Establishing a reasonable price for an orphan drug

Berdud, Mikel; Drummond, Michael; Towse, Adrian

doi:10.1186/s12962-020-00223-x

Cited by 60 publications

(58 citation statements)

References 25 publications

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“…Also, requiring OMPs to adhere to standard cost-effectiveness thresholds may motivate manufacturers to improve methods for the collection of robust study data and, subsequently, reduce the uncertainty regarding the clinical effectiveness of OMPs ( Berdud et al, 2020 ). Another strength of a standard economic evaluation (or an economic evaluation in general) is that it allows the flexibility to shift between different perspectives when considering costs to calculate the ICER.…”

Section: Resultsmentioning

confidence: 99%

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

et al. 2021

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Background: Decision-makers have implemented a variety of value assessment frameworks (VAFs) for orphan drugs in European jurisdictions, which has contributed to variations in access for rare disease patients. This review provides an overview of the strengths and limitations of VAFs for the reimbursement of orphan drugs in Europe, and may serve as a guide for decision-makers.Methods: A narrative literature review was conducted using the databases Pubmed, Scopus and Web of Science. Only publications in English were included. Publications known to the authors were added, as well as conference or research papers, or information published on the website of reimbursement and health technology assessment (HTA) agencies. Additionally, publications were included through snowballing or focused searches.Results: Although a VAF that applies a standard economic evaluation treats both orphan drugs and non-orphan drugs equally, its focus on cost-effectiveness discards the impact of disease rarity on data uncertainty, which influences an accurate estimation of an orphan drug’s health benefit in terms of quality-adjusted life-years (QALYs). A VAF that weighs QALYs or applies a variable incremental cost-effectiveness (ICER) threshold, allows the inclusion of value factors beyond the QALY, although their methodologies are flawed. Multi-criteria decision analysis (MCDA) incorporates a flexible set of value factors and involves multiple stakeholders’ perspectives. Nevertheless, its successful implementation relies on decision-makers’ openness toward transparency and a pragmatic approach, while allowing the flexibility for continuous improvement.Conclusion: The frameworks listed above each have multiple strengths and weaknesses. We advocate that decision-makers apply the concept of accountability for reasonableness (A4R) to justify their choice for a specific VAF for orphan drugs and to strive for maximum transparency concerning the decision-making process. Also, in order to manage uncertainty and feasibility of funding, decision-makers may consider using managed-entry agreements rather than implementing a separate VAF for orphan drugs.

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Section: Resultsmentioning

confidence: 99%

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

et al. 2021

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“…There are inherent difficulties in developing medicines for rare diseases including small population sizes in pivotal studies and consequently, limitations in the corresponding evidence package. It is recognised that orphan medicines often fail to meet the standard ICER threshold of £20–30,000 in the UK due to high acquisition costs and uncertainty in the evidence base [ 9 , 10 , 15 ].…”

Section: Discussionmentioning

confidence: 99%

“…Whilst the scope of our analysis did not allow for separation of oncology orphan medicines, there are indicators that these may be less disadvantaged than other orphan medicines. For example, they have been shown to have comparable evidence standards to non-orphan oncology products, and HTA decisions for oncology medicines can be influenced if they meet NICE criteria for End-of-Life [ 15 ]. Further analysis to identify the group of orphan medicines remaining at a disadvantage under the current NICE methods would be worthwhile to inform the NMR so that adequate provision can be made to ensure equity of access for all patients with rare conditions.…”

Section: Discussionmentioning

confidence: 99%

The impact of rarity in NICE’s health technology appraisals

Clarke

Ellis

Brownrigg

2021

Orphanet J Rare Dis

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Background In the absence of a framework designed to evaluate medicines for rare diseases in the UK, most orphan medicines are appraised by the National Institute for Health and Care Excellence (NICE) through the Single Technology Appraisal (STA) process. Results An analysis of STA appraisals of orphan and non-orphan medicines revealed that orphan medicines were subject to a significantly longer mean time in the NICE process than non-orphan medicines [370 days (n = 44) vs. 277 days (n = 118), p = < 0.0001]. A higher proportion of orphan STAs required more than one Appraisal Committee Meeting (ACM) versus non-orphan STAs, and orphan STAs were disadvantaged by worse outcomes with respect to positive recommendations than those orphan medicines assessed by Highly Specialised Technology evaluation (HST). Conclusions The uncertainties inherent to developing orphan medicines may contribute to these disadvantages. Improved understanding of the challenges in drug development for orphan medicines and clearer guidance for decision makers on navigating uncertainty in the HTA process may promote greater equity in access to medicines across rare and common conditions.

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“…The previously published model is based on average R&D costs and average probabilities of failure during the development (phase I, II and III) phases from the published literature[ 10 ].However R&D costs may vary for low prevalence orphan drugs, as recruitment of sufficient patients may require more effort and time, which leads to higher costs [ 16 ]. In addition, the costs and probabilities of failure may be higher for the first on class new innovative drug for a disease, for which where there has only been best supportive care to treat the symptoms and complications of a disease.…”

Section: Methodsmentioning

confidence: 99%

Pricing of orphan drugs in oncology and rare diseases

Nuijten

Capri

2020

Journal of Market Access & Health Policy

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Objective: The objective of this paper is to determine an upper price limit for an orphan drug by taken a broader perspective and, including also other monetary and non-monetary values for the society. Methods: This model is based on the expected free cash flows and the required minimum rate of return for the investor. In addition we calculated an innovation premium resulting from cost savings due to the substitution effect and the monetary gain in QALYs of a new medicine. We selected Spinraza®, a first in class drug with only best supportive care as comparator, and Perjeta®, a first in class drug with already an actual treatment as comparator. Results: The results show that Spinraza® leads to an innovation premium of € 78,966 and Perjeta® shows an innovation premium of € 4,388, because there were no cost savings. The analyses show the outcomes are sensitive to discount rate for QALYs. Conclusion: The break-even price from only an investor perspective may not reflect the value of drug from a broader perspective. This study shows drug prices based on an innovation premium may be more representative of the actual value of innovation for the society.

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Establishing a reasonable price for an orphan drug

Cited by 60 publications

References 25 publications

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

The impact of rarity in NICE’s health technology appraisals

Pricing of orphan drugs in oncology and rare diseases

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