VP21 Factors Associated With Recommendations On Drugs For Rare Diseases

2018 ◽  
Vol 34 (S1) ◽  
pp. 164-164
Author(s):  
Fernanda Inagaki Nagase ◽  
Jian Sun ◽  
Tania Stafinski ◽  
Devidas Menon
Keyword(s):  
Quality Of Life ◽  
Rare Diseases ◽  
External Validity ◽  
Treatment Cost ◽  
Orphan Drugs ◽  
Clinical Safety ◽  
Factors Associated ◽  
Drug Review ◽  
Positive Recommendation

Introduction:In Canada, reimbursement recommendations on drugs for common and rare indications (for example, orphan drugs) are made through the pan-Canadian Oncology Drug Review (pCODR) and the Common Drug Review (CDR). However, some stakeholders have called for a separate mechanism for orphan drugs, arguing that existing processes place too much weight on their high price tags. The purpose of this study was to examine factors associated with positive recommendations on drugs for rare diseases.Methods:Information was extracted from CDR and pCODR recommendations on drugs for diseases (prevalence of less than 1 in 2,000) up to April 2018. Univariate and multivariate logistic regression models were applied to explore the influence of the following variables on recommendations: year; prevalence; clinical safety and effectiveness (safety, quality of life, symptoms, surrogate outcomes, and survival); quality of evidence (availability of comparative data, external validity, and bias); unmet need; treatment cost; and incremental cost-effective ratio (ICER). Two-way interactions were also tested.Results:Of 128 recommendations, fifty-four (77 percent) and forty (69 percent) were positive for cancer and non-cancer indications, respectively. For cancer indications, all submissions reporting meaningful improvements in surrogate, quality of life, and survival outcomes were significantly more likely to have a positive recommendation. Submissions showing a lack of external validity were significantly less likely to receive a positive recommendation. For non-cancer indications, more recent submissions and those presenting no safety issues were associated with positive recommendations. Prevalence, treatment cost, and ICER were not determinants of positive or negative recommendations.Conclusions:For both cancer and non-cancer orphan drugs, impact on clinical safety and effectiveness, rather than cost, appears to be a key factor in the formulation of recommendations.

MALIGNANT TUMORS ASSOCIATED WITH METABOLIC SYNDROME: THE EXPRESSION PROFILE OF THE TRANSCRIPTIONAL AND GROWTH FACTORS

2017 ◽  
Vol 63 (6) ◽  
pp. 817-823
Author(s):  
Natalya Yunusova ◽  
Irina Kondakova ◽  
Sergey Afanasev ◽  
Larisa Kolomiets ◽  
Alena Chernyshova
Keyword(s):  
Quality Of Life ◽  
Metabolic Syndrome ◽  
Growth Factors ◽  
Cell Motility ◽  
Expression Profile ◽  
Malignant Tumors ◽  
Molecular Targets ◽  
Factors Associated ◽  
High Incidence

The study of the pathogenetic features of malignant tumors associated with metabolic syndrome (MS) is relevant because of high incidence of these tumors. Investigations of the mechanisms of involvement of MS in the pathogenesis of cancer reasonably supplemented by the study of transcription and growth factors associated with energy imbalance of the cell and involved in proliferation, apoptosis, angiogenesis, cell motility and inflammation. More research is needed to identify the most promising molecular targets for therapy of malignant tumors associated with MS with a view to increasing the survival and quality of life of these patients.

scholarly journals Factors Associated with Health‐Related Quality of Life in Late‐Stage Parkinson's Disease

2021 ◽  
Author(s):  
Kristina Rosqvist ◽  
Per Odin ◽  
Stefan Lorenzl ◽  
Wassilios G. Meissner ◽  
Bastiaan R. Bloem ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Parkinson’S Disease ◽  
Parkinson's Disease ◽  
Late Stage ◽  
Health Related Quality ◽  
Factors Associated ◽  
Related Quality ◽  
Health Related

scholarly journals AB0727 WORK OUTCOMES AND ASSOCIATED FACTORS IN ANKYLOSING SPONDYLITIS PATIENTS IN CHINA

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1658.1-1658
Author(s):  
L. Tu ◽  
Y. Xie ◽  
Q. Lv ◽  
M. Yang ◽  
Z. Liao ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Ankylosing Spondylitis ◽  
Disease Duration ◽  
Productivity Loss ◽  
Work Productivity ◽  
Work Outcomes ◽  
Activity Impairment ◽  
Factors Associated ◽  
The Impact

Background:Poorer work productivity due to pain and functional impairment is commonly seen in ankylosing spondylitis (AS) patients, which may contribute to huge social economic burden. However, data about work outcomes and associated factors in Chinese AS patients were barely reported.Objectives:To assess work outcomes and identify factors associated with poor work productivity in patients with AS in China.Methods:A cross-sectional study was conducted in China. Adult patients (aged ≥ 18 years) fulfilled the 1984 New York modified criteria of AS were enrolled from rheumatology center from Jan 2017 to Aug 2017. All participants completed questionnaires about socio-demographic characteristics, disease characteristics, quality of life and the Work productivity and activity impairment questionnaire in AS (WPAI:SpA) to accesses the impact of chronic health conditions on job performance and productivity. Factors associated with work outcomes were evaluated.Results:A total of 91 patients with AS were included: 87.8% males, 78.02% employed, mean age and disease duration of 30 and 10 years respectively. The mean (SD) activity impairment of all patients was 48.57% (22.02%). For patients with employed work, mean (SD) absenteeism, presenteeism and work productivity loss were 10.22% (19.44%), 43.86% (22.48%) and 47.92% (25.81%) respectively. In multivariable analysis, activity impairment was associated with Bath Ankylosing Spondylitis Functional Index (BASFI) (P<0.01) and Ankylosing Spondylitis Quality of Life (ASQoL) (P<0.01). Absenteeism was associated with disease duration (P=0.03). Presenteeism was associated with disease duration (P=0.04), BASFI (P<0.01) and ASQoL (P<0.01). Work productivity loss was associated with BASFI (P<0.01) and ASQoL (P<0.01).Conclusion:Longer disease duration, reduced physical function and poorer quality of life are associated with reduced work productivity in Chinese AS patients.References:[1]Boonen A, van der Heijde D, Landewe R, Spoorenberg A, Schouten H, Rutten-van Molken M, et al. Work status and productivity costs due to ankylosing spondylitis: comparison of three European countries. Annals of the rheumatic diseases. 2002;61(5):429-37.[2]Martindale J, Shukla R, Goodacre J. The impact of ankylosing spondylitis/axial spondyloarthritis on work productivity. Best Pract Res Clin Rheumatol. 2015;29(3):512-23.[3]Castillo-Ortiz JD, Ramiro S, Landewe R, van der Heijde D, Dougados M, van den Bosch F, et al. Work Outcome in Patients With Ankylosing Spondylitis: Results From a 12-Year Followup of an International Study. Arthritis Care Res (Hoboken). 2016;68(4):544-52.[4]Sag S, Nas K, Sag MS, Tekeoglu I, Kamanli A. Relationship of work disability between the disease activity, depression and quality of life in patients with ankylosing spondylitis. J Back Musculoskelet Rehabil. 2018;31(3):499-505.[5]Goh Y, Kwan YH, Leung YY, Fong W, Cheung PP. A cross-sectional study on factors associated with poor work outcomes in patients with axial spondyloarthritis in Singapore. Int J Rheum Dis. 2019;22(11):2001-8.Disclosure of Interests:None declared

scholarly journals Factors associated with anxiety and quality of life of the Wuhan populace during the COVID‐19 pandemic

2021 ◽  
Author(s):  
Cheng Liu ◽  
Ya‐Chen Lee ◽  
Ying‐Lien Lin ◽  
Shang‐Yu Yang
Keyword(s):  
Quality Of Life ◽  
Factors Associated

scholarly journals Health-Related Quality of Life in Asian Differentiated Thyroid Cancer Survivors

2021 ◽  
Vol 28 ◽  
pp. 107327482110297
Author(s):  
Wing-Lok Chan ◽  
Horace Cheuk-Wai Choi ◽  
Brian Lang ◽  
Kai-Pun Wong ◽  
Kwok-Keung Yuen ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Thyroid Cancer ◽  
Cancer Survivors ◽  
Differentiated Thyroid Cancer ◽  
Summary Score ◽  
Health Related Quality ◽  
Factors Associated ◽  
Related Quality ◽  
Health Related

Background: Health-related quality of life (HRQoL) is important for differentiated thyroid cancer survivors, but data for Asian survivors is lacking. This study aimed to have an overview of, and identify any disease-or treatment-related factors associated with, HRQoL in Asian differentiated thyroid cancer survivors. Patients and Methods: Thyroid cancer survivors were recruited from the thyroid clinics at Queen Mary Hospital, Hong Kong from February 2016 to December 2016. All adult differentiated thyroid cancer patients with stable disease more than or equal to 1 year received a survey on HRQoL using the European Organization for the Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) and Thyroid cancer specific quality of life (THYCA-QoL) questionnaire. Clinical information was collected retrospectively from the computerized clinical management system. To identify factors associated with poor HRQoL, univariable and stepwise multivariable regression analysis were performed. Results: A total of 613 survivors completed the questionnaires (response rate: 82.1%; female: 80.1%; median survivorship: 7.4 years (range: 1.0-48.2 years)). The QLQ-C30 summary score mean was 84.4 (standard deviation (SD): 12.7) while the THYCA-QoL summary score mean was 39.9 (SD: 9.7). The 2 highest symptom subscales were fatigue (mean: 26.4, SD: 20.6) and insomnia (mean: 26.2, SD: 27.6). Factors associated with worse HRQoL included serum thyrotropin (TSH) greater than 1.0 mIU/L, unemployment, and concomitant psychiatric disorders. Concomitant psychiatric illness (n = 40/613, 6.5%) also showed significant association with most of the symptom and functional subscales. Conclusions: Fatigue and insomnia were the 2 most common symptoms experienced by our differentiated thyroid cancer survivors. Long-term survivorship care with monitoring serum TSH level, supporting return-to-work and screening for concomitant psychiatric disorders should be offered.

scholarly journals Quality of Life in European Older Adults of SHARE Wave 7: Comparing the Old and the Oldest-Old

2021 ◽  
Vol 10 (13) ◽  
pp. 2850
Author(s):  
Amparo Oliver ◽  
Trinidad Sentandreu-Mañó ◽  
José M. Tomás ◽  
Irene Fernández ◽  
Patricia Sancho
Keyword(s):  
Quality Of Life ◽  
Older Adults ◽  
Measurement Invariance ◽  
Factor Structure ◽  
External Validity ◽  
Oldest Old ◽  
Age Groups ◽  
Depression Scale ◽  
Scalar Invariant

CASP-12 (Control, Autonomy, Self-realization, and Pleasure scale) is one of the most common internationally used measures for quality of life in older adults, although its structure is not clearly established. Current research aims to test the factor structure of the CASP-12, so as to provide evidence on reliability and external validity, and to test for measurement invariance across age groups. Data from 61,355 Europeans (≥60 years old) from the Survey of Health, Ageing and Retirement in Europe wave 7 were used. CASP-12, EURO-D (European depression scale), self-perceived health, and life satisfaction measurements were included. Reliability and validity coefficients, competing confirmatory factor models, and standard measurement invariance routine were estimated. A second-order factor model with the original factor structure was retained. The scale showed adequate reliability coefficients except for the autonomy dimension. The correlation coefficients for external validity were all statistically significant. Finally, CASP-12 is scalar invariant across age. We conclude that the best-fitting factor structure retained allows using CASP-12 either by factors, or as an overall score, depending on the research interests. Findings related to CASP-12 measurement invariance encourage its use in the oldest-old too. When comparing the dimensions across age groups, as people age, autonomy slightly increases and the rest of the dimensions decline.

Clinician modifiable factors associated with better quality of life in children with acquired brain injury undergoing rehabilitation

Brain Injury ◽  
2018 ◽  
Vol 32 (4) ◽  
pp. 423-430
Author(s):  
Tim Connell ◽  
Julia Paterson ◽  
Rachel M. Roberts ◽  
Parimala Raghavendra ◽  
Michael Sawyer ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Brain Injury ◽  
Acquired Brain Injury ◽  
Factors Associated ◽  
Modifiable Factors
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