This article presents the pharmaceutical industry's perspective on health technology assessment (HTA) with specific comments on the HTA systems in England and Wales, France, The Netherlands, and Sweden. The comments are focused on the following main themes: (i) The contributions of the HTA system to overall efficiency in the health-care system, (ii) HTA as a cost-driver for industry, patients, government, and society, and (iii) The various implementation barriers that currently exist for a successful implementation of HTA results.

South Africa has embarked on major health policy reform to deliver universal health coverage through the establishment of National Health Insurance (NHI). The aim is to improve access, remove financial barriers to care, and enhance care quality. Health technology assessment (HTA) is explicitly identified in the proposed NHI legislation and will have a prominent role in informing decisions about adoption and access to health interventions and technologies. The specific arrangements and approach to HTA in support of this legislation are yet to be determined. Although there is currently no formal national HTA institution in South Africa, there are several processes in both the public and private healthcare sectors that use elements of HTA to varying extents to inform access and resource allocation decisions. Institutions performing HTAs or related activities in South Africa include the National and Provincial Departments of Health, National Treasury, National Health Laboratory Service, Council for Medical Schemes, medical scheme administrators, managed care organizations, academic or research institutions, clinical societies and associations, pharmaceutical and devices companies, private consultancies, and private sector hospital groups. Existing fragmented HTA processes should coordinate and conform to a standardized, fit-for-purpose process and structure that can usefully inform priority setting under NHI and for other decision makers. This transformation will require comprehensive and inclusive planning with dedicated funding and regulation, and provision of strong oversight mechanisms and leadership.

Objectives: The need to consider the patient perspective in health technology assessments (HTA) has been widely recognized. In July 2012, the Finnish Medicines Agency (Fimea) published a national recommendation for integrating the patient perspective into the HTAs of pharmaceuticals. The aim of this study is to describe the development of the recommendation for integrating the patient perspective into the HTA process of pharmaceuticals in Finland.

Methods: The development of the recommendation was based on a review of international recommendations and experiences of patient and public involvement in HTA. The draft recommendation was tested in two focus group discussions (n = 7 patients) and three individual interviews among diabetes patients (type 1 or 2) using long-acting insulin treatment. The recommendation was open for public consultation in April 2012 and revised according to the comments received.

Results: Patients will be involved in multiple stages of Fimea's HTA process. The recommendation includes step-by-step instructions on how to assess the patient perspective. The main focus is on qualitative interviews, which will be conducted at the beginning of the assessments to gain information, particularly on patient preferences and values, including positive and negative outcomes important to patients and ethical and social aspects of the medicine's use.

Conclusions: The recommendation will act as a tool to integrate patients’ experiences, needs and preferences into Fimea's HTAs of pharmaceuticals.

Objectives: The aim of this study is to report on the experiences, benefits, and challenges of patient and public involvement and engagement (PPIE) from a publicly funded early awareness and alert (EAA) system in the United Kingdom.

Methods: Using email, telephone, a Web site portal, Twitter and focus groups, patients and the public were involved and engaged in the recognized stages of an EAA system: identification, filtration, prioritization, early assessment, and dissemination.

Results: Approaches for PPIE were successfully integrated into all aspects of the National Institute for Health Research Horizon Scanning Research and Intelligence Centre's EAA system. Input into identification activities was not as beneficial as involvement in prioritization and early assessment. Patients gave useful insight into the Centre's Web site and engaging patients using Twitter has enabled the Centre to disseminate outputs to a wider audience.

Conclusions: EAA systems should consider involving and engaging with patients and the public in identification, prioritization, and assessment of emerging health technologies where practicable. Further research is required to examine the value and impact of PPIE in EAA activities and in the early development of health technologies.

Objectives: The aim of this study was to examine the impact of the National Institute for Health and Clinical Excellence's (NICE's) negative and restricting technology appraisals on the number of prescription items dispensed and the corresponding total net ingredient costs for drugs from 2000 to 2004 in the ambulatory care of the National Health Service (NHS) in England and Wales. In addition, it is discussed whether the NICE approach could be a role model for Germany.

Methods: The number of prescription items dispensed and the net ingredient costs of thirty-one drugs reimbursed by the NHS were analyzed, thereof thirteen drugs descriptively and twenty-one drugs with regression analyses. Data were extracted from the “Prescription-Costs-Analysis-Statistics” for the ambulatory care of the British Department of Health (England 1993–2005). In the case of the twenty-one drugs analyzed by regression analyses, predictions were established how the prescribing and the costs would have developed without NICE's drug appraisal. Finally, conclusions were drawn whether NICE's negative and restricting drug appraisals had a decreasing effect or not.

Results: For 97 percent of the drugs analyzed in this study, the publication of NICE's fourteen negative and restricting technology appraisals of drugs between 2000 and 2004 did not reduce the number of prescription items dispensed and net ingredient costs in the ambulatory care of the NHS in England and Wales.

Conclusions: Cost-effectiveness appraisals as performed by NICE or the German Institute for Quality and Efficiency in Health Care (Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, IQWiG) are a useful and important tool to enhance the discussion about methods and acceptance of evidence-based medicine in general.

Objectives: This study evaluates the cost of dialysis care in Brazil, including costs of ambulatory care and hospital admissions due to all causes and cardiovascular events.

Methods: Data were analyzed for 200 patients with end-stage renal disease (ESRD) on chronic hemodialysis in Brazil between 2001 and 2004. Main end points were all-cause mortality, all-cause hospital admissions, and cardiovascular events. Direct costs of dialysis treatment and complications were computed from the perspective of two payers, the Ministry of Health (MoH) and private health insurance (PHI).

Results: Mean number of days of hospitalization was 12 per patient-year. There were 105 cardiovascular events; the most frequent events were coronary disease (n = 59, 56 percent) and congestive heart failure (n = 26, 25 percent). The rate of cardiovascular events was 193 per 1,000 patient-years. There were 43 deaths, and the death rate was 79 per 1,000 patient-years. Median cost per hospital admission was US$ 675 and US$ 932 from the perspective of the MoH and PHI. For admissions due to cardiovascular causes, the corresponding costs were US$ 1,639 and US$ 4,499, respectively. Mean global cost per patient-year for chronic hemodialysis therapy was US$ 7,980 and US$ 13,428 from the perspective of the MoH and PHI, respectively.

Conclusions: Patients on chronic hemodialysis care incur significant healthcare resources due to the costs of dialysis and complications, notably cardiovascular disease. New disease management programs aimed at reducing cardiovascular morbidity and efficient use of resources are critical to ensuring the sustainability of treatments for ESRD in Brazil.

Objectives: The objective is to describe the principles for the design of the economic evaluation of COLOR II, a randomized, multi-country study comparing laparoscopic and open surgery for rectal cancer.

Methods: By using the experiences gained in a recent economic evaluation in colon cancer, where the same surgical techniques were compared, we could improve the method for identifying and measuring resource use items and also accommodate the use of data from the global study population.

Results: In the design of the study, the uncertainty in the resource-use variables was reduced by considering (i) what aspects drive each variable, (ii) what resource use is related to the intervention, (iii) how data from different countries affects the variable.

Conclusions: The aim was to refine the data collection so that the economic research question could be answered in the best possible way, given the circumstances in the clinical study. Thus, (i) some variables were treated as stochastic variables and others as deterministic variables, (ii) aggregate key cost-driving resource items were developed that corresponded to clinical events, and (iii) a surrogate variable was selected, instead of the “obvious variable”, to reduce the impact of confounding factors for one particular resource unit.

On the basis of two case histories of medical device innovation, two aspects of demand are identified: (a) the prospect of new markets and (b) information about user needs. Conditions for direct interaction between the user and producer in the innovation process are identified. The article concludes with some normative implications for the publicly designed generation of new medical technology.

Initial functional ability (Barthel Index, mean 57) was found to be an important predictor of functional ability 1 year after stroke (mean 80) and for costs during the period. On average the total cost for a stroke patient was about SEK 200,000; the main expense, accommodation, averaged about SEK 140,000, while assistive devices amounted to SEK 2,600. Those who use assistive devices, although having achieved a high functional ability, perceive and rate their life situation (Nottingham Health Profile) considerably more impaired than those without assistive devices.

The purpose of this study was to identify the determinants of choice of surgical procedure (anterior colporrhaphy, colposuspension, or needle suspension) to treat stress incontinence in women. We used multilevel modeling of data on 271 patients in 18 hospitals in England in 1993–94. Patient-related factors included sociodemographic details, anatomical diagnosis, symptom severity, symptom impact, previous treatment, parity, comorbidity, and general health status. Surgeon-related factors were specialty, grade, and annual volume of procedures undertaken. Hospital teaching status was considered. Some patient-related factors were associated with choice of procedure: women with a concomitant genital prolapse, with a history of high parity, and with no previous nonsurgical treatment were more likely to undergo an anterior colporrhaphy than a colposuspension or needle suspension (although this finding could be confounded by surgical specialty). In addition, women were more likely to be treated by colposuspension if their surgeon specialized in incontinence surgery (measured by annual volume of cases). Finally, being treated by needle suspension depended on there being a consultant surgeon familiar with the procedure at the hospital attended. While choice of surgical procedure depends partly on the patient's anatomical diagnosis, it is also dependent on the specialty of the surgeon whom she consults and the hospital that she attends. This variability, in turn, could have implications for the patient (as the relative effectiveness of the different procedures is unknown) and for the purchasers of care (as the relative cost-effectiveness of procedures is also unknown).

Objectives: The aim of our study was to describe approaches to health technology assessment (HTA) for medicines in the Slovak healthcare system and the related decision-making processes concerning reimbursement for medicines.

Methods: Analysis of the Slovak legislative framework related to HTA and the reimbursement process for medicines was performed. Additionally, current practices of the Working Group for Pharmacoeconomics, Clinical Outcomes and Health Technology Assessment of the Slovak Ministry of Health were evaluated.

Results: In Slovakia, there is always at least one treatment available in each determined therapeutic class with no co-payment. HTA is becoming an established method for the evaluation of cost-effectiveness of medicines in Slovak healthcare policy. The majority of decision makers within Slovakia support the idea of increased use of and the quality and efficiency of HTA methods. However, it is crucial to overcome several practical barriers to facilitate progress in the field of HTA in the Slovak Republic.

Conclusions: It can be seen that participation within the European Network for Health Technology Assessment (EUnetHTA JA 2 and EUnetHTA JA 3 projects) has significantly improved the quality of the process of HTA in Slovakia. Further legislative activities in this field are required due to the approved strategy for European Union cooperation on HTA.

Objectives: Reducing sexually transmitted infections (STI) and teenage pregnancy through effective health education is a high priority for health policy. Behavioral interventions which teach skills to practice safer sex may reduce the incidence of STIs. We evaluated the cost-effectiveness of school-based behavioral interventions in young people.

Methods: We developed an economic model to estimate the total number of STI cases averted, consequent gain in health related quality of life (HRQoL) and savings in medical costs, based on changes in sexual behavior. The parameters for the model were derived from a systematic literature search on the intervention effectiveness, epidemiology of STIs, sexual behavior and lifestyles, HRQoL and health service costs.

Results: The costs of providing teacher-led and peer-led behavioral interventions were €5.16 and €18 per pupil, respectively. For a cohort of 1000 boys and 1000 girls aged 15 years, the model estimated that the behavioral interventions would avert two STI cases and save 0.35 Quality Adjusted Life Years (QALYs). Compared to standard education, the incremental cost-effectiveness of the teacher-led and peer-led interventions was €24,268 and €96,938 per QALY gained, respectively.

Conclusions: School-based behavioral interventions which provide information and teach young people sexual health skills can bring about improvements in knowledge and increased self-efficacy, though these may be limited in terms of impact on sexual behavior. There was uncertainty around the results due to the limited effect of the intervention on behavioral outcomes and paucity of data for other input parameters.

The FDA recently approved Ceredase™, a new treatment for Gaucher's disease, under the provisions of the Orphan Drug Act. Ceredase™ is unusually expensive, but there are no satisfactory alternative therapies. It appears likely that Ceredase™would not have become available without the protection of the Orphan Drug Act, but its expense and the lack of information about its long-term effects on health raise questions about whether the ODA provides appropriate incentives to develop cost-effective technologies.

In this paper we describe the present range of organizations that have a role in the early warning of new and emerging health care technologies in the United Kingdom. We discuss in more detail the processes and prioritization criteria used by the U.K. horizon-scanning project for the NHS Health Technology Assessment Programme, and the principal methods of technology identification for the horizon-scanning project are outlined. The United Kingdom plans to develop an integrated system for the identification of technologies for commercial planning, health service research prioritization, financial planning, and provision of information to policy makers, purchasers, and providers of health care.

Eight hundred and six medical and surgical patients who were hospitalized via the emergency ward were followed over their entire inpatient stays and rated in anterograde, double-blind fashion for inpatient incidents (falls, medication errors, other). Injuries were minor but affected 2.2% of admissions, a figure which is strikingly similar to studies in other hospitals. There was a statistical trend toward a higher-than-normal risk of hazardous in-hospital incidents for males age 20 to 40 admitted because of injury and for medically ill females over 60 years old.

Objectives: External experts can be consulted at different stages of an HTA. When using vague information sources, it is particularly important to plan, analyze, and report the information processing in a standardized and transparent way. Our objective was to search and analyze recommendations regarding where and how to include expert data in HTA.

Methods: We performed a systematic database search and screened the Internet pages of seventy-seven HTA organizations for guidelines, recommendations, and methods papers that address the inclusion of experts in HTA. Relevant documents were downloaded, and information was extracted in a standard form. Results were merged in tables and narrative evidence synthesis.

Results: From twenty-two HTA organizations, we included forty-two documents that consider the use of expert opinion in HTA. Nearly all documents mention experts in the step of preparation of the evidence report. Six documents address their role for priority setting of topics, fifteen for scoping, twelve for the appraisal of evidence and results, another twelve documents mention experts when considering the dissemination of HTA results.

During the assessment step, experts are most often asked to amend the literature search or to provide expertise for special data analyses. Another issue for external experts is to appraise the HTA results and refer them back to a clinical and social context. Little is reported on methods of expert elicitation when their input substitutes study data.

Conclusions: Despite existing recommendations on the use of expert opinion in HTA, common standards for elicitation are scarce in HTA guidelines.

Purpose: A peripherally inserted central catheter (PICC) is a useful option in providing secure venous access, which enables patients to be discharged earlier with the provision of home care. The objective was to identify the costs associated with having a PICC from a societal perspective, and to identify factors that are associated with total PICC costs.

Methods: Data were obtained from a retrospective cohort of 469 hospitalized pediatric patients with PICCs inserted. Both direct and indirect costs were estimated from a societal perspective. Insertion costs, complication costs, nurse and physician assessment costs, inpatient ward costs, catheter removal costs, home care costs, travel costs, and the cost associated with productivity losses incurred by parents were included in this study.

Results: Based on catheter dwell time, the median total cost associated with a PICC per patient per day (including inpatient hospital costs) was $3,133.5 ($2,210.7–$9,627.0) in 2017 Canadian dollars ($1.00USD = $1.25CAD in 2017). The adjusted mean cost per patient per day was $2,648.2 ($2,402.4–$2,920.4). Excluding inpatient ward costs, the median total and adjusted costs per patient per day were $198.8 ($91.8–$2,475.8) and $362.7($341.0–$386.0), respectively. Younger age, occurrence of complications, more catheter dwell days, wards with more intensive care, and the absence of home care were significant factors associated with higher total PICC costs.

Conclusions: This study has demonstrated the costs associated with PICCs. This information may be helpful for healthcare providers to understand PICC related cost in children and resource implications.

Background: The Institute for Quality and Efficiency in Health Care (IQWiG) was established in 2003 by the German parliament. Its legislative responsibilities are health technology assessment, mostly to support policy making and reimbursement decisions. It also has a mandate to serve patients’ interests directly, by assessing and communicating evidence for the general public.

Objectives: To develop a priority-setting framework based on the interests of patients and the general public.

Methods: A theoretical framework for priority setting from a patient/consumer perspective was developed. The process of development began with a poll to determine level of lay and health professional interest in the conclusions of 124 systematic reviews (194 responses). Data sources to identify patients’ and consumers’ information needs and interests were identified.

Results: IQWiG's theoretical framework encompasses criteria for quality of evidence and interest, as well as being explicit about editorial considerations, including potential for harm. Dimensions of “patient interest” were identified, such as patients’ concerns, information seeking, and use. Rather than being a single item capable of measurement by one means, the concept of “patients’ interests” requires consideration of data and opinions from various sources.

Conclusions: The best evidence to communicate to patients/consumers is right, relevant and likely to be considered interesting and/or important to the people affected. What is likely to be interesting for the community generally is sufficient evidence for a concrete conclusion, in a common condition. More research is needed on characteristics of information that interest patients and consumers, methods of evaluating the effectiveness of priority setting, and methods to determine priorities for disinvestment.