The prevalence of child-specific utilities in NICE appraisals for paediatric indications: rise of the economic orphans?

Montgomery, Stephen; Kusel, J.

doi:10.1080/14737167.2016.1179116

Cited by 9 publications

(11 citation statements)

References 16 publications

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“…A meta-analysis of childhood health utilities by Kwon et al 17 also found that although child-specific instruments are applied quite frequently, the most commonly used indirect valuation method was the Health Utilities Index Mark 3. The same observation was made by Montgomery and Kusel, 18 who found that in most published National Institute for Health and Care Excellence appraisals concerning child populations, adult utilities were used to inform the decision model. Nevertheless, as they also argue, children are not small adults and we cannot assume that their preferences are the same as in the adult population.…”

Section: Introductionmentioning

confidence: 56%

Valuing Health Status in the First Year of Life: The Infant Health-Related Quality of Life Instrument

et al. 2019

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Objectives: Efforts to evaluate HRQoL and calculate quality-adjusted life years (QALYs) for infants less than 12 months of age are hampered by the lack of preference-based HRQoL instruments for this group. To fill this gap, we developed the Infant Quality of life Instrument (IQI), which is administered through a mobile application. This article explains how weights were derived for the 4 levels of each health item. Methods:The IQI includes 7 health items: sleeping, feeding, breathing, stooling/poo, mood, skin, and interaction. In an online survey, respondents from the general population (n = 1409) and primary caregivers (n = 1229) from China, the United Kingdom, and the United States were presented with 10 discrete choice scenarios. Coefficients for the item levels were obtained with a conditional logit model. Results:The highest coefficients were found for sleeping, feeding, and breathing. All coefficients for these items were negative and logically ordered, meaning that more extreme levels were less preferred. Stooling, mood, skin, and interaction showed some irregularities in the ordering of coefficients. Results for caregivers and the general population were about the same. Conclusions:The IQI is the first generic instrument to assess overall HRQoL in infants up to 1 year of age. It is short and easy to administer through a mobile application. We demonstrated how to derive values for infant health states with a discrete choice methodology. Our next step will be to normalize these values into utilities ranging from 0 (dead) to 1 (best health state) and to collect IQI values in a clinical population.

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Section: Introductionmentioning

confidence: 56%

Valuing Health Status in the First Year of Life: The Infant Health-Related Quality of Life Instrument

et al. 2019

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“…A commentary in 2016 briefly reviewed the approaches to generate utility values in child populations in NICE technology appraisals over time 21 and 'found that most had used adult utilities to inform the model, due to the unavailability of suitable child derived utilities; furthermore, it was clear that there was no trend away from this over time' (p. 349). However, this statement was supported using only three illustrative case studies.…”

Section: Discussionmentioning

confidence: 99%

A Review of the Methods Used to Generate Utility Values in NICE Technology Assessments for Children and Adolescents

et al. 2020

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What is already known about the topic?There is little guidance or recommendations around the methods for generating health state utility values for children and adolescents in technology appraisals. What does the paper add to existing knowledge?This review examines previous NICE technology appraisals to identify the methods used to generate utility values for child and adolescent health statesThe review finds that most assessments (33 of 40) involved the use of adult EQ-5D, and few used a child or adolescent specific preference-based measure (10 of 40).This raises the issue of whether adult specific measures are appropriate and are the best approach for valuing the health of children and adolescents. A review of the methods used to generate utility values in NICE technology assessments for children and adolescentsObjective: This review summarises and critically examines methods used to generate utilities for child and adolescent health states in previous NICE (National Institute for Health and Care Excellence) technology assessments. Methods:We identified all NICE Technology Appraisal (TA) and NICE Highly Specialised Technologies (HST) guidance where the licensed indication for the technology included people under 18 years. Results:The review includes 40 NICE technology assessments. Most assessments generated utility values using EQ-5D scored using the adult version of the EQ-5D either exclusively (n=16) or alongside other utility measures and direct elicitation methods of patient own utility (n=17) and seven did not use the EQ-5D. Ten assessments used both adult and children population-specific measures: Health Utilities Index Mark 2 (n=6); a children and adolescents specific preference-based measure for atopic dermatitis (n=1); the youth version of the EQ-5D (EQ-5D-Y) generated using mapping and valued using the UK EQ-5D adult tariff (n=2), and the EQ-5D-Y using the EQ-5D adult tariff (n=1). The cost-effectiveness analyses used age adjustment (utility subtractions, weights and published mapping formulae) from adult EQ-5D UK population norms to reflect general population or disease-free health for children and adolescents (n=9), and one assessment assumed full health (utility value of 1). Conclusion:The review found very limited use of child and adolescent population-specific measures to generate health state utility values for children and adolescents in technology assessments submitted to NICE.Often they use an adult specific measure to reflect the health of children.

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“…First of all, the HTA and reimbursement agencies reported uncertainties in utility values and questioned the added value of nusinersen in SMA subtypes, in type 0, type IV, and even type III SMA. In the past, methodologies to determine utility values have been criticized for being ill-adapted to the needs of younger patients (Eiser and Morse, 2001;Matza et al, 2004;Prosser, 2009;Ungar, 2011;Lim et al, 2014;Montgomery and Kusel, 2016;Gissen et al, 2021). These shortcomings become more impactful considering the fact that 69.9% of rare diseases present themselves in a mainly young patient population, such as in the case of SMA (Nguengang Wakap et al, 2020).…”

Section: Barriers Towards the Economic Evaluation Of Nusinersenmentioning

confidence: 99%

Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries

et al. 2022

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Background: Nusinersen is an orphan drug intended for the treatment of spinal muscular atrophy (SMA), a severe genetic neuromuscular disorder. Considering the very high costs of orphan drugs and the expected market entry of cell and gene therapies, there is increased interest in the use of health technology assessment (HTA) for orphan drugs. This study explores the role of the economic evaluation and budget impact analysis on the reimbursement of nusinersen.Methods: Appraisal reports for nusinersen were retrieved from reimbursement and HTA agencies in Belgium, Canada, France, England and Wales, Germany, Italy, Ireland, Scotland, Sweden, the Netherlands, and the United States. Detailed information was extracted on the economic evaluation, the budget impact, the overall reimbursement decision, and the managed entry agreement (MEA). Costs were adjusted for inflation and currency.Results: Overall, the reports included limited data on budget impact, excluding information on the sources of data for cost and patient estimates. Only three jurisdictions reported on total budget impact, estimated between 30 and 40 million euros per year. For early-onset SMA, the incremental cost-effectiveness threshold (ICER) ranged from €464,891 to €6,399,097 per quality-adjusted life year (QALY) gained for nusinersen versus standard of care. For later-onset SMA, the ICER varied from €493,756 to €10,611,936 per QALY. Although none of the jurisdictions found nusinersen to be cost-effective, reimbursement was granted in each jurisdiction. Remarkably, only four reports included arguments in favor of reimbursement. However, the majority of the jurisdictions set up an MEA, which may have promoted a positive reimbursement decision.Conclusion: There is a need for more transparency on the appraisal process and conditions included in the MEA. Additionally, by considering all relevant criteria explicitly during the appraisal process, decision-makers are in a better position to justify their allocation of funds among the rising number of orphan drugs that are coming to the market in the near future.

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The prevalence of child-specific utilities in NICE appraisals for paediatric indications: rise of the economic orphans?

Cited by 9 publications

References 16 publications

Valuing Health Status in the First Year of Life: The Infant Health-Related Quality of Life Instrument

Valuing Health Status in the First Year of Life: The Infant Health-Related Quality of Life Instrument

A Review of the Methods Used to Generate Utility Values in NICE Technology Assessments for Children and Adolescents

Assessing the Value of Nusinersen for Spinal Muscular Atrophy: A Comparative Analysis of Reimbursement Submission and Appraisal in European Countries

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