Hypoglycemia: An overview of fear of hypoglycemia,quality-of-life,and impact on costs

Abstract

Background:

The clinical goal in the treatment of diabetes is to achieve good glycemic control. Tight glycemic control achieved with intensive glucose lowering treatment reduces the risk of long-term micro- and macro-vascular complications of diabetes, resulting in an improvement in quality-of-life for the patient and decreased healthcare costs. The positive impact of good glycemic control is, however, counterbalanced by the negative impact of an increased incidence of hypoglycemia.

Methods:

A search of PubMed was conducted to identify published literature on the impact of hypoglycemia, both on patient quality-of-life and associated costs to the healthcare system and society.

Results:

In people with type 1 or type 2 diabetes, hypoglycemia is associated with a reduction in quality-of-life, increased fear and anxiety, reduced productivity, and increased healthcare costs. Fear of hypoglycemia may promote compensatory behaviors in order to avoid hypoglycemia, such as decreased insulin doses, resulting in poor glycemic control and an increased risk of serious health consequences. Every non-severe event may be associated with a utility loss in the range of 0.0033–0.0052 over 1 year, further contributing to the negative impact.

Limitations:

This review is intended to provide an overview of hypoglycemia in diabetes and its impact on patients and society, and consequently it is not a comprehensive evaluation of all studies reporting hypoglycemic episodes.

Conclusion:

To provide the best possible care for patients and a cost-effective treatment strategy for healthcare decision-makers, a treatment that provides good glycemic control with a limited risk of hypoglycemia would be a welcome addition to diabetes management options.     

Medical economic impact of tracheotomy patients on a hospital system

Abstract

Background:

Tracheotomy patients are a small portion of hospitalizations, but account for disproportionately high risk and costs. There are many complex decisions that go into the care of these patients, and practice variation is expected to be compounded in a health system. This study sought to characterize the medical economic impact of tracheotomy patients on the hospital system.

Methods:

A retrospective review of the health system’s hospital billing software was performed for 2013, and pertinent outcomes measures were tabulated.

Results:

There were 829 tracheotomies performed in the health system of seven hospitals, with total costs of $128,883,865. Average length of stay was 36.74 days for principal procedures, and 43.36 days for tracheotomy as secondary procedures. Mortality was ～18% overall, and re-admissions were 10.93% for primary, and 14.36% for secondary procedures. A fairly wide variation in each category among the different hospitals was observed.

Conclusions:

There are potentially many factors that impact variations of care and outcomes in patients with tracheotomy. Due to their large economic impact and risks for morbidity and mortality, a formalized care pathway is warranted. Goals of the pathway should include understanding medical decisions surrounding these complex patients, monitoring pertinent outcomes, reducing practice variation, and improving the efficiency of compassionate care.     

Implications of Real-world Adherence on Cost-effectiveness Analysis in Multiple Sclerosis

Abstract

Objectives:

Adherence to medication is essential for optimal outcomes, especially for chronic diseases such as multiple sclerosis (MS). Studies in MS indicate that lower adherence is associated with an increased risk of relapse, hospitalization or emergency room (ER) visits, and higher medical costs. A previous investigation assessed the cost per relapse avoided for patients with MS receiving first-line disease modifying therapies (DMTs); however, the model assumed 100% adherence.

Methods:

Because real-world utilization patterns influence the actual effectiveness of medications, this analysis assessed the impact of real-world adherence from a US commercial payer perspective, using updated costs.

Results:

As was seen in the original study, in this revised model, fingolimod was associated with the lowest cost per relapse avoided ($90,566), followed by SC IFN β-1b (Extavia: $127,024), SC IFN β-1b (Betaseron: $137,492), SC IFN β-1a ($144,016), glatiramer acetate ($160,314), and IM IFN β-1a ($312,629). The model inputs that had the greatest impact on the results were adherence-adjusted relative relapse rate reduction (RRR) of fingolimod, the wholesale acquisition costs of fingolimod, and the average number of relapses in untreated patients with MS.

Limitations:

The estimates of DMT adherence are from a single claims database study of a large national pharmacy benefit manager that only measured adherence, not actual relapses, and the model does not incorporate manufacturer discounts and rebates, which are not publicly available.

Conclusion:

These results suggest that economic analyses of MS therapies should incorporate real-world adherence rates where available, rather than relying exclusively on trial-based efficacy estimates when considering the economic value of treatment alternatives, and that highly efficacious therapies with low adherence may yield real-world efficacy that is substantially lower than that observed in closely monitored clinical trials.     

Changing costs and the impact on RSV prophylaxis

Abstract

Objective:

Acquisition costs of palivizumab have increased in Canada since 2007. This analysis aims to re-evaluate the cost effectiveness of palivizumab in Canada for premature infants born between 32 and 35 weeks’ gestational age using updated 2010 healthcare costs compared to those used in a 2007 decision analytic model.

Methods:

New costs (CAN$) were acquired from the same Health Canada and Ontario Ministry of Health sources that were utilized in the previously published 2007 model. Palivizumab prices were acquired from Abbott Laboratories Ltd., current as of August 2010.

Results:

Incremental cost-effectiveness ratios (ICERs) rose by $742, going from $30,618/QALY to $31,360/QALY. ICER changes increased from a range of $801,297 to $820,701 for infants with zero risk factors to a decrease from $808 to $192 for infants with four or more risk factors.

Conclusions:

Palivizumab ICERs remained fairly stable from 2007 to 2010. The original recommendation stating that palivizumab is cost effective in infants born between 32 and 35 weeks’ GA with two or more risk factors, or who are at moderate-to-high risk based on a risk assessment model, does not change. Analyses founded on evolving country-specific variables are needed in order to accurately reassess the cost effectiveness of interventions as costs change worldwide.

Limitations:

There are a limited number of publications reporting mortality in premature Canadian infants with RSV as a primary outcome. In addition, conclusions drawn from this analysis are country-specific and limited to premature infants dwelling in Canada.     

The impact of rheumatoid arthritis on the burden of disease in urban China

Abstract

Objectives:

The aim of this study is to assess the burden of disease associated with the impact of rheumatoid arthritis in urban China. Burden of disease is considered from four perspectives: (i) health-related quality-of-life (HRQoL); (ii) health status; (iii) employment status; and (iv) absenteeism and presenteeism.

Methods:

Data are from the 2009 National Health and Wellness Survey (NHWS) of urban China. This is an internet-based survey and details the health experience of 13,007 respondents. The survey is representative of the urban China population at 18 years of age and over (18.1% of the total population). Of those responding to the survey, a total of 353 reported that they had been diagnosed with rheumatoid arthritis – an unweighted estimate of 2.65%. The sample design allows a comparison of those reporting rheumatoid arthritis with those not reporting this disease and, hence, a quantitative assessment of the burden of disease. Estimates of the quantitative impact of the presence of rheumatoid arthritis are through a series of generalized linear regression models. HRQoL is evaluated through the SF-12 instrument together with responses to the first item of the SF-12, self-reported health status. The SF-12 instrument generates three measures of HRQoL: the physical component summary (PCS), the mental component summary (MCS) and SF-6D utilities. Health status is captured as a self-report on a 5-point scale. Employment status is considered in terms of self-reported labor force participation, while absenteeism and presenteeism are estimated from the Work Productivity Activity Index (WPAI). Apart from a binary variable capturing the presence or absence of rheumatoid arthritis, control variables were included to capture the impact of other potential determinants of HRQoL and health status.

Results:

The presence of rheumatoid arthritis in urban China has a significant deficit impact on HRQoL as measured by the PCS and MCS components of the SF-12, SF-6D absolute utilities and on self-assessed health status. In the case of PCS, the deficit impact of rheumatoid arthritis is ?2.289 (95%CI: ?3.042 to ?1.536); for MCS ?1.472 (95%CI: ?2.338 to ?0.605) and for utilities ?0.025 (95% CI: ?0.036 to ?0.014). In the case of health status the odds ratio for the presence of rheumatoid arthritis is 1.275 (95%CI 1.031–1.576). The presence of rheumatoid arthritis has a marked negative effect, just under 8%, on the likelihood of workforce participation. Finally, the presence of rheumatoid arthritis is associated with an increased likelihood of absenteeism and presenteeism.

Limitations:

The NHWS survey has a number of limitations. As the NHWS is an internet-based survey, biases may be present due to the lack of internet penetration in the urban China population. The extent to which individuals and households have internet access is unknown. In addition, the NHWS relies upon respondents reporting they have been diagnosed with one or more specific disease states. These are not, given the nature of the survey, clinically verified. This also introduces a degree of uncertainty. Care should be taken in uncritically generalizing these results to the wider China population.

Conclusions:

The burden of disease associated with self-reported, diagnosed rheumatoid arthritis in urban China is substantial. Utilizing a series of multivariate models, substantial deficits are associated not only in reported HRQoL and health status but also in respect of employment status and, for those in employment, rates of absenteeism and presenteeism.     

Economic evaluation of anti-epileptic drug therapies with specific focus on teratogenic outcomes

Abstract

Background:

Anti-epileptic drugs are known to be teratogenic, yet many women do need to continue the anti-epileptic drug use during pregnancy.

Objectives:

To perform an economic evaluation of the anti-epileptic drug choice in young women who potentially wish to become pregnant. In particular, to estimate the impact of teratogenicity on the costs per quality adjusted life year (QALY).

Methods:

A decision-tree model is used to calculate the costs per QALY, taking into account the malformation risk in offspring due to the exposure to carbamazepine, lamotrigine or valproic acid, based on the European birth cohort of 2007. Probabilistic sensitivity analyses were performed using Monte Carlo simulation.

Results:

Valproic acid is dominated by carbamazepine after rank ordering on costs. The incremental cost-effectiveness of lamotrigine vs carbamazepine was estimated at €175,534 per QALY. Although valproic acid was dominated by carbamazepine in terms of costs and related effects, it is clinically relevant to compare lamotrigine with valproic acid. In particular, treatment options are dependent on several individual and clinical characteristics and these agents are therefore not always considered as interchangeable for all specified populations. The incremental cost-effectiveness for lamotrigine vs valproic acid was estimated at €13,370 per QALY. With assuming a willingness to pay threshold of €50,000 per QALY, results from the probabilistic analysis resulted in an acceptance level for lamotrigine vs carbamazepine and lamotrigine vs valproic acid of 4% and 99%, respectively.

Conclusion:

Based on epidemiological data it is advised to whenever possible avoid valproic acid during pregnancy. Both carbamazepine and lamotrigine are estimated to be cost-effective treatment options vs valproic acid if focused on teratogenicity.     

Hospital patients with severe wounds: early evidence on the impact of Medicare payment changes on treatment patterns and outcomes

Aims: This study examines the effects of recent changes in Medicare long-term care hospital (LTCH) payments on treatment patterns and outcomes for severe wound patients discharged from short-term acute care hospitals (STACHs).

Materials and methods: The rolling implementation of a new Medicare payment policy was used to develop a difference-in-difference model. The study population consisted of Medicare beneficiaries subjected to the payment policy changes and hospitalized for stage 3, 4, or unstageable wounds; non-healing surgical wounds; and fistula. Using 2015-Q1-2017 Medicare claims data, changes in outcomes were examined for severe wound patients exposed to the new policy (treatment) and those that were not (comparison). All outcomes were modeled using linear regressions and adjusted for patient clinical characteristics. Analysis was conducted in a full sample and a sample with high-LTCH-use propensity.

Results: Severe wound patients exposed to the new policy experienced 4.1 and 7.5 percentage point (pp) reductions in LTCH use relative to the comparison group in the full sample and high-LTCH-propensity sample, respectively (p?p?=?.039). No statistically significant change was found in 60-day mortality or Medicare spending after the policy change in the treatment group as compared to the comparison group (p?>?.10). However, among severe wound patients who are exposed to the new policy in the high-LTCH-propensity sample, readmission and post-discharge sepsis rates increased after the policy change relative to the comparison group (readmission rate = 8.1 pp, p?=?.075; sepsis rate = 7.0 pp, p?=?.033).

Limitations: The findings are based on data from a limited timeframe around the policy change and, thus, provide only early evidence on the effects of the new policy.

Conclusion: The new LTCH payment policy is associated with no changes in Medicare spending and mortality, but higher readmissions and post-discharge sepsis rates among severe wound patients with a high likelihood to use an LTCH.     

Quantifying the economic impact of a digital self-care behavioral health platform on Missouri Medicaid expenditures

Abstract

Aim: The primary objective of this study is to estimate the economic benefits relative to return on investment (ROI) of a state-wide initiative to integrate digital behavioral health (BH) self-care into the community BH system.

Methods: The observational study reviewed claims data of 799 people who registered with the digital tool (myStrength) and 715 matched control study participants. The control group was formed via coarsened exact match with blocking variables, including presence on the claims file, volume of health care utilization, participation in a medical health home, BH diagnosis and volume of psychotherapy claims. The primary study analysis of cost differences for the BH self-care tool group versus the control group were calculated by cost setting and the ROI of the BH self-care tool was estimated. Cost settings assessed include inpatient, emergency services, general and psychiatric outpatient, and outpatient psychotherapy.

Results: An incremental annual difference in difference reduction of $382 per user was observed over the 11 month study period in the self-care tool group. Sensitivity analysis indicated an ROI of between 142% and 695%.

Conclusions: Augmenting BH offerings to include digital BH self-care tools appears to generate positive ROI via reduced total cost of care.     

Predicting time to nursing home placement based on activities of daily living scores – a modelling analysis using data on Alzheimer's disease patients receiving rivastigmine or donepezil

Abstract

Objective: To quantify the impact of activities of daily living (ADL) scores on the risk of nursing home placement (NHP) in Alzheimer's disease (AD) patients.

Setting: Models predicting NHP for AD patients have depended on cognitive deterioration as the primary measure. However, there is increased recognition that both patient functioning and cognition are predictive of disease progression.

Methods: Using the database from a prospective, randomised, double-blind trial of rivastigmine and donepezil, two treatments indicated for AD, Cox regression models were constructed to predict the risk of NHP using age, gender, ADL and MMSE (Mini-Mental State Examination) scores as independent variables.

Participants: Patients aged 50–85 years, with MMSE scores of 10–20, and a diagnosis of dementia of the Alzheimer type.

Results: Cox regression analyses indicated that being female, older age, lower ADL score at baseline, and deterioration in ADL all significantly increased the risk of NHP. Over 2 years, risk of NHP increased by 3% for each 1-point deterioration in ADL score independent of cognition.

Conclusion: Data analyses from this long-term clinical trial established that daily functioning is an important predictor of time to NHP. Further research may be required to confirm whether this finding translates to the real world.     

The impact of frailty on the economic evaluation of geriatric surgery: hospital costs and opportunity costs based on meta-analysis

Abstract

Objectives: We used a systematic review and meta-analysis to analyze the difference in costs between surgery for frail and non-frail elderly patients. The opportunity cost of frailty in geriatric surgery is estimated using the results.

Methodology: Two literature reviews were carried out between 2000 and 2019: (1) studies comparing total hospital costs of frail and non-frail surgical patients; (2) studies evaluating the length of hospital stay and cost for surgical geriatric patients. We performed a meta-analysis of the items selected in the first review. We subsequently calculated the opportunity cost of frail patients, based on the design of a cost/time variable.

Results: Twelve articles in the first review were selected (272,717 non-frail and 16,461 frail). Fourteen articles were selected from the second review. Frail patients had higher hospital costs than non-frail patients (22,282.541 € and 16,388.844, p?<?.001) and a longer hospital stay (10.16 days and 8.4 (p?<?.001)). The estimated opportunity cost in frail patients is 1,019.56 € (cost/time unit factor of 579.30 €/day).

Conclusions: Frail surgical geriatric patients generate a higher total hospital cost, and an opportunity cost arising from not operating in the best possible state of health. Preoperatively treating the frailty of elderly patients will improve the use of health resources     

Evaluation of costs associated with tolvaptan-mediated length-of-stay reduction among heart failure patients with hyponatremia in the US,based on the EVEREST trial

Abstract

Background:

The Efficacy of Vasopressin Antagonism in Heart Failure Outcome Study with Tolvaptan (EVEREST) trial showed that tolvaptan use improved heart failure (HF) signs and symptoms without serious adverse events.

Objective:

To evaluate the potential cost savings associated with tolvaptan usage among hospitalized hyponatremic HF patients.

Methods:

The Healthcare Cost and Utilization Project (HCUP) 2008 Nationwide Inpatient Sample (NIS) database was used to estimate hospital cost and length of stay (LOS), for diagnosis-related group (DRG) hospitalizations of adult (age ≥18 years) HF patients with complications and comorbidities or major complications and comorbidities. EVEREST trial data for patients with hyponatremia were used to estimate tolvaptan-associated LOS reductions. A cost offset model was constructed to evaluate the impact of tolvaptan on hospital cost and LOS, with univariate and multivariate Monte Carlo sensitivity analyses.

Results:

Tolvaptan use among hyponatremic EVEREST trial HF patients was associated with shorter hospital LOS than placebo patients (9.72 vs 11.44 days, respectively); 688,336 hospitalizations for HF DRGs were identified from the HCUP NIS database, with a mean LOS of 5.4 days and mean total hospital costs of $8415. Using an inpatient tolvaptan treatment duration of 4 days with a wholesale acquisition cost of $250 per day, the cost offset model estimated a LOS reduction among HF hospitalizations of 0.81 days and an estimated total cost saving of $265 per admission. Univariate and multivariate sensitivity analysis demonstrated that cost reduction associated with tolvaptan usage is consistent among variations of model variables.

Conclusions:

The estimated LOS reduction and cost savings projected by the cost offset model suggest a clinical and economic benefit to tolvaptan use in hyponatremic HF patients.

Study Limitations:

The EVEREST trial data may not generalize well to the US population. Clinical trial patient profiles and relative LOS reductions may not be applicable to real-world patient populations.     

The impact of pain on labor force participation,absenteeism and presenteeism in the European Union

Abstract

Objectives:

The aims of this paper are to generate estimates of the association between the experience and burden of pain, by severity and frequency, with (1) labor force participation and workforce status in five EU countries (the UK, France, Spain, Germany and Italy) and (2) patterns of absenteeism and presenteeism for the employed workforce.

Methods:

Data are from the internet-based 2008 National Health and Wellness Survey (NHWS). This survey covers both those who report experiencing pain in the last month as well as the no pain population. A series of regression models are developed with the no pain group as the reference category. The impact of pain, categorized by severity and frequency reported, is assessed within a labor supply framework for (1) labor force participation and (2) absenteeism and presenteeism. In the former case both binomial and multinomial logistic models are estimated; in the latter case ordered logit models are estimated.

Results:

The results demonstrate that, in the context of health status, the experience of frequent severe and moderate pain has a dominant, independent and negative association with labor force participation and employment status as well as absenteeism and presenteeism. The presence of severe daily pain is associated with a 20-point reduction in the probability of being employed full-time; with moderate daily pain associated with a 10-point reduction. The impact of pain is far greater than the potential impact of other health status measures (e.g., chronic comorbidities and BMI). The experience of pain, notably severe and frequent pain, also outstrips the impact of other health status factors in absenteeism and presenteeism.

Conclusions:

The experience of pain, in particular severe daily pain, has a substantial negative association with labor force participation in these five European countries as well as reported absenteeism and presenteeism. As a measure of health status, it clearly outweighs other health status measures. Whether or not pain is considered as a disease in its own right, the experience of chronic pain, as defined here, presents policy makers with a major challenge. Programs to relieve the burden of pain in the community clearly have the potential for substantial benefits from societal, individual and employer perspectives.     

Impact of upper and lower gastrointestinal blood loss on healthcare utilization and costs: a systematic review

Abstract

Objectives:

Gastrointestinal (GI) blood loss is a common medical condition which can have serious morbidity and mortality consequences and may pose an enormous burden on healthcare utilization. The purpose of this study was to conduct a systematic review to evaluate the impact of upper and lower GI blood loss on healthcare utilization and costs.

Methods:

We performed a systematic search of peer-reviewed English articles from MEDLINE published between 1990 and 2010. Articles were limited to studies with patients ≥18 years of age, non-pregnant women, and individuals without anemia of chronic disease, renal disease, cancer, congestive heart failure, HIV, iron-deficiency anemia or blood loss due to trauma or surgery. Two reviewers independently assessed abstract and article relevance.

Results:

Eight retrospective articles were included which used medical records or claims data. Studies analyzed resource utilization related to medical care although none of the studies assessed indirect resource use or costs. All but one study limited assessment of healthcare utilization to hospital use. The mean cost/hospital admission for upper GI blood loss was reported to be in the range $3180–8990 in the US, $2500–3000 in Canada and, in the Netherlands, the mean hospital cost/per blood loss event was €11,900 for a bleeding ulcer and €26,000 for a bleeding and perforated ulcer. Mean cost/ hospital admission for lower GI blood loss was $4800 in Canada, and $40,456 for small bowel bleeding in the US.

Conclusions:

Our findings suggest that the impact of GI blood loss on healthcare costs is substantial but studies are limited. Additional investigations are needed which examine both direct and indirect costs as well as healthcare costs by source of GI blood loss focusing on specific populations in order to target treatment pathways for patients with GI blood loss.     

Cost effectiveness of rosuvastatin in patients at risk of cardiovascular disease based on findings from the JUPITER trial

Abstract

Objective:

This study assessed the long-term cost effectiveness of rosuvastatin therapy compared with placebo in reducing the incidence of major cardiovascular (CVD) events and mortality.

Methods:

A probabilistic Monte Carlo simulation model estimated long-term cost effectiveness of rosuvastatin therapy (20?mg daily) for the prevention of CVD mortality and morbidity. The model included three stages: (1) CVD prevention simulating the 4 years of the JUPITER trial, (2) initial CVD prevention beyond the trial, and (3) subsequent CVD event prevention. A US payer perspective was assessed reflecting direct medical costs, and up to a lifetime horizon. Sensitivity analyses tested the robustness of the model estimates.

Results:

For a hypothetical cohort of 100,000 patients at moderate and high risk of CVD events based on Framingham risk of ≥10%, estimated quality-adjusted life-years (QALYs) gained with rosuvastatin therapy compared with placebo was 33,480 over a lifetime horizon, and 25,380 and 9916 over 20-year and 10-year horizons, respectively. Approximately 12,073 events were avoided over the lifetime; 6,146 non-fatal MIs, 2905 non-fatal strokes, and 4030 CVD deaths avoided. Estimated incremental cost-effectiveness ratio (ICER) for cost per QALY was $7062 (lifetime), $10,743 (20-year horizon), and $44,466 (10-year horizon). For a hypothetical cohort similar to the overall JUPITER population, the cost per QALY ICER was $11,025 for the lifetime and $60,112 for a 10-year horizon.

Limitations:

The cost-effectiveness comparison of rosuvastatin 20?mg was against no active treatment (as opposed to an alternative statin) due to lack of comparative cardiovascular morbidity and mortality risk reduction data for other statins in a population similar to the JUPITER trial population. The analysis was conducted from the payer perspective and lack of inclusion of indirect costs limit interpretability of results from a societal perspective.

Conclusions:

Treatment with rosuvastatin 20?mg daily, is a cost-effective treatment alternative to no treatment in patients at a higher risk (Framingham risk ≥10%) of CVD.     

Systematic literature review of economics analysis on treatment of mild-to-moderate bleeds with aPCC versus rFVIIa

Abstract

Objective:

Two bypassing agents, activated prothrombin complex concentrates (aPCC) and recombinant factor VIIa (rFVIIa), have shown similar efficacy and safety in the treatment of bleeding episodes in patients with hemophilia and inhibitors as demonstrated through the only two head-to-head clinical trials. Given the economic burden of bypassing treatment, it is crucial to have a valid estimate of cost effectiveness of alternative treatments. The aims of this study were to conduct a systematic review of published pharmacoeconomic literature on the cost-effectiveness of aPCC versus rFVIIa to treat mild-to-moderate bleeds in patients with hemophilia and inhibitors, with a focus on the model assumptions and their impact on results.

Methods:

An English language search was conducted for original economic studies comparing aPCC and rFVIIa published between 1995 and July 2010. Detailed information on sponsorship, study design, assumptions and their impact on results was collected for each study.

Results:

A total of 11 economic studies were included in the review. Nine studies assessed cost per bleeding episode (eight cost-minimization analysis (CMA) and one cost-effectiveness analysis (CEA)). Two studies were from longitudinal perspective. Studies on cost per bleeding episode were evaluated and systematically compared. All studies were from a third-party payer perspective. Most analyses, except one study, used a similar decision-tree model. The assumptions for all CMA studies were obtained from non-comparable single-armed trials or observational data. All studies were sponsored by the two competing manufacturers of rFVIIa (seven studies) and aPCC (two studies). The crucial parameter assumptions on treatment efficacy and dosing drove their reported findings. Eight of these nine studies favored their sponsor’s product.

Conclusion:

With one exception, published economic studies tend to favor their sponsor’s product primarily by assuming a higher efficacy and lower dosing for the sponsored agent, even though the two existing head-to-head clinical studies do not support superior efficacy for either product.     

The effect of functional status impairment on nursing home admission risk among patients with advanced Parkinson’s disease

Abstract

Aims: To estimate the relationship between functional status (FS) impairment and nursing home admission (NHA) risk in Parkinson’s disease (PD) patients, and quantify the effect of advanced PD (APD) treatment on NHA risk relative to standard of care (SoC).

Materials and methods: PD patients were identified in the Medicare Current Beneficiary Survey (MCBS) (1992–2010). A working definition based on the literature and clinical expert input determined APD status. A logit model estimated the relationship between FS impairment and NHA risk. The effect of levodopa-carbidopa intestinal gel (LCIG) on NHA risk relative to SoC was simulated using clinical trial data (control: optimized oral levodopa-carbidopa IR, ClinicalTrials.gov NCT00660387 and NCT0357994).

Results: Non-advanced PD and APD significantly increased NHA risk when controlling for demographics (p?<?0.01). APD status was no longer significant after controlling for FS limitations, implying that FS limitations explain the increased NHA risk in APD patients. Reduced impairment in FS in patients with APD treated with LCIG reduced risk of NHA by 13.5% relative to SoC.

Limitations: This study applies clinical trial results to real-world data. LCIG treatment might have a different effect on NHA risk for the nationally representative population than the effect measured in the trial. Both data sources employ different instruments to measure FS, instrument wording and study follow-up differed, which might bias our estimates. Finally, there lacks consensus on a definition of APD. The prevalence of APD in this study is high, perhaps due to the specific definition used.

Conclusions: Patients with APD experience a higher risk in NHA than those with non-advanced disease. This increased risk in NHA in patients with APD is explained by greater limitations in FS. The relative reduction in risk of NHA for the APD population treated with LCIG is quantitatively similar to doubling Medicaid home care services.     

Economic and quality of life burden of anemia on patients with CKD on dialysis: a systematic review

Abstract

Aims: The overall cost and health-related quality of life (HRQoL) associated with current treatments for chronic kidney disease (CKD)-related anemia are not well characterized. A systematic literature review (SLR) was conducted on the costs and HRQoL associated with current treatments for CKD-related anemia among dialysis-dependent (DD) patients.

Materials and methods: The authors searched the Cochrane Library, MEDLINE, EMBASE, NHS EED, and NHS HTA for English-language publications. Original studies published between January 1, 2000 and March 17, 2017 meeting the following criteria were included: adult population; study focus was CKD-related anemia; included results on patients receiving iron supplementation, red blood cell transfusion, or erythropoiesis stimulating agents (ESAs); reported results on HRQoL and/or costs. Studies which included patients with DD-CKD, did not directly compare different treatments, and had designs relevant to the objective were retained. HRQoL and cost outcomes, including healthcare resource utilization (HRU), were extracted and summarized in a narrative synthesis.

Results: A total of 1,625 publications were retrieved, 15 of which met all inclusion criteria. All identified studies included ESAs as a treatment of interest. Two randomized controlled trials reported that ESA treatment improves HRQoL relative to placebo. Across eight studies comparing HRQoL of patients achieving high vs low hemoglobin (Hb) targets, aiming for higher Hb targets with ESAs generally led to modest HRQoL improvements. Two studies reported that ESA-treated patients had lower costs and HRU compared to untreated patients. One study found that aiming for higher vs lower Hb targets led to reduced HRU, while two other reported that this led to a reduction in cost-effectiveness.

Limitations: Heterogeneity of study designs and outcomes; a meta-analysis could not be performed.

Conclusions: ESA-treated patients undergoing dialysis incurred lower costs, lower HRU, and had better HRQoL relative to ESA-untreated patients. However, treatment to higher Hb targets led to modest HRQoL improvements compared to lower Hb targets.     

Assessing the impact of nocturia on health-related quality-of-life and utility: results of an observational survey in adults

Background and aim: The impact of nocturia (getting up at night to void) on health-related quality-of-life (HRQoL) is often under-estimated. This study investigated the relative burden in terms of HRQoL and utilities of nocturia in a real-world setting.

Methods: Patient data were collected from two surveys: a nocturia-specific, cross-sectional survey of physicians and their patients (DSP), and a general UK population health survey (HSFE). Utilities (EQ-5D-5L), productivity (Work Productivity and Activity Index), and the impact of nocturia symptoms (Nocturia Impact Diary and Overactive Bladder Questionnaires) were assessed against the number of voids. A robust linear regression model with propensity score weights was used to control for confounding factors in estimating utilities.

Results: Physician-recorded data were available from 8,738 patients across the US, Germany, Spain, France, and the UK; of these, 5,335 (61%) included patient-reported outcomes. In total, 6,302 controls were drawn from the two surveys and compared to 1,104 nocturia patients. Deterioration of HRQoL was associated with increasing number of night-time voids (p?p?p?Limitations: The cause of nocturia is multifactorial and the mostly elderly patients may have several concomitant diseases. The authors tried to adjust for the most common ones, but there may be diseases or unknown relationships not included.

Conclusions: Nocturia negatively affected HRQoL and patient utility. A clear effect is seen already at two voids per night. Every effort should, therefore, be made to reduce nocturia below the bother threshold of two voids per night.