Genomic testing transforms the diagnosis and management of rare conditions. However, uncertainty exists on how to best measure genomic outcomes for informing healthcare priorities. Using the ...HTA-preferred method should be the starting point to improve the evidence-base. This study explores the responsiveness of SF-6D, EQ-5D-5L and AQoL-8D following genomic testing across childhood and adult-onset genetic conditions.
Self-reported patient-reported outcomes (PRO) were obtained from: primary caregivers of children with suspected neurodevelopmental disorders (NDs) or genetic kidney diseases (GKDs) (carers' own PRO), adults with suspected GKDs using SF-12v2; adults with suspected complex neurological disorders (CNDs) using EQ-5D-5L; and adults with dilated cardiomyopathy (DCM) using AQol-8D. Responsiveness was assessed using the standardised response mean effect-size based on diagnostic (having a confirmed genomic diagnosis), personal (usefulness of genomic information to individuals or families), and clinical (clinical usefulness of genomic information) utility anchors.
In total, 254 people completed PRO measures before genomic testing and after receiving results. For diagnostic utility, a nearly moderate positive effect size was identified by the AQoL-8D in adult DCM patients. Declines in physical health domains masked any improvements in mental or psychosocial domains in parents of children affected by NDs and adult CNDs and DCM patients with confirmed diagnosis. However, the magnitude of the changes was small and we did not find statistically significant evidence of these changes. No other responsiveness evidence related to diagnostic, clinical, and personal utility of genomic testing was identified.
Generic PRO measures may lack responsiveness to the diagnostic, clinical and personal outcomes of genomics, but further research is needed to establish their measurement properties and relevant evaluative space in the context of rare conditions. Expected declines in the physical health of people experiencing rare conditions may further challenge the conventional application of quality of life assessments.
Purpose
EQ-5D and PROMIS-29 are both concise, generic measures of patient-reported outcomes accompanied by preference weights that allow the estimation of quality-adjusted life years (QALYs). Both ...instruments are candidates for use in economic evaluation. However, they have different features in terms of the domains selected to measure respondents’ self-perceived health and the characteristics of (and methods used to obtain) the preference weights. It is important to understand the relationship between the instruments and the implications of choosing either for the evidence used in decision-making. This literature review aimed to synthesise existing evidence on the relationship between PROMIS-29 (and measures based on it, such as PROMIS-29+2) and EQ-5D (both EQ-5D-3L and EQ-5D-5L).
Methods
A literature review was conducted in PubMed and Web of Science to identify studies investigating the relationship between PROMIS-29 and EQ-5D-based instruments.
Results
The literature search identified 95 unique studies, of which nine studies met the inclusion criteria, i.e. compared both instruments. Six studies examined the relationship between PROMIS-29 and EQ-5D-5L. Three main types of relationship have been examined in the nine studies: (a) comparing PROMIS-29 and EQ-5D as descriptive systems; (b) mapping PROMIS-29 domains to EQ-5D utilities; and (c) comparing and transforming PROMIS-29 utilities to EQ-5D utilities.
Conclusion
This review has highlighted the lack of evidence regarding the relationship between PROMIS-29 and EQ-5D. The impact of choosing either instrument on the evidence used in cost-effectiveness analysis is currently unclear. Further research is needed to understand the relationship between the two instruments.
The health shocks literature typically does not take into account the temporal patterns of loss since the time of the shock. This limits understanding of the long‐run impact of health shocks and the ...capacity of individuals to cope over time. This study estimates the dynamic effects of a noncommunicable disease shock on the economic well‐being of working‐age individuals in China up to 6 years after onset. We find that after a period of temporal loss, individuals and their families can insure consumption against the average noncommunicable disease shock over the long‐run. We observe significant heterogeneity according to the persistence of the disease, value of household wealth, and health insurance status. Individuals with consistent onset, with below median wealth, and without health insurance are least equipped to smooth consumption over the long‐term.
The rise of non-communicable diseases has placed enormous stress on health systems leading to calls for improved prevention. This article examines the association of risk factors and non-communicable ...disease diagnosis in China using longitudinal data which enables us to control for important simultaneity bias. Using three waves of the China Family Panel Studies (CFPS) survey (2010–2014) and a dynamic model conditional on not having an NCD in the first period, we find positive association of being obese, using solid cooking fuels, history of frequent drinking, and household consumption expenditure during the preceding period on non-communicable disease onset. We find significant heterogeneity in risks across the population suggesting that a targeted policy response is required to reduce the burden of non-communicable disease in China.
•We use a dynamic model to estimate risk factors of NCD diagnosis in China.•We find positive associations of obesity, solid fuel use, consumption and drinking history.•We find significant heterogeneity in risk factors across the population.•Targeted public health response is recommended to reduce the burden of NCDs in China.
The burden of disease attributable to obesity is rapidly increasing and becoming a public health challenge globally. Using a nationally representative sample in Australia, this study aims to examine ...the association of obesity with healthcare service use and work productivity across outcome distributions. We used Household, Income and Labour Dynamics Australia (HILDA) Wave 17 (2017-2018), including 11,211 participants aged between 20 and 65 years. Two-part models using multivariable logistic regressions and quantile regressions were employed to understand variations in the association between obesity levels and the outcomes. The prevalence of overweight and obesity was 35.0% and 27.6%, respectively. After adjusting for socio-demographic factors, low socioeconomic status was associated with a higher probability of overweight and obesity (Obese III: OR = 3.79; 95% CI 2.53-5.68) while high education group was associated with a lower likelihood of being high level of obesity (Obese III OR = 0.42, 95% CI 0.29-0.59). Higher levels of obesity were associated with higher probability of health service use (GP visit Obese, III: OR = 1.42 95% CI 1.04-1.93,) and work productivity loss (number of paid sick leave days, Obese III: OR = 2.40 95% CI 1.94-2.96), compared with normal weight. The impacts of obesity on health service use and work productivity were larger for those with higher percentiles compared to lower percentiles. Overweight and obesity are associated with greater healthcare utilisation, and loss in work productivity in Australia. Australia's healthcare system should prioritise interventions to prevent overweight and obesity to reduce the cost on individuals and improve labour market outcomes.
According to the reversal hypothesis, as a country's economic and social development progresses, the burden of NCDs and risk factors shifts from rich to poor. The aim of this research is to examine ...the reversal hypothesis in the Chinese setting.
Using data from the China Health and Retirement Longitudinal Study (CHARLS) in 2015, we explored whether the reversal hypothesis applies at the subnational level. Participants aged 45 years and older in 2015 were included. We examined five risk factors (smoking, heavy drinking, physical inactivity, overweight, and obesity) and three objectively measured NCDs (diabetes, hypertension, dyslipidemia). Binary logistic regressions were performed to examine outcomes across people of differing SES in provincial level, in urban and rural areas, and across generations.
Nationally, SES is positively associated with heavy drinking, obesity, diabetes and dyslipidemia, whereas it is negatively associated with physical inactivity. The association between SES and smoking and hypertension was not statistically significant. Except in the cases of diabetes and dyslipidemia, we found that risk factors of all kinds were more concentrated among richer people in rural than in urban areas. Across provinces with increasing GDP per capita, a downward trend in risk factors among those with high SES compared to those with low SES could be interpreted, while the opposite trend could be interpreted with respect to the metabolic syndrome conditions. Obesity and overweight exhibited slight downward trends (in line with those for risk factors) and upward trends (in line with those for metabolic syndrome conditions), respectively.
We conclude that China is at a relatively early stage of ‘reversal’, visible with respect to risk factors. If these patterns persist over time, the trend will likely feed through to metabolic disorders which will increasingly become diseases of the poor.
•China is at a relatively early stage of ‘reversal’. If it persists, the metabolic disorders will become diseases of the poor.
Multimorbidity (the presence of two or more non-communicable diseases) is a major growing challenge for many low-income and middle-income countries (LMICs). Yet, its effects on health care costs and ...financial burden for patients have not been adequately studied. This study investigates the effect of multimorbidity across the different percentiles of healthcare utilisation and out-of-pocket expenditure (OOPE).
We conducted a secondary data analysis of the 2014/2015 Indonesian Family Life Survey (IFLS-5), which included 13,798 respondents aged ≥40 years. Poisson regression was used to assess the association between sociodemographic characteristics and the total number of non-communicable diseases (NCDs), while multivariate logistic regression and quantile regression analysis was used to estimate the associations between multimorbidity, health service use and OOPE.
Overall, 20.8% of total participants had two or more NCDs in 2014/2015. The number of NCDs was associated with higher healthcare utilisation (coefficient 0.11, 95% CI 0.07-0.14 for outpatient care and coefficient 0.09 (95% CI 0.02-0.16 for inpatient care) and higher four-weekly OOPE (coefficient 27.0, 95% CI 11.4-42.7). The quantile regression results indicated that the marginal effect of having three or more NCDs on the absolute amount of four-weekly OOPE was smaller for the lower percentiles (at the 25th percentile, coefficient 1.0, 95% CI 0.5-1.5) but more pronounced for the higher percentile of out-of-pocket spending distribution (at the 90th percentile, coefficient 31.0, 95% CI 15.9-46.2).
Multimorbidity is positively correlated with health service utilisation and OOPE and has a significant effect, especially among those in the upper tail of the utilisation/costs distribution. Health financing strategies are urgently required to meet the needs of patients with multimorbidity, particularly for vulnerable groups that have a higher level of health care utilisation.
The co-occurrence of mental and physical chronic conditions is a growing concern and a largely unaddressed challenge in low-and-middle-income countries. This study aimed to investigate the ...independent and multiplicative effects of depression and physical chronic conditions on health-related quality of life (HRQoL) in China, and how it varies by age and gender. We used two waves of the China Health and Retirement Longitudinal Study (2011, 2015), including 9227 participants aged ≥ 45 years, 12 physical chronic conditions and depressive symptoms. We used mixed-effects linear regression to assess the effects of depression and physical multimorbidity on HRQoL, which was measured using a proxy measure of Physical Component Scores (PCS) and Mental Component Scores (MCS) of the matched SF-36 measure. We found that each increased number of physical chronic conditions, and the presence of depression were independently associated with lower proxy PCS and MCS scores. There were multiplicative effects of depression and physical chronic conditions on PCS (- 0.83 points, 95% CI - 1.06, - 0.60) and MCS scores (- 0.50 points, 95% CI - 0.73, - 0.27). The results showed that HRQoL decreased markedly with multimorbidity and was exacerbated by the presence of co-existing physical and mental chronic conditions.
The co-occurrence of mental and physical chronic conditions (mental-physical multimorbidity) is a growing and largely unaddressed challenge for health systems and wider economies in low-and ...middle-income countries. This study investigated the independent and combined (additive or synergistic) effects of mental and physical chronic conditions on disability, work productivity, and social participation in China.
Panel data study design utilised two waves of the China Health and Retirement Longitudinal Study (2011, 2015), including 5616 participants aged ≥45 years, 12 physical chronic conditions and depression. We used a panel data approach of random-effects regression models to assess the relationships between mental-physical multimorbidity and outcomes.
After adjusting for socio-economic and demographic factors, an increased number of physical chronic conditions was independently associated with a higher likelihood of disability (Adjusted odds ratio (AOR) = 1.39; 95% CI: 1.33, 1.45), early retirement (AOR = 1.37 1.26, 1.49) and increased sick leave days (1.25 days 1.16, 1.35). Depression was independently associated with disability (AOR = 3.78 3.30, 4.34), increased sick leave days (2.18 days 1.72, 2.77) and a lower likelihood of social participation (AOR = 0.57 0.47, 0.70), but not with early retirement (AOR = 1.24 0.97, 1.58). There were small and statistically insignificant interactions between physical chronic conditions and mental health on disability, work productivity and social participation, suggesting an additive effect of mental-physical multimorbidity on productivity loss.
Mental-physical multimorbidity poses substantial negative health and economic effects on individuals, health systems, and societies. More research that addresses the challenges of mental-physical multimorbidity is needed to inform the development of interventions that can be applied to the workplace and the wider community in China.
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