Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?

Postma, Maarten; Noone, Declan; Rozenbaum, Mark H; Carter, John A.; Botteman, Marc; Fenwick, Elisabeth; Garrison, Louis P.

doi:10.1186/s13023-022-02283-z

Cited by 17 publications

(12 citation statements)

References 78 publications

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“…It has been described before, that the criterium relevance is not always met in reimbursement decisions for orphan medicines [ 28 ]. Especially the arguments most often used to reason a reimbursement decision, cost-effectiveness, can be difficult to assess in orphan medicines [ 26 , 29 , 30 ].…”

Section: Discussionmentioning

confidence: 99%

The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy

Scheijmans

Zomers

Fadaei

et al. 2022

BMC Health Serv Res

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Background The reimbursement for expensive medicines poses a growing challenge to healthcare worldwide. In order to increase its control over the costs of medicines, the Dutch government introduced the Coverage Lock (CL) policy in 2015. The CL postpones decisions regarding reimbursement of expensive medicines until detailed advice on i.e., cost-effectiveness has been given. The CL has been in place for six years, has raised many questions and concerns, but currently, no evaluation is known to the authors. A better understanding of the effects of the CL on all stakeholders involved may contribute to reflections on the CL process and help find ways to improve it. An evaluation of Dutch policy will also be relevant for other countries that aim to optimize reimbursement procedures for expensive treatments. To perform this evaluation, we focused on the CL procedure for the medicine nusinersen. Nusinersen is the first treatment for spinal muscular atrophy (SMA). Following EMA approval in May 2017, it was placed in the CL. The analysis of cost-effectiveness and added therapeutic value resulted in an advice for reimbursement limited to children younger than 9.5 years at the start of treatment; this was implemented from August 2018 onwards. Methods Qualitative stakeholder perspective analysis of the CL procedure focusing on nusinersen with 15 stakeholders. Results Stakeholders raised key issues of the CL based on their experience with nusinersen: emotional impact of the CL, duration of the CL procedure, appropriateness of the CL procedure for different types of medicines, transparency of the CL, a wish for patient-centred decision-making and the lack of uniformity of access to expensive treatments. Discussion Stakeholders supported measures to control healthcare expenses and to ensure reasonable pricing. They considered the delay in access to therapies and lack of procedural transparency to be the main challenges to the CL. Stakeholders also agreed that the interests of patients deserve more attention in the practical implementation of the reimbursement decision. Stakeholders suggested a number of adjustments to improve the CL, such as a faster start with conditional reimbursement programs to ensure access and intensify European collaboration to speed up the assessment of the medicine.

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Section: Discussionmentioning

confidence: 99%

The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy

Scheijmans

Zomers

Fadaei

et al. 2022

BMC Health Serv Res

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“…Therapies for orphan diseases are typically much more expensive per QALY than therapies for more common diseases [100]. QALY methods are not well suited to assess the value of therapies for orphan diseases [101]. It may cost as much to develop a drug for an uncommon malignancy subtype or for an orphan disease as for a common disease, but the costs of drug development must then be recouped from a relatively small population when the drug is marketed [102].…”

Section: Controlling Drug Pricesmentioning

confidence: 99%

New Anticancer Drugs: Reliably Assessing “Value” While Addressing High Prices

Stewart,

Bradford,

Sehdev

et al. 2024

Current Oncology

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Countries face challenges in paying for new drugs. High prices are driven in part by exploding drug development costs, which, in turn, are driven by essential but excessive regulation. Burdensome regulation also delays drug development, and this can translate into thousands of life-years lost. We need system-wide reform that will enable less expensive, faster drug development. The speed with which COVID-19 vaccines and AIDS therapies were developed indicates this is possible if governments prioritize it. Countries also differ in how they value drugs, and generally, those willing to pay more have better, faster access. Canada is used as an example to illustrate how “incremental cost-effectiveness ratios” (ICERs) based on measures such as gains in “quality-adjusted life-years” (QALYs) may be used to determine a drug’s value but are often problematic, imprecise assessments. Generally, ICER/QALY estimates inadequately consider the impact of patient crossover or long post-progression survival, therapy benefits in distinct subpopulations, positive impacts of the therapy on other healthcare or societal costs, how much governments willingly might pay for other things, etc. Furthermore, a QALY value should be higher for a lethal or uncommon disease than for a common, nonlethal disease. Compared to international comparators, Canada is particularly ineffective in initiating public funding for essential new medications. Addressing these disparities demands urgent reform.

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“…Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose? (Postma et al, 2022) "The shortcomings of conventional CEA with respect to orphan drugs centers primarily on three points: a conflict between equity and equality, limitations of the conventional application of the QALY, and how to deal with uncertainty." Klungel, Leufkens, & Frederix, 2020;Qiu et al, 2022).…”

Section: Aggregate Affordability and Sustainabilitymentioning

confidence: 99%

A review of economic issues for gene‐targeted therapies: Value, affordability, and access

Garrison

Lo²,

Finkel

et al. 2023

American J of Med Genetics Pt C

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The National Center for Advancing Translational Sciences' virtual 2021 conference on gene-targeted therapies (GTTs) encouraged multidisciplinary dialogue on a wide range of GTT topic areas. Each of three parallel working groups included social scientists and clinical scientists, and the three major sessions included a presentation on economic issues related to their focus area. These experts also coordinated their efforts across the three groups. The economics-related presentations covered three areas with some overlap: (1) value assessment, uncertainty, and dynamic efficiency;(2) affordability, pricing, and financing; and (3) evidence generation, coverage, and access. This article provides a synopsis of three presentations, some of their key recommendations, and an update on related developments in the past year. The key high-level findings are that GTTs present unique data and policy challenges, and that existing regulatory, health technology assessment, as well as payment and financing systems will need to adapt. But these adjustments can build on our existing foundation of regulatory and incentive systems for innovation, and much can be done to accelerate progress in GTTs. Given the substantial unmet medical need that exists for these oft-neglected patients suffering from rare diseases, it would be a tragedy to not leverage these exciting scientific advances in GTTs.

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Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?

Cited by 17 publications

References 78 publications

The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy

The reimbursement for expensive medicines: stakeholder perspectives on the SMA medicine nusinersen and the Dutch Coverage Lock policy

New Anticancer Drugs: Reliably Assessing “Value” While Addressing High Prices

A review of economic issues for gene‐targeted therapies: Value, affordability, and access

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