Cost-of-Illness in Rare Diseases

Linertová, Renata; García-Pérez, Lidia; Gorostiza, Iñigo

doi:10.1007/978-3-319-67144-4_17

Cited by 15 publications

(12 citation statements)

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“…The progressive and changing nature of certain conditions, such as Huntington disease, also adds challenging elements to the analysis, including an accurate staging of the disease at a specific point in time, the time-related effects of its treatment or the aspects of continuity of care that could overall improve the HRQoL of people living with rare long-term disorders ( Calvert et al, 2013 ; van Walsem et al, 2017 ). Similar conclusions could be drawn with regards to the inadequacy of COI studies to take account of the benefits of treatments ( Linertová et al, 2017 ). As for the content of COI studies, several observations can be made.…”

Section: Discussionsupporting

confidence: 66%

Valuing the “Burden” and Impact of Rare Diseases: A Scoping Review

2022

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Introduction: Rare diseases (RDs) are a severe, chronic, degenerative and often life-threatening group of conditions affecting more than 30 million people in Europe. Their impact is often underreported and ranges from psychological and physical symptoms seriously compromising quality of life. There is then a need to consolidate knowledge on the economic, social, and quality of life impacts of rare diseases.Methods: This scoping review is the result of 9 qualitative interviews with experts and a literature search on Cost-of-Illness (COI) studies and quality of life (QoL) studies following the PRISMA methodology. Grey literature was also included to complement findings. Results. 63 COI studies were retrieved, covering 42 diseases and a vast majority of them using a prevalence-based approach (94%). All studies included medical costs, while 60% included non-medical costs, 68% productivity losses and 43% informal care costs. 56 studies on QoL were retrieved, mostly from Europe, with 30 different measurement tools. Grey literature included surveys from the pharmaceutical industry and patient organisations.Discussion: The majority of studies evaluating the impact of RDs on the individual and society use the COI approach, mostly from a societal perspective. Studies often vary in scope, making them difficult to consolidate or compare results. While medical costs and productivity losses are consistently included, QoL aspects are rarely considered in COI and are usually measured through generic tools.Conclusion: A comprehensive study on impact of rare disease across countries in Europe is lacking. Existing studies are heterogeneous in their scope and methodology and often lack a holistic picture of the impact of rare. Consensus on standards and methodology across countries and diseases is then needed. Studies that consider a holistic approach are often conducted by pharmaceutical companies and patient organisations exploring a specific disease area but are not necessarily visible in the literature and could benefit from the sharing of standards and best practices.

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Section: Discussionsupporting

confidence: 66%

Valuing the “Burden” and Impact of Rare Diseases: A Scoping Review

2022

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“…Prevalence-based cost-of-illness analysis has the advantage of incorporating measurements of total annual health care expenditure, which is particularly relevant for chronic conditions such as MG that require long-term treatment. In this context, a bottom-up costing approach was applied to estimate total and average annual costs ( 14 ).…”

Section: Methodsmentioning

confidence: 99%

Socio-Economic Burden of Myasthenia Gravis: A Cost-of-Illness Study in Bulgaria

Ignatova

Kostadinov²,

Vassileva³

et al. 2022

Front. Public Health

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BackgroundMyasthenia gravis (MG) is a chronic autoimmune disorder, which is characterized by fatigable muscle weakness with frequent ocular signs and/or generalized muscle fatigue, and occasionally associated with thymoma. MG patients and their families face a significant socio-economic burden. This population is often experiencing unemployment, unwilling job transfers and decreased income.ObjectiveThis study aimed to estimate the annual costs from a societal perspective in a triple dimension of direct health care costs, direct non-health care costs (formal and informal care) and labor productivity losses in MG patients from Bulgaria, as well as to identify the main clinical and demographical cost drivers.MethodsA bottom-up, cross-sectional, cost-of-illness analysis of 54 adult MG patients was carried out in 2020. To collect data on demographic characteristics, health resource utilization, informal care and productivity losses, questionnaires were administered to and completed by patients.Results and ConclusionMedian annual costs of MG in Bulgaria were 4,047 EUR per patient. Direct costs slightly outweighed indirect costs, with drugs cost item having the biggest monetary impact. Despite the zero-inflated median, hospitalizations also influenced the direct costs by an estimated amount of 1,512 EUR in the 3rd quartile. Social services and professional caregiver costs were found to be almost missing, with the vast majority of patients reporting reliance on informal caregivers. Severe generalized disease, disease crises, and recurrent infections were confirmed as statistically significant cost driving factors. There were no severe generalized MG patients in the bottom quartile of the total costs distribution. It should be noted that in both cases of crises or infections, the overall increase in the total costs was mainly due to higher indirect costs observed. Reliance on family members as informal caregivers is routine among Bulgarian MG patients. This phenomenon is likely due to the lack of access to appropriate social services. Moreover, it is directly related with higher disease burden and significant inequalities. There is a need for further research on MG in Bulgaria in order to design targeted health policies that meet the needs and expectations of these patients.

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“…-Götz, M. 2000). Így jelentős társadalmi terhek jelentkeznek minden ágazat ellátórendszerében, valamint az ellátórendszereken kívül (Linertová et al 2017).…”

Section: A Kihívás: Problémák Az Egészségügy éS a Szociális üGy Területénunclassified

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2021

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Tartalom TEMATIKUS TANULMÁNYOK Betegségekkel való megküzdés esélyeinek növelése. Az életminőség javításának lehetőségei Vendégszerkesztő: Pék Győző 3 Vajsz Kornél -Horváth Márton -Simon Lajos -Vass Edit -Sztancsik Veronika: Stigmatizáció és időskori mentális betegségek 20 Kosztáné Hadházi Tünde -Pék Győző: Demenciával élők életminőségének javítása demenciabarát szemléletű foglalkoztatásokkal 37 Pék Győző -Kosztáné Hadházi Tünde -Orbánné Gellén Marianna -Tóthné Görög Edit: Támogató Bálint-csoport demenciával élő családtagot gondozó hozzátartozók számára 49 Pogány Gábor-Dupcsik Csaba: A ritka betegségek szociálpolitikai jelentősége, a RIROSZ törekvései TANULMÁNYOK 62 Paksi Borbála -Magi Anna -Gurály Zoltán: Hajléktalan emberek pszichoaktív szerhasználata 92 Győri Péter "Így kallódunk el szépen egymás után". Állami gondozottak voltak, ma hajléktalanok 112 Berényi Eszter Különleges oktatási időszakok: felvételizők és szüleik tapasztalatai a digitális tanrendről KEREKASZTAL-BESZÉLGETÉS 132 Kiss László Filozófia -tudományok -gondolkodás | 3

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Cost-of-Illness in Rare Diseases

Cited by 15 publications

References 34 publications

Valuing the “Burden” and Impact of Rare Diseases: A Scoping Review

Valuing the “Burden” and Impact of Rare Diseases: A Scoping Review

Socio-Economic Burden of Myasthenia Gravis: A Cost-of-Illness Study in Bulgaria

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