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Summary: Background: There are no data to assess the value associated with a treat‐to‐target (T2T) strategy based on tight control of mild–moderate ulcerative colitis (UC). Aim: To assess the cost‐effectiveness of a T2T approach based on the normalisation of clinical signs and faecal calprotectin (FC) Methods: A decision analytical Markov model was developed to compare T2T algorithm combining clinical symptoms and FC levels to define treatment response and the possible switch to the next treatment line (T2T‐FC), and the reference strategy based only on symptoms. The model included five treatment lines and was conducted from the Italian national health service (NHS) perspective using a 3‐year time horizon. The model calculated the incremental cost‐effectiveness ratio as € per relapse avoided. Deterministic and probabilistic sensitivity analyses were conducted. Results: The cost‐effectiveness analysis produced an increased time spent by a patient in clinical remission and FC ≤ 100 level (+0.177 years; about 2 months) and a decreasing number of relapses (−0.1937; −20.9%) per patient using a T2T‐FC approach compared to only symptoms. Furthermore, the T2T‐FC was associated with higher cost (+€1795). The ICER estimated was €9263 per relapse avoided. These results were confirmed by sensitivity analyses. Conclusions: T2T‐FC approach resulted in a higher benefit for mild–moderate UC patients in terms of time in remission and incidence of relapse but was associated with higher costs. Clinical trials and real‐world clinical studies are needed to provide additional data on the cost–benefit of this approach. [ABSTRACT FROM AUTHOR]

Both the human capital approach and the friction cost approach are frequently used to quantify the productivity costs associated with illness, disability or death in health economic evaluations. In this paper we argue that these approaches have one major, but common shortcoming: they only capture partial equilibrium (PE) effects and therefore underestimate the true potential productivity costs associated with health conditions. They neglect the sizable, indirect, ripple effects in the economy captured by general equilibrium (GE) models. To demonstrate our point, we compare a traditional PE with a GE approach for the application to nocturia, a condition characterized by the need to frequently wake up at night to urinate. Nocturia is associated with substantial impairment of daytime functioning and work productivity. We employ large‐scale United Kingdom (UK) employer‐employee survey data to estimate the prevalence and productivity loss. These estimates are then used as shared inputs to drive both approaches. We find that the traditional PE approach underestimates the annual productivity cost of clinically relevant nocturia by around 16%. We propose a generalized GE/PE multiplier to approximate the GE effect for other health conditions. Our findings stress the importance of accounting for sizable GE effects when conducting health economic evaluations. [ABSTRACT FROM AUTHOR]

Objectives: To investigate the long‐term efficacy, quality of life (QoL), and safety of desmopressin orally disintegrating tablets (ODTs) in Japanese patients with nocturia. Methods: A long‐term, multicenter phase 3 study was conducted that enrolled Japanese male and female patients with nocturia (NCT03051009). Male patients received desmopressin 25‐ or 50‐μg ODTs, and female patients received desmopressin 25‐μg ODTs for up to 1 year. The primary endpoint was safety. Secondary endpoints included change from baseline in number of nocturnal voids, time to first awakening to void, and QoL assessments (nocturia‐specific zQoL [N‐QoL], Insomnia Severity Index [ISI], and Hsu bother score). Results: Overall, 503 patients were enrolled. Reductions from baseline in mean number of nocturnal voids were observed in all treatment groups from week 1 (−0.62 to −1.00), with improvements continuing through week 52 (−1.39 to −1.71). Changes from baseline above or approximating a clinically meaningful improvement were seen by week 52 in the disease‐specific N‐QoL total score (improved by 11.5‐22.6), ISI (improved by −3.9 to −7.1), and Hsu bother scores (improved by −1.5 to −2.0). Adverse events (AEs) were reported in 54.9% of desmopressin‐treated patients. Most AEs were mild or moderate in severity. Conclusions: Desmopressin ODTs (25 and 50 μg) demonstrated long‐term efficacy, improved QoL, and were well tolerated in Japanese male and female patients with nocturia treated for up to 1 year. Clinically meaningful improvements in patients' QoL, assessed by N‐QoL, sleep quality, and bother, occur later than objective symptom improvements, such as voids. [ABSTRACT FROM AUTHOR]