Mapping of the Insomnia Severity Index and other sleep measures to EuroQol EQ-5D health state utilities

<p>Abstract</p> <p>Background</p> <p>This study sought to map the Insomnia Severity Index (ISI) and symptom variables onto the EQ-5D.</p> <p>Methods</p> <p>A cross-sectional survey was conducted among adult US residents with self-reported sleep problems. Respondents provided demographic, comorbidity, and sleep-related information and had completed the ISI and the EQ-5D profile. Respondents were classified into ISI categories indicating no, threshold, moderate, or severe insomnia. Generalized linear models (GLM) were used to map the ISI's 7 items (Model I), summary scores (Model II), clinical categories (Model III), and insomnia symptoms (Model IV), onto the EQ-5D. We used 50% of the sample for estimation and 50% for prediction. Prediction accuracy was assessed by mean squared errors (MSEs) and mean absolute errors (MAEs).</p> <p>Results</p> <p>Mean (standard deviation) sleep duration for respondents (N = 2,842) was 7.8 (1.9) hours, and mean ISI score was 14.1 (4.8). Mean predicted EQ-5D utility was 0.765 (0.08) from Models I-III, which overlapped with observed utilities 0.765 (0.18). Predicted utility using insomnia symptoms was higher (0.771(0.07)). Based on Model I, predicted utilities increased linearly with improving ISI (0.493 if ISI = 28 vs. 1.00 if ISI = 0, p < 0.01). From Model II, each unit decrease in ISI summary score was associated with a 0.022 (p < 0.001) increase in utility. Predicted utilities were 0.868, 0.809, 0.722, and 0.579, respectively, for the 4 clinical categories, suggesting that lower utility was related to greater insomnia severity. The symptom model (Model IV) indicated a concave sleep-duration function of the EQ-5D; thus, utilities diminished after an optimal amount of sleep. The MSEs/MAEs were substantially lower when predicting EQ-5D > 0.40, and results were comparable in all models.</p> <p>Conclusions</p> <p>Findings suggest that mapping relationships between the EQ-5D and insomnia measures could be established. These relationships may be used to estimate insomnia-related treatment effects on health state utilities.</p

Assessing the value of orphan drugs using conventional cost-effectiveness analysis:Is it fit for purpose?

Conventional cost-effectiveness analysis-i.e., assessing pharmaceuticals through a cost per quality-adjusted life year (QALY) framework-originated from a societal commitment to maximize population health given limited resources. This "extra-welfarist" approach has produced pricing and reimbursement systems that are not well- aligned with the unique considerations of orphan drugs. This framework has been slow to evolve along with our increased understanding of the impact of rare diseases, which in turn has complicated the assessment of orphan drugs meant to treat rare diseases. Herein, we (i) discuss the limitations of conventional cost-effectiveness analysis as applied to assessing access to, as well as the pricing and reimbursement of, orphan drugs, (ii) critically appraise alternative and supplemental approaches, and (iii) offer insights on plausible steps forward

Patterns of Clinical Management of Atopic Dermatitis in Infants and Toddlers: A Survey of Three Physician Specialties in the United States

ObjectiveTo describe atopic dermatitis (AD) management patterns in children ≤36 months old as reported by pediatricians, dermatologists, and allergists in the US.Study designA nationally-representative survey was administered to pediatricians (n = 101), dermatologists (n = 26), and allergists (n = 26). Main outcomes included referrals to health care professionals, suggested/ordered laboratory tests, management approach (dietary, pharmacologic, or combination of both) by age, AD location, and severity.ResultsSignificant differences were observed in referrals to healthcare professionals (P < .001). Pediatricians more frequently referred to dermatologists than allergists in mild (52.4% vs 32.0%) and moderate/severe (60.6% vs 38.1%) cases. Dermatologists referred to allergists less frequently for mild (9.1%) than moderate/severe (40.7%) AD cases. Pediatricians (59%), allergists (61.5%), and dermatologists (26.9%) reported treating at least some of their patients with AD with dietary management (infant formula change) alone (with or without emollients). Soy-based formulas were often used. For mild AD, the most commonly reported first-line pharmacologic treatments included topical emollients, topical corticosteroids, and barrier repair topical therapy/medical devices. Over 80% of physicians used a dietary and pharmacologic combination approach. Dermatologists were most likely to manage AD symptoms with a pharmacologic-only approach. AD lesion location influenced pharmacologic treatment in >80% of physicians.ConclusionsSignificant and distinct differences in AD treatment approach exist among physicians surveyed. Most pediatricians and allergists use formula change as a management strategy in some patients, whereas dermatologists favor a pharmacologic approach. This diversity may result from inadequate evidence for a standard approach. Consistent methods for managing AD are needed

Economic value of atopic dermatitis prevention via partially-hydrolyzed whey-based infant formula (PHF-W) use in high-risk, non-exclusively breastfed, Indonesian urban infants: results of a cost-effectiveness model

Background:  Early nutritional intervention with partially-hydrolyzed whey-based formula (PHF-W) instead of standard cow’s milk formula (CMF) has been found to reduce the risk of atopic dermatitis (AD) development in non-exclusively breastfed infants with familial heredity of AD.Objective:  To estimate the 6-year economic impact of this nutritional intervention in non-exclusively breastfed Indonesian urban infants with family history of AD.Methods:  A mathematical model simulated AD incidence and burden of using PHF-W vs. CMF in the target population from birth to age 6. The model integrated literature, current cost and market catalogues, and expert clinician opinion. Modeled outcomes included AD risk, time spent post-AD diagnosis, days without flare, quality-adjusted life-years, and costs.Results: Using PHF-W instead of CMF resulted in an estimated absolute 14% (95% CI: 4%, 23%) AD risk reduction, a 0.69 year (95% CI: 0.26, 1.13) per-child reduction in time spent post-AD diagnosis, a 38 (95% CI: 12, 67) increase in days without AD flare, and a 0.046 gain in quality-adjusted life-years. The AD-related 6-year cost estimates when feeding high-risk urban infants with PHF-W were Indonesian Rupiah (IDR) 8,695,057 (95% CI: IDR 4,519,447, IDR13,995,605) and IDR13,139,569 (95% CI: IDR 7,098,794, IDR 19,216,068) per child, respectively, resulting in a net per-child difference of IDR 4,444,512 (95% CI: IDR1,893,080, IDR 8,557,946) favoring PHF-W.Conclusion: PHF-W for the first 17 weeks of non-exclusively breastfed Indonesian urban infants with a hereditary risk of AD demonstrated a reduction in AD incidence, increased days without flare, and increased quality-adjusted life-years and net cost reductions

Eliciting health state utilities for Dupuytren's contracture using a discrete choice experiment

Background and purpose An internet-based discrete choice experiment (DCE) was conducted to elicit preferences for a wide range of Dupuytren’s contracture (DC)-related health states. An algorithm was subsequently developed to convert these preferences into health state utilities that can be used to assess DC’s impact on quality of life and the value of its treatments. Methods Health state preferences for varying levels of DC hand severity were elicited via an internet survey from a sample of the UK adult population. Severity levels were deined using a combination of contractures (0, 45, or 90 degrees) in 8 proximal interphalangeal and metacarpophalangeal joints of the index, middle, ring, and little ingers. Right-handed, left-handed, and ambidextrous respondents indicated which hand was preferable in each of the 10 randomly-selected hand-pairings comparing different DC severity levels. For consistency across comparisons, anatomically precise digital hand drawings were used. To anchor preferences onto the traditional 0–1 utility scale used in health economic evaluations, unaffected hands were assigned a utility of 1.0 whereas the utility for a maximally affected hand (i.e., all 8 joints set at 90 degrees of contracture) was derived by asking respondents to indicate what combination of attributes and levels of the EQ-5D-5L proile most accurately relects the impact of living with such hand. Conditional logistic models were used to estimate indirect utilities, then rescaled to the anchor points on the EQ-5D-5L. Results Estimated utilities based on the responses of 1,745 qualiied respondents were 0.49, 0.57, and 0.63 for completely affected dominant hands, non-dominant hands, or ambidextrous hands, respectively. Utility for a dominant hand with 90-degree contracture in t h e metacarpophalangeal joints of the ring and little ingers was estimated to be 0.89. Separately, reducing the contracture of metacarpophalangeal joint for a little inger from 50 to 12 degrees would improve utility by 0.02. Interpretation DC is associated with substantial utility decrements. The algorithms presented herein provide a robust and lexible framework to assess utility for varying degrees of DC severity

Understanding the Disease Course and Therapeutic Benefit of Tafamidis Across Real-World Studies of Hereditary Transthyretin Amyloidosis with Polyneuropathy: A Proof of Concept for Integrative Data Analytic Approaches

<p></p><p><b>Article full text</b></p> <p><br> </p> <p>The full text of this article can be found here<b>.</b> <a href="https://link.springer.com/article/10.1007/s40120-018-0096-x">https://link.springer.com/article/10.1007/s40120-018-0096-x</a></p><p></p><p></p><p> </p><p><br></p> <p><b>Provide enhanced content for this article</b></p> <p><br></p> <p>If you are an author of this publication and would like to provide additional enhanced content for your article then please contact <a href="http://www.medengine.com/Redeem/”mailto:[email protected]”"><b>[email protected]</b></a>.</p> <p><br></p> <p>The journal offers a range of additional features designed to increase visibility and readership. All features will be thoroughly peer reviewed to ensure the content is of the highest scientific standard and all features are marked as ‘peer reviewed’ to ensure readers are aware that the content has been reviewed to the same level as the articles they are being presented alongside. Moreover, all sponsorship and disclosure information is included to provide complete transparency and adherence to good publication practices. This ensures that however the content is reached the reader has a full understanding of its origin. No fees are charged for hosting additional open access content.</p> <p><br></p> <p>Other enhanced features include, but are not limited to:</p> <p><br></p> <p>• Slide decks</p> <p>• Videos and animations</p> <p>• Audio abstracts</p> <p>• Audio slides</p><br><p></p

Cost-Effectiveness Model Of Long-Acting Risperidone in Schizophrenia in the US

Background: Schizophrenia is a devastating and costly illness that affects 1% of the population in the US. Effective pharmacological therapies are available but suboptimal patient adherence to either acute or long-term therapeutic regimens reduces their effectiveness. The availability of a long-acting injection (LAI) formulation of risperidone may increase adherence and improve clinical and economic outcomes for people with schizophrenia. Objective: To assess the cost effectiveness of risperidone LAI compared with oral risperidone, oral olanzapine and haloperidol decanoate LAI over a 1-year time period in outpatients with schizophrenia who had previously suffered a relapse requiring hospitalisation. Perspective: US healthcare system. Methods: Published medical literature, unpublished data from clinical trials and a consumer health database, and a clinical expert panel were used to populate a decision-analysis model comparing the four treatment alternatives. The model captured: rates of patient compliance; rates, frequency and duration of relapse; incidence of adverse events (bodyweight gain and extrapyramidal effects); and healthcare resource utilisation and associated costs. Primary outcomes were: the proportion of patients with relapse; the frequency of relapse per patient; the number of relapse days per patient; and total direct medical cost per patient per year. Costs are in year 2002 US dollars. Results: Based on model projections, the proportions of patients experiencing a relapse requiring hospitalisation after 1 year of treatment were 66% for haloperidol decanoate LAI, 41% for oral risperidone and oral olanzapine and 26% for risperidone LAI, while the proportion of patients with a relapse not requiring hospitalisation were 60%, 37%, 37% and 24%, respectively. The mean number of days of relapse requiring hospitalisation per patient per year was 28 for haloperidol decanoate LAI, 18 for oral risperidone and oral olanzapine and 11 for risperidone LAI, while the mean number of days of relapse not requiring hospitalisation was 8, 5, 5 and 3, respectively. This would translate into direct medical cost savings with risperidone LAI compared with oral risperidone, oral olanzapine and haloperidol decanoate LAI of $US397,$US1742, and $US8328, respectively. These findings were supported by sensitivity analyses. Conclusion: The use of risperidone LAI for treatment of outpatients with schizophrenia is predicted in this model to result in better clinical outcomes and lower total healthcare costs over 1 year than its comparators, oral risperidone, oral olanzapine and haloperidol decanoate LAI. Risperidone LAI may therefore be a cost saving therapeutic option for outpatients with schizophrenia in the US healthcare setting.Cost-effectiveness, Haloperidol, Haloperidol-decanoate, Olanzapine, Risperidone, Schizophrenia

Understanding what matters to metastatic castration‐resistant prostate cancer (mCRPC) patients when considering treatment options: A US patient preference survey

Abstract Background Understanding how patients perceive the efficacy, safety, and administrative burden of treatments for metastatic castration‐resistant prostate cancer (mCRPC) can facilitate shared‐decision making for optimal management. This study sought to elicit patient preferences for mCRPC treatments in the US. Methods We conducted a cross‐sectional survey using the discrete‐choice experiment method. Participants were asked to state their choices over successive sets of treatment alternatives, defined by varying levels of treatment attributes: overall survival (OS), months until patients develop a fracture or bone metastasis, likelihood of requiring radiation to control bone pain, fatigue, nausea, and administration (i.e., oral/IV injection/IV infusion). Using mixed logit models, we determined the value (i.e., preference weights) that respondents placed on each attribute. Relative attribute importance (RAI) and marginal rates of substitution (MRS) were calculated to understand patients' willingness to make tradeoffs among different attributes. Results The final data set numbered 160 participants, with a mean age of 71.6 years old and a mean of 8.96 years since prostate cancer diagnosis. Participants' treatment preferences were as follows: OS (RAI: 31%), bone pain control (23%), nausea (16%), delaying fracture or bone metastasis (15%), fatigue (11%), and administration (3%). The MRS demonstrated that respondents were willing to trade 1.9 months of OS to eliminate moderate nausea and 3.3 months of OS for a reduction in fatigue from severe to mild. Conclusions Improving OS is the highest priority for patients with mCRPC, but they are willing to trade some survival to reduce the risk of requiring radiation to control bone pain, delay a fracture or bone metastasis, and experience less severe nausea and fatigue