Pharmaceutical sales totaled an estimated USD 700 billion in OECD countries in 2011, approximately one-fifth of total healthcare spending (5). In the past, pharmaceutical expenditure has increased faster than total healthcare expenditure in many countries. The increase in pharmaceutical expenditure has slowed down in recent years due to the economic recession, patent expirations of important blockbusters, and cost-containment measures already taken. However, pharmaceutical expenditure still continues to rise, albeit at a reduced rate. Alongside an increased consumption of medicines, an important reason for growing costs is the adoption of new—and typically more expensive—products. To ensure that pharmaceutical expenditure does not become excessive and that patients have access to medicines they need, most countries now weigh up what products are to be included in the reimbursement system and at what price. Both the criteria to assess products and the processes to make reimbursement decisions vary from country to country and so far there have been no analytically oriented comparison studies.

Objectives: The aim of our study is to compare five European drug reimbursement systems, describe similarities and differences, and obtain insight into their strengths and weaknesses and formulate policy recommendations.

Methods: We used the analytical Hutton Framework to assess in detail drug reimbursement systems in Austria, Belgium, France, the Netherlands, and Sweden. We investigated policy documents, explored literature, and conducted fifty-seven interviews with relevant stakeholders.

Results: All systems aim to balance three main objectives: system sustainability, equity and quality of care. System impact, however, is mainly assessed by drug expenditure. A national reimbursement agency evaluates reimbursement requests on a case-by-case basis. The minister has discretionary power to alter the reimbursement advice in Belgium, France, and the Netherlands. All systems make efforts to increase transparency in the decision-making process but none uses formal hierarchical reimbursement criteria nor applies a cost-effectiveness threshold value. Policies to deal with uncertainty vary: financial risk-sharing by price/volume contracts (France, Belgium) versus coverage with evidence development (Sweden, the Netherlands). Although case-by-case revisions are embedded in some systems for specific groups of drugs, systematic (group) revisions are limited.

Conclusions: As shared strengths, all systems have clear objectives reflected in reimbursement criteria and all are prepared to pay for drugs with sufficient added value. However, all systems could improve the transparency of the decision-making process; especially appraisal lacks transparency. Systems could increase the use of (systematic) revisions and could make better use of HTA (among others cost-effectiveness) to obtain value for money and ensure system sustainability.

Objectives. In a democratic system, decision makers are accountable for the reasonableness of their decisions. This presumes (i) transparency, (ii) relevance of the decision criteria, (iii) revisability of decisions, and (iv) enforcement/regulation. We aim to (i) evaluate the extent to which drug reimbursement decision-making processes in different contexts meet these conditions and (ii) develop, starting from these findings, a framework for improving the transparency and the relevance of used decision criteria.

Methods. We evaluated the Austrian, Belgian, French, Dutch, and Swedish drug reimbursement systems. Based on this evaluation, we developed a framework for improving the transparency of drug reimbursement decision-making processes. It makes explicit the questions often addressed implicitly during decision-making processes as well as criteria for answering each question.

Results. Transparency of appraisal processes varies across systems. Justification with explicit criteria is generally limited. Although relevant criteria are similar across systems, their operationalization varies and their role in the appraisal process is not always clear. All systems seem to implicitly address five key questions, relating to (i) the medical, therapeutic, and societal need for treatment; (ii) preparedness to pay for treating the condition as a principle and (iii) for using the treatment under consideration; (iv) preparedness to pay more compared with alternatives; and (v) actual willingness to pay from public resources.

Conclusions. Transparency of the appraisal process can be improved by using an explicit decision framework. Systematic use of such a framework enhances consistency across decisions, allows justification of value judgments, and thus enhances legitimacy of societal decision making.

Objectives: The aim of this study is to examine key informants’ perceptions of how the New Zealand Pharmaceutical Management Agency (PHARMAC) operates in New Zealand.

Methods: We carried out qualitative analysis of semi-structured interviews with key informants. We obtained ethics approval from the University of Otago School of Pharmacy, and all participants gave informed consent. We digitally recorded the interviews, which were then transcribed, and coded in NVivo. The data were analyzed by theme using constant comparison methods. Twenty informants who had previously published research or commentary on New Zealand's access to medicines, acted as spokespersons for interest groups, or held positions that gave them key insights into New Zealand's medicines system agreed to participate. Informants were purposefully selected to ensure a wide range of views, including five people working in medicine, four in pharmacy, three Members of Parliament from different parties, and two each from PHARMAC and the pharmaceutical industry.

Results: Respondents saw PHARMAC as an organization that contained medicine costs effectively, was politically neutral, and resistant to lobbying. It enjoyed broad political support and, with extremely rare exceptions, had been allowed to carry out its functions independently regardless of who was in government. As a result of this political stability, the relationship between PHARMAC and the pharmaceutical industry has been improving.

Conclusion: PHARMAC's longevity and increasing influence are largely due to political choices made to prioritize containing pharmaceutical expenditure and to respecting PHARMAC's independence. This may be difficult to replicate in other countries.

There has been an increased focus on the relationship between health technology assessment (HTA) and regulatory assessments and how regulatory, HTA and coverage bodies, and industry can work better together to improve efficiency and alignment of processes. There is increasingly agreement across sectors that improved communication and coordination could contribute to facilitating timely patient access to effective, affordable treatments that offer value to the health system. Discussions on aspects of this relationship are being held in different forums and various forms of coordination and collaboration are being developed or piloted within several jurisdictions. It is therefore both timely and of value to stakeholders to describe and reflect on current initiatives intended to improve interactions between regulatory, HTA and coverage bodies, and industry. Drawing on 2011 meetings of the HTAi Policy Forum and the Center for Innovation in Regulatory Science (CIRS), this study aims to describe and compare initiatives, and point to success factors and challenges that are likely to inform future work and collaboration.

Background: The cost-effectiveness of triptans in the treatment of migraine has not been assessed since generic sumatriptan entered the Finnish market in 2008.

Methods: Using systematic review and mixed treatment comparison, the effectiveness of triptans was estimated with regard to 2-hour response, 2-hour pain-free, recurrence, and any adverse event, using published clinical data. Direct and indirect costs (2010 EUR, societal perspective) and quality-adjusted life-years (QALYs) were evaluated over one acute migraine attack using a decision-tree model.

Results: The meta-analysis combined data from fifty-six publications. The highest probability of achieving the primary outcome, “sustained pain-free, no adverse event” (SNAE), was estimated for eletriptan 40 mg (20.9 percent). Sumatriptan 100 mg was the treatment with lowest estimated costs (€20.86), and the incremental cost-effectiveness ratio of eletriptan 40 mg compared with sumatriptan 100 mg was €43.65 per SNAE gained (€19,659 per QALY gained).

Conclusion: Depending on the decision-maker's willingness-to-pay threshold, either sumatriptan 100 mg or eletriptan 40 mg is likely to be cost-effective.

Background: The benefits of pharmaceutical innovations are widely diffused; they accrue to the healthcare providers, patients, employers, and manufacturers. We estimate the societal monetary benefits of simvastatin in Canada and its distribution among different beneficiaries overtime.

Methods: Monetary benefits to developing and generic manufacturers were estimated by calculating public and private revenues minus the development costs of simvastatin and the contribution toward further research and development. We used a dynamic Markov model to estimate monetary benefits to healthcare and employment sectors in terms of cost avoidance associated with prevented cardiovascular events, including stroke and myocardial infarction, and lost productivity due to disability and premature death in working population.

Results: Cumulative monetary benefits of simvastatin from 1990 to 2009 were $4.8 billion (2010 CA$), of which developing and generic manufacturers, and healthcare and employment sectors accounted for 32 percent, 27 percent, 32 percent, and 9 percent, respectively. The yearly trend showed that after the patent expired in 2002 the generic manufacturers became dominant in the market. Benefits for the healthcare sector started to decrease from 2003 corresponding to the decreasing population taking simvastatin during the same time period. Sensitivity analysis showed the higher the compliance or the efficacy, the larger the benefits to healthcare and employment sectors, while monetary benefits for manufacturers were unchanged.

Conclusions: Societal monetary benefits of simvastatin are significant and the distributions of the benefits have changed overtime. Patent, compliance, and efficacy play a vital role in the estimation of the benefits. Analysis of all beneficiaries separately overtime is important when assessing the value of pharmaceutical innovation.

Background: Peginterferon alfa and ribavirin combination therapy is an effective treatment for many patients with chronic hepatitis C virus (HCV). Reducing the length of treatment may be advantageous. We performed a systematic review and economic evaluation to assess shorter treatment duration of this regimen.

Methods: We searched fourteen bibliographic databases (including The Cochrane Library, Medline, and Embase) from 2000 to October 2009 and consulted experts and drug manufacturers. Eligible articles were randomized controlled trials (RCTs) selected according to predefined criteria. We undertook an economic evaluation to assess the cost-effectiveness of shortened treatment versus standard treatment in the UK.

Results: Six trials were included. In the sub-group of patients who had low viral load (LVL) and a rapid virological response (RVR), there were no statistically significant differences in sustained virological response (SVR) rates between patients who received standard treatment (range, 83 percent to 100 percent) and those who received shortened courses (range 84 percent to 96 percent) (24 weeks for genotype 1, 16 weeks for genotype 2/3). Shortened treatment resulted in cost savings, but in some scenarios also resulted in poorer outcome, compared with standard treatment. This requires a judgment to be made on the value of the quality-adjusted life-year loss resulting from adopting a shorter treatment regimen, if shorter treatment is associated with a lower SVR than standard treatment duration.

Conclusions: For chronic HCV patients who have LVL and achieve an RVR, shortened peginterferon and ribavirin combination therapy could be considered as a viable treatment option.

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.

Objectives: The European Code Against Cancer recommends individuals aged ≥50 should participate in colorectal cancer screening. CT-colonography (CTC) is one of several screening tests available. We systematically reviewed evidence on, and identified key factors influencing, cost-effectiveness of CTC screening.

Methods: PubMed, Medline, and the Cochrane library were searched for cost-effectiveness or cost-utility analyses of CTC-based screening, published in English, January 1999 to July 2010. Data was abstracted on setting, model type and horizon, screening scenario(s), comparator(s), participants, uptake, CTC performance and cost, effectiveness, ICERs, and whether extra-colonic findings and medical complications were considered.

Results: Sixteen studies were identified from the United States (n = 11), Canada (n = 2), and France, Italy, and the United Kingdom (1 each). Markov state-transition (n = 14) or microsimulation (n = 2) models were used. Eleven considered direct medical costs only; five included indirect costs. Fourteen compared CTC with no screening; fourteen compared CTC with colonoscopy-based screening; fewer compared CTC with sigmoidoscopy (8) or fecal tests (4). Outcomes assessed were life-years gained/saved (13), QALYs (2), or both (1). Three considered extra-colonic findings; seven considered complications. CTC appeared cost-effective versus no screening and, in general, flexible sigmoidoscopy and fecal occult blood testing. Results were mixed comparing CTC to colonoscopy. Parameters most influencing cost-effectiveness included: CTC costs, screening uptake, threshold for polyp referral, and extra-colonic findings.

Conclusion: Evidence on cost-effectiveness of CTC screening is heterogeneous, due largely to between-study differences in comparators and parameter values. Future studies should: compare CTC with currently favored tests, especially fecal immunochemical tests; consider extra-colonic findings; and conduct comprehensive sensitivity analyses.

Objectives: Information on the impact of computed tomography coronary angiography (CTCA) on patterns of care is scarce. In particular, it is not clear if, and to what extent, its adoption actually leads to a reduction in the use of other diagnostics tools. The aim of this study was to evaluate whether the adoption of CTCA in Emilia-Romagna (an Italian region with a population of 4.4 million) had any effect on utilization rates of myocardial perfusion scintigraphy (MPS) and coronary angiography (CA).

Methods: Interrupted time series (ITS) were applied to monthly volumes of MPS and CA tests performed from 2003 to 2010, to assess trends in usage rates for those procedures before and after CTCA was adopted by all the healthcare organizations operating in the region.

Results: After an increase in the first year of CTCA introduction, its use remained stable over the study period. After September 2006, a significant decrease in MPS volumes (31 percent; p < .0001) and a much less tangible decrease in CA volumes (5 percent; p < .0001), were detected by ITS analyses.

Conclusions: This study demonstrates that the use of CTCA had a greater impact on MPS usage rates than on CA.

Objectives: Cardiac resynchronization therapy (CRT) has recently been shown to reduce both mid-term and long-term mortality in patients with mild heart failure. Although proven effective, it is unclear whether CRT is cost-effective in low and middle-income countries (LMIC). Therefore, we set out to analyze the cost-effectiveness of CRT in Argentina in patients with New York Heart Association (NYHA) functional class (FC) I or II heart failure (HF). We chose to compare patients receiving optimal medical treatment (OMT) and CRT with those patients receiving only OMT.

Methods: We constructed a Markov model with a cohort simulation, and a life-time horizon to assess costs, life-years, and quality-adjusted life-year (QALY) gained as a result of treatment with both CRT and OMT from an Argentine third party payer perspective. We included patients who met the following criteria: left ventricular ejection fraction (LVEF) ≤ 40 percent, sinus rhythm with a QRS ≥ 120 msec, and NYHA FC I-II HF. The results were expressed as cost per life-year and QALY gained in international dollars (ID$) for the year 2009.

Results: For the base case analysis performed, we started at a fixed age of 65. After applying a 3 percent annual discount rate, the incremental cost-effectiveness ratio (ICER) was 38.005 ID$ per year of life gained and 34.185 ID$ per QALY gained.

Conclusions: Long-term treatment with CRT appears to be cost-effective in Argentina compared with patients treated solely with OMT. Similar analysis should be performed to determine if this treatment option is cost-effective in other LMIC.

Objectives: The aim of this study was to evaluate the long-term cost-utility of liraglutide versus glimepiride as add-on therapy to metformin in patients with type 2 diabetes mellitus (T2DM), based on the results of clinical trial conducted in Asian population.

Methods: The validated UKPDS Outcomes Model was used to project life expectancy, quality adjusted life-years (QALYs), incidence of diabetes-related complication and cost of complications in patients receiving those regimens. Baseline cohort characteristics and treatment effects were derived from an Asian study. China-specific complication costs and utility score were taken from local studies. Patients’ outcomes were modeled for 30 years and incremental cost-effectiveness ratios were calculated for liraglutide compared with glimepiride from the healthcare system perspective. Both future costs and clinical benefits were discounted at 3 percent. Sensitivity analyses were performed.

Results: Over a period of 30 years, compared with glimepiride, liraglutide 1.8 mg was associated with improvements in life expectancy (0.1 year) and quality adjusted life-year (0.168 QALY), and a reduced incidence of diabetes-related complications leading to an incremental cost-effectiveness ratio per QALY gained versus glimepiride of CNY 25,6871 (DEC 2010, 1 USD = 6.6227 CNY).

Conclusions: Long-term projections indicated that liraglutide was associated with increased life expectancy, QALYs, and reduced complication incidences comparing with glimepiride. When the UK cost of liraglutide was discounted by 38 percent, liraglutide would be a cost-effective option in China from the healthcare system perspective using the 3X GDP/capita per QALY as the WTP threshold.

Objectives: The objective of this study is to review the implementation of health technology assessment (HTA) at the local and hospital levels in low- and middle-income countries (LMIC). This review will provide a starting point for identifying the conditions for HTA implementation in hospitals in LMIC through the analysis of experiments conducted in these countries.

Methods: A systematic review of the literature was conducted to document the local-/hospital-level HTA experiments performed in LMIC.

Results: This systematic review showed that few experiments of local HTA in LMIC have been published to date, with only five articles found in our survey. These documents report studies of clinical effectiveness and economic evaluation at the local level in certain Asian and Latin American countries. In addition, pharmaceuticals and medical devices were the most common topics covered by HTA at the local level in these countries.

Conclusions: Currently, HTA plays an increasingly important role in healthcare systems in supporting decision making for healthcare policies and practices. This systematic review contributes to identify priorities in the process and methodology of HTA implementation at the local/hospital level in LMIC. The paucity of HTA in LMIC is often assumed to be due to the lack of formally tasked HTA agencies, to politics and to shortage of resources.

Background and Objectives: Horizon scanning systems need to handle a wide range of sources to identify new or emerging health technologies. The objective of this study is to develop a validated Medline bibliographic search strategy (PubMed search engine) to systematically identify new or emerging health technologies.

Methods: The proposed Medline search strategy combines free text terms commonly used in article titles to denote innovation within index terms that make reference to the specific fields of interest. Efficacy was assessed by running the search over a period of 1 year (2009) and analyzing its retrieval performance (number and characteristics). For comparison purposes, all article abstracts published during 2009 in six preselected key research journals and eight high impact surgery journals were scanned. Sensitivity was defined as the proportion of relevant new or emerging technologies published in key journals that would be identified in the search strategy within the first 2 years of publication.

Results: The search yielded 6,228 abstracts of potentially new or emerging technologies. Of these, 459 were classified as new or emerging (383 truly new or emerging and 76 new indications). The scanning of 12,061 journal abstracts identified 35 relevant new or emerging technologies. Of these, twenty-nine were located within the Medline search strategy during the first 2 years of publication (sensitivity = 83 percent).

Conclusions: The current search strategy, validated against key journals, has demonstrated to be effective for horizon scanning. Even though it can require adaptations depending on the scope of the horizon scanning system, it could serve to simplify and standardize scanning processes.

Objective: To develop and test a decision-support tool for prioritizing new competing Health Technologies (HTs) after their assessment using the mini-HTA approach.

Methods: A two layer value/risk tool was developed based on the mini-HTA. The first layer included 12 mini-HTA variables classified in two dimensions, namely value (safety, clinical benefit, patient impact, cost-effectiveness, quality of the evidence, innovativeness) and risk (staff, space and process of care impacts, incremental costs, net cost, investment effort). Weights given to these variables were obtained from a survey among decision-makers (at National/Regional level and hospital settings). A second layer included results from mini-HTA (scored as higher, equal or lower), which compares the performance of the new HT (in terms of the abovementioned 12 variables) with the available comparator. An algorithm combining the first (weights) and second (scores) layers was developed to obtain an overall score for each HT, which was then plotted in a value/risk matrix. The tool was tested using results from the mini-HTAs for three new HTs (Surgical Robot, Platelet Rich Plasma, Deep Brain Stimulation).

Results: No significant differences among decision-makers were observed as regards the weights given to the 12 variables, therefore, the median aggregate weights from decision-makers were introduced in the first layer. The dot plot resulting from the mini-HTA presented good power to visually discriminate between the assessed HTs.

Conclusion: The decision-support tool developed here makes possible a robust and straightforward comparison of different competing HTs. This facilitates hospital decision-makers deliberations on the prioritization of competing investments under fixed budgets.