Health Technology Assessment of Gene Therapies in Europe and the USA: Analysis and Future Considerations

Qiu, Tingting; Hanna, E; Dabbous, Monique; Borislav, Borisov; Toumi, Mondher

doi:10.18609/cgti.2019.112

Cited by 8 publications

(7 citation statements)

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“…Despite this, to date, most gene therapies have successfully been granted reimbursement, with more or less delay in terms of assessment timelines, as described elsewhere. 44 In less extent, ethical and social aspects related to the use of genetic therapy also seem to impact patient access. It became clear that the more serious a medical condition is, the more likely the patient is willing to use gene therapy.…”

Section: Discussionmentioning

confidence: 99%

“…44 45 In Scotland, Kymriah was accepted for B-cell acute lymphoblastic leukaemia treatment with a patient access scheme, while Yescarta and Kymriah for diffuse large B-cell lymphoma were rejected due to unjustified cost-effectiveness estimates. 44 In Germany, three GTMPs had 'non-quantifiable added benefit' due to insufficient data (Glybera, Yescarta and Kymriah) and Imlygic had 'no-added benefit' due to inappropriate comparator use. However, this did not limit its reimbursement.…”

Section: Current Gtmp Patient Access Landscape In Europe and Usamentioning

confidence: 99%

“…However, this did not limit its reimbursement. 44 45 Three GTMPs were reimbursed in France (Yescarta, Kymriah and Luxturna), while Glybera was not recommended, as it was considered to have 'insufficient' benefit due to its unsustainable and heterogeneous treatment effects. 44 45 In Italy, one GTMP was reimbursed for hospital use with managed entry agreement (Strimvelis).…”

Section: Current Gtmp Patient Access Landscape In Europe and Usamentioning

confidence: 99%

“…45 In the USA, Kymriah, Yescarta, Luxturna and Zolgensma were evaluated as having substantial net health benefits. However, a high certainty of conclusion for the assessment of Zolgensma was established 44 . No data on Zynteglo were available for any of the EU5 countries either because the assessment is in progress or not assessed at all.…”

Section: Current Gtmp Patient Access Landscape In Europe and Usamentioning

confidence: 99%

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Patient access to gene therapy medicinal products: a comprehensive review

2020

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BackgroundGene therapies have the potential to be a curative approach to a large number of genetic diseases. However, granting of a positive marketing authorisation does not equal patient access to therapy.ObjectivesThe purpose of this paper is to identify a full set of hurdles potentially preventing patient access to gene therapies based on the available literature.MethodsA review of the literature using systematic approach in two distinct databases was performed by identifying relevant, peer-reviewed publications, between 2012 and 2018.ResultsSeven major topics were identified as potential patient access hurdles, namely affordability, assessment of value, development of therapy, ethical/social factors, evidence generation, operational implementation and regulatory hurdles. From these, 25 additional subthemes were further identified. The most frequently mentioned obstacle in the literature is related to the affordability aspect especially focusing on high cost of therapy (84%) and therapy payment/reimbursement (51%). Importantly, the evidence generation focusing on limited trial outcomes (81%) seems as a strong obstacle in patient access to these therapies.ConclusionsA growing number of gene therapies are expected to be developed and made available to patients and healthcare professionals. Improvement of patient access to gene therapies can only be achieved by understanding all hurdles, in a complete and integrated fashion, so that strategies are timely established to ensure gene therapies’ benefits are provided to patients and to the society.

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Section: Discussionmentioning

confidence: 99%

Section: Current Gtmp Patient Access Landscape In Europe and Usamentioning

confidence: 99%

Section: Current Gtmp Patient Access Landscape In Europe and Usamentioning

confidence: 99%

Section: Current Gtmp Patient Access Landscape In Europe and Usamentioning

confidence: 99%

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Patient access to gene therapy medicinal products: a comprehensive review

2020

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“…Gene therapies (GTs) have shown extraordinary promise for the mitigation or potential cure of a broad spectrum of life-threatening or debilitating diseases, such as cancers and retinal disorders (1). Yet, despite the innovative treatment paradigm and exceptional clinical benefits potentially provided by some GTs (2,3), numerous obstacles to efficient market access prevail (4). These obstacles range from resource-consuming manufacturing processes to reimbursement and funding challenges (4)(5)(6)(7)(8).…”

Section: Introductionmentioning

confidence: 99%

Gene Therapy Evidence Generation and Economic Analysis: Pragmatic Considerations to Facilitate Fit-for-Purpose Health Technology Assessment

Qiu

Pochopien²,

Liang

et al. 2022

Front. Public Health

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Gene therapies (GTs) are considered to be a paradigm-shifting class of treatments with the potential to treat previously incurable diseases or those with significant unmet treatment needs. However, considerable challenges remain in their health technology assessment (HTA), mainly stemming from the inability to perform robust clinical trials to convince decision-makers to pay the high prices for the potential long-term treatment benefits provided. This article aims to review the recommendations that have been published for evidence generation and economic analysis for GTs against the feasibility of their implementation within current HTA decision analysis frameworks. After reviewing the systematically identified literature, we found that questions remain on the appropriateness of GT evidence generation, considering that additional, broader values brought by GTs seem insufficiently incorporated within proposed analytic methods. In cases where innovative methods are proposed, HTA organizations remain highly conservative and resistant to change their reference case and decision analysis framework. Such resistances are largely attributed to the substantial evidence uncertainty, resource-consuming administration process, and the absence of consensus on the optimized methodology to balance all the advantages and potential pitfalls of GTs.

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