiered medical service system, secondary hospitals will play a more important role in the future. This study aims to explore the cost-benefit of computed tomography (CT) in secondary hospitals in China, with a view to providing information for overall economic management in hospitals as well as for regional planning of medical equipment in different areas.

Fifty-eight secondary hospitals from six provinces located in the eastern, central, and western regions of China were selected as the study sample. Questionnaires were used to collect information on the cost structure, efficiency, and benefits of CT in the secondary hospitals in the past 5 years. Cost analysis was conducted from the perspective of the hospitals, which mainly referred to direct fixed costs and variable costs. We analyzed the investment recovery years a, cost recovery rate b, and benefit-cost ratio to evaluate the economic benefits of CT. We also analyzed the technological benefits of CT based on its effective utilization rate c and positive detection rate.

1. a: Investment recovery years = total original investment / (annual net income + annual depreciation expense)

2. b: Cost recovery rate = average income per check / average cost per check

3. c: Effective utilization rate = single equipment utilization rate * positive detection rate

4. (Single equipment utilization rate = actual working time / rated working time)

Depreciation costs (36.3 percent) were the largest proportion of all costs over the 5-year period, followed by material costs (22.2 percent), maintenance costs (18.2 percent), labor costs (17.1 percent), and electricity consumption (1.2 percent). The investment recovery periods of CT in the eastern, central, and western regions were 2.5, 2.8, and 3.1 years, respectively; the cost recovery rates were 186.5 percent, 172.0 percent, and 174.1 percent, respectively; the benefit-cost ratios were 1.9, 1.7, and 1.7, respectively; the effective utilization rates were 46.1 percent, 58.3 percent, and 71.2 percent, respectively; and the positive detection rates were 52.3 percent, 60.5 percent, and 73.3 percent, respectively.

The current study indicates that the cost-benefit of CT is good in secondary hospitals, especially in terms of economic benefits. But to achieve greater technological benefits in all three regions, more appropriate utilization of CT is needed.

The increase in travelers and refugees combined with global warming may soon lead to the development of tropical diseases such as Schistosoma or Strongyloides infections in some European countries.

Those intestinal parasites may persist for decades with subclinical infections or low-grade disease with nonspecific manifestations. In the presence of immunosuppression, strongyloidiasis can rapidly evolve into life-threatening disseminated disease, whereas chronic schistosomiasis can lead to complications causing future morbidity and death.

Currently in France, an update of diagnostic tests reimbursed for those tropical diseases is ongoing to fully cover diagnostic needs.

Our aim was to assess the clinical relevance of tests used in schistosomiasis’ or strongyloidiasis’ diagnosis and include the most relevant in the national list of reimbursed tests.

The assessment involves a critical analysis of national and international guidelines identified by a systematic literature search, and stakeholders’ views.

This work identifies several autochthonous outbreaks of those diseases in France; such as urogenital schistosomiasis that occurred in Corsica, in summer 2013. Also it enlightens the increase of strongyloides serological tests performed in the past years. Those facts prove the potential development of those infections in Europe.

It underlines that, serology is the first diagnostic test line for most cases and is more sensitive than stool microscopy which represents however the final diagnostic investigation to confirm the intestinal infection.

It confirms the main indications of those two diagnostic tools.

It relies on a tropical infectious disease expert network including the French army health service. They have brought further clarification of diagnostic tests clinical relevance for travelers or autochthonous cases.

This new use of Health Technology Assessment has allowed updating and listing the relevant diagnostic tools which might be crucial to better follow those diseases and it may help the health system to face the increase of tropical infections.

This study explored the cost-effectiveness of expanding Hepatitis C Virus (HCV) screening and subsequent treatment in Swiss custodial settings, given the availability of rapid antibody saliva tests (Oraquick®) and dried blood spot tests (semi-quantitative viremia and viral genotype), and recent therapeutic advances which have higher cure rates and shorter treatment courses (1).

A comprehensive strategy offering screening to all detainees was compared to the current setup of screening high-risk individuals (for example, from endemic countries, active or former injecting drug users). A decision tree simulated the diagnosis pathway, and results from a Markov model were included to predict treatment effects and natural progression over a lifetime time-horizon. Input data were derived from clinical studies, literature reviews, custodial health services and expert opinion (2). The net monetary benefit (NMB) and incremental cost-effectiveness ratio (ICER) of comprehensive compared to current screening were calculated. Deterministic and probabilistic sensitivity analyses were performed to explore parameter uncertainty and whether variations informed by expert opinion changed the cost-effectiveness of comprehensive screening.

At a willingness-to-pay threshold of CHF100,000 (USD99,500) per Quality-Adjusted Life-Year (QALY), comprehensive screening had an 83 percent probability of being cost-effective, with a corresponding NMB of CHF33,451,972 (USD33,284,712) and ICER of CHF7,168/QALY (USD7,132/QALY). Results were most sensitive to the QALYs gained from the treatment model (both treatment and no treatment arms), respective HCV prevalence in the current and comprehensive screening populations, treatment initiation rates, and screening offer acceptance rates. Compared to the current practice of screening high-risk individuals, comprehensive screening is likely to be cost-effective due to the increase in testing rates, which were conservatively estimated in this study. Furthermore, comprehensive HCV screening of prisoners may prove more cost-effective in countries where prisoners are not routinely screened.

Comprehensive screening programs could be considered in prison units with a large proportion of high-risk individuals and where detainees are incarcerated for enough time to complete a treatment course during their sentence.

Drug overuse in healthcare settings is common in China. Clinical pathways are tools that provide the link between the best available evidence and clinical practice. This study aimed to determine if the clinical pathway of community-acquired pneumonia (CAP) had effects on the antibiotic use in patients with CAP.

The study was conducted in Shanghai, Hubei Province, and Gansu Province to represent high, middle, and low levels of socioeconomic status in 2015. In each region, three public tertiary general hospitals and three public secondary general hospitals were selected for chart review of antibiotics’ utilization in the patients with CAP during 2014. A multilevel logistic regression model was used in the study, with a dependent variable of appropriate utilization of antibiotics (right time, right type, and right combined use) and independent variables of hospital adoption of clinical pathway and patient characteristics (sex, age, severity of disease, and number of comorbidities).

Twelve surveyed hospitals (66.67 percent) adopted CAP clinical pathways and 354 cases (66.29 percent) were from these twelve hospitals (CP group). Among the total utilization of antibiotics (796 times) in eighteen types of antibiotics used in patients with CAP, the five recommended types of antibiotics accounted for 82.16 percent.

The percentages of cases that got initial antibiotics in time were 90.60 percent in the CP group and 76.11 percent in the non-CP group. The compliance rate for appropriate types of antibiotic utilization was 88.36 percent in CP group, much higher than that in non-CP group (70.22 percent). For 244 cases that used combined antibiotics, the compliance rate for the recommended combinations of antibiotics was 20.12 percent in the CP group, but 1.25 percent in the non-CP group. After controlling patients’ characteristics, the patients in the CP group got more appropriate antibiotics than those in the non-CP group.

Adoption of the CAP clinical pathway in hospitals can improve antibiotics' utilization.

Medtech innovation briefings (MIBs) are intended to support National Health Service (NHS) decision makers and staff who are considering using new innovative medical devices and in-vitro diagnostics. MIBs are produced in support of the NHS 5-Year Forward View, specifically to accelerate innovation in new treatments and diagnostics. This project aimed to evaluate the extent to which published MIBs address national priorities set by NHS England, including in six clinical areas: cancer, mental health, dementia, diabetes, learning disabilities, and maternity.

Data was extracted from eighty-seven MIBs downloaded from the National Institute for Health and Care Excellence (NICE) website including: study design, amount of evidence, date of CE mark, population, cost, manufacturer, device class, publication date, and category of conditions and disease (as prescribed by NICE). Descriptive analysis was done for each variable and frequency tables were produced for MIBs by disease category.

Cardiovascular disease (n = 19) and cancer (n = 12) were the two most common conditions addressed by MIB-evaluated devices. The four medical conditions with the fewest MIBs (n = 1 each) were: diabetes, liver conditions, neurological conditions, and fertility, pregnancy and childbirth. Of the eighty-five MIBs with stated device classifications, just over half were Class IIa and IIb devices and 18 percent were in-vitro diagnostics. The earliest original CE mark was 1997, and approximately half of the devices obtained or updated their CE mark after 2010.

Chronic conditions such as cancer, cardiovascular disease, and diabetes accounted for 89 percent of total deaths in the UK in 2014, thus, the most commonly published MIBs aptly address these issues. However, MIBs are lacking in five out of six NHS priority areas. There is opportunity for innovative technologies to be reviewed via MIBs and alternative NICE pathways in the areas of diabetes, maternity, mental health, and learning disabilities and dementia.

Existing literature shows evidence on the differences in drug reimbursement decisions across countries. These differences are the reason for this study. The main aim of this research is to model the impact of drug reimbursement decisions on health outcomes (that is, life expectancy, healthy life years and mortality rates). In particular, this study is looking at countries that have different acceptance, restriction and rejection rates for drug reimbursement decisions.

The current study is based on a longitudinal dataset with data from nine European countries from 2002 to 2014. This dataset is formed of primary data on drug reimbursement decisions (that is, cancer drugs) collected in the Advancing and strengthening the methodological tools and practices relating to the application and implementation of Health Technology Assessment (ADVANCE-HTA) project and secondary data on life tables and indicators of health and socioeconomic status (from Eurostat and World Bank). Following the longevity model defined by Lichtenberg (1), a panel data model with country and year fixed-effects is run on this dataset in order to model the impact of the level of access to drugs on health outcomes.

The results show that the rate of adoption of new drugs into a national health system does not have any significant effect on life expectancy. However, more restrictive systems are positively and significantly related with healthy life years. Finally, for mortality rates, higher rejection rates are associated with lower deaths.

To conclude, contrary to the public opinion, results show that a more restrictive drug reimbursement system is not related with a worse health outcome, it is either associated with a positive outcome or it is not related.