Is the world ready for gene therapy?

Garrison, Louis P.; Kleinermans, Diane

doi:10.1111/hae.14439

Cited by 5 publications

(4 citation statements)

References 20 publications

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“…The key high‐level findings are that GTTs present unique data and policy challenges, and that existing regulatory, HTA, as well as payment and financing systems will need to adapt. Essential changes will include greater patient‐centeredness, flexibility in evidence requirements and payment models, and collaboration among stakeholders (Garrison & Kleinermans, 2022). But these adjustments can build on our existing foundation of regulatory and incentive systems for innovation, and much can be done to accelerate progress in this important field as illustrated by the recommendations of the three working groups.…”

Section: Discussionmentioning

confidence: 99%

A review of economic issues for gene‐targeted therapies: Value, affordability, and access

Garrison

Lo²,

Finkel

et al. 2023

American J of Med Genetics Pt C

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The National Center for Advancing Translational Sciences' virtual 2021 conference on gene-targeted therapies (GTTs) encouraged multidisciplinary dialogue on a wide range of GTT topic areas. Each of three parallel working groups included social scientists and clinical scientists, and the three major sessions included a presentation on economic issues related to their focus area. These experts also coordinated their efforts across the three groups. The economics-related presentations covered three areas with some overlap: (1) value assessment, uncertainty, and dynamic efficiency;(2) affordability, pricing, and financing; and (3) evidence generation, coverage, and access. This article provides a synopsis of three presentations, some of their key recommendations, and an update on related developments in the past year. The key high-level findings are that GTTs present unique data and policy challenges, and that existing regulatory, health technology assessment, as well as payment and financing systems will need to adapt. But these adjustments can build on our existing foundation of regulatory and incentive systems for innovation, and much can be done to accelerate progress in GTTs. Given the substantial unmet medical need that exists for these oft-neglected patients suffering from rare diseases, it would be a tragedy to not leverage these exciting scientific advances in GTTs.

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Section: Discussionmentioning

confidence: 99%

A review of economic issues for gene‐targeted therapies: Value, affordability, and access

Garrison

Lo²,

Finkel

et al. 2023

American J of Med Genetics Pt C

Self Cite

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“…The question that the scientific community often discusses is: Are we ready for gene therapy? 24 , 25 In other words, how can we insure a smooth transition from existing practice to gene therapy?…”

Section: Are We Ready For Gene Therapy?mentioning

confidence: 99%

How to translate and implement the current science of gene therapy into haemophilia care?

Hermans

Gruel

Frenzel

et al. 2023

Therapeutic Advances in Hematology

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Gene-based therapy opens an entirely new paradigm in managing people with haemophilia (PWH), offering them the possibility of a functional cure by enabling continuous expression of factor VIII (FVIII) or factor IX (FIX) after transfer of a functional gene designed to replace the PWH’s own defective gene. In recent years, significant advances in gene therapy have been made, resulting in clotting factor activity attaining near-normal levels, as reflected by ‘zero bleeding rates’ in previously severely inflicted patients following a single administration of adeno-associated viral (AAV) vectors. While this new approach represents a major advancement, there are still several issues that must be resolved before applying this technology in clinical practice. First, awareness, communication, and education about the therapeutic potential and modalities of gene therapy must be further strengthened. To this end, objective, unbiased, transparent, and regularly updated information must be shared, in an appropriate way and understandable language with the support of patients’ organizations. Second, healthcare providers should adopt a patient-centred approach, as the ‘one size fits all’ approach is inappropriate when considering gene therapy. Instead, a holistic patient view taking into account their physical and mental dimensions, along with unexpressed expectations and preferences, is mandatory. Third, the consent procedure must be improved, ensuring that patients’ interests are maximally protected. Finally, gene therapy is likely to be first delivered in a few centres, with the highest expertise and experience in this domain. Thus, patients should be managed based on a hub-and-spoke model, taking into account that the key to gene therapy’s success lies in an optimal communication and collaboration both within and between haemophilia centres sharing their experiences in the frame of international registries. This review describes recent progress and explains outstanding hurdles that must be tackled to ease the implementation of this paradigm-changing new therapy.

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“…Affecting approximately 1:3,500 births worldwide [4] , there are approximately 1.25 million men in the world with haemophilia, 420,000 of whom have severe haemophilia [5] . Current treatments consist of factor replacement, coagulation pathway rebalancing or, in the near future, gene therapy [6][7][8] . Haemophilia and its treatment come with significant individual, family and organisational burdens [9,10] .…”

Section: Case Study Simon Fletchermentioning

confidence: 99%

“I didn’t know women could have haemophilia”: A qualitative case study

Fletcher

2022

The Journal of Haemophilia Practice

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Introduction There is a historic but persistent belief in haemophilia care that women do not suffer with the condition, they merely carry and transmit it. However, around 250 women worldwide are known to have factor levels within the severe to moderate haemophilia range (<1 IU/dL to 5 IU/dL), and the true figure may be greater than this. The experience of these women may be the same as or similar to those of men with similar factor levels, but there may be significant differences. What these differences are and what they mean to the women affected are not well understood as their voices are not heard. This case study highlights the issues and experiences of one woman living severe haemophilia. Methods A single semi-structured qualitative interview was undertaken to explore the experiences of a young woman who has factor VIII levels of <1 IU/dL. The interview was recorded, transcribed and thematically analysed. Results Four interlinked themes were identified: recognition, self-advocacy, identity and access to treatment. Conclusion This case study indicates that, despite recent attempts to improve the diagnostic nomenclature, women and girls with haemophilia continue to find it difficult to access similar levels of care to men and boys. As such, they may fail to achieve parity in terms of safety, integrity and wellbeing, and have a reduced quality of life. If women and girls affected by haemophilia are to receive levels of treatment comparable to men, diagnostic criteria need to change further. Focusing on genotype, levels of factor expressed and phenotypical presentation rather than biological sex will acknowledge and validate their experiences, and improve treatment for all people with haemophilia in the future.

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Is the world ready for gene therapy?

Cited by 5 publications

References 20 publications

A review of economic issues for gene‐targeted therapies: Value, affordability, and access

A review of economic issues for gene‐targeted therapies: Value, affordability, and access

How to translate and implement the current science of gene therapy into haemophilia care?

“I didn’t know women could have haemophilia”: A qualitative case study

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