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Purpose: During the past decade, intravitreally administered biologic drugs have advanced the treatment of retinal diseases, such as wet age‐related macular degeneration (AMD), diabetic macular oedema and retinal venous occlusions. The drugs as well as the necessary disease management imply considerable economic burden on healthcare systems. This Norwegian study documents the rates of use of intravitreal therapies and intercounty variation over a 5‐year period. Methods: We collected data from the Norwegian Patient Register for all episodes of care encompassing intravitreal therapy during the period 2011–2015. For each episode, we received information on patient age, sex, county of residence, diagnosis and name of drug injected. Results: During the study period, 21 277 patients had in total 236 857 episodes of care. The number of intravitreal injections doubled from 2011 to 2015, reaching 63 601 injections in 2015, of which 77% were for diagnosed wet AMD. In 2015, the age‐adjusted number of episodes varied from 19 to 55 per 1000 population aged 50+ across Norway's 19 counties. The age‐adjusted number of patients treated per 1000 population aged 50+ varied from 5.22 to 8.35. Conclusion: The use of intravitreal injections increased rapidly with wet AMD as the most frequent diagnosis and with varying utilization across Norway's 19 counties. The causes of the varying use of intravitreal therapies could not be established but may reflect variation in disease prevalence, treatment capacity, travel distance to the nearest ophthalmic service and lack of national treatment guidelines. The geographic variation in utilization may challenge policy goals of equitable care and warrants further studies. [ABSTRACT FROM AUTHOR]

A better understanding of the risk concept in relation to treatment of chronic disease processes is warranted. Consequences of therapies may be described in terms of risk reductions or postponement of adverse outcomes. This review argues that for most interventions, neither of these measures adequately characterize the distribution of treatment benefit across patients. More focus should be placed on choosing the context-relevant outcome measure and on developing techniques for extracting and communicating 2D outcome measures where the distribution of outcomes across patients is explicitly reflected. Since the distribution of outcomes affects patients' valuation of interventions, more research into risk communication is likely to lead to improved decision making. [ABSTRACT FROM AUTHOR]

Background The number needed to treat (NNT) has been promoted as the preferred effect measure when patients and physicians share decision making. Our aim was to explore the impact of the NNT on laypeople’s decisions about preventive drug therapies. Methods Two thousand subjects were selected for the survey; 1201 (60%) responded for a representative sample of the Norwegian population. Respondents were allocated to scenarios with random combinations of a disease to be prevented, drug treatment costs, and effect size in terms of NNT. They were interviewed about their hypothetical consent to the therapy, then randomized to different interpretations of NNT and asked to reconsider their initial responses. Results The proportions consenting varied from 76% when the NNT was 50 to 67% when the NNT was 1600 (P for trend = .06). When faced with the prospect of avoiding lethal disease, stroke, myocardial infarction, or hip fracture, the proportions consenting were 84%, 76%, 68%, and 53%, respectively (P<.01). Across different treatment costs ($37, $68, $162, and $589) the proportions consenting varied from 78% to 61% (P for trend <.01). Twenty-four percent of the respondents changed their decision when informed about how to interpret the NNT, and 93% of those switched from positive to negative decisions, regardless of the magnitude of NNT. Conclusions Respondents’ decisions were influenced by the type of disease to be prevented and the cost of the intervention, but not by the effect size in terms of NNT. This suggests that NNT is difficult to understand and that other effect formats should be considered for shared decision making. [ABSTRACT FROM AUTHOR]

The "number needed to treat" is assumed to be readily understood, but empirical evidence to support this assumption is sparse. 72% of medical doctors recommended a preventive drug therapy when NNT was 50 compared to 52% when NNT was 200. 77% of doctors recommending against a preventive drug therapy thought that only one out of NNT patients benefits from therapy. Since this assumption may be misleading, we suggest that the NNT should be used with caution in clinical practice. Objective - While the number needed to treat (NNT) is in widespread use, empirical evidence that doctors or patients interpret the NNT adequately is sparse. The aim of our study was to explore the influence of the NNT on medical doctors' recommendation for or against a life-long preventive drug therapy. Design - Cross-sectional study with randomisation to different scenarios. Setting - Postal questionnaire presenting a clinical scenario about a hypothetical drug as a strategy towards preventing premature death among healthy people with a known risk factor. Benefit after 5 years of treatment was presented in terms of NNT, which was set at 50 for half of the respondents and 200 for the other half. Subjects - Representative sample (n=1616) of Norwegian medical doctors. Main outcome measures - Proportion of doctors that would prescribe the drug. Reasons for recommending against the therapy. Results - With NNT set at 50, 71.6% (99% CI 66.8-76.4) of the doctors would prescribe the drug, while the proportion was 52.3% (99% CI 47.5-57.1) with an NNT of 200 (χ 2 =50.7, p<0.001). Multivariate logistic regression analysis indicated that the effect of NNT on the likelihood for recommending the therapy was age-dependent; young doctors (<36 of age) were more sensitive to the difference in NNTs than older doctors. Thirty-six percent (n=464) of the doctors would not prescribe the drug, and 77.4% (99% CI 68.5-86.2) of those agreed with an argument stating that only one out of NNT patients would benefit from the treatment. Conclusion - Medical doctors appear to be sensitive to the magnitude of the NNT in their clinical recommendations. However, many doctors believe that only one out of NNT patients benefits from therapy. Clinical recommendations based on this assumption may be misleading. [ABSTRACT FROM AUTHOR]

The article discusses a study of the sensitivity of physicians to the magnitude of number needed to treat (NNT) and gain in disease-free survival when considering a lipid-lowering drug therapy. Forty-seven percent of physicians recommend neostatin after being presented with gain in disease-free survival information. Results also showed the insensitivity of lay people to effect size in terms of NNT. The strength of the study is its randomised design.

The article focuses on a survey which examined the views of the society on the treatment of rare diseases in older people in Norway. Survey respondents are Norwegians who are aged 40 to 67, who were asked to chose between funding rare disease treatment or funding treatment for a common disease. It discovered that there is no societal preference for rare diseases.

The article examines ways to describe the benefits of risk-reducing treatments. It aims to determine a laypersons responses to scenarios that describe benefits as postponing an adverse event or the equivalent the number needed to treat (NNT) with the use of cross-sectional survey with random allocation to different scenarios.

Background: There is ample evidence for several pharmaceutical treatments that adherence in terms of treatment duration and dose is suboptimal. The actual drug intake cannot be observed directly in prescription databases, which only register drug redeemed and a limited number of patient characteristics. Consequently, the actual dose and duration of treatment must be inferred from observed redemptions. Persistence can then be expressed as treatment duration (also referred to as drug survival). Method: We used data from the Norwegian Prescription Database (NorPD) on redemptions of statins (ATCcode C10AA) for the period 2010-2019 to explore three methods for determining prescription durations and in turn persistence (treatment duration): (i) The DDD-method using the number of DDD redeemed; (ii) The dose-unit approach using the number of tablets redeemed; (iii) The reverse waiting time distribution method (WTD), which estimates prescription duration as the 90th percentile of the distribution within which patients in ongoing treatment will have a new subsequent redemption. The three methods for estimating prescription duration were then used to estimate treatment duration using Kaplan Meier (KM) survival functions. For the DDD-method and the dose-unit approach we conducted sensitivity analyses assuming that one DDD or one tablet would last for 1.00, 1.25 or 2.00 days. We also tested the impact of grace periods in sensitivity analyses. Results: Treatment duration and drug survival varied substantially for the same patients depending on the chosen method, duration of a DDD or a tablet, and inclusion of grace periods. The 25th percentile of treatment duration was 100 days for the DDD approach with one DDD per day, 100 days with the dose-unit approach with one tablet per day and 453 days with the WTD approach. Conclusion: When estimating treatment duration from prescription databases one should be aware that these measures of persistence are highly influenced by the chosen methodology. The choice of method should be informed by the clinical context with a preference for use of methods based on a formal model. [ABSTRACT FROM AUTHOR]

Background: Information on the benefits of therapeutic interventions can ve expressed in various ways, including relative risk reduction, absolute risk reduction, and number needed to treat (NNT). An alternative to such risk-based measures is postponement of an adverse outcome (eg, hip fracture in the case of osteoporosis).Objective: The goal of this study was to examine whether laypersons'' perception of the benefit of an osteoporosis therapy differs when it is presented in terms of the NNT to avoid 1 hip fracture compared with the duration of postponement of hip fracture.Methods: This was a cross-sectional, randomized, controlled trial. Face-to-face interviews of a representative sample of the Danish population were conducted in respondents'' homes. Respondents were randomized to receive information about the benefits of a hypothetical osteoporosis intervention either in terms of different magnitudes of NNT (10, 50, 100, or 400) or different durations of postponement of hip fracture (1 month, 6 months, 1 year, or 4 years). Participants were subsequently asked if they would consent to the intervention.Results: A total of 1728 individuals were contacted at home and asked if they would take part in a face-to-face interview; 967 (56%) were successfully interviewed. The age (mean age, 44.5 years; range, 20–74 years) and sex distrivution (51% male, 50% female) of the sample was similar to that of the general Danish population. Based on NNTs of 10, 50, 100, and 400, the proportions of responsiveness [Copyright &y& Elsevier]