In this issue of the Journal, Panteli et al. (6) provide insight into the extent to which a gender-sensitive approach is adopted by sixty health technology assessment (HTA) agencies worldwide. Their findings should make all of us involved in the production of HTA take pause: less than a handful of the agency Web sites that were examined by Panteli's team made any mention of gender as an explicit consideration in priority setting processes or in the HTA methods used (6). This is despite the fact that gender is recognized as a social determinant of health (1) and despite best practices that acknowledge the need to account for equity issues—of which gender is one—in the design, conduct, and reporting of HTA (3;4). Assuming we take the findings of Panteli et al. at face value, this does seem to be a case of “do as I say, not as I do.”

During the 26th year of the International Journal of Technology Assessment in Health Care (IJTAHC), 154 original manuscripts were evaluated. Of these, 70 percent were from Europe and 14 percent from North America, as judged by the address of the first author. At the end of September 2010, sixty-four papers (42 percent of submissions) had been accepted for publication, seventy-two (47 percent) had been rejected, fourteen (9 percent) had been invited to submit a new version, and four (3 percent) were still in review.

Scientific reports should be orderly, clear, and unambiguous. Clarity is even more important for health technology assessments (HTAs), which aim at supplying reliable and up-to-date evidence on the effectiveness, safety, and cost of healthcare interventions. Increasingly assessments also provide information about the effects of technologies on organization of care, as well as relevant legal, social, and ethical considerations.

HTA can be described as a research-based, practice-oriented assessment of relevant evidence and knowledge on the direct and intended effects of healthcare technologies, as well as the indirect and unintended consequences (4). It is also viewed as a multidisciplinary field of policy analysis that studies the medical, ethical, social, and economic implications of the development, diffusion, and use of a health technology (5).

Objectives: Previous research has suggested people with impaired fasting glucose (IFG) are less likely to develop Type 2 diabetes (T2DM) if they receive prolonged structured diet and exercise advice. This study examined the within-trial cost-effectiveness of such lifestyle interventions.

Methods: Screen-detected participants with either newly diagnosed T2DM or IFG were randomized 2:1 to intervention versus control (usual care) between February and December 2009, in Norfolk (UK). The intervention consisted of group based education, physiotherapy and peer support sessions, plus telephone contacts from T2DM volunteers. We monitored healthcare resource use, intervention costs, and quality of life (EQ-5D). The incremental cost per quality-adjusted life-year (QALY) gain (incremental cost effectiveness ratio [ICER]), and cost effectiveness acceptability curves (CEAC) were estimated.

Results: In total, 177 participants were recruited (118 intervention, 59 controls), with a mean follow-up of 7 months. Excluding screening and recruitment costs, the mean cost was estimated to be £551 per participant in the intervention arm, compared with £325 in the control arm. The QALY gains were –0.001 and –0.004, respectively. The intervention was estimated to have an ICER of £67,184 per QALY (16 percent probability of being cost-effective at the £20,000/QALY threshold). Cost-effectiveness estimates were more favorable for IFG participants and those with longer follow-up (≥4 months) (ICERs of £20,620 and £17,075 per QALY, respectively).

Conclusions: Group sessions to prevent T2DM were not estimated to be within current limits of cost-effectiveness. However, there was a large degree of uncertainty surrounding these estimates, suggesting the need for further research.

Objectives: The aim of this study was to assess the test performance and clinical effectiveness of photodynamic diagnosis (PDD) compared with white light cystoscopy (WLC) in people suspected of new or recurrent bladder cancer.

Methods: A systematic review was conducted of randomized controlled trials (RCTs), nonrandomized comparative studies, or diagnostic cross-sectional studies comparing PDD with WLC. Fifteen electronic databases and Web sites were searched (last searches April 2008). For clinical effectiveness, only RCTs were considered.

Results: Twenty-seven studies (2,949 participants) assessed test performance. PDD had higher sensitivity than WLC (92 percent, 95 percent confidence interval [CI], 80–100 percent versus 71 percent, 95 percent CI, 49–93 percent) but lower specificity (57 percent, 95 percent CI, 36–79 percent versus 72 percent, 95 percent CI, 47–96 percent). For detecting higher risk tumors, median range sensitivity of PDD (89 percent [6–100 percent]) was higher than WLC (56 percent [0–100 percent]) whereas for lower risk tumors it was broadly similar (92 percent [20–95 percent] versus 95 percent [8–100 percent]). Four RCTs (709 participants) using 5-aminolaevulinic acid (5-ALA) as the photosensitising agent reported clinical effectiveness. Using PDD at transurethral resection of bladder tumor (TURBT) resulted in fewer residual tumors at check cystoscopy (relative risk [RR], 0.37, 95 percent CI, 0.20–0.69) and longer recurrence-free survival (RR, 1.37, 95 percent CI, 1.18–1.59), compared with WLC.

Conclusions: PDD detects more bladder tumors than WLC, including more high-risk tumors. Based on four RCTs reporting clinical effectiveness, 5-aminolaevulinic acid–mediated PDD at TURBT facilitates a more complete resection and prolongs recurrence-free survival.

Objectives: In a practice setting where casting is considered the standard of care, the aim of this study was to assess the cost-effectiveness of wrist splints compared with routine casting in children with acceptably angulated distal radius greenstick or transverse fractures.

Methods: A cost-effectiveness analysis was conducted alongside a randomized controlled trial (RCT). One hundred children with acceptably angulated distal radius greenstick or transverse fractures received either a wrist splint or cast. Information on health care provider and patient and family resource use as well as productivity cost was collected. Resource use was costed using unit costs from local administrative data sources and expense diaries. Effectiveness was assessed at 6 weeks using the performance version of the Activities Scale for Kids (ASKp) questionnaire. Cost-effectiveness analysis related differential costs to differential ASKp scores.

Results: Mean total cost was $877.58 in the splint group and $950.35 in the cast group, with a mean difference of $−72.76 (standard error [SE] 45.88). Mean total healthcare cost was $670.66 in the splint group and $768.22 in the cast group, with a mean difference of $−97.56 (SE 9.24). Mean (SE) ASKp was 92.8 in the splint group and 91.4 in the cast group, with a mean difference of 1.439 (SE 1.585). Therefore, splint management was more effective and cheaper. After accounting for uncertainty, the probability of splint being cost-effective compared with cast was 94 percent for a willingness-to-pay threshold value of $0 for one-unit gain in ASKp score and exceeded 82 percent for all threshold values.

Conclusions: In this RCT, splint management was cost-effective compared with casting in children with acceptably angulated distal radius greenstick or transverse fractures. This study challenges the existing standard of care for children with this type of fracture and provides justification on clinical and economic grounds for a change in routine practice.

Objectives: To explore the cost-effectiveness of early biologic treatment, followed by dose-reduction in the case of remission, of active rheumatoid arthritis (RA), compared with standard treatment with methotrexate (MTX) in Sweden.

Methods: Effectiveness (function, disease activity, erosions) in early RA for both alternatives was taken from a clinical trial comparing etanercept (ETA) combined with MTX to MTX alone. Patients discontinuing treatment can switch to another or their first biologic treatment. For patients in remission (Disease Activity Score [DAS28] < 2.6), ETA is reduced to half the dose. Return to full dose occurs when DAS28 reaches ≥ 3.2 again. Costs and utilities by level of functional capacity from an observational study are used. The model is analyzed as a micro-simulation and results are presented from the societal perspective for Sweden, for 10 years; costs (€2008) and effects are discounted at 3 percent. Sensitivity analysis was performed for the perspective, the time horizon, switching, and dose-reduction.

Results: The main analysis conservatively assumes 50 percent switching at discontinuation. The cost per quality-adjusted life-year (QALY) gained with early ETA/MTX treatment is €13,500 (societal perspective, incremental cost of €15,500 and incremental QALYs of 1.15). With 75 percent switching, the cost per QALY gained was €10,400. Over 20 years, the cost per QALY gained was €8,200. Results were further sensitive to the time patients remained on half dose and the perspective.

Conclusions and Policy Implications: This study combines clinical trial and clinical practice data to explore cost-effective treatment scenarios in early RA, including the use of biologics. Our results indicate that a situation where a considerable proportion of patients achieve remission, dose-adjustments will increase the cost-effectiveness of treatment.

Objectives: The aim of this study was to determine adults’ Willingness To Pay (WTP) for CardioVascular Disease (CVD) intervention programs of different intensities.

Methods: Three hundred fourteen participants were randomized to two study conditions: (i) CVD risk assessment/communication; (ii) CVD risk assessment/communication + a behavior change program. The behavior change program was aimed at increasing physical activity, reducing saturated fat intake and smoking cessation. It consisted of a tailored Web site and individual coaching with a self-selected dose. At post-assessment, WTP and perceived autonomy support items were included. The intervention dose was registered throughout the trial and post-hoc intervention dose groups were created. Pearson Chi-Square tests, Student's t-tests, one-way analyses of variance were used to examine WTP-differences between the study conditions and intervention dose groups.

Results: Twenty-four months after baseline, 61 and 135 participants of the control and intervention condition, respectively, completed the questionnaires. No WTP difference was found between the study conditions. However, participants that selected a higher intervention dose were willing to pay significantly more for their program (p < .05).

Conclusions: In general, people want to pay the same amount of money for a CVD prevention program, irrespective of the inclusion of a behavior change program. However, there seems to be an association between the self-selected dose of the latter program and the WTP.

Background: We calculated the daily cost of implantable cardioverter-defibrillators (ICDs) based on their actual longevity to prove whether the up-front cost is a reliable parameter for the ICD purchasing-process.

Methods. Longevity of single chamber (SC), double chamber (DC), and biventricular (BiV) ICDs from Medtronic (MDT), Guidant (GDT), and St. Jude Medical (SJM) was measured in all the patients implanted in years 2000, 2001, 2002 who reached device replacement within December 31, 2009. The cost of each ICD (device + lead/s) was normalized for its own longevity. Data are expressed as median (25th–75th percentile).

Results: A total of 123/153 patients completed the study, 70 percent being alive 8 years after implantation. MDT devices had a superior longevity compared with GDT and SJM (p < .001). Fifty-eight percent of replaced ICDs had a service life at least 1 year shorter than the manufacturers' prediction. Longer-lasting devices had a significantly lower daily cost: €4.8 (4.6–5.7) versus €6.8 (6.2–9.2) and €6.9 (6.2–7.6) for SC (p < .001); €6.9 (6.8–7.7) versus €12.6 (11.8–13.3) and €13.4 (10.3–16.1) for DC; €8.5 (8.3–10.3) versus €15.4 (15.1–15.8) and €14.6 (14.1–14.9) for BiV (p < .005).

Conclusions: The true cost of ICD treatment is strictly dependent on device longevity, whereas device up-front cost is unreliable. This aspect should be valued in the technology purchasing process, and could set the basis for an outcome-based reimbursement system. Our observations may be the benchmark respectively for ICD longevity and daily ICD cost in future comparisons. Independent observations in the real-life scenario are needed to properly value newer technologic improvements.

Background: In unselected patients, the incidence of restenosis is lower after placement of drug-eluting stents (DES) than bare-metal stents (BMS) without difference in safety at a time horizon of 4 years. However, DES appears less effective in “off label” patients.

Objectives: The aim of the study was to assess available evidence of DES efficacy and safety by patient category to establish when DES placement may be recommended for reimbursement by the French national health insurance.

Methods: Based on a systematic review by patient category (January 2002 to August 2009), two health technology assessment (HTA) reports and thirty-eight clinical studies not covered by the HTA reports (eleven meta-analysis including ours, eleven randomized trials and sixteen cohort studies) were selected. After assessment of the methodological quality, the studies mostly comparing DES with BMS were reviewed by a panel of health professionals who defined a priori the most relevant end points of safety and efficacy.

Results: Seven to fourteen patients treated with DES were needed to avoid one target lesion revascularization (TLR) in patients with lesions >15 mm long, vessel diameter <3 mm, or diabetes, and with some complex lesions (total coronary occlusion, BMS in-stent restenosis multivessel disease, unprotected left main stenosis). DES appeared as safe as other alternatives over a follow-up of up to 4 years when dual antiplatelet therapy was continued for at least 1 year, but statistical power remains limited to conclude for some clinical features.

Conclusions: For reimbursement, DES use should be limited to certain categories of patients. Treatment of particular cases requires a multidisciplinary approach.

Objectives: Recent concerns have been raised for the safety after drug-eluting stents (DES) implantation compared with the use of bare-metal stents (BMS) in patients with ST-elevation acute myocardial infarction (STEMI). The objective of this study was to estimate the relative impact of DES versus BMS on mortality, myocardial infarction (MI), target vessel revascularization (TVR), and stent thrombosis (ST) in STEMI patients by performing comprehensive meta-analyses of randomized controlled trials (RCTs) and observational studies.

Methods: We performed an electronic search and manual search of studies presented through September 2009, without language restrictions. An approach of “using systematic reviews” was used. Two independent reviewers extracted prespecified data from each study. A random-effects model was used to combine trials and to perform stratified analyses based on study designs and the duration of follow-up.

Results: Fourteen RCTs were identified (N = 7,654). Compared with BMS, DES significantly reduced TVR (risk ratio [RR], 0.48; 95 percent confidence interval [CI], 0.41–0.56) and MI (RR, 0.77; 95 percent CI, 0.61–0.97), without increasing mortality (RR, 0.88; 95 percent CI, 0.70–1.10) and ST (RR, 0.93; 95 percent CI, 0.72–1.21). Among 35 observational studies (N = 44,849), the use of DES was associated with a significant reduction in mortality (RR, 0.85; 95 percent CI, 0.79–0.91) and TVR (RR, 0.61; 95 percent CI, 0.48–0.77). MI and ST were significantly lower in the DES group within 1-year follow-up, but there were no differences within 2 years of follow-up. There was no evidence of statistical heterogeneity and publication bias.

Conclusions: These data in aggregate suggest that using DES in STEMI patients is safe and efficacious, but there are differences between RCT and observational data comparing DES and BMS.

Objectives: This article examines the challenges for health technology assessment (HTA) in the light of new developments of personalized health care, focusing on European HTA perspectives.

Methods: Using the example of the Integrated Genome Research Network – Mutanom (IG Mutanom) project, with focus on personalized cancer diagnostics and treatment, we assess the scope of current HTA and examine it prospectively in the context of the translation of basic and clinical research into public health genomics and personalized health care.

Results: The approaches developed within the IG-Mutanom project are based on innovative technology potentially providing targeted therapies for cancer; making translation into clinical practice requires a novel course of action, however. New models of HTA are needed that can account for the unique types of evidence inherent to individualized targeted therapies. Using constructive health technology assessment (CTA) models is an option, but further suitable models should be developed.

Conclusions: Integrative, systems biology-based approaches toward personalized medicine call for novel assessment methods. The translation of their highly innovative technologies into the practice of health care requires the development of new HTA concepts.

Objectives: In clinical trials of new cancer drugs, reliable data for progression-free survival will often become available far sooner than reliable data for overall survival. The aim of this study was to determine how many months it would be expected that any given new drug for metastatic breast or colorectal cancer will add to overall survival times given that the number of months the drug adds to progression-free survival times relative to a standard drug is roughly already known.

Methods: A literature search was conducted over Medline for randomized controlled trials (RCTs) published between January 1980 and August 2008 that assessed the effect of a drug treatment in comparison to an alternative drug treatment on patients with either metastatic breast or metastatic colorectal cancer.

Results: The literature search found 95 and 74 RCTs for metastatic breast and colorectal cancer, respectively, that satisfied the study's inclusion criteria. The results from these trials are consistent, in the case of each of these two metastatic cancers, with gains in time to disease progression being generally associated with no gains or with very slight gains or losses in post-progression survival (i.e., the time between disease progression and death).

Conclusions: It would appear that drugs for metastatic breast or colorectal cancer that extend, by a given amount, the time period between the start of treatment and disease progression (i.e., time to progression) have a strong tendency to extend, by roughly the same amount, the period between the start of treatment and death (i.e., overall survival).

Background: Using multiples of India's per capita gross domestic product (GDP) as the threshold for economic value as suggested by the World Health Organization (WHO), decision analysis modeling was used to estimate a more affordable monthly cost in India for a hypothetical new cancer drug that provides a 3-month survival benefit to Indian patients with metastatic colorectal cancer (mCRC).

Methods: A decision model was developed to simulate progression-free and overall survival in mCRC patients receiving chemotherapy with and without the new drug. Costs for chemotherapy and side-effects management were obtained from both public and private hospitals in India. Utility estimates measured as quality-adjusted life-years (QALY) were determined by interviewing twenty-four oncology nurses using the Time Trade-Off technique. The monthly cost of the new drug was then estimated using a target threshold of US$9,300 per QALY gained, which is three times the Indian per capita GDP.

Results: The base-case analysis suggested that a price of US$98.00 per dose would be considered cost-effective from the Indian public healthcare perspective. If the drug were able to improve patient quality of life above the standard of care or survival from 3 to 6 months, the price per dose could increase to US$170 and US$253 and offer the same value.

Conclusions: The use of the WHO criteria for estimating the cost of a new drug based on economic value for a developing country like India is feasible and can be used to estimate a more affordable cost based on societal value thresholds.

Objectives: Recent introduction of a quadrivalent human papillomavirus (HPV) vaccine for girls in New Zealand is expected to decrease the incidence of HPV infection as well as resultant cytological abnormalities and cervical cancer. This may affect the cost-effectiveness of the national cervical screening program by reducing the incidence of lesions detected. This study investigates the cost-effectiveness of the current cervical screening policy with and without the HPV vaccine and compares these results with the cost-effectiveness of a range of other screening strategies.

Methods: A Markov state-transition model was built based on the natural history of HPV and cervical carcinogenesis. The model followed a hypothetical cohort of girls from 12 years to 85 years of age or death, through screening and treatment pathways. The model compared a “no vaccine and current screening” strategy with a selection of screening strategies with different age ranges and frequency intervals.

Results: The most cost-effective cervical screening strategy in the presence of the HPV vaccine would be screening women aged 30–60 every 5 years. Moving to this screening strategy from the base case of no vaccine and the current New Zealand strategy of screening women aged 20–69 every 3 years is predicted to have an incremental cost per quality-adjusted life-year gained of NZ$9,841 (€4,428).

Conclusions: Reducing screening intensity from 3 to 5 years as well as narrowing the screening age range for the vaccinated cohort once they reach mid-twenties is recommended. The importance of achieving a high vaccine uptake in New Zealand remains high.

Objectives: Immediate post-mastectomy breast reconstruction (IBR) is a procedure that has proven advantages, but it also entails risks. The aim of this study was to identify risk factors for reconstruction failure.

Methods: A review was made of all the IBR carried out at a general hospital from 2002 to 2009. Retrospective information was obtained about postoperative complications and the characteristics of patients and treatments applied. The minimum follow-up period was 9 months. Cox's regression analysis was performed on the variables related to the reconstruction failure requiring the removal of the prosthesis, with an explanatory model in which all the study variables were introduced and a predictive model that contained only the variables known before the intervention.

Results: A total of 115 IRB interventions carried out on 112 women with breast cancer were analyzed. The mean follow-up period was 25.5 months. In sixty cases (52.2 percent), there were no complications; in sixteen cases (13.9 percent) minor complications appeared, and in 39 (33.9 percent) the complications were moderate. In twenty-six cases (22.6 percent), a reconstruction failure occurred. Cox's regression model revealed that the reconstruction failures were related to the patient's age (Hazard Ratio 1.08), to neoadjuvant chemotherapy (HR 6.24) and to postoperative tamoxifen (HR 3.10). The predictive model included the age of the patient (HR 1.05) and the use of neoadjuvant chemotherapy (HR 5.11).

Conclusions: A significant proportion of the patients receiving IBR developed reconstruction failure. Multivariate analysis identified three variables related to this complication, two of which were known before the intervention.

Objectives: Health systems frequently make decisions regarding acquisition and use of new technologies. It is desirable to base these decisions on clinical evidence, but often these technologies are used for multiple indications and evidence of effectiveness for one indication does not prove effectiveness for all. Here, we describe two examples of evidence inventory reports that were performed for the purposes of identifying how much and what type of published clinical evidence was available for a given technology, and the contexts in which those technologies were studied.

Methods: The evidence inventory reports included literature searches for systematic reviews and health technology assessment (HTA) reports, and systematic searches of the primary literature intended to count and categorize published clinical studies. The reports did not include analysis of the primary literature.

Results: The inventory reports were completed in 3 to 4 days each and were approximately ten pages in length, including references. Reports included tables listing the number of reported studies by specific indication for use, and whether or not there were randomized trials. Reports also summarized findings of existing systematic reviews and HTA reports, when available. Committees used the inventory reports to decide for which indications they wanted a full HTA report.

Conclusions: Evidence inventory reports are a form of rapid HTA that can give decision makers a timely understanding of the available evidence upon which they can base a decision. They can help HTA providers focus subsequent reports on topics that will have the most influence on healthcare decision making.

Objectives: The aim of this study was to review international experiences of patient or public involvement in the field of health technology assessment (HTA).

Methods: A systematic review of the scientific literature was conducted. A literature search was performed across nine databases. Other literature was identified through citation tracking, government websites (HTA agencies), and Internet search engines. Characteristics of the studies, description of the activities related to patient or public involvement, impact of these activities on the HTA process, and factors facilitating or limiting involvement were abstracted independently by two reviewers.

Results: A total of 1,441 potentially relevant papers were identified by the main search strategy. Among these, seventeen papers met the inclusion criteria; other search strategies identified seven additional documents. The findings reveal that patient or public involvement in HTA activities was reported in two domains, research and HTA process. In the research domain, patients are consulted to gather evidence about their perspectives, experiences, or preferences about a health technology. These perspectives could add key dimensions to the evaluation of health technologies that might otherwise be overlooked. In the domain of the HTA process, patients or public representatives participate in different stages of this process: prioritization, evidence assessment, or dissemination of findings.

Conclusions: There are few published examples of experiences involving patients and the public in HTA. These examples show that patients' or the public's perspectives could add important dimensions to the evaluation of health technologies. However, there is a need to develop more systematic approaches to considering patients' and the public's perspectives in HTA.

Objectives: Extended-release therapies avoid the need for children with attention-deficit/hyperactivity disorder (ADHD) to take medication at school. Recently a transdermal delivery system has been developed which can allow symptom control all day long but with greater dosing flexibility. This study explored the parents' preferences regarding oral and transdermal therapy.

Methods: A nonsystematic and qualitative literature review and in-depth interviews with parents and physicians helped identify salient treatment attributes for a discrete choice experiment. Treatment attributes included mode of administration (tablet or transdermal), speed of onset (30–90 min); duration (lasts until 3–9 pm) and ability to tailor the drug to different needs (no flexibility, limited flexibility, easy to adjust to different days). A convenience sample of parents of children treated for ADHD (n = 200) were recruited using a recruitment agency. Data were analyzed using generalized estimating equations (GEE).

Results: Parents' preferred once-a-day oral therapy (odds ratio [OR] = 1.76 [95 percent confidence interval {CI}, 1.43 – 2.18]); rapid speed of onset (OR = 1.22 [95 percent CI, 1.07 – 1.39]), and symptom control until 9 pm (OR = 3.79 [95 percent CI, 2.98 – 4.82]). Analyses identified that 30 percent of parents preferred transdermal treatment and this subgroup preferred treatments with a fast onset of action.

Conclusions: This survey demonstrates that parents of ADHD children have different preferences for the ADHD treatments prescribed for their children. A distinct subgroup of parents prefer the transdermal therapy. These parents were less likely to be working and so monitoring compliance and doing after school activities may have been easier.