Humanistic Burden Research Articles

PMU123 DO SYSTEMATIC REVIEWS SYSTEMATICALLY ASSESS RISK OF BIAS IN REAL-WORLD EVIDENCE STUDIES?

Formal risk-of-bias (RoB) assessment is essential in any rigorous systematic literature review (SLR). The Cochrane RoB tool is widely accepted for assessing RoB in SLRs of randomized controlled trials. However, there seems to be no obvious consensus on approaches for assessing RoB in SLRs of observational studies. To explore this issue, we conducted an SLR on the use of RoB tools in SLRs of disease burden. MEDLINE and EMBASE were searched to identify SLRs (published from 2017 onwards) of the epidemiological, economic, or humanistic burden in ischaemic heart disease, stroke, or chronic obstructive pulmonary disease—three diseases causing highest mortality, according to the World Health Organization. We extracted and narratively synthesised information on whether or how these SLRs assessed RoB. The database searches identified 742 records, among which 20 SLRs (13 represented only in conference abstracts) met our inclusion criteria. RoB was reported for six studies. Three SLRs used validated tools (GRADE, CHEERS, the Newcastle-Ottawa scale) and one used a custom checklist. The authors of another SLR stated they assessed study quality with the STROBE reporting checklist (which is not a RoB assessment tool) and that low-quality studies were excluded following RoB assessment. However, the exclusion criteria used, and the number of studies excluded were not reported. Only one conference abstract reported RoB assessment, and without naming the tool used. Most of the identified SLRs evaluating disease burden did not report any RoB assessment, so potentially limiting interpretation of their findings. This may be particularly problematic where such SLRs are used to inform healthcare decision-making. There was considerable variation in the choice of RoB instrument across studies that assessed this characteristic. Given the ever-increasing importance of insights from real-world data, there needs to be greater standardization of how RoB is assessed in SLRs of disease burden.

PCN331 HUMANISTIC BURDEN OF NEWLY DIAGNOSED VERSUS RELAPSED/REFRACTORY ACUTE MYELOID LEUKEMIA

Acute Myeloid Leukemia (AML) is an aggressive leukemia associated with poor overall survival. Patients with AML experience significant disease symptoms and treatment-related consequences that diminish health-related quality of life (HRQoL) and psychosocial well-being. The objective of this targeted literature review was to assess humanistic burden evidence in patients with newly diagnosed and relapsed/refractory (R/R) AML. Medline, Embase, and the Cochrane Library databases were searched via the Ovid platform for all relevant humanistic burden studies between 2013 and 2018. The search strategy combined terms including AML and disease burden, quality of life (QOL), HRQoL, patient preference among others. 11 unique humanistic burden studies including randomized clinical trials (n=3), prospective studies (n=5) and patient surveys (n=3) from North America and Europe met eligibility criteria. Studies included adult patients with AML (mean age: 52 to 73.2 years). The European Organisation for the Research and Treatment of Cancer core 30-item questionnaire (EORTC QLQ-C30) was reported in ∼50% of all QoL studies (n=5/11). Fatigue and pain were among the most common and severe symptoms reported across all studies. Patients with secondary or R/R AML reported worse HRQoL than patients with denovo AML. Achievement of remission was associated with markedly improved HRQoL after one year with similar trends for younger versus older patients (<60 vs ≥60 years). Younger and older patients (<60 vs ≥60 years) one year from diagnosis reported no differences in global health or fatigue. Patients obtaining complete response perceived improvement in global health, physical and emotional functioning, and symptoms burden. AML is a life-threatening condition. The disease and treatment-related toxicities cause distressing symptoms that worsen overall health status, significantly impair daily functioning, role-fulfillment and emotional health. Treatments with the potential to induce long-term remission and complete responses are needed to improve outcomes for this deadly disease.

PRO150 A SYSTEMATIC LITERATURE REVIEW OF HEALTH STATE UTILITY VALUATIONS IN HAEMOPHILIA

Health state utility values (HSUVs) are an important input to economic evaluations. This systematic literature review (SLR) evaluated HSUVs for people with haemophilia (PWH) to facilitate the economic evaluation of novel treatments being developed, including gene therapies. Building on a previous review (Grosse et al. 2015), we conducted a SLR to March 2019 through a search of electronic databases, including MEDLINE®, Web of Science, Cochrane Library, and the School of Health and Related Research Health Utilities Database (SCHARRHUD). Major clinical, patient, and pharmacoeconomic conferences in 2016-2019 were also queried. Studies were double screened by independent reviewers, after which data extraction was performed. Of 1,511 titles and abstracts screened, 20 studies and 12 conference abstracts were included. The studies identified applied a mix of direct and indirect health utility elicitation techniques. Two studies applied direct time trade-off (TTO) methodology and the remaining 30 studies adopted indirect valuation methodologies. HSUVs were found to decrease with increasing disease severity. For example, in Hoxer et al. (2018), mean (standard deviation) HSUV were 0.80 (0.21), 0.73 (0.22) and 0.67 (0.25) in people with mild, moderate, and severe haemophilia, respectively. Utility values were also found to vary by severity of musculoskeletal damage, bleed frequency, inhibitors, haemophilia subtype, treatment regimen and adherence. Interestingly, HSUVs derived from valuations from the general public were found to be valued lower than those derived from PWH for similar health states. For example, in Carlsson et al. (2017), general population participants consistently rated significantly lower HSUVs for haemophilia disease states compared to PWH (range: 0.54-0.60 vs. 0.67-0.73). This SLR shows significant impact of haemophilia on health utilities among PWH. The substantial humanistic burden experienced by PWH highlights unmet needs remaining in haemophilia. Our review findings also suggest potential disease state adaptation among PWH, which warrants further research using robust patient preference studies.

PDB99 UNDERSTANDING THE HUMANISTIC BURDEN AND PATIENT IMPACT IN NON-ALCOHOLIC STEATOHEPATITIS (NASH)

NASH is a chronic and progressive form of non-alcoholic fatty liver disease (NAFLD) leading to cirrhosis and hepatocellular carcinoma (HCC). NAFLD is associated with significant impairment in health-related quality of life (HRQoL), but the humanistic burden of NASH is still unclear. This review aimed to understand the evidence defining the impact of NASH on patients’ QoL. A structured literature review across multiple databases was conducted to investigate the QoL, patient reported outcomes (PRO) and productivity in NASH patients. Searches were restricted to publications reporting on NASH, English language and articles published between January 2009 and May 2019. A grey literature search was also conducted. In total, only 13 publications were found reporting on the humanistic burden of NASH - the majority of studies demonstrated an impairment in HRQoL with poor physical and mental health across multiple PRO measures. No publications reported on the HRQoL in patients with early stages of NASH; however, four publications reported that NASH patients overall experience a range of symptoms, including pain, fatigue, anxiety and depression, and two publications reported a loss in productivity. A recent study showed that NASH patients with cirrhosis (F4) had lower physical health-related scores than with bridging fibrosis (F3) in all but one domain of CLDQ-NASH, SF-36, and EQ-5D HRQoL instruments (p≤0.01). Cirrhosis also poses a substantial emotional and financial burden to patients and caregivers, but not this has not been investigated specifically in NASH. Overall, HRQoL is significantly impaired in NASH patients; however, there is a gap in the literature for HRQoL in NASH with early stages of fibrosis. Additionally, there is a lack of data on the wider humanistic burden of NASH, including loss of productivity, activities of daily living and caregiver burden, highlighting that further research is needed.

PCN468 SYSTEMATIC REVIEW OF UTILITY ESTIMATES IN TRIPLE-NEGATIVE BREAST CANCER PATIENTS

This study was conducted to gather data from the literature on health state utility values (HSUVs) relevant to triple-negative breast cancer (TNBC) patients. Two systematic literature reviews (SLRs) were completed in April 2019, one to identify studies measuring health-related quality of life (HRQoL) outcomes and the other to identify economic studies in TNBC patients. The SLRs covered EMBASE, MEDLINE, EconLit, and CENTRAL as well as conference proceedings and health technology assessment (HTA) databases. Of the 4 bibliographic databases (2007-2019), 7 congresses (2017-2019), and 11 HTA databases (2007-2019) reviewed, 4 studies that reported HRQoL outcomes and 9 studies that reported economic outcomes were identified. None of the included HRQoL studies reported HSUVs in TNBC patients. Of the 9 economic studies identified, 4 (a value of information analysis, two cost-utility analyses, and an HTA submission) included HSUVs. In all 4 studies, the utility values were derived from published literature on the general breast cancer (BC) population. The utilities values reported were associated with a variety of health states. In the HTA submission, the mean utility values derived using a standard gamble technique for progression-free survival, response, stable disease, and progressive disease associated with metastatic BC were 0.73, 0.81, 0.65, and 0.45, respectively. The other economic evaluations used utility estimates for relapse (0.73), disease-free/well (0.78-1.0), and breast cancer in general (0.77). The methods for selection and synthesis of these HSUVs were not well-reported in the economic analyses. An SLR employing a highly sensitive search strategy for humanistic burden did not identify any HSUVs in TNBC patients. Existing economic models for TNBC employ utilities for different health states in the general breast cancer population; further research is necessary to derive HSUVs specific to TNBC that can be used in economic evaluations.

PUK33 PATIENT INSIGHTS FOR COMPLEMENT 3 GLOMERULOPATHY (C3G) USING SOCIAL MEDIA LISTENING

Published evidence on the humanistic burden of C3G, an ultra-rare renal condition, are scarce. This social media study was conducted to understand the patient journey, quality of life (QoL) and unmet needs from the patients’ perspective. Social media data from September 2015–September 2018 were captured based on a predefined search strategy using MeSH terms, through social media data aggregator. C3G specific social media posts in English language from the US and the UK, on open channels such as twitter, forums, blogs and newswires were analyzed, post anonymization. A total of 791 relevant posts were analyzed. The focus of the discussions, mainly driven by patients and caregivers, were on seeking or giving advice in regards to diagnosis and treatments, obtaining information on C3G and looking for support groups. The major reasons that led patients to physician visits were edema, fatigue, presence of blood in the urine and hypertension. Diagnosis was reported mainly in patients aged ≤20 years, through biopsy. Nearly 50% of mentions involved initial treatments with anti-hypertensives followed by steroids and/or immunosuppressants. Dialysis and transplant were mentioned as treatment options, adding significant emotional and physical burden. In addition, symptoms like feeling very weak and tired, swellings and always getting sick affected the patients’ day to day activities. C3G patients and their caregivers felt uncertainty about transplant success and anxiety around their future. The absence of proper information in the public domain and counseling support emerged as unmet needs which caused more emotional stress among patients and caregivers. The symptoms and emotions associated with the disease reduced the QoL of both patients and caregivers. Furthermore, these patients’ insights shed light on the disease burden from a patients’ perspective and can be taken into account for future drug development programs.

PRO144 HEALTH-RELATED QUALITY OF LIFE AND PATIENT REPORTED OUTCOMES MEASURES IN IDIOPATHIC MULTICENTRIC CASTLEMAN'S DISEASE: A SYSTEMATIC REVIEW

Idiopathic Multicentric Castleman’s disease (iMCD) is a rare disease that causes distressing and debilitating symptoms which have a negative impact on patients’ lives and may lead to life-threatening complications. This systematic review aimed to identify and quantify the humanistic burden of disease in adults with MCD who are not infected with HIV and HHV-8. Medline and Embase databases were searched in December 2018. There were no limits on publication language, but only studies from 2008 were included. Two reviewers screened articles against pre-defined criteria, a third reviewer resolved discrepancies. One analyst extracted data and another quality-controlled it. The DistillerSR Systematic Review Software was used in the studies selection process. Five studies (four in the same cohort, all with treatment with siltuximab) were identified. Patient reported outcomes and health-related quality of life (HRQoL) were measured using the Multicentric Castleman Disease Symptom Scale (MCD-SS) (n=4), the SF-36 (n=3), and the FACIT-Fatigue questionnaire (n=2). MCD-SS outcomes reported most frequently were very mild and mild: shortness of breath (40%), lack of energy (33%), feeling weak (33%), tiredness (32%) and pain (32%). The FACIT questionnaire showed that at baseline, >50% patients reported lower fatigue scores than the normal population. In a comparative study, both siltuximab and placebo treated patients had comparable mean total FACIT score at baseline (32.4 vs 31), while siltuximab-treated patients reported much higher mean values than the patients on placebo during follow-up (38.6 vs 26.9, respectively). Treatment with siltuximab more often resulted in an SF-36 score equal to, or greater than the population norm at final visit compared to placebo. Generally, siltuximab was associated with better fatigue and depressive symptoms scores. Data is limited, but MCD is associated with poor patient reported outcomes. Available data showed that siltuximab was associated with improvements in fatigue, functioning and well-being compared with placebo.

Disease burden of Crigler-Najjar syndrome: Systematic review and future perspectives.

Crigler-Najjar syndrome (CNS) results from biallelic mutations of UGT1A1 causing partial or total loss of uridine 5'-diphosphate glucuronyltransferase activity leading to unconjugated hyperbilirubinemia and its attendant risk for irreversible neurological injury (kernicterus). CNS is exceedingly rare and has been only partially characterized through relatively small studies, each comprising between two and 57 patients. A systematic literature review was conducted to consolidate data on the patient, caregiver, and societal burden of CNS. Twenty-eight articles on clinical aspects of CNS were identified, but no published data on its humanistic or economic burden were found. In patients with complete UGT1A1 deficiency (type 1 CNS [CNS-I]), unconjugated bilirubin levels increase 3-6mg/dL/day during the newborn period and reach neurologically dangerous levels between 5 and 14days of age. Phototherapy is the mainstay of treatment but poses significant challenges to patients and their families. Despite consistent phototherapy, patients with CNS-I have worsening hyperbilirubinemia with advancing age. Liver transplantation is the only definitive therapy for CNS-I and is increasingly associated with excellent long-term survival but also incurs high costs, medical and surgical morbidities, and risks of immunosuppression. Crigler-Najjar syndrome is associated with a substantial burden, even with existing standards of care. The development of novel disease-modifying therapies has the potential to reduce disease burden and improve the lives of CNS patients and their families.

Humanistic and economic burden among caregivers of patients with cancer in Japan

Aims: As the number of cancer patients increases in Japan, and people are living longer with cancer, the need for caregivers of cancer patients is expected to increase substantially. This study intended to reveal the humanistic and economic burden among caregivers of cancer patients, and to compare it with the burden among caregivers of patients with other conditions (other caregivers) and non-caregivers.Materials and methods: This cross-sectional analysis used data from the Japan National Health and Wellness Survey 2017. Outcome measures included the Short Form 12-item Health Survey for health-related quality of life (HRQoL), EuroQol 5-dimension scale (EQ-5D) for health states utilities, Work Productivity and Activity Impairment questionnaire for the impact of health on productivity and activity, and indirect costs. Multivariate analysis was used to compare across groups, with adjustment for potential confounding effects.Results: A total of 251 caregivers of cancer patients, 1,543 other caregivers, and 27,300 non-caregivers were identified. Caregivers of cancer patients (average 48.0 years old) tended to be younger than non-caregivers (51.5) and other caregivers (54.4) and had the highest education level (57.8% completed university education). Fewer non-caregivers had stress-related comorbidities than caregivers. Non-caregivers had significantly higher EQ-5D index scores than caregivers (average 0.81 vs. 0.73 vs. 0.74). Caregivers of cancer patients had significantly lower mental component summary scores than non-caregivers (40.18 vs. 46.70), and the difference indicated a clinically meaningful decrease in HRQoL. Caregivers of cancer patients had significantly higher presenteeism (37.31% vs. 20.43%), total work productivity impairment (38.85% vs. 21.98%), and activity impairment (40.94% vs. 25.78%) than non-caregivers. Additionally, caregivers of cancer patients had significantly higher total indirect costs (36.34% vs. 20.03% of average annual income).Conclusions: These results have implications for future healthcare planning, suggesting the importance of healthcare systems in Japan to consider the substantial burden borne by caregivers of cancer patients.

1171P - The humanistic burden reported by patients diagnosed with recurrent/metastatic squamous cell carcinoma of the head and neck (R/M SCCHN) in Europe

BackgroundR/M SCCHN is associated with poor prognosis and low survival rate. Further, there is a lack of evidence of how the disease impacts patients’ quality of life (QoL) and ability to perform activities of daily living (ADL). This study examines patients’ experience of living with R/M SCCHN. MethodsA cross-sectional study was conducted in the EU5 from Jan to May 2019. Medical oncologists recruited patients to complete a survey, including the Functional Assessment of Cancer Therapy – Head and Neck Cancer (FACT-H&N), a 39-item instrument evaluating patients’ QoL (score range 0-148; higher scores indicating better QoL), the European Quality of Life – 5 Dimension Questionnaire (EQ-5D-3L; utility scores range -0.59-1, VAS scores 0-100; with higher scores indicating better health status) and questions on daily life impact using a 7-point scale (7= extremely high impact). Physicians also completed a case report form (CRF) for each patient. This analysis is based on interim data. Results191 patients completed the survey. Median age was 66 years (45% < 65, 55% ≥ 65), 77% were male, the majority (82%) had an ECOG score 0-1 and 39% required caregiver support for daily needs (mean 32.7hours per week). At data capture, 9% of patients were in paid work, 21% on sick leave and 48% retired. Most patients (93%) received at least one therapy line following R/M. Patients reported diminished health status, with a mean EQ-5D utility score of 0.62 and a mean VAS score of 58. Mean FACT-H&N score was low at 74.0. Almost half of patients reported high impact (score 5-7) on ADL and family/social life (43% and 46%, respectively). For patients with a lower FACT-H&N score (lowest score quartile [26–60.2]), 75% reported high impact on ADL, versus 15% patients in the highest FACT-H&N score quartile (87.5–120). 81% patients in the lowest FACT-H&N quartile reported high family/social life impact, versus 15% patients in the highest quartile. ConclusionsIn addition to considerable impact on health status and QoL, patients report high impact on ability to perform ADL, with a high caregiving burden. There is a clear relationship between reduced QoL and restricted ADL and social life, highlighting the need to consider novel approaches to improve QoL in R/M SCCHN. Legal entity responsible for the studyBristol-Myers Squibb. FundingBristol-Myers Squibb. DisclosureP. Singh: Full / Part-time employment: Bristol-Myers Squibb. B. Bennett: Full / Part-time employment: Bristol-Myers Squibb. M.C. Contente: Full / Part-time employment: Bristol-Myers Squibb. All other authors have declared no conflicts of interest.

P1.16-28 The Humanistic Burden of ALK+ NSCLC: Findings from the ALKConnect Patient Insight Network and Research Platform

P1.16-28 The Humanistic Burden of ALK+ NSCLC: Findings from the ALKConnect Patient Insight Network and Research Platform

The clinical and economic impact of three-monthly long-acting formulation of paliperidone palmitate versus the one-monthly formulation in the treatment of schizophrenia in France: A cost-utility study

ObjectivesSchizophrenia entails a considerable humanistic and economic burden. Improved treatment continuity to antipsychotic therapy is paramount to reduce the risk of relapse. The novel three-monthly paliperidone palmitate treatment (PP3M) offers the longest dosing interval currently available in France. This study assesses its cost-effectiveness, versus the currently available one-monthly long-acting treatment (PP1M) in French schizophrenic patients. MethodsA Markov model with monthly cycles was developed and adapted. It encompassed [a] administration of PP3M or PP1M in first-line, [b] a period where the patient does not receive any active treatment, and [c] a follow-up treatment line consisting of a treatment mix reflecting French clinical practice. Relapse rates in first-line were based on a pivotal non-inferiority head-to-head trial, and treatment discontinuation rates were based on French real-world data. Accounting for differences in drug exposure, time-dependent monthly relapse rates were applied following discontinuation to first line. The impact of a less frequent injection schedule for PP3M in QoL was accounted for through the application of a utility differential. The collective perspective was adopted throughout a 5-year time horizon. Four percent discount rates were applied on costs and outcomes. ResultsPP3M was dominant when compared to PP1M, featuring an incremental QALY of 0.123 and a cost saving effect (−669€) resulting from reduced therapy costs (drug acquisition, administration and monitoring) and relapse-related costs. Sensitivity analysis supported the robustness of the results. ConclusionWith slightly better QALY outcomes and a cost-saving effect when compared to PP1M, introducing PP3M is an improvement to the current treatment in France.

Management Of Glaucoma In Developing Countries: Challenges And Opportunities For Improvement.

Glaucoma is the leading cause of blindness in the developed and developing world. Not only is the clinical impact of this disease considerable, but associated economic and humanistic burdens – affecting patients, caregivers, and society – are substantial. Since glaucoma is an age-related disorder and populations in many developing countries are aging at a faster pace than in the developed world, increasing attention is being focused on ways to ameliorate the burdens of illness. In this paper, we examine the burdens of glaucoma with particular focus on developing countries, discuss some of the challenges that exist in delivering optimal glaucoma management within budget constraints, and bring into perspective how we could improve current healthcare systems, leverage technology, and strike an appropriate balance between cost and quality of care, thereby offering considerations to payors and policymakers in these countries that may result in longer-term cost savings, while concurrently striving to achieve the WHO Vision on the prevention of blindness and visual impairment.

Burden Of Treatment Among Patients Undergoing Intravitreal Injections For Diabetic Macular Oedema In Australia.

AimThe incidence and prevalence of diabetes mellitus (DM) in Australia is increasing. Thus, it is essential that practitioners appreciate the impending effect that increasing incidence of diabetes has on patients and the wider community. Accordingly, this study examines the humanistic burden of intravitreal injections for the treatment of diabetic macular oedema (DMO) among several health variables.MethodsSurvey data from a representative sample of Australian adults undergoing treatment for DMO were examined. Respondents participated via an online survey recruited by means of a national online consumer panel and the New South Wales and Victorian Diabetes Foundations. The online survey included questions relating to the humanistic burden of disease, such as the emotional and physical impact of intravitreal injection therapy; the practical impacts of injection therapy; and to identify potential improvements to treatment regimens.ResultsSixty-five participants took part in the online survey. Of these, 49% had their most recent injection <1 month prior to completing the survey. The mean age was 52.5 years, with the majority of patients in full-time work. A substantial proportion of participants had several comorbidities, with a significantly high Charlson comorbidity index of 2.7. Participants reported the main burden of DMO care was the direct cost of medical treatment and the time burden demanded upon their carers. Results suggest that the overall burden is significant for those with diabetes and increases as additional complications of diabetes occur.ConclusionThese results suggest that treatment strategies for DMO should consider clinical, humanistic and economic burden and patients should be educated on the roles of complications in disease outcomes. Less frequent treatment regimens could also reduce the economic burden and assist in decreasing the effect on health care resources, relevant to the escalation in the prevalence of diabetes.

An online survey of burden of illness in families with mucopolysaccharidosis type II children in the United States

Mucopolysaccharidosis type II (MPS II or Hunter's syndrome) is a rare X-linked recessive genetic disease caused by lack of the enzyme iduronate sulfatase, which leads to an accumulation of glycosaminoglycans (GAGs) throughout the body. As an X-linked disorder, MPS II occurs almost exclusively in males, although rarely females have been diagnosed. The disease has both severe and attenuated presentations, but there is a wide spectrum of clinical severity [1]. Severity of disease is usually based on the presence or absence of progressive neurological involvement and behavioral problems [[2], [3], [4]]. In severe MPS II children, coarse facial features, short stature, skeletal deformities, joint stiffness, cardiopulmonary disfunction, and neurocognitive deficits are typical. Disease onset occurs between two and four years of age, with somatic signs such as chronic ear infections, facial dysmorphism, enlarged tongue and tonsils, enlarged liver/spleen, and joint stiffness [2,[5], [6], [7]]. Cognitive development and adaptive ability begin to decline around 4 years of age, accompanied by severe speech and language delay [5,8]. Hearing loss begins around the age of 2 years, which affects speech and language development [2,9]. In addition, abnormal behavior such as hyperactivity, frustration, and impulsivity starts around 4 years of age [2,8]. Current treatment for MPS II in the United States (US) consists of enzyme replacement therapy (ERT), idursulfase, given intravenously (IV) usually on a weekly basis. The results of a phase II/III placebo-controlled clinical study of ERT with idursulfase demonstrated that weekly infusions produced significant improvements based on the six-minute walk test and pulmonary function as measured by the percentage of predicted forced vital capacity (FVC) [10]. However, these trials enrolled children with higher functioning who were likely not neuronopathic. Therefore, while ERT was shown to improve endurance and pulmonary function in these trials, the long-term effect of ERT on neurocognitive function specifically in neuronopathic children is not known, especially since IV ERT does not cross the blood-brain barrier. As severe MPS II is a chronic and debilitating condition, families with MPS II children experience significant economic, social, and humanistic burdens. Healthcare utilization, educational needs for the child, and caregiver burden in the US have not been well documented in the literature. A few non-US studies have assessed the burden of a variety of MPS types in terms of healthcare use, cost of care, caregiver time, and impact on work and family life. In France, a study assessed healthcare utilization in 51 MPS type II patients, primarily (69.2%) those with severe subtype [11]. Among respondents, 51% of the patients had been admitted to the hospital in the last 12 months for reasons associated with their disease, with a mean length of stay of 10.4 days (range 1–120 days), and 25.5% had been to the emergency room a mean number of 2 times, primarily for respiratory symptoms. In a 12-month period, 49% had seen an ear, nose and throat specialist, 30% had seen an orthopedic surgeon, and 29% had seen an ophthalmologist at least once. Eighty-four percent had seen a physical therapist (for an average of 7.4 times/year) and 40% had seen a speech therapist (for an average of 5.3 times/year). More than two-thirds (72.5%) reported they used social services assistance for help with home care aides and financial assistance. A study conducted by Pentek et al. in seven European countries assessed disease burden in 120 patients (74 children and 46 adults) and 66 caregivers among the MPS community, across many disease types, as part of a project referred to as BURQOL-RD (www.burqol-rd.org) [12]. Most caregivers (65 of 66) responding to the survey were the parent of the patient. These caregivers reported spending similar amounts of time per week caring for adult patients with MPS as the time reported caring for children with MPS (52 vs 51 h respectively), indicating that challenges to perform activities of daily living likely continue throughout life. The purpose of this study was to gather, via an online survey, real-world data from families in the US caring for sons with severe MPS II to quantify children's healthcare resource use, to describe educational needs, and to assess parents' work productivity and caregiving time.

Humanistic and Economic Impact of Moderate to Severe Plaque Psoriasis in Brazil.

IntroductionPsoriasis is an immune-mediated, chronic, inflammatory disease, which has a substantial humanistic and economic burden. This study aimed to assess the impact of this disease on health-related quality of life (HRQoL), work productivity, and direct and indirect costs from a societal perspective among Brazilian patients.MethodsThis is a cross-sectional, observational, multicenter study, enrolling patients with moderate to severe plaque psoriasis according to physician evaluation. Data collection was performed from December 2015 to November 2016 through face-to-face interviews using a structured questionnaire and five standardized patient-reported outcomes instruments. Direct costs were estimated by multiplying the amount of resources used (12-month recall period) by the corresponding unit cost. Indirect costs were grouped in two time horizons: annual costs (income reduction and absenteeism) and lifetime costs (demission and early retirement).ResultsA total of 188 patients with moderate to severe plaque psoriasis were included, with mean age of 48.0 (SD 13.1). “Anxiety and depression” and “pain and discomfort” were the most impaired dimensions, according to the EuroQol Five-Dimension-Three-Level (EQ-5D-3L). The highest effect was found for “symptoms and feelings” [mean (SD) 2.4 (1.7)] Dermatology Life Quality Index (DLQI) subscale. Psoriatic arthritis (PsA) presence and biologic-naïve status were associated with worse HRQoL. Presenteeism was more frequent than absenteeism, according to the Work Productivity and Activity Impairment questionnaire-General Health (WPAI-GH) [17.4% vs. 6.3%], while physical demands and time management were the most affected Work Limitations Questionnaire (WLQ) subscales [means (SD) 23.5 (28.5) and 17.7 (24.9), respectively]. The estimated annual cost per patient was USD 4034. Direct medical costs accounted for 87.7% of this estimate, direct non-medical costs for 2.4%, and indirect costs for 9.9%.ConclusionsResults evidenced that moderate to severe plaque psoriasis imposes substantial costs to society. Our data showed that this disease negatively affects both work productivity and HRQoL of Brazilian patients. Subgroups with PsA and biologic-naïve patients presented lower HRQoL, showing the impact of this comorbidity and the relevance of biologics in psoriasis treatment.FundingNovartis Biociências S.A.Electronic supplementary materialThe online version of this article (10.1007/s12325-019-01049-7) contains supplementary material, which is available to authorized users.

The humanistic and economic burden of treatment-resistant depression in Europe: a cross-sectional study

BackgroundA patient is considered to suffer from treatment resistant depression (TRD) when consecutive treatment with two products of different pharmacological classes, used for a sufficient length of time at an adequate dose, fail to induce a clinically meaningful effect (inadequate response). The primary aim of the current study was to examine the humanistic and economic burden of TRD in five European countries, France, Germany, Italy, Spain and the United Kingdom, by comparing with non-treatment resistant depression (nTRD) and general population respondents.MethodsThe sample for this retrospective observational study was taken from the 2017 National Health and Wellness Survey conducted in five European countries. Demographic and patient characteristics were examined for TRD patients compared to respondents with nTRD and to the general population using chi-square tests or one-way analysis of variance for categorical or continuous variables, respectively. Generalized linear models were performed to examine group differences after adjusting these estimates for confounders.ResultsA total 52,060 survey respondents were examined, of which 2686 and 622 were considered to have non-treatment resistant and treatment-resistant depression, respectively. Relative to the general population, nTRD and TRD respondents reported significant decrements in health-related quality of life, including lower adjusted mental (− 12.1 vs. -18.1) and physical (− 2.5 vs. -5.4) component scores of the SF-12v2 and increased adjusted relative risk for work (2.2 vs. 2.7) and activity (1.9 vs. 2.5) impairment (all p < 0.001). Additionally, healthcare resource utilization was significantly higher for TRD patients more so than nTRD, compared to the general population, especially for healthcare professional visits (odds ratio nTRD = 5.4; TRD = 15.9, p < 0.001).ConclusionsIn conclusion, TRD patients had significantly lower quality of life, greater work productivity and activity impairment, and increased healthcare resource utilization as compared with nTRD and general population. The study findings suggest an unmet need exists among TRD patients in Europe.

Clinical, humanistic, and economic burden of osteoarthritis among noninstitutionalized adults in the United States

To estimate the burden of osteoarthritis (OA) among noninstitutionalized adults (≥18 years of age) in the US. Weighted nationally representative data from the 2015 Medical Expenditure Panel Survey were used to estimate OA prevalence in noninstitutionalized adults and compare adults with OA to those without OA for clinical (pain interference with activities [PIA], functional limitations), humanistic (health-related quality-of-life [HRQoL]) and economic outcomes (healthcare costs, wage loss). Productivity/wage loss was estimated among employed working-age adults (18-64 years). Multivariable regression analyses examined the associations between OA and outcomes. In 2015, 10.5% (25.6 million) of noninstitutionalized US adults reported having any OA. Regression analyses indicated that adults with OA were significantly more likely than those without OA to report moderate (adjusted odds ratios [AOR] 1.99; 95% confidence interval [CI] 1.65-2.40] or severe PIA (AOR 2.59; 95% CI 2.21-3.04), any functional limitation (AOR 2.51; 95% CI 2.21-2.85), and poorer HRQoL on the SF-12 version 2 Physical Component Summary score (adjusted beta [standard error]-3.88 [0.357]; P<0.001). Adjusted incremental annual total healthcare costs and lost wages among adults with OA relative to those without OA were $1778 and $189 per person, respectively, resulting in estimated national excess costs of $45 billion and $1.7 billion, respectively. OA affects approximately 10% of noninstitutionalized adults in the US, resulting in substantial clinical, humanistic, and economic burdens.

070 Burden of migraine is australia: a systematic literature review

IntroductionMigraine is a disabling neurological disease characterised by recurrent attacks of moderate to severe headaches. This systematic literature review (SLR) aimed to investigate the clinical, humanistic, and economic burden of chronic migraine (CM), episodic migraine (EM), and of current preventive migraine treatments in Australia.MethodsThe methodology of this SLR was aligned with the National Institute for Health and Care Excellence (NICE) guidelines. An electronic database search was conducted in Embase, MEDLINE and the Cochrane Library, with a time frame of 2008 to 2018.ResultsIn total, 1,122 records were identified and 168 of these were included for data extraction. The prevalence of migraine in Australia is estimated at 18.9%. Of those, 44% of people with EM and 86% of people with CM reported moderate-to-severe disability. Over one-third (36%) of people with EM and nearly two-thirds (64%) of people with CM reported visiting a healthcare provider in the previous three months. No data relating to the economic burden of migraine were returned by the searches. In people with EM and CM, anti-calcitonin gene-related peptide (anti-CGRP) preventive treatments for migraine safely, effectively and significantly reduced the mean number of monthly migraine and/or headache days from baseline compared with placebo.ConclusionsMigraine is associated with a substantial burden, and people living with migraine feel the impact in their day-to-day lives. Anti-CGRPs are a promising class of preventive treatments for all people with migraine. Longer-term studies are needed to determine if the positive effects of anti-CGRPs are sustained over greater time periods.

Conceptual Model to Illustrate the Symptom Experience and Humanistic Burden Associated With Atopic Dermatitis in Adults and Adolescents.

Atopic dermatitis (AD) is a pruritic or painful dermatologic disease characterized by xerosis and eczema lesions. The symptoms/signs of AD can significantly impact patients' health-related quality of life (HRQoL). This study aimed to qualitatively explore the adult and adolescent experience of AD. A targeted literature review and qualitative concept elicitation interviews with clinicians (n = 5), adult AD patients (n = 28), and adolescent AD patients (n = 20) were conducted to elicit AD signs/symptoms and HRQoL impacts experienced. Verbatim transcripts were analyzed using thematic analysis. Twenty-nine symptoms/signs of AD were reported, including pruritus, pain, erythema, and xerosis. Atopic dermatitis symptoms/signs were reported to substantially impact HRQoL. Scratching was reported to influence the experience of symptoms and HRQoL impacts. Four proximal impacts (including discomfort and sleep disturbance) were reported. Ten domains of distal impact were reported, including impacts on psychological and social functioning and activities of daily living. A conceptual model was developed to summarize these findings. This study highlights the range of symptoms and HRQoL impacts experienced by adults and adolescents with AD. To our knowledge, this study was first to explore the lived experience of AD in both adult and adolescent patients, providing valuable insight into the relatively unexplored adolescent experience of AD.