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BACKGROUND: To estimate the potential benefits in terms of avoided complications and cost reduction if the Spanish health system would encourage the intensification of treatment for better glycaemic control in adults with Type 2 diabetes from the current HbA1c target used in clinical practice of 68 mmol/mol to a target of 53 mmol/mol.
METHODS: The IQVIA Core Diabetes Model (version 9.0) was used to model the impact of these changes in respect of micro- and macrovascular complications and the associated costs. The modelling was based on data derived from the SIDIAP-Q population database from Catalonia, taking a random cohort of 10,000 people with type 2 diabetes and dividing it into sub-groups based on their baseline HbA1c.
RESULTS: The CDM modelling showed that the average cost reduction per person varies depending on baseline HbA1c. The model estimates that after 25 years, people with a baseline HbA1c between 48 and 58 mmol/mol and > 75 mmol/mol show an average cost reduction of €6027 and €11,966, respectively. Applying the per-person cost reduction to the cohorts of the prevalent population in Spain (1,910,374) the overall estimated cost reduction was €14.7 billion over 25 years. The improvements in outcomes resulted in an estimated reduction of more than 1.2 million complications cumulatively over 25 years, of which more than 550,000 relate to diabetic foot and more than 170,000 related to renal disease.
CONCLUSION: Over a 25 year period, Spain could considerably reduce costs and avoid major complications if, on a population level, more ambitious glycaemic control, according to Spanish or EU guidelines, could be achieved among people with type 2 diabetes by reducing the HbA1c threshold for treatment intensification. Although there is a slower trajectory for benefits in earlier years, there is a much more rapid benefit gain between years 5 and 15.
BACKGROUND: Psoriasis is a chronic inflammatory skin condition that impacts quality of life and requires long-term treatment and effective symptom management. Interleukin-23 (IL-23) has emerged as a key player in the pathogenesis of psoriasis and tildrakizumab and guselkumab are both immunomodulatory agents that inhibit the p19 subunit of IL-23. In its pivotal Phase III trial, tildrakizumab demonstrated greater efficacy than etanercept in moderate-to-severe psoriasis. However, there are no head-to-head trials comparing tildrakizumab with guselkumab.
METHODS: We conducted a systematic literature review and Bucher indirect comparison of tildrakizumab and guselkumab, using placebo as a common comparator. We searched MEDLINE, MEDLINE In-Process, MEDLINE(R) Daily Epub Ahead of Print, and Cochrane Central Register of Controlled Trials for Phase III randomized controlled trials between 1946 and November 2018. Inclusion criteria were adult patients =18 years with moderate-to-severe chronic plaque psoriasis, and intervention with tildrakizumab or guselkumab compared to placebo or best supportive care. Outcomes included were severity of psoriasis as defined by the Psoriasis Area and Severity Index (PASI) 75 and PASI 90, frequency of serious adverse events (SAEs), and treatment discontinuations. Outcomes were evaluated at Weeks 12 to 16 and 24 to 28. Analysis was based on the intent-to-treat population and, for all outcomes, the number of events reported were analyzed as a proportion of the number of patients randomized to ensure consistency across trials.
RESULTS: Overall, 154 unique records were identified. Five studies met the eligibility criteria and were included in the analysis; two tildrakizumab trials (reSURFACE 1 and reSURFACE 2) and three guselkumab trials (VOYAGE 1, VOYAGE 2, and a Japanese study). There was no statistically significant difference between guselkumab and tildrakizumab for PASI 75, PASI 90, SAEs, and rate of discontinuations at either timepoint.
CONCLUSIONS: This study assessed the comparative efficacy of tildrakizumab and guselkumab for the treatment of moderate-to-severe psoriasis. Limitations included the limited number of publications, imputation of placebo arm values for Weeks 24 to 28, and limited relevance of the Japanese study. This indirect comparison does not provide evidence that one treatment is superior to the other.
PURPOSE: To generate UK health-related quality-of-life (HRQoL) data for adult patients with moderate-to-severe limbal stem cell deficiency (LSCD), unilateral or bilateral, due to physical or chemical ocular burns to help inform economic evaluations of treatments.
PATIENTS AND METHODS: EQ-5D-3L with vision bolt-on scores was prospectively measured for one of five clinical scenarios of LSCD described in vignettes in a demographically representative population of 520 UK adults. These were converted to health state utilities using three different UK value sets. A standard gamble (SG) was then undertaken using 12 SG scenarios to examine the component drivers of health utility for the treatment of LSCD.
RESULTS: For the EQ-5D-3L scenarios, the mean disutility for LSCD with poor visual acuity, pain and disfigurement in both eyes compared to one eye was - 0.084 (range=- 0.156 to - 0.045 across the value sets). The mean disutility of bilateral LSCD with pain, disfigurement, and poor visual acuity compared to unilateral LSCD with only poor visual acuity in one eye was - 0.104 (range=- 0.151 to - 0.078). Similarly, where one eye was affected, pain and disfigurement in combination were associated with a greater mean disutility than improvements in visual acuity alone: - 0.011 (range=- 0.04 to 0.005). Mean SG utilities were within a narrow range (0.682- 0.765). Where one eye was affected, the main driver was disfigurement: mean utility was 0.731 (0.709- 0.753) compared to 0.682 (0.659- 0.704) when disfigurement was removed compared to vision restored to normal. For bilateral LSCD, mean utilities were 0.693 (0.672- 0.715) for normal vision and 0.75 (0.73- 0.771) when disfigurement and pain were removed.
CONCLUSION: Improvements in pain and disfigurement appeared to be the main factors driving differences in health utilities associated with symptom profiles in LSCD, with improvements in visual acuity having lesser impact.
BACKGROUND: High prices of pharmaceutical products are an increasing challenge in high- and low-income countries. Governments in many countries have implemented pricing policies to ensure affordability of medicines to patients and healthcare systems. The World Health Organization published in 2015 the Guideline on Country Pharmaceutical Pricing Policies, which was based on a series of evidence reviews in the preceding years.
As part of the ongoing update of this guideline, we present a protocol for 10 systematic literature reviews on pharmaceutical pricing policies to be covered by the updated guideline.
METHODS: The systematic literature reviews will be undertaken according to the principles embodied in the Cochrane Handbook and Centre for Reviews and Dissemination. The interventions studied are pharmaceutical pricing policies implemented by public institutions or a group of purchasing organizations/individuals (e.g. health services). Studies reporting price, volume, availability and/or affordability as the primary outcomes will be eligible for inclusion. Studies in any country or jurisdiction, in any language and in any setting published in 2004 or later are eligible. Eligible study designs are randomized and non-randomized trials, and observational studies including cohort studies, panel data analyses, comparative time series design (including interrupted time-series and repeated measures studies), and controlled before-after studies. A list of 21 databases of peer-reviewed and grey literature will be searched, along with supplementary searches of relevant national and international organizational and governmental websites. Risk of bias will be assessed according to the Cochrane Effective Practice and Organisation of Care (EPOC) guidelines. A summary table according to the EPOC Worksheets for preparing a Summary of Findings table (SoF) using GRADE will be provided.
DISCUSSION: The results of the review will be used as part of the update of the WHO Guideline on Country Pharmaceutical Pricing Policies. The current protocol may serve as an example for performing systematic literature reviews to inform policy makers.
The health economic evidence of nutrition tends to be limited. An important reason is that nutritionals do not always fall under the coverage requirements for reimbursement, like pharmaceuticals, which often require health economic data. The objective of this paper is to assess the relevance of proactively generating cost-effectiveness data from a nutrition company perspective. After a general introduction into health economics, we will address the key question of this paper: Are cost-effectiveness data in nutrition a double edged sword for nutrition companies?
The conclusion is that, as things currently stand, nutrition companies should be cautious in proactively generating cost-effectiveness data in terms of cost per QALY. Disaggregated economic and outcomes data may be more relevant and less risky for nutrition companies.
BACKGROUND: Local oestrogens, the current treatment for vulvar and vaginal atrophy (VVA), are not suitable for all women. Standard of care (SoC) consists of over-the-counter lubricants and moisturisers. Senshio® (ospemifene) provides a treatment option for postmenopausal women who are not candidates for local vaginal oestrogen therapy who would otherwise have an unmet clinical need.
OBJECTIVES: The aim of this study was to estimate the cost-effectiveness of ospemifene, a selective oestrogen receptor modulator, for the treatment of moderate to severe symptomatic VVA in postmenopausal women who are not candidates for local vaginal oestrogen therapy.
METHODS: The Scottish Medicines Consortium (SMC) recently evaluated the clinical and cost-effectiveness evidence of ospemifene plus SoC compared with SoC alone. A cost-effectiveness study, from a National Health Service (NHS) Scotland perspective over a lifetime time horizon, was submitted to the SMC. The cohort-based Markov model used robust clinical evidence from two large pivotal phase III randomised controlled studies and included four health states classified by dyspareunia symptom severity: none, mild, moderate and severe. The movement of women between health states was dependent on the effectiveness of treatment in reducing dyspareunia. Extensive sensitivity analyses were undertaken to assess the level of confidence associated with the base-case results.
RESULTS: Treatment with ospemifene was associated with an additional cost of £847 per patient and an increase in quality-adjusted life-years (QALY) of 0.06 per patient. Ospemifene had an incremental cost-effectiveness ratio of £14,138 per QALY. In the probabilistic sensitivity analysis, there was a probability of 89% that ospemifene was cost-effective at a threshold of £20,000 per QALY gained. Ospemifene remained cost-effective under all scenario analyses. The SMC reviewed the clinical and economic evidence and judged that the evidence demonstrated a robust case to support prescribing ospemifene in NHS Scotland.
CONCLUSION: Ospemifene is a cost-effective intervention that has recently been accepted by the SMC for the treatment of postmenopausal women with moderate to severe VVA who are not candidates for local oestrogen.
BACKGROUND: Lower gastrointestinal symptoms are common in the general population and it can be difficult to discriminate between inflammatory bowel disease (IBS) and irritable bowel syndrome (IBD) due to overlap of symptoms. The York Fecal Calprotectin Care Pathway (YFCCP) was introduced in 2016 as an alternative to the NICE fecal calprotectin pathway (DG11). This analysis uses the prospective data from the first 1005 patients in the YFCCP. Previous analysis demonstrated the YFCCP may be cost-saving when compared with the DG11 pathway. This analysis examined the short-term health-related quality of life (HRQoL) impact for patients in the YFCCP for IBD and IBS.
METHODS: A decision tree model was used to estimate the proportion of people presenting with lower gastrointestinal symptoms that were correctly or falsely diagnosed with IBS and IBD. Time to diagnosis data was estimated and HRQoL data was estimated from published sources. Costs and QALYs were calculated for the YFCCP and each comparator.
RESULTS: The YFCCP was cost-effective at a £20,000 threshold when compared with the current NICE recommended pathways and was cost-saving with a QALY gain (dominant)in four of the five comparators. Conclusions: The YFCCP demonstrated a QALY benefit when compared with all alternative pathways.
