Research Topic

Assessing the Value of Novel Gene Therapies: The role of Health Technology Assessment

About this Research Topic

There is expected to be an explosion of novel gene therapies hitting the market in the coming years. Gene therapies have the potential for substantial patient and caregiver benefit, for disease transformation or cure, often in rare, severely disabling and life threatening diseases. However, they come with a high upfront price tag. Demonstrating cost-effectiveness is difficult and highly uncertain to the point that conventional health technology assessment (HTA) using cost utility analysis and cost per quality-adjusted life year (QALY) gained threshold criteria, whilst feasible to perform, is itself costly and arguably of limited value for reliable decision making.

Work performed by HTA bodies such as the National Institute for Health and Care Excellence (NICE) in the UK and the Institute for Clinical and Economic Review (ICER) in the US has demonstrated that it is possible to perform conventional economic evaluation on gene therapies. However, an alternative proposal is to bring key stakeholders together (payers, clinicians, patients) in order to negotiate a fair and affordable price, and a payment plan that can spread costs over time whilst monitoring patient outcomes.

Is there a halfway compromise, involving the use of wider value frameworks than is typically encompassed by conventional economic evaluation methods? This Research Topic will explore these issues and dilemmas of how best to move forward with the economic evaluation and health technology assessment of gene therapies. Manuscripts (including empirical, methods, policy based, or reviews, commentaries and opinion papers) that address one or more of the following or related issues are welcomed:

• The conduct or role of conventional HTA and economic evaluation to aid decision making for access to new gene therapies?
• Alternative models to conventional HTA for determining access to new gene therapies;
• Cost-effectiveness analyses of gene therapies;
• The value criteria that could be taken into account when appraising new gene therapies for market access;
• The role of patient stakeholder in the evaluation of new gene therapies;
• How to pay for new gene therapies: access versus affordability;
• The measurement of long term outcomes and benefits of gene therapies;
• Discounting of costs and benefits of gene therapies in economic evaluations;
• Challenges of designing clinical trials of gene therapies to be useful for HTA.

Francis Pang is employed by Orchard Therapeutics. Keith Tolley has previously received funding from pharmaceutical companies involved in gene therapy for research projects and consultancy (most notably Audentes Therapeutics). All other Topic Editors have no conflicts of interest to declare.


Keywords: Gene therapy, HTA, economic evaluation, cost, cure


Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.

There is expected to be an explosion of novel gene therapies hitting the market in the coming years. Gene therapies have the potential for substantial patient and caregiver benefit, for disease transformation or cure, often in rare, severely disabling and life threatening diseases. However, they come with a high upfront price tag. Demonstrating cost-effectiveness is difficult and highly uncertain to the point that conventional health technology assessment (HTA) using cost utility analysis and cost per quality-adjusted life year (QALY) gained threshold criteria, whilst feasible to perform, is itself costly and arguably of limited value for reliable decision making.

Work performed by HTA bodies such as the National Institute for Health and Care Excellence (NICE) in the UK and the Institute for Clinical and Economic Review (ICER) in the US has demonstrated that it is possible to perform conventional economic evaluation on gene therapies. However, an alternative proposal is to bring key stakeholders together (payers, clinicians, patients) in order to negotiate a fair and affordable price, and a payment plan that can spread costs over time whilst monitoring patient outcomes.

Is there a halfway compromise, involving the use of wider value frameworks than is typically encompassed by conventional economic evaluation methods? This Research Topic will explore these issues and dilemmas of how best to move forward with the economic evaluation and health technology assessment of gene therapies. Manuscripts (including empirical, methods, policy based, or reviews, commentaries and opinion papers) that address one or more of the following or related issues are welcomed:

• The conduct or role of conventional HTA and economic evaluation to aid decision making for access to new gene therapies?
• Alternative models to conventional HTA for determining access to new gene therapies;
• Cost-effectiveness analyses of gene therapies;
• The value criteria that could be taken into account when appraising new gene therapies for market access;
• The role of patient stakeholder in the evaluation of new gene therapies;
• How to pay for new gene therapies: access versus affordability;
• The measurement of long term outcomes and benefits of gene therapies;
• Discounting of costs and benefits of gene therapies in economic evaluations;
• Challenges of designing clinical trials of gene therapies to be useful for HTA.

Francis Pang is employed by Orchard Therapeutics. Keith Tolley has previously received funding from pharmaceutical companies involved in gene therapy for research projects and consultancy (most notably Audentes Therapeutics). All other Topic Editors have no conflicts of interest to declare.


Keywords: Gene therapy, HTA, economic evaluation, cost, cure


Important Note: All contributions to this Research Topic must be within the scope of the section and journal to which they are submitted, as defined in their mission statements. Frontiers reserves the right to guide an out-of-scope manuscript to a more suitable section or journal at any stage of peer review.

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Submission Deadlines

30 June 2020 Abstract
30 September 2020 Manuscript

Participating Journals

Manuscripts can be submitted to this Research Topic via the following journals:

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Topic Editors

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Submission Deadlines

30 June 2020 Abstract
30 September 2020 Manuscript

Participating Journals

Manuscripts can be submitted to this Research Topic via the following journals:

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