Rare Lung Disease Research

Vuga, Louis J.; Aggarwal, Neil R.; Reineck, Lora A.; Kalantari, Roya; Banerjee, Koyeli; Kiley, James P.

doi:10.1016/j.chest.2019.05.004

Cited by 4 publications

(3 citation statements)

References 23 publications

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“…About 1 in 12 Canadians, or approximately 3 million people, live with a rare condition (CIHR, 2016;Divino et al, 2016;Kvancz, 2016). The majority of rare diseases are severe and chronic, with many being degenerative and life-threatening (Vuga et al, 2019). Therefore, this paper will focus on exploring objectivity in the research of chronic, serious, and potentially progressive rare diseases.…”

Section: R Are D Is E a S E Re S E Archmentioning

confidence: 99%

“…Therefore, this paper will focus on exploring objectivity in the research of chronic, serious, and potentially progressive rare diseases. Similarly, the natural history of the disease in an individual may not correlate with the expected journey-even within an individual, the course of the disease may change unexpectedly (Bygum, Busse, Caballero, & Maurer, 2017;Divino et al, 2016;Vuga et al, 2019;Whicher, Philbin, & Aronson, 2018). The heterogeneity of disease presentation varies not only between diseases but may also vary between individuals with the same disease (as can occur in hereditary angioedema or hemophilia) (Bygum et al, 2011;Carcao, Berg, Ljung, & Mancuso, 2013).…”

Section: R Are D Is E a S E Re S E Archmentioning

confidence: 99%

“…The heterogeneity of disease presentation varies not only between diseases but may also vary between individuals with the same disease (as can occur in hereditary angioedema or hemophilia) (Bygum et al, 2011;Carcao, Berg, Ljung, & Mancuso, 2013). Similarly, the natural history of the disease in an individual may not correlate with the expected journey-even within an individual, the course of the disease may change unexpectedly (Bygum, Busse, Caballero, & Maurer, 2017;Divino et al, 2016;Vuga et al, 2019;Whicher, Philbin, & Aronson, 2018).…”

Section: R Are D Is E a S E Re S E Archmentioning

confidence: 99%

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Objectivity in rare disease research: A philosophical approach

2019

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Individuals living with rare conditions are faced with important challenges derived from the rarity of their conditions and aggravated by the low priority given to rare disease research. However, current realities of rare disease research require consideration of the relationship between subjectivity and 'traditional' objectivity.Objectivity in research has traditionally been associated with processes and descriptions that are independent of the investigator. The need for researchers to provide unbiased knowledge and achieve a balance between objectivity and the underlying values in nursing and scientific research requires an examination of how objectivity is conceptualized within the context of rare disease research. The aim of this paper is to examine scientific objectivity in rare disease research from a philosophical viewpoint and, in doing so, demonstrate the need to redefine it to reflect the current scientific environment. As such, healthcare providers working on this field need to redefine objectivity around ethical and moral obligation to advance science in an equitable manner with the end goal to produce knowledge that is trustworthy and beneficial to our patients. K E Y W O R D Sethics, moral, nursing practice, philosophy, philosophy of science, research implementation, research in practice

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Section: R Are D Is E a S E Re S E Archmentioning

confidence: 99%

Section: R Are D Is E a S E Re S E Archmentioning

confidence: 99%

Section: R Are D Is E a S E Re S E Archmentioning

confidence: 99%

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Objectivity in rare disease research: A philosophical approach

2019

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Financing and Reimbursement Models for Personalised Medicine: A Systematic Review to Identify Current Models and Future Options

Koleva-Kolarova

Buchanan

Vellekoop

et al. 2022

Appl Health Econ Health Policy

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Background The number of healthcare interventions described as ‘personalised medicine’ (PM) is increasing rapidly. As healthcare systems struggle to decide whether to fund PM innovations, it is unclear what models for financing and reimbursement are appropriate to apply in this context. Objective To review financing and reimbursement models for PM, summarise their key characteristics, and describe whether they can influence the development and uptake of PM. Methods A literature review was conducted in Medline, Embase, Web of Science, and Econlit to identify studies published in English between 2009 and 2021, and reviews published before 2009. Grey literature was identified through Google Scholar, Google and subject-specific webpages. Articles that described financing and reimbursement of PM, and financing of non-PM were included. Data were extracted and synthesised narratively to report on the models, as well as facilitators, incentives, barriers and disincentives that could influence PM development and uptake. Results One hundred and fifty-three papers were included. Research and development of PM was financed through both public and private sources and reimbursed largely through traditional models such as single fees, Diagnosis-Related Groups, and bundled payments. Financial-based reimbursement, including rebates and price-volume agreements, was mainly applied to targeted therapies. Performance-based reimbursement was identified mainly for gene and targeted therapies, and some companion diagnostics. Gene therapy manufacturers offered outcome-based rebates for treatment failure for interventions including Luxturna ® , Kymriah ® , Yescarta ® , Zynteglo ®, Zolgensma ® and Strimvelis ® , and coverage with evidence development for Kymriah ® and Yescarta ® . Targeted testing with OncotypeDX ® was granted value-based reimbursement through initial coverage with evidence development. The main barriers and disincentives to PM financing and reimbursement were the lack of strong links between stakeholders and the lack of demonstrable benefit and value of PM. Conclusions Public-private financing agreements and performance-based reimbursement models could help facilitate the development and uptake of PM interventions with proven clinical benefit. Supplementary Information The online version contains supplementary material available at 10.1007/s40258-021-00714-9.

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The Trouble With Group 3 Pulmonary Hypertension in Interstitial Lung Disease

King

Shlobin

2020

Chest

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Rare Lung Disease Research

Cited by 4 publications

References 23 publications

Objectivity in rare disease research: A philosophical approach

Objectivity in rare disease research: A philosophical approach

Financing and Reimbursement Models for Personalised Medicine: A Systematic Review to Identify Current Models and Future Options

The Trouble With Group 3 Pulmonary Hypertension in Interstitial Lung Disease

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