Background: The evidence supporting the use of new, or established, interventions may be derived from either (or both) experimental or observational study designs. Although a rigorous examination of the evidence base for clinical and cost-effectiveness is essential, it is never sufficient, and those undertaking a health technology assessment (HTA) also have to exercise judgments.

Methods: The basis for this discussion is largely from the author's experience as chairman of the national Institute for Health and Clinical Excellence (NICE).

Results: The judgments necessary for HTA to make are twofold. Scientific judgments relate to the interpretation of the science. Social value judgments are concerned with the ethical principles, preferences, culture, and aspirations of society.

Conclusions: How scientific and social value judgments might be most appropriately captured is a challenge for all HTA agencies. Although competent HTA bodies should be able to exercise scientific judgments they have no legitimacy to impose their own social values. These must ultimately be informed by the general public.

Rationale: Bayesian methods provide an interesting approach to assessing an implantable medical device (IMD) that has evolved through successive versions because they allow for explicit incorporation of prior knowledge into the analysis. However, the literature is sparse on the feasibility and reliability of elicitation in cases where expert beliefs are used to form priors.

Objectives: To develop an Internet-based method for eliciting experts’ beliefs about the success rate of an intracranial stenting procedure and to assess their impact on the estimated benefit of the latest version.

Study Design and Setting: The elicitation questionnaire was administered to a group of nineteen experts. Elicited experts’ beliefs were used to inform the prior distributions of a Bayesian hierarchical meta-analysis model, allowing for the estimation of the success rate of each version. RESULTS: Experts believed that the success rate of the latest version was slightly higher than that of the previous one (median: 80.8 percent versus 75.9 percent). When using noninformative priors in the model, the latest version was found to have a lower success rate (median: 83.1 percent versus 86.0 percent), while no difference between the two versions was detected with informative priors (median: 85.3 percent versus 85.6 percent).

Conclusions: We proposed a practical method to elicit experts’ beliefs on the success rates of successive IMD versions and to explicitly combine all available evidence in the evaluation of the latest one. Our results suggest that the experts were overoptimistic about this last version. Nevertheless, the proposed method should be simplified and assessed in larger, representative samples.

Objectives: Health economic models are developed as part of the health technology assessment process to determine whether health interventions represent good value for money. These models are often used to directly inform healthcare decision making and policy. The information needs for the model require the use of other types of information beyond clinical effectiveness evidence to populate the model's parameters. The purpose of this research study was to explore issues concerned with the identification and use of information for the development of such models.

Methods: Three focus groups were held in February 2011 at the University of Sheffield with thirteen UK HTA experts. Attendees included health economic modelers, information specialists and systematic reviewers. Qualitative framework analysis was used to analyze the focus group data.

Results: Six key themes, with related sub-themes, were identified dealing with decisions and judgments; searching methods; selection and rapid review of evidence; team communication; modeler experience and clinical input and reporting methods. There was considerable overlap between themes.

Conclusions: Key issues raised by the respondents included the need for effective communication and teamwork throughout the model development process, the importance of using clinical experts as well as the need for transparent reporting of methods and decisions.

Introduction: Genetic factors affecting warfarin maintenance dose have been well established, but patient acceptance is a necessary consideration for the successful clinical implementation of warfarin pharmacogenetic testing (WPGT). In this study, we aimed to determine the attitudes toward WPGT among Singaporean Chinese.

Methods: A total of 194 warfarin patients and 187 members of the public completed a structured survey on paper and the Internet, respectively. Attitudes were expressed as willingness to undergo WPGT (single item with 5-point response) and expectations and concerns about WPGT (two multi-item scales). Relationships between attitudes and socio-demographic and clinical variables were explored using Fisher's exact test, Student's t-test, one-way analysis of variance or Pearson's correlation.

Results: Majority of respondents were willing to or neutral about undergoing WPGT. Both patients and public had relatively high expectations (mean [SD]: 3.77 [0.63], and 3.97 [0.55], respectively) and moderately high concerns (mean [SD]: 3.30 [0.69] and 3.33 [0.68], respectively) about WPGT. Willingness to undergo WPGT was associated with gender, educational status, length of warfarin treatment, and number of chronic diseases among warfarin patients, and with history of adverse drug reactions and number of chronic diseases among the public. Higher expectation of WPGT was associated with higher willingness (p < .001 in both populations), while higher concern was associated with lower willingness to undergo WPGT among the public (p = .004) but not among patients (p = .072).

Conclusion: Patient acceptance is not a major barrier to clinical implementation of WPGT but patient education is necessary and the ethical, social, and legal issues should be addressed.

Objectives: The aim of this study was to analyze the experience of the National Institute for Health and Care Excellence (NICE) in identifying new procedures entering the United Kingdom (UK) healthcare system, for assessment and publication of recommendations on their use. This system is designed to provide guidance in an area where regulation is lacking worldwide.

Methods: Retrospective analysis of all procedures notified to the Interventional Procedures Programme (NICE) between 2002 and 2012. Notifications were analyzed year by year for their source (who notified them), clinical specialties involved, and whether guidance was subsequently published.

Results: A total of 1,094 procedures were notified by clinicians (51 percent), and by others, including hospitals (6 percent), horizon scanners (5 percent), patients (4 percent), private health insurers (4 percent), and medical device manufacturers (3 percent). Guidance was published on 44 percent of procedures notified to the program. There was a decrease in the numbers of procedures notified during 2003–2012 (p = .049). There were notifications across all specialties, with the largest numbers in general surgery (125), urology (104), orthopedics (99), interventional radiology (93), cardiology (82), and obstetrics and gynecology (82).

Conclusions: The “open” NICE Web portal allows anyone to notify new procedures, aiming to maximize the opportunity of identifying all those procedures entering clinical practice. This has resulted in identification of large numbers of procedures from across the whole range of medical specialties. The fact that similar proportions of procedures notified from diverse sources have been selected for assessment and publication of practice recommendations suggests that this inclusive approach is worthwhile.

Objectives: This study reports on the development of a critical process for health technologies incorporation concerning an Argentinean Provincial Ministry of Health (MOH) in collaboration with the University of Lanús from 2008 to 2010.

Methods: We describe the approach developed to adapt selected international experiences to provincial scenario. Bibliographic review, regulations examination, key informants interviews and iterative adjustments after various stages of consultation and consensus building with main local players, contribution from foreign experts, and piloting of process and instruments for ultimate fine-tuning are described. We examine final proposal in the light of new updated studies.

Results: Analysis of regulations revealed that rules governing the provincial system were historically linked to administrative resolutions in relation to procurement with poor consideration to clinical, epidemiological, organizational, and health policy aspects. Key informants from hospitals, MOH, and other governmental agencies agreed on the lack of a process capable of guaranteeing a decision about health technology incorporation based on a transparent use of the best available information, ready to deal with competitive pressures. This adaptation provided a structured and explicit process (introduction, implementation, and development) as well as essential and supporting tools.

Conclusions: MOH adopted the proposal for its progressive implementation while institutional evaluation capacity develops. Further studies are needed on the value placed on health technology assessment–based processes and recommendations by clinicians, managers, policy makers, and patients.

The affiliation listed for author Sergio Iannazzo for the article “Economic Evaluation of Intravenous Iodinated Contrast Media in Italy” was incorrect. The study and writeup were completed while Sergio Iannazzo was an IMS Health employee, and the author's correct affiliation for this paper is “IMS Health Italy.” The publisher regrets the error.

Objectives: The aim of this study was to determine the prevalence and methods of expert knowledge elicitation (EKE) for specifying input parameters in health economic decision models (HEDM).

Methods: We created two samples using the National Health System Economic Evaluations Database: (1) 100 randomly selected HEDM studies to determine prevalence of EKE and (2) sixty studies using a formal EKE process to determine methods used.

Results: Fifty-seven (57 percent) of the random sample included at least one EKE-derived parameter. Of these, six (10 percent) used a formal expert process. Thirty-four studies from our second sample of sixty studies (57 percent) described at least one aspect of the process (e.g., elicitation method) with reasonable clarity. In approximately two-thirds of studies the external experts estimated parameters de novo; the remainder confirmed or modified initial estimates provided by authors, or the method was unclear. The majority of elicitations obtained point estimates only, although a few studies asked experts to estimate ranges of parameter values.

Conclusions: The use of EKE for parameter estimation is common in HEDMs, although there is room for improvement in the methods used.

In some countries, reimbursement of drugs is based on cost-effectiveness analysis (CEA), in others not. In times of ageing populations, increasing number of possible interventions, and limited resources, it seems likely that CEA will be more and more important as a basis for decision making.

Objectives: The aim of this study was to determine the economic costs and the level of catastrophic health expenditure (CHE) due to childhood epilepsy.

Methods: The study was conducted at the Paediatric Neurology Clinic of the University of Nigeria Teaching Hospital, Enugu. Data were collected using pre-tested questionnaires that were administered to caregivers of the children. The indirect and direct expenditure due to childhood epilepsy were computed. A 40 percent of monthly non-food expenditure was used to estimate CHE.

Results: The average annual direct and indirect expenditures were USD 162.6 and USD 82.3, respectively. Most of direct costs were drugs (25.4 percent versus 35.3 percent) and investigations (48.7 percent versus 61.3 percent) for out-patient and in-patient, respectively. CHE was 34.1 percent and 63.6 percent for out-patient and in-patient care, respectively. The total annual costs: (direct and indirect), for childhood epilepsy of USD244.9. Considering the estimated 190,000 epileptic children in Nigeria, it will amount to USD46.53 million annually, approximately 0.018 percent of Nigeria Gross Domestic Product (GDP). All payments were made out-of-pocket with no health insurance for financial risk protection.

Conclusions: The cost of treatment of childhood epilepsy is high and catastrophic for many households. There was lack of usage of health financial risk mechanisms. Scale-up use of health financial risk protection mechanisms such as health insurance can reduce the economic burden.

In our article “Romiplostim and eltrombopag for immune thrombocytopenia: methods for indirect comparison” published in the Journal (1), we presented an indirect comparison of the effectiveness of eltrombopag and romiplostim in raising platelet counts in patients with immune thrombocytopenia (ITP). Indirect comparison analyses are recommended by the National Institute for Health and Care Excellence (NICE) in cases where randomized head-to-head studies do not exist, and were used by NICE in their guidance for the eltrombopag Single Technology Appraisal submission (2).