An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models

Pochopien, M; Paterak, Ewelina; Clay, E.; Janik, Justyna; Aballéa, Samuel; Biernikiewicz, Małgorzata; Toumi, Mondher

doi:10.1080/20016689.2021.2002006

Cited by 11 publications

(11 citation statements)

References 3 publications

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“…The economic evaluation for gene therapy products faces several challenges, including demonstrating clinical effectiveness and safety, extrapolating effects beyond clinical trial duration, and the inclusion of equity considerations 37. Concerns also include the heterogenicity of patients evaluated the validity and reliability of outcomes and cost estimations derived from incomplete clinical trials, the time horizon used, and the data extrapolation to predict the effect of gene therapies on long-term quality of life 37,38. Despite all the challenges, many gene therapy products are deemed cost-effective by different health technology assessment bodies in the USA and some European countries and are reimbursed based on this 36,39–43.…”

Section: Discussionmentioning

confidence: 99%

“…37 Concerns also include the heterogenicity of patients evaluated the validity and reliability of outcomes and cost estimations derived from incomplete clinical trials, the time horizon used, and the data extrapolation to predict the effect of gene therapies on long-term quality of life. 37,38 Despite all the challenges, many gene therapy products are deemed costeffective by different health technology assessment bodies in the USA and some European countries and are reimbursed based on this. 36,[39][40][41][42][43] Nevertheless, there is a need for a standardized economic model to assess the value of gene therapies for patients and society.…”

Section: Discussionmentioning

confidence: 99%

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Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency

et al. 2023

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Background: Gene therapy, altering the genes inside human cells, has recently emerged as an alternative for preventing and treating disease. Concerns have been expressed about the clinical value and the high cost of gene therapies.Objective: This study assessed the characteristics of the clinical trials, authorizations, and prices of gene therapies in the United States and the European Union.Research Design: We collected regulatory information from the Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and manufacturer-listed prices from the United States, UK, and Germany. Descriptive statistics and t tests were conducted in the study.Results: As of January 1, 2022, the FDA and EMA authorized 8 and 10 gene therapies, respectively. The FDA and EMA granted orphan designation to all gene therapies except talimogene laherparepvec. Pivotal clinical trials were nonrandomized, open level, uncontrolled, phase I-III, and included a limited number of patients. Study primary outcomes were mainly surrogate endpoints without demonstration of direct patient benefit. The price of gene therapies at market entry ranged from $200,064 to $2,125,000 million.Conclusions: Gene therapy is used to treat incurable diseases that affect only a small number of patients (orphan diseases). Based on this, they are approved by the EMA and FDA with insufficient clinical evidence to ensure safety and efficacy, in addition to the high cost.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency

et al. 2023

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“…The challenges with structured value assessments and downstream implementation remain largely unaddressed for precision child health. A review of HTAs for gene therapies across various agencies found discrepancies between their economic evaluations due to the lack of a generalized framework for gene therapies [107]. While this is currently being overcome by adjusting frameworks to accommodate for circumstances where evidence generation is challenging, the unsystematic methodology creates barriers to public access.…”

Section: Discussionmentioning

confidence: 99%

Translating Precision Health for Pediatrics: A Scoping Review

et al. 2023

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Precision health aims to personalize treatment and prevention strategies based on individual genetic differences. While it has significantly improved healthcare for specific patient groups, broader translation faces challenges with evidence development, evidence appraisal, and implementation. These challenges are compounded in child health as existing methods fail to incorporate the physiology and socio-biology unique to childhood. This scoping review synthesizes the existing literature on evidence development, appraisal, prioritization, and implementation of precision child health. PubMed, Scopus, Web of Science, and Embase were searched. The included articles were related to pediatrics, precision health, and the translational pathway. Articles were excluded if they were too narrow in scope. In total, 74 articles identified challenges and solutions for putting pediatric precision health interventions into practice. The literature reinforced the unique attributes of children and their implications for study design and identified major themes for the value assessment of precision health interventions for children, including clinical benefit, cost-effectiveness, stakeholder values and preferences, and ethics and equity. Tackling these identified challenges will require developing international data networks and guidelines, re-thinking methods for value assessment, and broadening stakeholder support for the effective implementation of precision health within healthcare organizations. This research was funded by the SickKids Precision Child Health Catalyst Grant.

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“…Benefits are usually expressed in terms of quality-adjusted life-years (QALYs), a measure incorporating both the quality and quantity of life, created by multiplying every life year with a utility weight (ranging between 0 and 1) reflecting healthrelated quality of life [59]. The time horizon for the assessment varies; however, for gene therapies, and other interventions with expected long-term benefits, the full lifetime of the patient is usually considered relevant [52,60] (across which benefits and costs are measured and discounted to present values [61]). In the case above, the outcome of the economic evaluation is expressed as the incremental costeffectiveness ratio (ICER), C/ E, or "cost per QALY gained".…”

Section: Health Technology Assessment Of Gene Therapiesmentioning

confidence: 99%

“…Similar to assessment informing regulatory approval, the lack of robust efficacy and safety data for many gene therapies is a challenge also for HTA. Moreover, because of their very high prices, gene therapies are generally not found to be cost-effective when tested against conventional WTP thresholds [60,62]. For these reasons, voices have been raised to also considered other factors in the reimbursement appraisal of gene therapies to facilitate patient access, including but not limited to the potential additional value of (1) curing a disease (as opposed to chronic treatment administration), (2) treating a very severe disease (as opposed to a nonfatal, less debilitating illness), (3) treating a rare disease (as opposed to a common condition), and (4) treating a disease with a very high unmet medical need (as opposed to an illness with many efficacious treatment options).…”

Section: Health Technology Assessment Of Gene Therapiesmentioning

confidence: 99%

Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives

Landfeldt

2022

Journal of Neuromuscular Diseases

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Highly efficacious, potentially curative gene therapies holds immense clinical promise, but also present complex challenges. At the time of regulatory approval and health technology assessment (HTA), evidence of efficacy and safety of gene therapies is often uncertain. In addition, research, development, and manufacturing costs, small pools of eligible patients, and the fact that many gene therapies are administered only once means that they frequently are associated with very high “one-off” price points. Although only a limited number of products have been brought to market globally, hundreds of clinical trials of gene therapies, including several of monogenetic neuromuscular diseases, are currently ongoing. Over time, as more and more conditions become amendable to gene therapy, the number of transformative, high-cost treatments is likely to increase considerably. For these reasons, concerns have been raised regarding the suitability of current health policy systems, including HTA frameworks, in ensuring appropriate access to these therapeutic innovations while simultaneously safeguarding value for taxpayers’ money, as well as affordability and sustainability. This review provides a summary overview of current challenges and future perspectives of gene therapies for neuromuscular diseases from a health economic point of view.

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An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models

Cited by 11 publications

References 3 publications

Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency

Analysis of the Gene Therapies Authorized by the United States Food and Drug Administration and the European Medicines Agency

Translating Precision Health for Pediatrics: A Scoping Review

Gene Therapy for Neuromuscular Diseases: Health Economic Challenges and Future Perspectives

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