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Pragmatic or practice-oriented comparative effectiveness trials may be conducted to fill the evidence gaps that are revealed after the private sector has performed the trials needed for bringing their product to the market. A tool of increasing importance to identify such evidence gaps is resulting from health technology assessments (HTA) whereby the data derived from clinical research are examined in a systematic manner with reference to effect, safety, as well as additional parameters. Practice-oriented trials are informative for healthcare decision makers, practice-changing and may even be cost-saving for the healthcare payers. There are however only a limited number of funding sources for such trials. Public and private healthcare payers should stimulate the conduct of practice-oriented trials in their effort to maximize patient benefit within the limitation of the available resources. Pragmatic randomized trials can be performed at low cost when based on existing coded electronic health records and as well health registries. Public health decision makers are increasingly taking advantage of results from health technology assessments to support priority setting. In accordance with this it would appear reasonable that decision makers should get more involved in priority setting and funding also in the field of clinical research in order to provide further evidence needed for assessments, reassessments, and subsequent qualified decisions and resource allocations in health care. A closer dialogue and collaboration between the clinical research and HTA communities would facilitate a more efficient utilization of such opportunities. [ABSTRACT FROM AUTHOR]

Objectives: We studied the cost-effectiveness of tenofovir and entecavir in e antigen positive (CHBe+) and negative (CHBe-) chronic hepatitis B.Methods: Using a multicenter survey including 544 patients we measured patient quality of life and attributable costs by clinical disease stage. Natural disease progression was studied in 278 patients in a single center. A Markov model was constructed to follow hypothetical cohorts of treated and untreated 40-year-old CHBe+ and CHBe- patients and 50-year-old patients with compensated cirrhosis.Results: We did not find an improvement in quality of life when viral load was reduced under treatment. Transition rates to liver cirrhosis were found to be age-dependent. Assuming equal effectiveness, tenofovir dominates the entecavir strategy because of its lower price in Belgium. The incremental cost-effectiveness ratio (ICER) of tenofovir after 20 years is more favorable for treating Caucasian cirrhotic patients (mean ICER €29,000/quality-adjusted life-year [QALY]) compared with treating non-cirrhotic patients (mean ICER €110,000 and 131,000/QALY for CHB e+ and e-, respectively). Within the non-cirrhotic patients the ICER decreases with increasing cohort starting age from 30 to 50 years.Conclusions: Results of long-term models for tenofovir or entecavir treatment of CHB need to be interpreted with caution as long-term trials with hard end points are lacking. Especially the effect on HCC remains highly uncertain. Based on cost-effectiveness considerations such antiviral treatment should be targeted at patients with cirrhosis or at risk of rapid progression to this disease stage. [ABSTRACT FROM AUTHOR]

Objectives: High-quality clinical evidence is most often lacking when novel high-risk devices enter the European market. At the same time, a randomized controlled trial (RCT) is often initiated as a requirement for obtaining market access in the US. Should coverage in Europe be postponed until RCT data are available? We studied the premarket clinical evaluation of innovative high-risk medical devices in Europe compared with the US, and with medicines, where appropriate.Methods: The literature and regulatory documents were checked. Representatives from industry, Competent Authorities, Notified Bodies, Ethics Committees, and HTA agencies were consulted. We also discuss patient safety and the transparency of information.Results: In contrast to the US, there is no requirement in Europe to demonstrate the clinical efficacy of high-risk devices in the premarket phase. Patients in Europe can thus have earlier access to a potentially lifesaving device, but at the risk of insufficiently documented efficacy and safety. Variations in the stringency of clinical reviews, both at the level of Notified Bodies and Competent Authorities, do not guarantee patient safety. We tried to document the design of premarket trials in Europe and number of patients exposed, but failed as this information is not made public. Furthermore, the Helsinki Declaration is not followed with respect to the registration and publication of premarket trials.Conclusions: For innovative high-risk devices, new EU legislation should require the premarket demonstration of clinical efficacy and safety, using an RCT if possible, and a transparent clinical review, preferably centralized. [ABSTRACT FROM PUBLISHER]

Objectives: The cost-effectiveness of adding a human papillomavirus (HPV) vaccination program in 12-year-old females to the recommended cervical cancer screening in Belgium is examined. Moreover, the health and economic consequences of a potential decline in screening uptake after initiation of a HPV vaccination program are investigated. Methods: A static Markov model is developed to estimate the direct effect of vaccination on precancerous lesions and cervical cancers. Results: Vaccination is estimated to avoid 20 percent of the cervical cancers occurring in a 12-year-old girls' cohort and to cost €32,665 per quality-adjusted life-year (QALY) gained (95 percent credibility interval [CrI]: €17,447 to €68,078), assuming a booster injection after 10 years, a limited duration of protection and discounting costs and effects at 3 percent and 1.5 percent, respectively. Assuming lifelong protection, HPV vaccination is estimated to cost €14,382 (95 percent CrI: €9,238 to €25,644) per QALY gained, while avoiding 50 percent of the cervical cancer cases. In the base-case, a 10 percent reduction in screening compliance after vaccination obliterates the effect of vaccination on cervical cancer cases avoided, whereas further declines in the level of screening compliance even turned out to be detrimental for the cohort's health, inducing a mean loss in QALYs and life-year gained compared with the situation prevaccination. Conclusions: An HPV vaccination program should only be considered if the level of screening after vaccination can be maintained. [ABSTRACT FROM AUTHOR]