Prolapse affects 30-40% of women. Those using a pessary for prolapse usually receive care as an outpatient. This trial determined effectiveness and cost-effectiveness of pessary self-management (SM) vs clinic-based care (CBC) in relation to condition-specific quality of life (QoL).Parallel-group, superiority randomised controlled trial, recruiting from 16 May 2018 to 7 February 2020, with follow-up to 17 September 2021. Women attending pessary clinics, ≥18 years, using a pessary (except Shelf, Gellhorn or Cube), with pessary retained ≥2 weeks were eligible. Limited manual dexterity; cognitive deficit; pregnancy; or requirement for non-English teaching were exclusions. SM group received a 30-min teaching session; information leaflet; 2-week follow-up call; and telephone support. CBC group received usual routine appointments. The primary clinical outcome was pelvic floor-specific QoL (PFIQ-7), and incremental net monetary benefit for cost-effectiveness, 18 months post-randomisation. Group allocation was by remote web-based application, minimised on age, user type (new/existing) and centre. Participants, intervention deliverers, researchers and the statistician were not blinded. The primary analysis was intention-to-treat based. Trial registration: https://doi.org/10.1186/ISRCTN62510577.The requisite 340 women were randomised (169 SM, 171 CBC) across 21 centres. There was not a statistically significant difference between groups in PFIQ-7 at 18 months (mean SM 32.3 vs CBC 32.5, adjusted mean difference SM-CBC -0.03, 95% CI -9.32 to 9.25). SM was less costly than CBC. The incremental net benefit of SM was £564 (SE £581, 95% CI -£576 to £1704). A lower percentage of pessary complications was reported in the SM group (mean SM 16.7% vs CBC 22.0%, adjusted mean difference -3.83%, 95% CI -6.86% to -0.81%). There was no meaningful difference in general self-efficacy. Self-managing women were more confident in self-management activities. There were no reported suspected unexpected serious adverse reactions, and 31 unrelated serious adverse events (17 SM, 14 CBC).Pessary self-management is cost-effective, does not improve or worsen QoL compared to CBC, and has a lower complication rate.National Institute for Health and Care Research, Health Technology Assessment Programme (16/82/01).
Background Pelvic organ prolapse is estimated to affect 41–50% of women aged > 40 years. A multicentre randomised controlled trial of individualised pelvic floor muscle training found that pelvic floor muscle training was effective in reducing symptoms of prolapse, improved quality of life and showed clear potential to be cost-effective. Provision of pelvic floor muscle training for prolapse has continued to vary across the UK, with limited availability of specialist physiotherapists to deliver it. Objectives This project aimed to study the implementation and outcomes of different models of delivery to increase the service provision of pelvic floor muscle training, and to follow up treatment outcomes for the original trial participants. Design A realist evaluation of pelvic floor muscle training implementation conducted within three full case study sites and two partial case study sites; an observational prospective cohort study comparing patient-reported outcomes pre and post intervention in all five sites; and a long-term follow-up study linking previous trial participants to routine NHS hospital data. Setting The setting for the realist evaluation was pelvic floor muscle training service delivery models in three NHS sites. The setting for the patient-reported outcome measures study was pelvic floor muscle training services in five NHS sites. Methods Realist evaluation qualitative data were collected at four time points in three case study sites to understand the implementation models, uptake, adherence and impact. Interviews involved service managers/leads, consultants, staff delivering pelvic floor muscle training and women receiving pelvic floor muscle training. Main outcomes measures Patient-reported outcomes were collected at baseline and at 6 and 12 months across five sites, including the Pelvic Organ Prolapse Symptom Score, health-related quality of life (measured using the EuroQol-5 Dimensions, five-level version, questionnaire), prolapse severity (measured using the Pelvic Organ Prolapse Quantification System), urinary incontinence (measured using International Consultation on Incontinence Questionnaire – Urinary Incontinence Short Form) and need for further treatment. Results A total of 102 women were recruited to the patient-reported outcome measures cohort study (target, n = 120), and 65 women had matched baseline and 6-month Pelvic Organ Prolapse Symptom Scores. The mean Pelvic Organ Prolapse Symptom Score was 10.18 (standard deviation 5.63) at baseline and 6.98 (standard deviation 5.23) at 6 months, representing a statistically significant and clinically meaningful difference. There was no statistically significant difference between the outcomes obtained from delivery by specialist physiotherapists and the outcomes obtained from delivery by other health-care professionals (mean change in Pelvic Organ Prolapse Symptom Score: –3.95 vs. –2.81, respectively). Services delivered using higher-band physiotherapists only were more costly than services delivered using other staff mixes. The effect of the original pelvic floor muscle training intervention, over a post-intervention period of > 10 years, was a reduction in the odds of any treatment during follow-up (odds ratio 0.61, 95% confidence interval 0.37 to 0.99). The realist evaluation revealed stark differences in implementation. The site with a specialist physiotherapy service resisted change because of perceived threats to the specialist role and concerns about care quality. Pelvic floor muscle training delivery by other health-care staff was easier when there was a lack of any existing specialist service; staff had prior training and interest in pelvic health; staff had support, autonomy, time and resources to deliver pelvic floor muscle training as part of their core role; and surrounding services supported a flow of pelvic floor muscle training referrals. Limitations The number of available matched pre and post outcomes for women and the lack of Pelvic Organ Prolapse Quantification System examinations were limitations of this study. Conclusions It is possible to train different staff to effectively deliver pelvic floor muscle training to women. Women’s self-reported outcomes significantly improved across all service models. Training should be adequately tailored to differential skill mix needs. Future work Future work should include further implementation of pelvic floor muscle training and should include pre- and post-outcome data collection using the Pelvic Organ Prolapse Symptom Score. Study registration This study is registered as Research Registry 4919. Funding This project was funded by the National Institute for Health Research (NIHR) Health Services and Delivery Research programme and will be published in full in Health Services and Delivery Research ; Vol. 8, No. 47. See the NIHR Journals Library website for further project information.
Background Age-related macular degeneration is the most common cause of sight impairment in the UK. In neovascular age-related macular degeneration (nAMD), vision worsens rapidly (over weeks) due to abnormal blood vessels developing that leak fluid and blood at the macula. Objectives To determine the optimal role of optical coherence tomography (OCT) in diagnosing people newly presenting with suspected nAMD and monitoring those previously diagnosed with the disease. Data sources Databases searched: MEDLINE (1946 to March 2013), MEDLINE In-Process & Other Non-Indexed Citations (March 2013), EMBASE (1988 to March 2013), Biosciences Information Service (1995 to March 2013), Science Citation Index (1995 to March 2013), The Cochrane Library (Issue 2 2013), Database of Abstracts of Reviews of Effects (inception to March 2013), Medion (inception to March 2013), Health Technology Assessment database (inception to March 2013). Review methods Types of studies: direct/indirect studies reporting diagnostic outcomes. Index test: time domain optical coherence tomography (TD-OCT) or spectral domain optical coherence tomography (SD-OCT). Comparators: clinical evaluation, visual acuity, Amsler grid, colour fundus photographs, infrared reflectance, red-free images/blue reflectance, fundus autofluorescence imaging, indocyanine green angiography, preferential hyperacuity perimetry, microperimetry. Reference standard: fundus fluorescein angiography (FFA). Risk of bias was assessed using quality assessment of diagnostic accuracy studies, version 2. Meta-analysis models were fitted using hierarchical summary receiver operating characteristic curves. A Markov model was developed (65-year-old cohort, nAMD prevalence 70%), with nine strategies for diagnosis and/or monitoring, and cost–utility analysis conducted. NHS and Personal Social Services perspective was adopted. Costs (2011/12 prices) and quality-adjusted life-years (QALYs) were discounted (3.5%). Deterministic and probabilistic sensitivity analyses were performed. Results In pooled estimates of diagnostic studies (all TD-OCT), sensitivity and specificity [95% confidence interval (CI)] was 88% (46% to 98%) and 78% (64% to 88%) respectively. For monitoring, the pooled sensitivity and specificity (95% CI) was 85% (72% to 93%) and 48% (30% to 67%) respectively. The FFA for diagnosis and nurse-technician-led monitoring strategy had the lowest cost (£39,769; QALYs 10.473) and dominated all others except FFA for diagnosis and ophthalmologist-led monitoring (£44,649; QALYs 10.575; incremental cost-effectiveness ratio £47,768). The least costly strategy had a 46.4% probability of being cost-effective at £30,000 willingness-to-pay threshold. Limitations Very few studies provided sufficient information for inclusion in meta-analyses. Only a few studies reported other tests; for some tests no studies were identified. The modelling was hampered by a lack of data on the diagnostic accuracy of strategies involving several tests. Conclusions Based on a small body of evidence of variable quality, OCT had high sensitivity and moderate specificity for diagnosis, and relatively high sensitivity but low specificity for monitoring. Strategies involving OCT alone for diagnosis and/or monitoring were unlikely to be cost-effective. Further research is required on (i) the performance of SD-OCT compared with FFA, especially for monitoring but also for diagnosis; (ii) the performance of strategies involving combinations/sequences of tests, for diagnosis and monitoring; (iii) the likelihood of active and inactive nAMD becoming inactive or active respectively; and (iv) assessment of treatment-associated utility weights (e.g. decrements), through a preference-based study. Study registration This study is registered as PROSPERO CRD42012001930. Funding The National Institute for Health Research Health Technology Assessment programme.
Background An expertise-based randomised trial design, where participating health professionals only provide the intervention in which they have expertise, has been proposed to overcome challenges faced when evaluating skill based interventions. Health professionals (e.g. surgeons/therapists) often have differing levels of expertise in the interventions, conduct only one routinely and/or have strong preferences. However understanding of this design is limited.
Abstract Background The treatment of patients with varicose veins constitutes a considerable workload and financial burden to the National Health Service. This study aimed to assess the cost-effectiveness of ultrasound-guided foam sclerotherapy (UGFS) and endovenous laser ablation (EVLA) compared with conventional surgery as treatment for primary varicose veins. Methods Participant cost and utility data were collected alongside the UK CLASS multicentre randomized clinical trial, which compared EVLA, surgery and UGFS. Regression methods were used to estimate the effects of the alternative treatments on costs to the health service and quality-adjusted life-years (QALYs) at 6 months. A Markov model, incorporating available evidence on clinical recurrence rates, was developed to extrapolate the trial data over a 5-year time horizon. Results Compared with surgery at 6 months, UGFS and EVLA reduced mean costs to the health service by £655 and £160 respectively. When additional overhead costs associated with theatre use were included, these cost savings increased to £902 and £392 respectively. UGFS produced 0·005 fewer QALYs, whereas EVLA produced 0·011 additional QALYs. Extrapolating to 5 years, EVLA was associated with increased costs and QALYs compared with UGFS (costing £3640 per QALY gained), and generated a cost saving (£206–439) and QALY gain (0·078) compared with surgery. Applying a ceiling willingness-to-pay ratio of £20 000 per QALY gained, EVLA had the highest probability (78·7 per cent) of being cost-effective. Conclusion The results suggest, for patients considered eligible for all three treatment options, that EVLA has the highest probability of being cost-effective at accepted thresholds of willingness to pay per QALY.
Denosumab offers an alternative, or additional, treatment for the prevention of skeletal-related events (SREs) in patients with bone metastases from solid tumours.The aim of this review was to assess the clinical effectiveness and cost-effectiveness of denosumab, within its licensed indication, for the prevention of SREs in patients with bone metastases from solid tumours.Databases searched were MEDLINE (1948 to April 2011), EMBASE (1980 to March 2011), The Cochrane Library (all sections; Issue 1, 2011) and Web of Science with Conference Proceedings (1970 to May 2011).Only randomised controlled trials (RCTs) assessing denosumab, bisphosphonates (BPs) or best supportive care (BSC) in patients with bone metastases were included. Systematic reviews and observational studies were used for safety and quality-of-life assessments. Study quality was assessed using the Cochrane risk of bias tool. Studies suitable for meta-analysis were synthesised using network meta-analysis (NMA). A systematic review was conducted for cost, quality-of-life and cost-effectiveness studies. The results of this informed the cost-utility modelling. This principally estimated the cost-effectiveness of denosumab relative to zoledronic acid for when BPs are currently recommended and relative to BSC when BPs are not recommended or are contraindicated.A literature search identified 39 studies (eight suitable for NMA). Denosumab was effective in delaying time to first SRE and reducing the risk of multiple SREs compared with zoledronic acid. Generally speaking, denosumab was similar to zoledronic acid for quality of life, pain, overall survival and safety. The NMA demonstrated that denosumab was more effective in delaying SREs than placebo, but was limited by numerous uncertainties. Cost-utility modelling results for denosumab relative to zoledronic acid were driven by the availability of the patient access scheme (PAS) for denosumab. Without this, denosumab was not estimated to be cost-effective compared with zoledronic acid. With it, the cost-effectiveness ranged between dominance for breast and prostate cancer, to between £5400 and £15,300 per quality-adjusted life-year (QALY) for other solid tumours (OSTs) including non-small cell lung cancer (NSCLC) and £12,700 per QALY for NSCLC. Owing to small patient gains estimated, the cost-effectiveness of denosumab was very sensitive to the zoledronic acid price. Denosumab was not estimated to be cost-effective compared with BSC.Only subgroup data were available for denosumab for NSCLC, and OSTs excluding NSCLC. The NMA was subject to numerous uncertainties. Owing to small patient gains estimated, the cost-effectiveness of denosumab was very sensitive to the zoledronic acid price.Denosumab, compared with zoledronic acid and placebo, is effective in delaying SREs, but is similar with regard to quality of life and pain. Cost-effectiveness showed that without the PAS denosumab was not estimated to be cost-effective relative to either zoledronic acid or BSC. With the PAS, denosumab was estimated to be cost-effective relative to zoledronic acid but not BSC.PROSPERO number CRD42011001418.The National Institute for Health Research Health Technology Assessment programme.
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