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PROMS as predictors of healthcare use in multiple sclerosis

  • November 2017
  • Conference: HTAi Rome 2017
  • At: Rome
Authors:
Michela Tinelli at The London School of Economics and Political Science
Michela Tinelli
Olina Efthymiadou at The London School of Economics and Political Science
Olina Efthymiadou
Jean Mossman at The London School of Economics and Political Science
Jean Mossman
Panos Kanavos at The London School of Economics and Political Science
Panos Kanavos
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Title: PROMS as predictors of healthcare use in multiple sclerosis
Authors: M Tinelli, O Efthymiadou, J Mossman, P Kanavos.
Topic: Stakeholder involvement, patient involvement
Summary:
Patient-relevant-outcome-measures (PROMS) are primarily used to measure individual disability and
quality of life in people with MS. However, their relationship with f health care resources utilisation
(as well as productivity loss) is less clear. The IMPRESS data showed that PROMs may be useful tools
to predict the economic impact of MS on healthcare providers and wider society.
Full abstract:
Introduction: MS is a highly disabling chronic disorder affecting young adults with long term
economic consequences on society that escalate as MS disability increases.1-3 In the long-term,
progression of MS results in increased level of disability and most patients will eventually experience
some degree of functional impairment of the nervous system that impacts on mobility as well as
sensory and = coordination issues, bladder and sexual functioning, and mood and cogniton.4-6 This is
usually accompanied by a deterioration of their quality of life. Patient relevant outcome measures
(PROMS) are largely used to measure individual disability, and quality of life in MS.7-8 International
evidence from the IMPRESS9 was used to quantify the relationship between health care resources
utilisation and disability, quality of life in individuals with MS.
Methods: Multivariable logistic regression was performed in order to identify patient-related
variables reporting disability (Barthel) and utility (EQ-5D) that predict use of healthcare services
(visits to GP, specialists, nurses, hospitalisation and DMT treatment) and work limitation within the
participants of the IMPRESS.
Results: Reponses were collected from 1152 individuals across 21 countries of which 74.3% (856)
were useful for analysis. Preliminary findings indicated that for the pooled data sets both EQ-5D and
Barthel scores were predictors of healthcare resource use, both before and after adjustment for
sociodemographic status and diagnosis, across different categories (p<0.05), except for nurse visits
(Barthel only; p<0.09). Overall the association between PROM data and use of healthcare resources
appeared to be stronger with EQ-5D compared to Barthel score. EQ-5D appeared to also predict the
impact of MS on loss of productivity (in terms of work limitation; p<0.05).
Conclusion: PROMs can be used to predict the economic consequences of MS on healthcare
providers and society, but more research is needed to confirm the robustness of the evidence and its
validity across individual healthcare system settings.
References:
1. Ernstsson O, Gyllensten H, Alexanderson K, Tinghög P, Friberg E, Norlund A (2016) Cost of
Illness of Multiple Sclerosis - A Systematic Review. PLoS ONE 11(7): e0159129.
doi:10.1371/journal.pone.0159129.
2. Coleman CI, Sidovar MF, Roberts MS, Kohn C. Impact of mobility impairment on indirect costs
and health-related quality of life in multiple sclerosis. PloS one. 2013;8(1):e54756. Epub
2013/01/29. doi: 10.1371/journal.pone.0054756. pmid:23355896
3. Naci H, Fleurence R, Birt J, Duhig A. Economic burden of multiple sclerosis: a systematic review
of the literature. PharmacoEconomics. 2010;28(5):363–79. Epub 2010/04/21. doi:
10.2165/11532230-000000000-00000. pmid:20402540
4. Naci H, Fleurence R, Birt J, . The impact of increasing neurological disability of multiple
sclerosis on health utilities: A systematic review of the literature. J Med Econ 2010; 13: 78–89
5. Karatepe GA et al. Quality of life in patients with multiple sclerosis: the impact of depression,
fatigue, and disability. Int J Rehabil Res. 2011 Dec;34(4):290-8.
6. Ahmad H et al. The impact of multiple sclerosis severity on health state utility values: Evidence
from Australia. Mult Scler. 2016 Oct 3. pii: 1352458516672014. [Epub ahead of print]
7. Khurana V et al. Patient Reported Outcomes in Multiple Sclerosis: A Review of Literature.
Neurology April 5, 2016 vol. 86 no. 16 Supplement P2.134
8. Cohen JA et al. Disability outcome measures in multiple sclerosis clinical trials: current status
and future prospects. Lancet Neurol. 2012 May;11(5):467-76.
9. Kanavos P, Tinelli M, Efthymiadou O, Visintin E, Grimaccia F, Mossman J. Towards better
outcomes in multiple sclerosis by addressing policy change. The International MultiPlE
Sclerosis Study (IMPrESS). The London School of Economics and Political Science March 2016.
ResearchGate has not been able to resolve any citations for this publication.
Towards better outcomes in multiple sclerosis by addressing policy change The International MultiPlE Sclerosis Study (IMPrESS)
Technical Report
Full-text available
  • Mar 2016
Executive summary Multiple sclerosis (MS) is a serious and disabling condition, which affects people in early adulthood. It is the second most common cause of disability among central nervous system diseases and epidemiological data suggests that between 3 and 7 people per 100,000 population are newly diagnosed with MS each year. Neurological damage leads to problems with bodily functions, including impairment of muscle coordination, vision and sensation, and also results in cognitive and psychological dysfunction, sleep disorders, fatigue and pain. MS is associated with a high cost of illness, both in terms of direct and indirect costs. Given that the onset of MS is in early adult life (average onset at 29 years of age) lasting over an individual’s lifetime, there are huge costs relating to productivity losses. There is also a significant impact on the families of people with MS (PWMS). Based on WHO data at global level in 2012, MS was estimated to cause 1,165,000 disability-adjusted life years (DALYs), of which 387,000 were attributable to the European region and 282,000 to the Americas. It is becoming common practice to use magnetic resonance imaging (MRI) to diagnose and monitor disease activity in patients on DMTs. Current recommendations of ‘treat to target’ mean treating until ‘no evidence of disease activity’ is reached, including no relapses, no increase in disability and no new or active (enhancing) lesions on their MRI scans. Meeting this objective implies regular monitoring of not only clinical relapse and disability progression, but also MRI activity. However, regular use of MRI to monitor disease activity and the effects of treatment is still not universal, though it is increasingly used as an outcome measure for clinical trials. A number of senior clinicians from different countries confirmed that MRI is routinely available in their practice, but this does not necessarily mean that it is common practice across clinical settings. There are therapies, which modify the course of the illness, known as disease modifying treatments (DMTs); that is, their effect is to slow disability and disease progression. However, considerable neurological damage (some of which may be permanent) can occur if PWMS are not given the appropriate treatment early enough. There is increasing focus on finding ways to identify disease progression as early as possible so that treatments can be adapted to prevent or delay further neurological damage. There is an urgent need to achieve better outcomes for PWMS and the evidence suggests that this is possible if policy makers address the following issues. 1- Diagnosis, treatment and management goals should be set to provide the best health outcome for every person with MS. ● Early diagnosis and treatment are needed to secure the best outcomes for PWMS, to prevent or avoid irreversible health deterioration and disability progression. ● Diagnostic imaging is an effective way of capturing disease activity early and should be routinely available in the management of PWMS. ● Newer and more effective DMTs should be used both earlier and routinely while real world data on their long-term impact is collected. ● Health systems should involve more actively PWMS in decisions about their disease management. ● MS specialists should be involved in drawing up a treatment plan for each PWMS. 2- (Further) robust evidence should be generated and used in order to make appropriate decisions about care management in MS strategies. ● Robust epidemiological, clinical and disease management data are needed internationally to inform better decisions for priority setting in MS. ● National registries should be in place and the data from them should be routinely used; the production of such evidence should be adequately resourced. ● Data should be updated on an ongoing basis and should incorporate dimensions for which little validated information currently exists; for example, registry data should be amended to allow for collection and use of standardised information on MRI use across country settings. ● Updated and internationally comparable evidence on the use of diagnostic imaging as a means of capturing disease activity should be generated as a priority. ● Health gain and quality of life data should take account of dimensions that patients say have a significant impact on their daily lives; many of these items are not captured by generic tools often used by HTA agencies internationally (e.g. EuroQol 5 dimensions 5 levels (EQ-5D-5L) questionnaire). ● Greater consistency is needed in collecting economic data and evaluating the economic impact of MS to ensure that comparisons across settings can be made. 3- Improve the responsiveness of health care systems to new evidence on MS. ● Healthcare systems need to be able to respond dynamically as new evidence emerges on the diagnosis, treatment and psychosocial support of MS patients as well as the long-term economic evidence on the impact of MS. ● Updated guidance on MS management should be developed as new evidence becomes available on the use of imaging and disease modifying therapies and should be implemented promptly. ● Incentives should support improvement in clinical practice and the incorporation of new evidence on MS management in health care decision-making, especially if such evidence is linked to improvements in quality of care and health outcomes.
Cost of Illness of Multiple Sclerosis - A Systematic Review
Article
Full-text available
Background: Cost-of-illness (COI) studies of Multiple Sclerosis (MS) are vital components for describing the economic burden of MS, and are frequently used in model studies of interventions of MS. We conducted a systematic review of studies estimating the COI of MS, to compare costs between studies and examine cost drivers, emphasizing generalizability and methodological choices. Material and method: A literature search on studies published in English on COI of MS was performed in PubMed for the period January 1969 to January 2014, resulting in 1,326 publications. A mapping of studies using a bottom-up approach or top-down approach, respectively, was conducted for the 48 studies assessed as relevant. In a second analysis, the cost estimates were compared between the 29 studies that used a societal perspective on costs, human capital approach for indirect costs, presenting number of patients included, time-period studied, and year of price level used. Results: The mapping showed that bottom-up studies and prevalence approaches were most common. The cost ratios between different severity levels within studies were relatively stable, to the ratio of 1 to 2 to 3 for disability level categories. Drugs were the main cost drivers for MS-patients with low disease severity, representing 29% to 82% of all costs in this patient group, while the main cost components for groups with more advanced MS symptoms were production losses due to MS and informal care, together representing 17% to 67% of costs in those groups. Conclusion: The bottom-up method and prevalence approach dominated in studies of COI of MS. Our findings show that there are difficulties in comparing absolute costs across studies, nevertheless, the relative costs expressed as cost ratios, comparing different severity levels, showed higher resemblance. Costs of drugs were main cost drivers for less severe MS and informal care and production losses for the most severe MS.
Impact of Mobility Impairment on Indirect Costs and Health-Related Quality of Life in Multiple Sclerosis
Article
Full-text available
This study was conducted to estimate the indirect costs and health-related quality of life (HRQoL) (utilities) of multiple sclerosis (MS) patients in the United States (US), and to determine the impact of worsening mobility on these parameters. In collaboration with the North American Research Committee on Multiple Sclerosis (NARCOMS) registry we conducted a cross-sectional study of participants who completed the biannual update and supplemental spring 2010 survey. Demographic, employment status, income, mobility impairment, and health utility data were collected from a sample of registry participants who met the study criteria and agreed to participate in the supplemental Mobility Study. Mean annual indirect costs per participant in 2011US$ and mean utilities for the population and for cohorts reporting different levels of mobility impairment were estimated. Analyses included 3,484 to 3,611 participants, based on survey completeness. Thirty-seven percent of registrants were not working or attending school and 46.7% of these reported retiring early. Indirect costs per participant per year, not including informal caregiver cost, were estimated at $30,601±31,184. The largest relative increase in indirect costs occurred at earlier mobility impairment stages, regardless of the measure used. Participants' mean utility score (0.73±0.18) was lower than that of a similarly aged sample from the general US population (0.87). As with indirect costs, larger decrements in utility were seen at earlier mobility impairment stages. These results suggest that mobility impairment may contribute to increases in indirect costs and declines in HRQoL in MS patients.
Quality of life in patients with multiple sclerosis: The impact of depression, fatigue, and disability
Article
Full-text available
  • Sep 2011
The aim of this study was to assess the quality of life (QoL) in patients with multiple sclerosis (MS), and to evaluate its association with disability and psychosocial factors especially depression and fatigue. Demographic characteristics, education level, disease severity, and disease duration were documented for each patient. QoL, fatigue level, cognitive status, and depression level of patients were assessed by Multiple Sclerosis Quality of Life-54, Fatigue Severity Scale, Mini Mental State Scale, and Beck Depression Inventory, respectively. Seventy-nine patients with MS were included in the study. There was a moderate degree of impairment in the QoL scores of MS patients. The most affected parts of QoL were included: role limitation-related physical and emotional problems and physical and social functions. Both physical and mental health components of QoL showed a positive correlation with the educational level and employment status; a negative correlation with the level of disability, fatigue, and depression. Depression, disability level, and fatigue were the strongest variables associated with QoL, and the most important predictor of QoL was depression. Our results have shown that both physical and mental health components of QoL were negatively affected by MS. The most important predictor of QoL was depression followed by disability and fatigue. To improve the QoL for MS patients, in addition to physical disability, the influences of depression and fatigue on QoL should be taken into consideration.
The impact of multiple sclerosis severity on health state utility values: Evidence from Australia
Article
  • Oct 2016
Background: The measurement of health state utility values (HSUVs) for a representative sample of Australian people with multiple sclerosis (MS) has not previously been performed. Objectives: Our main aim was to quantify the HSUVs for different levels of disease severities in Australian people with MS. Method: HSUVs were calculated by employing a 'judgement-based' method that essentially creates EQ-5D-3L profiles based on WHOQOL-100 responses and then applying utility weights to each level in each dimension. A stepwise linear regression was used to evaluate the relationship between HSUVs and disease severity, classified as mild (Expanded Disability Status Scale (EDSS) levels: 0-3.5), moderate (EDSS levels: 4-6) and severe (EDSS levels: 6.5-9.5). Results: Mean HSUV for all people with MS was 0.53 (95% confidence interval (CI): 0.52-0.54). Utility decreased with increasing disease severity: 0.61 (95% CI: 0.60-0.62), 0.51 (95% CI: 0.50-0.52) and 0.40 (95% CI: 0.38-0.43) for mild, moderate and severe disease, respectively. Adjusted differences in mean HSUV between the three severity groups were statistically significant. Conclusion: For the first time in Australia, we have quantified the impact of increasing severity of MS on health utility of people with MS. The HSUVs we have generated will be useful in further health economic analyses of interventions that slow progression of MS.
Economic Burden of Multiple Sclerosis A Systematic Review of the Literature
Article
  • Jan 2010
Multiple sclerosis (MS) is a disease of the CNS, typically striking adults during the primary productive time of their life. The symptoms of MS can restrict the individual's physical activity and income-earning ability, resulting in a major financial burden on the patient, family, health system and society. This systematic literature review was conducted to document the economic burden of MS. Employing pre-defined search terms and inclusion/exclusion criteria, systematic searches were conducted in MEDLINE, EMBASE, PsycINFO, the Health Economic Evaluations Database (HEED), the NHS Economic Evaluation Database (EED) and the UK National Institute for Health and Clinical Excellence (NICE) website as well as conference abstracts. We identified 29 cost-of-illness studies that met the a priori inclusion criteria. The cost categories responsible for the majority of costs associated with MS varied across countries. There was a significant increase in costs associated with an increase in disease severity as measured by the Kurtzke Expanded Disability Status Scale (EDSS) score. The increase in magnitude was coupled with changes in the distribution of costs; although direct medical costs were important contributors in earlier stages of disease, they were outweighed by indirect costs in later stages, mainly due to relapses and productivity losses. Considering the increased costs associated with relapse occurrence and increasing disease severity, pharmaceutical or non-pharmaceutical interventions aimed at delaying the progression of disease may help to reduce the economic burden of MS.
Disability outcome measures in multiple sclerosis clinical trials: Current status and future prospects
Article
  • May 2012
Many of the available disability outcome measures used in clinical trials of multiple sclerosis are insensitive to change over time, inadequately validated, or insensitive to patient-perceived health status or quality of life. Increasing focus on therapies that slow or reverse disability progression makes it essential to refine existing measures or to develop new tools. Major changes to the expanded disability status scale should be avoided to prevent the loss of acceptance by regulators as a measure for primary outcomes in trials that provide substantial evidence of effectiveness. Rather, we recommend practical refinements. Conversely, although substantial data support the multiple sclerosis functional composite as an alternative measure, changes to its component tests and scoring method are needed. Novel approaches, including the use of composite endpoints, patient-reported outcomes, and measurement of biomarkers, show promise as adjuncts to the current disability measures, but are insufficiently validated to serve as substitutes. A collaborative approach that involves academic experts, regulators, industry representitives, and funding agencies is needed to most effectively develop disability outcome measures.
The impact of increasing neurological disability of multiple sclerosis on health utilities: A systematic review of the literature
Article
Multiple sclerosis (MS) is a debilitating disease, accompanied by neurological symptoms of varying severity. Utilities are a key summary index measure used in assessing health-related quality of life in individuals with MS. To provide a systematic review of the literature on utilities of relapsing-remitting MS (RRMS) and secondary progressive MS (SPMS) patients and to review changes in utilities associated with the increasing neurological disability of different stages of MS, as measured by the Expanded Disability Status Scale (EDSS). Employing pre-defined search terms and inclusion/exclusion criteria, systematic searches of the literature were conducted in EMBASE, MEDLINE, PsycINFO, the Health Economic Evaluation Database (HEED), and the NHS Economic Evaluations Database (NHS/EED). Proceedings for the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), the European Society for Treatment and Research in MS (ECTRIMS), the American Society for Treatment and Research in MS (ACTRIMS), and the Latin American Society for Treatment and Research in MS (LACTRIMS) were reviewed in addition to the UK National Institute for Health and Clinical Excellence website and the table of contents of PharmacoEconomics and Value in Health. This review identified 18 studies reporting utilities associated with health states of MS. Utilities ranged from 0.80 to 0.92 for patients with an EDSS score of 1, from 0.49 to 0.71 for patients with an EDSS score of 3, from 0.39 to 0.54 for patients with an EDSS score of 6.5, and from -0.19 to 0.1 for patients with an EDSS score of 9. Limitations: Several of the studies reviewed relied on data from patient organizations, which may not be fully representative of the general patient populations. Additionally, the majority of the studies relied on retrospective data collection. Utilities decrease substantially with increasing neurological disability. Cross-country differences are minimal with utility scores following a similar pattern across countries for patients at similar disease severity levels. This consistency in findings is noteworthy, as there is a reliable evidence base for selecting utility values for economic evaluation analyses. However, more research is needed to explore potential differences in utilities between RRMS and SPMS patients.
Patient Reported Outcomes in Multiple Sclerosis: A Review of Literature
  • Apr 2016
  • NEUROLOGY
  • V Khurana
Khurana V et al. Patient Reported Outcomes in Multiple Sclerosis: A Review of Literature. Neurology April 5, 2016 vol. 86 no. 16 Supplement P2.134