Comparison of the cost-effectiveness of sequential treatment with abaloparatide in US men and women at very high risk of fractures.

BACKGROUND: Osteoporotic-related fractures represent an increasing burden to patients, health care systems and society. AIMS: This study estimated cost-effectiveness of sequential treatment with abaloparatide (ABL) followed by alendronate (ALN) compared to relevant alternative strategies in US men and women aged 50 to 80 years at very high fracture risk (bone mineral density T-score ≤ - 2.5 and a recent fracture). METHODS: A lifetime Markov-based microsimulation model was used to estimate healthcare costs and quality-adjusted life years (QALYs). Comparators were sequential treatment with unbranded teriparatide (TPTD)/ALN, generic ALN monotherapy, and no treatment. Analyses were conducted based on initial fracture site (hip, vertebral, or any fracture) and treatment efficacy data (derived from clinical trials or a recent network meta-analysis). RESULTS: From all analyses completed, sequential ABL/ALN demonstrated more QALYs for lower healthcare costs versus unbranded TPTD/ALN. No treatment was dominated (higher costs for less QALYs) versus ALN monotherapy. Sequential ABL/ALN resulted in favorable cost-effectiveness (at US threshold of $150,000/QALY) versus generic ALN monotherapy in men aged ≥ 50 years with any fracture type, women aged ≥ 65 years with any fracture type, and women aged ≥ 55 years having a hip or vertebral fracture. DISCUSSION: Similar cost-effectiveness of sequential ABL/ALN versus unbranded TPTD/ALN, ALN monotherapy, and no treatment was observed in both US men and women at very high fracture risk, with a moderate improvement in cost-effectiveness in men versus women and in patients with a hip or vertebral fracture. CONCLUSIONS: Sequential therapy with ABL/ALN was cost-effective in US men and women at very high risk of fractures.

Economic evaluations of nonpharmacological treatments for drug-resistant epilepsy: A systematic review.

This study was undertaken to systematically identify and critically appraise all published full economic evaluations assessing the cost-effectiveness of nonpharmacological interventions for patients with drug-resistant epilepsy. The Population, Intervention, Comparison, Outcome, Study criteria was used to design search strategies for the identification and selection of relevant studies. Literature search was performed using the MEDLINE (via PubMed), Embase, International Health Technology Assessment, National Institute for Health Research Economic Evaluation Database, and Cost-Effectiveness Analysis Registry databases to identify articles published between January 2000 and May 2023. Web of Science was additionally used to perform forward and backward referencing. Title, abstract, and full-text screening was performed by two independent researchers. The Consensus Health Economic Criteria (CHEC) checklist and Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 were applied for quality assessment. A total of 4470 studies were identified, of which 18 met our inclusion criteria. Twelve of the studies conducted model-based economic evaluation, and others were trial-based. Three studies showed that epilepsy surgery was cost-effective in adults, whereas this remained inconclusive for children (two positive, three negative). Three studies showed negative economic outcome for ketogenic diet in children. One of four studies showed positive results for self-management. For vagus nerve stimulation, one study showed positive results in adults and another one negative results in children. One recent study showed cost-effectiveness of responsive neurostimulation (RNS) in adults. Finally, one study showed promising but inconclusive results for deep brain stimulation (DBS). The mean scores for risk of bias assessment (based on CHEC) and for reporting quality (CHEERS 2022) were 95.8% and 80.5%, respectively. This review identified studies that assessed the cost-effectiveness of nonpharmacological treatments in both adults and children with drug-resistant epilepsy, suggesting that in adults, epilepsy surgery, vagus nerve stimulation, and RNS are cost-effective, and that DBS and self-management appear to be promising. In children, the cost-effectiveness of epilepsy surgery remains inconclusive. Finally, the use of ketogenic diet was shown not to be cost-effective. However, limited long-term data were available for newer interventions (i.e., ketogenic diet, DBS, and RNS).

Advocating uniformity in spine surgery: a practical disease-specific guideline for trial-based economic evaluations.

OBJECTIVES: Despite the availability of general and national guidelines for the conduct and reporting of economic evaluations, there is heterogeneity in economic evolutions concerning spine surgery. This is partly the result of differing levels of adherence to the existing guidelines and the lack of disease-specific recommendations for economic evaluations. The extensive heterogeneity in study design, follow-up duration and outcome measurements limit the comparability of economic evaluations in spine surgery. This study has three objectives: (1) to create disease-specific recommendations for the design and conduct of trial-based economic evaluations in spine surgery, (2) to define recommendations for reporting economic evaluations in spine surgery as a complement to the Consolidated Health Economic Evaluation Reporting Standards (CHEERS) 2022 checklist and (3) to discuss methodological challenges and defining the need for future research. DESIGN: A modified Delphi method according to the RAND/UCLA Appropriateness Method. SETTING: A four-step process was followed to create and validate disease-specific statements and recommendations for the conduct and reporting of trial-based economic evaluations in spine surgery. Consensus was defined as >75% agreement. PARTICIPANTS: A total of 20 experts were included in the expert group. Validation of the final recommendations was obtained in a Delphi panel, which consisted of 40 researchers in the field who were not included in the expert group. PRIMARY AND SECONDARY OUTCOME MEASURES: The primary outcome measure is a set of recommendations for the conduct and reporting, as a complement to the CHEERS 2022 checklist, of economic evaluations in spine surgery. RESULTS: A total of 31 recommendations are made. The Delphi panel confirmed consensus on all of the recommendations in the proposed guideline. CONCLUSION: This study provides an accessible and practical guideline for the conduct of trial-based economic evaluations in spine surgery. This disease-specific guideline is a complement to existing guidelines, and should aid in reaching uniformity and comparability.

Stakeholder perspectives on cooperation in the clinical and nonclinical health technology assessment domains.

OBJECTIVES: The aim of this study was to deliver insights from multiple stakeholders into actual and future collaboration for health technology assessment (HTA) in general and in oncology in particular. METHODS: Eighteen semi-structured interviews were conducted with experts from European HTA bodies (HTAbs), former board members of the European Network for Health Technology Assessment (EUnetHTA), and representatives from the pharmaceutical industry, a regulatory agency, academia, and patient organizations. The stakeholders were asked about their support of the EUnetHTA's intent, about the general strengths and challenges of the EUnetHTA and its Joint Action 3 (JA 3), the strengths and challenges of the clinically oriented HTA collaboration in oncology during JA 3 across the technology life cycle, about future challenges to HTA in oncology with consequences for collaboration, and about collaboration in the economic domains of HTA. The transcribed interviews were analyzed qualitatively. RESULTS: The participants perceived the intention and work quality of the EUnetHTA as positive. The experts described methodological, procedural, and capacity challenges in early dialogues (EDs) and rapid relative effectiveness assessments (REAs) meant to analyze clinical effectiveness in oncology. The majority attached increasing importance to collaboration in the future to cope with the uncertainty of HTA. Several stakeholders also proposed the incorporation of joint postlaunch evidence generation (PLEG) activities. Some gave sporadic suggestions for voluntary nonclinical collaboration as well. CONCLUSION: Stakeholders' continued readiness to discuss the remaining challenges to and sufficient resources for implementing HTA regulation, as well as further cooperative expansion along the technology life cycle, are necessary for improved HTA collaboration in Europe.

Economic Evaluation of Endoscopic vs Open Vein Harvesting.

BACKGROUND: Endoscopic vein harvesting is an alternative to open vein harvesting during coronary artery bypass grafting. Although endoscopic vein harvesting includes significant clinical benefits, few long-term cost-effectiveness studies have been performed, limiting its use in the United Kingdom. In this study, we assessed the cost-effectiveness of endoscopic vein harvesting compared with open vein harvesting from the United Kingdom's National Health Service perspective. METHODS: A Markov model was developed to estimate the cost-effectiveness of endoscopic vein harvesting vs open vein harvesting by investigating the incremental lifetime costs per quality-adjusted life-year gained. A scoping literature review was conducted to inform the development of the model. One-way and probabilistic sensitivity analyses examined the robustness of the results. RESULTS: Compared with open vein harvesting, endoscopic vein harvesting leads to cost savings of £68.46 and quality-adjusted life-year gains of 0.206 per patient over a lifetime perspective. Thus, endoscopic vein harvesting is a dominant treatment option over open vein harvesting (net monetary benefit: £6248.46). In the scenario analysis, which accounted for a high-risk population with respect to leg wound infections, the net monetary benefit was £7341.47. The probabilistic sensitivity analysis showed that endoscopic vein harvesting has a 62.3% probability of being cost-effective at a threshold of £30,000 per quality-adjusted life-year, highlighting uncertainties resulting from follow-up event rates. CONCLUSIONS: Endoscopic vein harvesting is a cost-effective method of harvesting a saphenous vein graft. Further clinical data beyond 5 years of follow-up are required to confirm the long-term cost-effectiveness.

Methodology of economic evaluations in spine surgery: a systematic review and qualitative assessment.

OBJECTIVES: The present study is a systematic review conducted as part of a methodological approach to develop evidence-based recommendations for economic evaluations in spine surgery. The aim of this systematic review is to evaluate the methodology and quality of currently available clinical cost-effectiveness studies in spine surgery. STUDY DESIGN: Systematic literature review. DATA SOURCES: PubMed, Web of Science, Embase, Cochrane, Cumulative Index to Nursing and Allied Health Literature, EconLit and The National Institute for Health Research Economic Evaluation Database were searched through 8 December 2022. ELIGIBILITY CRITERIA FOR SELECTING STUDIES: Studies were included if they met all of the following eligibility criteria: (1) spine surgery, (2) the study cost-effectiveness and (3) clinical study. Model-based studies were excluded. DATA EXTRACTION AND SYNTHESIS: The following data items were extracted and evaluated: pathology, number of participants, intervention(s), year, country, study design, time horizon, comparator(s), utility measurement, effectivity measurement, costs measured, perspective, main result and study quality. RESULTS: 130 economic evaluations were included. Seventy-four of these studies were retrospective studies. The majority of the studies had a time horizon shorter than 2 years. Utility measures varied between the EuroQol 5 dimensions and variations of the Short-Form Health Survey. Effect measures varied widely between Visual Analogue Scale for pain, Neck Disability Index, Oswestry Disability Index, reoperation rates and adverse events. All studies included direct costs from a healthcare perspective. Indirect costs were included in 47 studies. Total Consensus Health Economic Criteria scores ranged from 2 to 18, with a mean score of 12.0 over all 130 studies. CONCLUSIONS: The comparability of economic evaluations in spine surgery is extremely low due to different study designs, follow-up duration and outcome measurements such as utility, effectiveness and costs. This illustrates the need for uniformity in conducting and reporting economic evaluations in spine surgery.

Associations between muscle mass/strength and healthcare costs/use for patients with cancer: A systematic literature review.

PURPOSE: To review published scientific evidence evaluating the potential associations between muscle mass/strength and healthcare use/costs for patients with cancer. METHODS: In accordance with the predefined protocol for a systematic literature review, studies assessing potential associations between muscle mass/strength and healthcare costs/use in cancer patients were searched on MEDLINE (via Ovid) and on the NHS Economic Evaluation Database in September 2021. Study selection, data extraction and quality assessment were performed by two independent reviewers. RESULTS: Of 613 studies identified, five met our inclusion criteria. Various outcomes were investigated: for length of hospital stay, one out of three studies reported an association between lower muscle mass and longer hospital stay; for hospital admission, the two identified studies did not highlight muscle weakness as a predictor of hospital admission; for hospital readmission, one out of two studies reported that patients with lower muscle mass had higher rates of hospital readmission; for costs and cost-effectiveness, results of two randomized controlled trials were mixed, with total costs of the intervention higher in one study and lower in the other, leading to opposite cost-effectiveness results. CONCLUSION: Only five studies evaluating potential associations between mass/strength and healthcare use/costs have been highlighted within this systematic review. The amount of evidence is limited but the studies are also very heterogeneous in regards of study designs, sample size, and type of population included. This important heterogeneity prevents drawing strong conclusions. Because of limited data available, more high quality longitudinal studies are needed to further investigate the relationship between muscle mass/strength and healthcare costs/use.

Does the Structure Matter? An External Validation and Health Economic Results Comparison of Event Simulation Approaches in Severe Obesity.

OBJECTIVES: As obesity-associated events impact long-term survival, health economic (HE) modelling is commonly applied, but modelling approaches are diverse. This research aimed to compare the events simulation and the HE outcomes produced by different obesity modelling approaches. METHODS: An external validation, using the Swedish obesity subjects (SOS) study, of three main structural event modelling approaches was performed: (1) continuous body mass index (BMI) approach; (2) risk equation approach; and (3) categorical BMI-related approach. Outcomes evaluated were mortality, cardiovascular events, and type 2 diabetes (T2D) for both the surgery and the control arms. Concordance between modelling results and the SOS study were investigated by different state-of-the-art measurements, and categorized by the grade of deviation observed (grades 1-4 expressing mild, moderate, severe, and very severe deviations). Furthermore, the costs per quality-adjusted life-year (QALY) gained of surgery versus controls were compared. RESULTS: Overall and by study arm, the risk equation approach presented the lowest average grade of deviation (overall grade 2.50; control arm 2.25; surgery arm 2.75), followed by the continuous BMI approach (overall 3.25; control 3.50; surgery 3.00) and by the categorial BMI approach (overall 3.63; control 3.50; surgery 3.75). Considering different confidence interval limits, the costs per QALY gained were fairly comparable between all structural approaches (ranging from £2,055 to £6,206 simulating a lifetime horizon). CONCLUSION: None of the structural approaches provided perfect external event validation, although the risk equation approach showed the lowest overall deviations. The economic outcomes resulting from the three approaches were fairly comparable.

Optimising Access Surgery in Senior Haemodialysis Patients (OASIS): study protocol for a multicentre randomised controlled trial.

INTRODUCTION: Current evidence on vascular access strategies for haemodialysis patients is based on observational studies that are at high risk of selection bias. For elderly patients, autologous arteriovenous fistulas that are typically created in usual care may not be the best option because a significant proportion of fistulas either fail to mature or remain unused. In addition, long-term complications associated with arteriovenous grafts and central venous catheters may be less relevant when considering the limited life expectancy of these patients. Therefore, we designed the Optimising Access Surgery in Senior Haemodialysis Patients (OASIS) trial to determine the best strategy for vascular access creation in elderly haemodialysis patients. METHODS AND ANALYSIS: OASIS is a multicentre randomised controlled trial with an equal participant allocation in three treatment arms. Patients aged 70 years or older who are expected to initiate haemodialysis treatment in the next 6 months or who have started haemodialysis urgently with a catheter will be enrolled. To detect and exclude patients with an unusually long life expectancy, we will use a previously published mortality prediction model after external validation. Participants allocated to the usual care arm will be treated according to current guidelines on vascular access creation and will undergo fistula creation. Participants allocated to one of the two intervention arms will undergo graft placement or catheter insertion. The primary outcome is the number of access-related interventions required for each patient-year of haemodialysis treatment. We will enrol 195 patients to have sufficient statistical power to detect an absolute decrease of 0.80 interventions per year. ETHICS AND DISSEMINATION: Because of clinical equipoise, we believe it is justified to randomly allocate elderly patients to the different vascular access strategies. The study was approved by an accredited medical ethics review committee. The results will be disseminated through peer-reviewed publications and will be implemented in clinical practice guidelines. TRIAL REGISTRATION NUMBER: NL7933. PROTOCOL VERSION AND DATE: V.5, 25 February 2021.

Mogamulizumab for Previously Treated Mycosis Fungoides and Sézary Syndrome: An Evidence Review Group Perspective of a NICE Single Technology Appraisal.

The National Institute for Health and Care Excellence (NICE) invited the manufacturer (Kyowa Kirin) of mogamulizumab (Poteligeo®), as part of the single technology appraisal process, to submit evidence for its clinical and cost-effectiveness for previously treated mycosis fungoides (MF) and Sézary syndrome (SS). Kleijnen Systematic Reviews Ltd, in collaboration with Maastricht University Medical Centre, was commissioned to act as the independent evidence review group (ERG). This paper summarises the company submission (CS), presents the ERG's critical review of the clinical and cost-effectiveness evidence in the CS, highlights the key methodological considerations and describes the development of the NICE guidance by the Appraisal Committee. Based on a systematic literature review, one randomised controlled trial, MAVORIC, was identified showing favourable results in patients with MF and SS. However, MAVORIC compared mogamulizumab to vorinostat, which is not standard care in the NHS, and there is uncertainty due to the study design, specifically crossover of patients. Based on a "naïve comparison of results from the vorinostat arm of the MAVORIC study and the physician's choice arm (methotrexate or bexarotene i.e. United Kingdom [UK] standard treatments) of the ALCANZA study as well as comparison to Phase II bexarotene data", the company considered vorinostat to be "a reasonable proxy for current standard of care in the NHS". The ERG considered, based on the limited data available, that the comparability of vorinostat (MAVORIC) and physician's choice (ALCANZA) could not be established. In response to the Appraisal Consultation Document, the company provided an unanchored matched adjusted indirect comparison (MAIC) of mogamulizumab with UK standard care by analysing Hospital Episode Statistics (HES) data. However, given the high risk of bias of an unanchored MAIC, these results needed to be regarded with a considerable degree of caution. The economic analysis suffered from uncertainty because there was no trial evidence on the comparator in the England and Wales National Health Service (NHS), and it was unclear to what extent the trial (MAVORIC) comparator (vorinostat) was comparable to standard care, referred to as established clinical management (ECM) in the NHS. The evidence for overall survival had not reached maturity and was confounded by treatment switching, for which different crossover adjustment methods produced large variations in life years. Caregiver utilities were applied in the analysis, but there was a lack of guidance on their application and whether these were indicated in this appraisal. After consultation, the company updated the economic analysis with the MAIC. Incremental cost-effectiveness ratios comparing mogamulizumab against ECM were (depending on whether the HES or MAVORIC comparison were used) £31,030 or £32,634 per quality-adjusted life years (QALYs) gained according to the company's base case and £38,274 or £80,555 per QALY gained according to the ERG's base case. NICE did not recommend mogamulizumab for treating MF or SS in adults who have had at least one previous systemic treatment. This decision was subsequently appealed, and an appeal decision has been reached.

Evidence-based recommendations for economic evaluations in spine surgery: study protocol for a Delphi consensus.

INTRODUCTION: Considering the rising global healthcare expenses, economic evaluations are more important than ever. Even though the number of studies regarding costs and cost-effectiveness is increasing, the quality of these studies remains relatively low. This is mainly caused by abundant heterogeneity in methods used for determining, calculating and reporting cost data, despite current general guidelines for the conduct of economic evaluations. Disease-specific recommendations for the conduct of economic evaluations in the field of spine surgery, as complement to existing general guidelines, will ameliorate overall research quality, comparability and interpretability and thus, the overall quality. We aim to provide expert-based recommendations for the design, conduct, and reporting of economic evaluations in spine surgery. METHODS AND ANALYSIS: A modified Delphi study will be conducted to formulate expert-based recommendations. The following steps will be taken:(1) The conduct of a systematic review to identify relevant publications and identify relevant authors. Formation of an expert group and a Delphi-panel. (2) Drafting of statements based on articles included in the systematic literature review. Validation of drafted statements by the expert group. Step 2 can be repeated up to three times, statements can be discarded and adjusted in these rounds. Statements with more than 75% agreement will be accepted as consensus statements. (3) Validation of statements by the Delphi-panel. (4) Final recommendations. ETHICS AND DISSEMINATION: The underlying work is based on existing literature and published data and does not include participation of patients, and thus does not require ethical review approval. The final recommendations are intended for (clinical) researchers in the field of cost-effectiveness in spine surgery. The Delphi method ensures that the final output reflects the opinions of international participants and gives insight in the adherence level to the recommendations. The aim is to reach uniformity in design, conduct and reporting of these studies, as is currently lacking. This will provide a solid basis to determine cost-effectiveness of spine surgeries and consequently aid to limit the rising healthcare costs. The findings of this study and the final recommendations will be disseminated in conferences and seminars and will be published in an international peer-reviewed journal.

Three years pilot of spinal muscular atrophy newborn screening turned into official program in Southern Belgium.

Three new therapies for spinal muscular atrophy (SMA) have been approved by the United States Food and Drug Administration and the European Medicines Agency since 2016. Although these new therapies improve the quality of life of patients who are symptomatic at first treatment, administration before the onset of symptoms is significantly more effective. As a consequence, newborn screening programs have been initiated in several countries. In 2018, we launched a 3-year pilot program to screen newborns for SMA in the Belgian region of Liège. This program was rapidly expanding to all of Southern Belgium, a region of approximately 55,000 births annually. During the pilot program, 136,339 neonates were tested for deletion of exon 7 of SMN1, the most common cause of SMA. Nine SMA cases with homozygous deletion were identified through this screen. Another patient was identified after presenting with symptoms and was shown to be heterozygous for the SMN1 exon 7 deletion and a point mutation on the opposite allele. These ten patients were treated. The pilot program has now successfully transitioned into the official neonatal screening program in Southern Belgium. The lessons learned during implementation of this pilot program are reported.

Effect of direct oral feeding following minimally invasive esophagectomy on costs and quality of life.

AIMS: Following (minimally invasive) esophagectomy, patients often rely on tube feeding, since oral intake is often delayed. Consequently, additional support by a dietician and home care is needed until oral intake is commenced. In this study, the effects of direct start of oral feeding compared with tube feeding following an esophagectomy was evaluated on treatment costs and health-related quality of life (QoL). METHODS: Patients undergoing a minimally invasive esophagectomy were randomized in the NUTRIENT II study between controls (nil-per-mouth during 5 days and subsequent tube feeding) and a group in whom oral feeding was started directly postoperatively. Total hospital costs (including readmission and outpatient costs) and home care data for a period of 6 months after surgery were analyzed. QoL (measured using EORTC-QLQ-C30 and EORTC OG-25) was assessed preoperatively and 6 weeks, 12 weeks, and 6 months postoperatively. RESULTS: A total 132 patients were included (n = 65 direct oral feeding group and n = 67 control group). Mean patient hospital costs were €26,014 in the intervention group over a 6-month period compared to €26,989 in the control group (p = .825). Furthermore, people with direct oral feeding required significantly less home care assistance; i.e. 23 (48.9%) intervention patients versus 37 (77.1%) control patients (p = .004). Also, QoL in patients with direct oral feeding progressed more quickly when compared to the control group. LIMITATIONS: Hospital costs were derived from a single hospital unit whereas costs from all the participating units may be a better reflection of the cost deviation. Availability of homecare data was limited, leading to difficulty in detecting differences in costs. CONCLUSION: This study suggests that direct oral feeding leads to similar total costs and a significantly reduced need for home care assistance. Furthermore, QoL in intervention group increased more quickly when compared to the control group.

Parent, provider and vaccinee preferences for HPV vaccination: A systematic review of discrete choice experiments.

OBJECTIVES: To systematically review, appraise and evaluate available evidence regarding discrete-choice experiments (DCEs) for the human papilloma virus (HPV) vaccination in order to support policymakers in making reasonable and effective vaccination program implementation decisions. METHODS: A systematic literature review was conducted using the databases PubMed and Embase for DCEs in HPV up to May 2019. Extracted data was tabulated and two checklists were used for the quality appraisal of the included studies. All attributes were categorized in outcome, process or costs attributes and the relative importance of attributes was calculated using the range method. RESULTS: Out of 164 identified studies, 12 met the inclusion criteria. Eight were from high income countries (HICs) and four from low and middle-income countries (LMICs). Five studies each examined vaccinee and parent preferences, while only two assessed the providers' preferences. The studies were rather heterogenous in terms of the populations investigated, the attributes included and the methodologic approach. Overall, outcome measures were the most prominent attributes and effectiveness consistently yielded high relative importance scores. But also process factors, such as the age at vaccination, played an important role for decision making. Discrepancies between HICs and LMICs were most prominent for cost attributes. CONCLUSION: The heterogenous preferences this review elicited highlight the importance of context when making decisions grounded on consumer preferences. Especially the lack of evidence from LMICs, where the burden of cervical cancer is highest, is worrisome. In order to increase uptake, close vaccination gaps and reduce current inequities in (reproductive) healthcare, policy makers need to understand the features that drive individual vaccination decisions and adapt national and clinical guidelines accordingly. Future research therefore needs to focus on LMICs in order to elicit preferences of those most vulnerable populations.

The clinical and economical impact of postoperative ileus in patients undergoing colorectal surgery.

BACKGROUND: Colorectal surgery is associated with postoperative ileus (POI). Despite its widespread manifestation, the influence of POI on recovery, quality of life (QoL), and costs is largely unknown. The aim of this study was to assess whether the inflammatory processes found in experimental studies are also evident in patients undergoing colorectal surgery. In addition, the impact of POI on short and long-term QoL and costs was investigated. METHODS: We analyzed the outcomes of the SANICS-II trial, including prospective evaluation of inflammatory parameters in blood samples, costs from a societal perspective and QoL, using validated questionnaires. Outcomes were compared between patients with and without POI, and in particular patients with POI as unique complication. KEY RESULTS: A total of 265 patients (POI, n = 66 vs non-POI, n = 199) were included and 38/66 had POI as only complication. CRP levels were significantly increased on postoperative day (POD) 1, 2, 3, and 4 in patients with POI. Furthermore, plasma levels of cytokines IL-6, Il-8 and IL-10 were significantly increased the first 2 days after resection. Patients with POI had a higher overall complication rate and a reduced QoL 3 months postoperatively, even in the only POI group. Moreover, mean societal cost per patient with POI was 38%-47% higher at 3 months postoperatively. CONCLUSIONS & INFERENCES: Supporting findings from experimental studies, inflammatory parameters were increased in patients with only POI and comparable with all patients with POI. These results demonstrate the impact and large contribution of POI in postoperative inflammation, costs and QoL in patients undergoing colorectal surgery.

Cost-effectiveness of sequential treatment with abaloparatide followed by alendronate vs. alendronate monotherapy in women at increased risk of fracture: A US payer perspective.

OBJECTIVES: Emerging evidence supports sequential therapy with anabolic followed by antiresorptive in patients at high-risk of fragility fractures. This study assessed the cost-effectiveness of sequential treatment with abaloparatide (ABL) followed by alendronate (ALN) [(ABL/ALN)] compared to ALN monotherapy and to sequential treatment starting with antiresorptive therapy (ALN/ABL/ALN). METHODS: A previously validated Markov microsimulation model was used to estimate the cost-effectiveness of sequential ABL/ALN compared to ALN monotherapy and to sequential ALN/ABL/ALN from a lifetime US payer perspective. In line with practice guidelines, patients were assumed to receive ABL for 18 months followed by 5 years of ALN, or ALN monotherapy for 5 years, or a sequence of ALN for 2 years followed by 18 months of ABL and then by 3 years ALN. Evaluation was conducted for patients aged 50-80 years old with a BMD T-score ≤-3.5 and without a history of prior fracture, or with a T-score between -2.5 and -3.5 and a history of ≥ 1 osteoporotic fracture. RESULTS: Sequential ABL/ALN was cost-effective (threshold of US$150,000 per QALY) vs generic ALN monotherapy in women ≥60 years with a BMD T-score ≤-3.5 and in women with BMD T-score between -2.5 and -3.5 and history of osteoporotic fracture. In all simulated populations, sequential ABL/ALN therapy was dominant (lower costs, more QALYs) compared with sequential ALN/ABL/ALN, resulting from limited effect of ABL in patients previously treated with an antiresorptive agent. CONCLUSIONS: Sequential ABL/ALN therapy is cost-effective vs ALN monotherapy for US postmenopausal women aged ≥60 years at increased risk of fractures.

Cost-effectiveness of sequential treatment with abaloparatide vs. teriparatide for United States women at increased risk of fracture.

OBJECTIVES: There is emerging evidence supporting sequential therapy with an osteoanabolic followed by an antiresorptive in patients at high-risk of fragility fractures. This study assessed the cost-effectiveness of sequential treatment with abaloparatide (ABL) followed by alendronate (ALN) [(ABL/ALN)] compared with teriparatide (TPTD) followed by ALN (TPTD/ALN). METHODS: A previously validated Markov microsimulation model was adapted to estimate the cost-effectiveness of sequential ABL/ALN compared with sequential TPTD/ALN and no treatment with a lifetime horizon from the US payer perspective. Patients were assumed to receive ABL or TPTD for 18 months followed by 5 years of ALN in line with clinical recommendations. The effects of ABL on fracture risk were derived from the ACTIVExtend trial. The effects of TPTD were assumed to be maintained during subsequent ALN treatment, consistent with ACTIVExtend findings for ABL. Evaluation was completed for patients, aged 50-80 years with a BMD T-score ≤ -3.5 or with a T-score between -2.5 and -3.5 and a history of ≥ one osteoporotic fracture. RESULTS: In all simulated populations, sequential ABL/ALN therapy was dominant (lower costs, higher QALYs) compared with sequential TPTD/ALN therapy, resulting from the improved efficacy and lower drug price of ABL. Probabilistic sensitivity analyses suggested that ABL/ALN was dominant in at least 99% of the simulations. Compared to no treatment, the cost per QALY gained of ABL/ALN was always below $130,000. CONCLUSIONS: Sequential ABL/ALN therapy is a cost-effective (dominant) strategy compared with sequential TPTD/ALN therapy for the treatment of US women at increased risk of fractures.

Methods for Measuring Multiple Medication Adherence: A Systematic Review-Report of the ISPOR Medication Adherence and Persistence Special Interest Group.

BACKGROUND: A broad literature base exists for measuring medication adherence to monotherapeutic regimens, but publications are less extensive for measuring adherence to multiple medications. OBJECTIVES: To identify and characterize the multiple medication adherence (MMA) methods used in the literature. METHODS: A literature search was conducted using PubMed, PsycINFO, the International Pharmaceutical Abstracts, the Cumulative Index to Nursing and Allied Health Literature and the Cochrane Library databases on methods used to measure MMA published between January 1973 and May 2015. A two-step screening process was used; all abstracts were screened by pairs of researchers independently, followed by a full-text review identifying the method for calculating MMA. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines were followed to conduct this systematic review. For studies that met the eligibility criteria, general study and adherence-specific characteristics and the number and type of MMA measurement methods were summarized. RESULTS: The 147 studies that were included originated from 32 countries, in 13 disease states. Of these studies, 26 used proportion of days covered, 23 used medication possession ratio, and 72 used self-reported questionnaires (e.g., the Morisky Scale) to assess MMA. About 50% of the studies included more than one method for measuring MMA, and different variations of medication possession ratio and proportion of days covered were used for measuring MMA. CONCLUSIONS: There appears to be no standardized method to measure MMA. With an increasing prevalence of polypharmacy, more efforts should be directed toward constructing robust measures suitable to evaluate adherence to complex regimens. Future research to understand the validity and reliability of MMA measures and their effects on objective clinical outcomes is also needed.

A discrete choice experiment to investigate patient preferences for HIV testing programs in Bogotá, Colombia.

BACKGROUND: To assess patients' preferences for HIV testing in Colombia. METHODS: A discrete choice experiment was used to assess preferences of patients diagnosed with HIV, for HIV testing in two HIV clinics in Bogotá, Colombia. Patients were asked to choose repeatedly between two hypothetical HIV testing options that varied with respect to five attributes: distance to testing site, confidentiality, testing days, sample collection method, and the services if HIV positive. A random parameter model was used to analyze the data. RESULTS: A total of 249 questionnaires were eligible for data analysis. Respondents showed a preference for testing on weekdays, nobody being aware, a sample taken from the arm, and receiving medications through a referral. The respondents showed a high negative preference for many people being aware, followed by testing during the weekend and home testing. Subgroup analyses by gender and prior testing history did not reveal significant differences. CONCLUSION: This study suggests that patients' preferences for HIV testing focused especially on confidentiality, availability during weekdays, and using a sample from the arm. This information could be useful to improve uptake of HIV testing in Bogotá, Colombia.