The payers' perspective on gene therapies

Touchot, Nicolas; Flume, Mathias

doi:10.1038/nbt.3332

Cited by 20 publications

(10 citation statements)

References 3 publications

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“…Although most of the gene therapy programmes remain in the early stages of development, healthcare economists are generating models to cost treatments, which might provide lifelong cures. A pay-for-performance system has been proposed with yearly-capped annuity paid to the pharmaceutical company if criteria of a metabolic control of the disease are met (Touchot and Flume 2015). These criteria might reflect cost-effectiveness and not only cost-saving.…”

Section: Current Challengesmentioning

confidence: 99%

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

Baruteau

Waddington

Alexander

et al. 2017

J of Inher Metab Disea

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Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have achieved sustained clinical improvement after a single systemic injection of adeno-associated virus (AAV) derived vectors encoding the human factor IX cDNA. These trials demonstrate the potential of AAV technology to provide long-lasting clinical benefit in the treatment of monogenic liver disorders. Indeed, with more than ten ongoing or planned clinical trials for haemophilia A and B and dozens of trials planned for other inherited genetic/metabolic liver diseases, clinical translation is expanding rapidly. Gene therapy is likely to become an option for routine care of a subset of severe inherited genetic/metabolic liver diseases in the relatively near term. In this review, we aim to summarise the milestones in the development of gene therapy, present the different vector tools and their clinical applications for liver-directed gene therapy. AAV-derived vectors are emerging as the leading candidates for clinical translation of gene delivery to the liver. Therefore, we focus on clinical applications of AAV vectors in providing the most recent update on clinical outcomes of completed and ongoing gene therapy trials and comment on the current challenges that the field is facing for large-scale clinical translation. There is clearly an urgent need for more efficient therapies in many severe monogenic liver disorders, which will require careful risk-benefit analysis for each indication, especially in paediatrics.

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Section: Current Challengesmentioning

confidence: 99%

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

Baruteau

Waddington

Alexander

et al. 2017

J of Inher Metab Disea

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“…It is therefore likely that price structures and valuation models may change in the future. For example, payers have suggested innovative pricing strategies for gene therapies which may lead to price levels that are more sustainable in the long term [55]. The question of how to fund new therapies equitably is a major issue at present, and is likely to become even more challenging in the future, with the advent of expensive, innovative, breakthrough treatments.…”

Section: Discussionmentioning

confidence: 99%

Ethical Hurdles in the Prioritization of Oncology Care

Groot¹,

Capri

Castanier³

et al. 2016

Appl Health Econ Health Policy

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With finite resources, healthcare payers must make difficult choices regarding spending and the ethical distribution of funds. Here, we describe some of the ethical issues surrounding inequity in healthcare in nine major European countries, using cancer care as an example. To identify relevant studies, we conducted a systematic literature search. The results of the literature review suggest that although prevention, access to early diagnosis, and radiotherapy are key factors associated with good outcomes in oncology, public and political attention often focusses on the availability of pharmacological treatments. In some countries this focus may divert funding towards cancer drugs, for example through specific cancer drugs funds, leading to reduced expenditure on other areas of cancer care, including prevention, and potentially on other diseases. In addition, as highly effective, expensive agents are developed, the use of value-based approaches may lead to unacceptable impacts on health budgets, leading to a potential need to re-evaluate current cost-effectiveness thresholds. We anticipate that the question of how to fund new therapies equitably will become even more challenging in the future, with the advent of expensive, innovative, breakthrough treatments in other therapeutic areas.Electronic supplementary materialThe online version of this article (doi:10.1007/s40258-016-0288-4) contains supplementary material, which is available to authorized users.

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“…Cell and gene therapies have very high price (22,23) making challenging for public and private healthcare systems to provide coverage of those product. (24,25) Alipogene tiparvovec was withdrawn from the market in 2017 after the Health Technology Assessment agencies of Germany and France rejected public health insurance coverage of this therapy indicated for lipoprotein lipase deficiency, an ultra-rare disease in Europe. (26,27) And the product was not assessed for public health insurance coverage in any other European country.…”

Section: Market Discontinuations Of Cell and Gene Therapy Productsmentioning

confidence: 99%

The Landscape of Cellular and Gene Therapy Products: Authorization, Discontinuations, and Cost

Shukla

Seoane‐Vazquez

Fawaz

et al. 2019

Human Gene Therapy Clinical Development

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The payers' perspective on gene therapies

Cited by 20 publications

References 3 publications

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects

Ethical Hurdles in the Prioritization of Oncology Care

The Landscape of Cellular and Gene Therapy Products: Authorization, Discontinuations, and Cost

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