Using a stated preference discrete choice experiment to assess societal value from the perspective of decision-makers in Europe. Does it work for rare diseases?

López-Bastida, Julio; Ramos-Goñi, Juan Manuel; Aranda-Reneo, Isaac; Trapero-Bertran, Marta; Kanavos, Panos; Martín, Beatriz

doi:10.1016/j.healthpol.2018.11.015

Cited by 12 publications

(22 citation statements)

References 23 publications

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“…Within our article sample, we encountered similar DCE applications within health economics indeed, e.g. Gyrd-Hansen (2004) and López-Bastida et al (2019), again indicating routinization.…”

Section: Practicesmentioning

confidence: 60%

Public values assessment as a practice: integration of evidence and research agenda

Huijbregts

George

Bekkers

2021

Public Management Review

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“…Within our article sample, we encountered similar DCE applications within health economics indeed, e.g. Gyrd-Hansen (2004) and López-Bastida et al (2019), again indicating routinization.…”

Section: Practicesmentioning

confidence: 60%

Public values assessment as a practice: integration of evidence and research agenda

Huijbregts

George

Bekkers

2021

Public Management Review

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“…As treatments for rare diseases are unlikely to be cost effective given their high prices, additional criteria are already being used to inform reimbursement decisions in some countries. One relevant study analyzed use of public funds for orphan drugs in five European countries from the decision-maker's point of view [ 57 ]. Another study was conducted in Italy from the patient's point of view focusing on two diseases, cystic fibrosis and hemophilia; it also quantified individual preferences [ 58 ].…”

Section: Discussionmentioning

confidence: 99%

“…As several criteria are relevant, a multi-criteria decision analysis can constitute a valuable solution for decision-making. It allows the influence of each criterion on the decision and relative importance to be defined, going beyond the simple QALY analysis [ 57 , 58 ].…”

Section: Discussionmentioning

confidence: 99%

Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments

Dangouloff

Botty

Beaudart

et al. 2021

Orphanet J Rare Dis

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Background Spinal muscular atrophy (SMA) is a rare and devastating condition for which new disease-modifying treatments have recently been approved. Given the increasing importance of economic considerations in healthcare decision-making, this review summarizes the studies assessing the cost of SMA and economic evaluations of treatments. A systematic review of the literature in PubMed and Scopus up to 15 September 2020 was conducted according to PRISMA guidelines. Results Nine studies reporting the annual cost of care of patients with SMA and six evaluations of the cost-effectiveness of SMA treatments were identified. The average annual cost of SMA1, the most frequent and severe form in which symptoms appear before the age of 6 months were similar according to the different studies, ranged from $75,047 to $196,429 per year. The yearly costs for the forms of the later-onset form, called SMA2, SMA3, and SMA4, which were usually pooled in estimates of healthcare costs, were more variable, ranging from $27,157 to $82,474. The evaluations of cost-effectiveness of treatment compared nusinersen treatment against standard of care (n = 3), two treatments (nusinersen and onasemnogene abeparvovec) against each other and no drug treatment (n = 1), nusinersen versus onasemnogene abeparvovec (n = 1), and standard of care versus nusinersen with and without newborn screening (n = 1). The incremental cost-effectiveness ratio (ICER) of nusinersen compared to standard of care in SMA1 ranged from $210,095 to $1,150,455 per quality-adjusted life years (QALY) gained and that for onasemnogene abeparvovec ranged from $32,464 to $251,403. For pre-symptomatic patients, the ICER value ranged from $206,409 to $735,519. The ICERs for later-onset forms of SMA (2, 3 and 4) were more diverse ranging from $275,943 to $8,438,049. Conclusion This review confirms the substantial cost burden of standard of care for SMA patients and the high cost-effectiveness ratios of the approved drugs at the current price when delivered in post-symptomatic patients. Since few studies have been conducted so far, there is a need for further prospective and independent economic studies in pre- and post-symptomatic patients.

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“…The most important attributes for this group of patients have similarities with the group of decision-makers that we have tested in a similar study [7]. In addition, the results in this study are in line with earlier work from England, where face-to-face interviews were used on a sample from the general population, and where improvement in health and value for money were the attributes that provided the strongest indication of social value (preference) [5].…”

Section: Discussionmentioning

confidence: 99%

“…A DCE was carried out in accordance with previously used methods [5]. More details about the design are provided elsewhere [7]. The study followed the approach recommended by Street and Burgess [8].…”

Section: Methodsmentioning

confidence: 99%

Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy

López-Bastida¹,

Ramos-Goñi²,

Aranda-Reneo³

et al. 2019

Orphanet J Rare Dis

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Background Decision makers have huge problems when attempting to attribute social value to the improvements achieved by new drugs, especially when considering the use of orphan drugs for rare diseases. We present the results of a pilot study aimed to investigate patient preferences regarding public funding for drugs used to treat rare diseases. Methods An online questionnaire was used as a discrete choice experiment (DCE) survey to explore the preferences of patients with cystic fibrosis and haemophilia in Italy. The questionnaire focused on relevant issues that were defined in a review of the literature. A conditional logistic model showed preferences for specific attributes. Results A total of 54 questionnaires (20% response rate) were completed. The issues that received the greatest attention were improvement in health, treatment cost and value for money. However, disease severity and the availability of other treatments were important social values that could not be ignored. Conclusions The findings presented here provide evidence as to what patients with cystic fibrosis or haemophilia think are the most important considerations on which to base decisions in health technology scenarios, and regarding the priorities for funding.

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Using a stated preference discrete choice experiment to assess societal value from the perspective of decision-makers in Europe. Does it work for rare diseases?

Cited by 12 publications

References 23 publications

Public values assessment as a practice: integration of evidence and research agenda

Public values assessment as a practice: integration of evidence and research agenda

Systematic literature review of the economic burden of spinal muscular atrophy and economic evaluations of treatments

Using a stated preference discrete choice experiment to assess societal value from the perspective of patients with rare diseases in Italy

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