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.

The Brazilian health system has not been able to enforce the constitutional Right to Health for the entire population, leaving litigation as the last alternative for the fulfillment of the right. In order to harmonize and underpin the decision making of federal judges, an Academic Detailing (AD) program with concepts of evidence-based medicine and health policies for federal judges will be conducted nationwide in Brazil. AD is a strategy to provide information, combining an interactive outreach approach with the best evidence. This study reports the method used to define key messages to be used during visits.

Government, federal judges and academy representatives were invited to a workshop on health litigation in Brazil. They were divided into six groups to discuss five hypothetical scenarios. In each scenario, groups listed two possible key messages to disseminate during AD, addressing the legal, scientific, economic or ethical dimensions. After the definition, a vote was taken, according to the importance that each participant attributed to them (1 to 10), and then a score was generated.

Of the thirty-one participants, five were from the judiciary, three prosecutors, one health insurance representative, nine managers and thirteen from the academy. From the case study presented, fifty-five key messages were suggested. After removing duplicates, twenty-five were selected to assign importance values by each participant. The key messages were sorted from highest to lowest score, the most important being “Explain what evidence-based medicine means and what scientific evidence means”.

Knowledge of theoretical concepts of evidence-based medicine, health policies and the technology incorporation process seem to be of fundamental importance to federal judges. This information will serve as a basis for decision making regarding litigious proceedings involving the constitutional Right to Health in Brazil. From the selected key messages, bulletins to the academic detailing program to federal judges shall be produced.

A number of economic evaluations of gender-neutral human papillomavirus (HPV) vaccination have been published, generally finding that the cost-effectiveness is sensitive to the uptake rate in girls. In Ireland there is a girls-only program in place, but the initial high uptake rate (>85 percent) was substantially impacted by high profile negative publicity concerning perceived vaccine safety issues. Efforts to address perceived safety concerns have recently yielded a partial recovery in uptake rates. The aim of this study was to estimate the cost-utility of extending the program to include boys and explore the impact of fluctuating uptake rates.

A previously published cost-utility model used in the United States of America and Norway was adapted to the Irish setting and populated with Irish epidemiological and cost data. Comparators included no vaccination, and girls-only and gender-neutral vaccination, both with either a 4-valent or 9-valent vaccine. Vaccination is at age 12 years and oropharyngeal and penile cancers were excluded in the base case analysis. Additional analyses were used to incorporate fluctuating uptake rates into the model.

A 9-valent girls-only program dominated the existing girls-only 4-valent program. The incremental cost-effectiveness ratio (ICER) for a gender-neutral 9-valent program was EUR 50,823/quality-adjusted life year (QALY). Gender-neutral vaccination would be cost-effective at a willingness-to-pay threshold of EUR 45,000/QALY when the uptake rate is below 78 percent. The ICER decreased to between EUR 41,000 and EUR 42,000/QALY when the uptake rate was allowed to fluctuate across six to 12 yearly cycles.

The cost-effectiveness of gender-neutral HPV vaccination is highly sensitive to the assumed uptake rate in girls. Large fluctuations in HPV vaccine uptake rates have been observed in a number of countries in the last decade. Incorporating fluctuating uptake rates in the model shows that a gender-neutral program may be more cost-effective than when a stable uptake is assumed.

Consistently high-quality health care is expected throughout Europe while concurrently, financial resources of member states are decreasing. National Health Technology Assessment (HTA) institutes are informing evidence-based reimbursement decisions in the national context, leading to redundancies in HTA production and tying up limited resources. Since 2006, the European Union project, the European Network for HTA (EUnetHTA) is aiming at enhancing the efficient use of HTA resources and facilitating transnational collaboration. Our aim is to present previous experience in joint assessment of medical devices. Furthermore, possible benefits of European collaboration for stakeholders will be discussed.

Processes and challenges of the completed EUnetHTA Joint Action (JA) 2 are summarized and discussed. Benefits, aims and opportunities of the ongoing EUnetHTA JA 3 are described.

Six rapid assessments of medical devices, focusing on the assessment of effectiveness and safety, were published during EUnetHTA JA 2. Challenges in European medical device assessment encompass the choice of topics, the time point of assessments and the lack of European standards for systematic patient involvement. Characteristics of medical devices, like learning curves, call for monitoring them throughout their lifecycle.

The benefit of European collaboration for stakeholders is manifold: uncertainty with regard to actual added value of a technology is minimized through Early Dialogues; harmonized and transparent assessment processes increase the quality of reports; work division among HTA organizations allows a resource-efficient assessment of a bigger amount of technologies; patient involvement ensures consideration of patient relevant endpoints.

The importance of cross-border collaboration in HTA is shown in the continuation of the EUnetHTA project, which aims to sustainably strengthen international collaboration even after expiration of EU-funding.

European collaboration in medical device assessment can ensure cross-border health care and efficient cooperation of national health systems. The focus should be set on a wide implementation of jointly established methods and quality standards. The European collaboration can lead to a concrete benefit for various stakeholders.

Clinical background: Until recently, populations living in remote areas did not have access to specialist care and quality diagnostic services and thus depended on the low response capacity of their local health system. Subsequently, there were equity issues between urban and rural populations. Therefore it was considered telediagnosis applications should be directed towards developing better equity in the provision of services in remote locations without access to specialists. This study has evaluated the results of a new telemedicine system in remote public hospitals in Paraguay, in order to show how the response capacity of the local integrated health service delivery networks has been improved by providing access to tertiary level diagnostic services by specialists. Objective: This study aims to evaluate the utility of telemedicine as a tool for developing better equity in the provision of services in remote locations.

This was a descriptive study, where the results of using telemedicine for diagnosis in remote public hospitals were evaluated as a tool to improve access to diagnostic services countrywide between 2014–2017. For these purposes, type and frequency of pathology diagnosed was determined.

A total of 311,562 telediagnoses were performed in fifty-seven hospitals. The 191,435 electrocardiogram diagnosis performed in the fifty-five hospitals were mainly normal (62.1%), unspecified arrhythmias (12.5%), and sinus bradycardia (10.4%). Also 115,924 teletomography tests were performed in twelve hospitals, where 54.4 percent corresponded to head as a consequence of accidents (motorcycles) and cerebrovascular diseases, 13.8 percent to chest, and the rest the other anatomical regions. Regarding the 4,184 electroencephalogram tests performed, antecedents of seizure (54.3%), evolutionary controls (14.0%), and headache (11.5%), were mainly diagnosed. The nineteen ultrasound studies corresponded to prenatal controls.

Despite the results of the telediagnosis implemented in the public health to develop better equity in the provision of services in remote locations, a widespread use-assessment should be analyzed before this tool is adopted.

We conducted an analysis of the key factors triggering cost-sharing mechanisms to understand the status of out-of-pocket (OOP) healthcare expense in the United States (US), Europe, and emerging markets and better appreciate the implications of OOP healthcare expense on patients’ health management.

A review of literature and databases including The Organisation for Economic Co-operation and Development (OECD) and World Bank was performed to understand different cost-sharing mechanisms, factors triggering OOP expenditure and the country-wise trends of OOP expenditure. Additionally, the impact of OOP expenditure on healthcare budget and on patients in terms of medication adherence, uptake of newer therapies and generic substitution was explored.

The findings reveal that patients are concerned about rising healthcare OOP costs, and we observed an increase of 134 percent in the number of articles published on OOP from 2005 to 2017. The percentage of household spending that goes OOP as healthcare expense is higher in Brazil, Russia, India, and China (BRIC countries; ~11 percent) compared to France, Germany, Italy, United Kingdom, US, Japan, and Canada (G7 countries; ~2 percent). In addition, OOP expenditure increased with age (1.9 percent of take home income in 55-64 age group versus 1.2 percent in 18-25 age group) and is higher in the low-income population (2.8 percent of take home income versus 1 percent in high-income group). Whereas, increasing OOP expenditure reduces the overall healthcare expenditure due to generic substitution (28 percent reduction) and reduction in excessive consumption of supplementary medicines, it also reduces patient adherence (~20 percent decline in dispensed prescriptions) and may foster a reluctance to adopt newer therapies.

The population groups most impacted by increasing OOP expense are the older population, those in the low-income bracket and in poorer countries. While OOP expense may help in the effective and judicious utilization of healthcare system resources and medicines usage, its implementation requires a cautious and considered approach.

There are enormous expectations for e-health solutions to support high quality healthcare services, with accessibility, and effectiveness as key goals. E-health encompasses a wide range of information and communication technologies applied to health care, and focuses on combining clinical activity, technical development, and political requirements. Hence, e-health solutions must be evaluated in relation to the desired goals, to justify the high costs of such solutions.

Health technology assessment (HTA) aims to produce rational decisions for purchasing new technologies and evaluating healthcare investments, like drugs and medical equipment, by measuring added value in relation to clinical effectiveness, safety, and cost effectiveness. It is desired to also apply HTA assessment on large scale e-health solutions, but traditional quantitative HTA methodology may not be applicable to complex e-health systems developed and implemented as ongoing processes over years. Systematic reviews and meta-analyses of these processes risk being outdated when published, therefore action research designed to work with complex, large scale programs may be a more suitable approach.

In the project, we followed the development of a new process-oriented electronic patient record system (EPR) in northern Norway. Part of the process was structuring clinical data to be used in electronic forms within the system. This was the first time a health region structured the clinical data and designed the forms; receiving feedback alongside the process was very important. The goal was to use structured forms as a basis for reusing EPR data within and between systems, and to enable clinical decision support.

After designing a prototype of a structured form, we wrote an assessment report focusing on designing a methodology for such development, which stakeholders to include, and how to divide the work between the health region and the system vendor. The answers to such questions will have both practical and economic consequences for designing the next phase of the process.

This study aimed to introduce a pilot program for hospital-based health technology assessment (HB-HTA) in China and present the participants’ experiences based on seven case studies from seven tertiary hospitals.

One-year pilot projects were initiated at the beginning of 2018. Seven pilot hospitals were closely followed from the beginning until the completion of their pilot HTA project. Regular interviews were conducted with the hospital managers leading the HB-HTA projects and key members of the special HTA teams. Observations were made based on field trips and written HTA reports.

Three pilot projects evaluated the use of medical consumables, three evaluated the use of surgical or medical interventions, and one evaluated an innovative management model for ventilators. Real-world data were collected from all the pilot projects to assist with the assessments. Most HB-HTA pilot projects achieved remarkable results such as improvements in economic efficiency; however, there were also obvious deficiencies such as the lack of a necessary cost-effectiveness analysis.

The results varied among the seven HB-HTA pilot projects. The HB-HTA pilot program was implemented to promote the use of HB-HTA in hospital decision making in China. At the same time, HB-HTA in China faces challenges. We have made some policy recommendations based on the findings of the pilot projects.

The aim of this study was to find ways of bridging the gap in opinions concerning health technology assessment (HTA) in reimbursement submission between manufacturers and payers to avoid access delays for patients of vital medicines such as oncology drugs. This was done by investigating differences and similarities of opinion among key stakeholders in Australia.

The survey comprised of nine sections: background demographics, general statements on HTA, clinical claim, extrapolations, quality of life, costs and health resource utilization, agreements, decision making, and capability/capacity. Responses to each question were summarized using descriptive statistics and comparisons were made using chi-square statistics.

There were ninety-seven respondents in total, thirty-seven from the public sector (academia/government) and sixty from the private sector (industry/consultancies). Private and public sector respondents had similar views on clinical claims. They were divided when it came to extrapolation of survival data and costs and health resource utilization. However, they generally agreed that rebates are useful, outcomes-based agreements are difficult to implement, managed entry schemes are required when data are limited, and willingness to pay is higher in cancer compared to other therapeutic areas. They also agreed that training mostly takes place through on the job training and that guideline updates were a least favored opportunity for continued training.

Private sector respondents favor methods that reduce the incremental cost-effectiveness ratio when compared to the public sector respondents. There still exist a number of challenges for HTA in oncology and many research opportunities as a result of this study.