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- Landfeldt, Erik, et al.
(författare)
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Health-related quality of life of children with spinal muscular atrophy in Sweden : A prospective cohort study in the era of disease-modifying therapy
- 2023
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Ingår i: European journal of paediatric neurology. - : Elsevier. - 1090-3798 .- 1532-2130. ; 46, s. 67-73
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Tidskriftsartikel (refereegranskat)abstract
- The objective of this study was to estimate change over time in health-related quality of life (HRQoL) of children with spinal muscular atrophy (SMA) in Sweden. Children with SMA were identified via the National Patient Register by the National Board of Health and Welfare in Sweden. Patient HRQoL was caregiver proxy-assessed using the Pediatric Quality of Life Inventory 4.0 Generic Core Scales at baseline, as well as at 6, 12, and 18 months of follow-up. Results were stratified by SMA type. Mothers and fathers to 27 children with SMA (mean patient age: 9.17 years; 59% female) participated in the study. All patients received disease-modifying therapy. At baseline, across SMA types, the mean total score was estimated at between 52.68 and 59.19, Physical Functioning score at between 26.39 and 40.34, Emotional Functioning score at between 66.82 and 68.57, Social Functioning score at between 55.00 and 70.45, and School Functioning score at between 70.45 and 78.33. The mean annual total score change was estimated at −2.03 for SMA type I, 4.11 for SMA type II, and 1.12 for SMA type III. In conclusion, we show that SMA has a detrimental impact on HRQoL that extends above and beyond somatic disability. Children with SMA type II experienced a dramatic increase in HRQoL over time, predominantly related to improvement in physical and social functioning. Our data helps quantify the patient burden of disease and adds to the rapidly expanding body of evidence of the effectiveness of recently approved disease-modifying therapies for SMA.
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- Landfeldt, Erik, et al.
(författare)
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Patient preferences for characteristics differentiating ovarian stimulation treatments
- 2012
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Ingår i: Human Reproduction. - : Oxford University Press (OUP): Policy B1. - 0268-1161 .- 1460-2350. ; 27:3, s. 760-769
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Tidskriftsartikel (refereegranskat)abstract
- BACKGROUND Little is known concerning patient preferences for IVF treatments. The objective of this study was to elicit patient preferences for characteristics differentiating ovarian stimulation treatments. METHODS Women undergoing IVF were recruited from six clinics in Sweden between May 2010 and December 2010. Included patients completed a study questionnaire consisting of one contingent valuation (CV) question (with six different bids) and 16 conjoint analysis (CA) questions formulated as discrete choices between two hypothetical ovarian stimulation treatments (defined in terms of manufacturing method, method of administration, time required for administration, dose variability and hypothetical price). Patient preferences were derived using multinomial logit modelling. RESULTS The final study population consisted of 294 women (mean age of 35). Respondents were willing to pay €360 [95% confidence interval (CI): €340-€390] to receive FSH derived from DNA technology instead of highly purified extract from urine from post-menopausal women, €300 (95% CI: €280-€320) to administer the FSH using a prefilled injection pen instead of a conventional syringe, €30 (95% CI: €20-€40) per saved minute required for administration and €530 (95% CI: €500-€570) to reduce the dose variability from 10-20% to 1-2%(P< 0.001 for all estimates). The result from the CV was similar to the CA. CONCLUSIONS Women undergoing IVF place significant value on characteristics differentiating ovarian stimulation treatments. Product-specific aspects should be taken into account by decision-makers when discriminating between commercial gonadotrophins in clinical practice to align health-care decision-making with patient preferences and potentially improve the effectiveness of IVF interventions through enhanced patient satisfaction and treatment compliance. Preferences for treatment characteristics should also be considered in evaluations of ovarian stimulation products to capture their true value from a patient perspective.
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| 4. |
- Landfeldt, Erik
(författare)
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The health economics of Duchenne muscular dystrophy
- 2016
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Doktorsavhandling (övrigt vetenskapligt/konstnärligt)abstract
- Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease characterized by progressive muscle degeneration resulting in loss of independent ambulation, serious multisystem complications, and a mean life expectancy at birth of about 25 years. Several therapies are on the horizon for DMD and it has thus become urgent to better understand the health economic context of the disease to enable assessments of cost-effectiveness and inform decisions regarding reimbursement of new health technologies. However, to date, little is known of the burden of DMD, including cost of illness and impact on health-related quality of life (HRQoL). The aim of this thesis was to estimate and describe the health economics of DMD, develop a tool measuring disease progression, and construct a model framework for economic evaluation of new treatments. Paper I to IV were based on data collected in a cross-sectional, observational study. Patients with DMD from Germany, Italy, the UK, and the US were identified and recruited through the Translational Research in Europe – Assessment and Treatment of Neuromuscular Diseases (TREAT-NMD) network and invited with a caregiver to complete a study questionnaire online. Paper V was a modelling study synthesizing data from the preceding papers. In paper I to III, we estimated direct and indirect costs of DMD and measured patient and caregiver HRQoL and burden using several generic and disease-specific instruments. In paper IV, we developed the Duchenne muscular dystrophy Functional Ability Self-Assessment Tool (DMDSAT), a new rating-scale measuring functional ability in DMD. Item selection for the DMDSAT was led by neuromuscular specialists and the psychometric properties of the instrument were examined using Rasch analysis. In paper V, we synthesized our health economic evidence and developed a decision-analytic model framework for the assessment of the cost-effectiveness of treatments for DMD based on the DMDSAT. For comparison, we also developed two models based on stages of disease as defined in the international DMD clinical care guidelines and patients’ ventilation status, respectively. In paper I, we show that DMD is associated with a substantial cost burden to society and affected households. Across countries, the mean per-patient annual direct cost of illness was found to be 7 to 16 times higher than the mean per-capita health expenditure. Indirect and informal care costs of DMD were substantial, each component constituting between 18% and 43% of total costs. In paper II, we show that HRQoL (as valued by the general population) in DMD is substantially impaired in relation to general population reference values, strongly negatively associated with disease progression, and in good agreement with the caregivers’ subjective rating of patients’ current health and mental status. Still, most children and young adults with DMD were perceived as happy and in good health by their caregivers, indicating that some domains of HRQoL remain intact through the progression of the disease. In paper III, we show that caring for a person with DMD can be associated with a substantial burden and impaired HRQoL. Our findings suggest that caregivers to patients with DMD should be screened for depression and emphasize the need for a holistic approach to family mental health in the context of chronic childhood disease. In paper IV, the administered version of the new rating-scale, the DMDSAT, comprised a total of eight questions covering four domains (arm function, mobility, transfers, and ventilation status). Results from the psychometric analysis show that the DMDSAT is an instrument fit for purpose to measure functional ability in ambulant and non-ambulant patients with DMD. In paper V, we found that model structure and perspective of analysis have a substantial impact on assessments of cost-effectiveness of treatments for DMD. Our results show that the DMDSAT represents a sensitive and clinically relevant option for modelling DMD across the entire trajectory of disease in economic evaluations compared with frameworks based on conventional staging of disease progression. In summary, this thesis provides a description of the previously unknown health economic context of DMD, including a portfolio of cost and utility data, a new tool designed to measure DMD disease severity, and a fully populated decision-analytic model framework for cost-effectiveness analysis. These data and tools should be helpful to inform health technology assessments and health economic programmes of new treatments for DMD.
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- Lochmueller, Hanns, et al.
(författare)
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The Position of Neuromuscular Patients in Shared Decision Making. Report from the 235th ENMC Workshop : Milan, Italy, January 19-20, 2018
- 2019
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Ingår i: JOURNAL OF NEUROMUSCULAR DISEASES. - : IOS Press. - 2214-3599 .- 2214-3602. ; 6:1, s. 161-172
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Tidskriftsartikel (refereegranskat)abstract
- In the era of patient-centered medicine, shared decision-making (SDM) - in which healthcare professionals and patients exchange information and preferences and jointly reach a decision - has emerged as the gold standard model for the provision of formal healthcare. Indeed, in many geographical settings, patients are frequently invited to participate in choices concerning the design and delivery of their medical management. From a clinical perspective, benefits of this type of patient involvement encompass, for example, enhanced treatment satisfaction, improved medical compliance, better health outcomes, and maintained or promoted quality of life. Yet, although the theory and enactment of SDM in healthcare are well-described in the literature [1-3], comparatively less attention has been devoted to contextualizing questions relating to if, when, and how to include patients in decisions within medical research. In this context, patient involvement would be expected to be potentially relevant for and applicable to a wide range of activities and processes, from the identification of research priorities and development of grant applications, to the design of patient information and consent procedures, formulation of interventions, identification and recruitment of study sample populations, feasibility of a clinical trial, identification, selection, and specification of endpoints and outcomes in clinical trials and observational studies, data collection and analysis, and dissemination of results. To this end, 45 clinicians, healthcare professionals, researchers, patients, caregivers, and representatives from regulatory authorities and pharmaceutical companies from 15 different countries met to discuss the level of involvement of patients with neuromuscular diseases, specifically in the following settings of medical research for neuromuscular diseases: i) registries and biobanks; ii) clinical trials; and iii) regulatory processes. In this report, we present summaries of the talks that were given during the workshop, as well as discussion outcomes from the three topic areas listed above.
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