Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies

Garrison, Louis P.; Jiao, Boshen; Dabbous, O.

doi:10.18553/jmcp.2021.27.5.674

Cited by 19 publications

(22 citation statements)

References 37 publications

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“…Gene therapy could cause a great upheaval in the treatment of patients with hemophilia and other congenital diseases. Although more work is needed to increase treatment efficacy and reduce adverse events such as immunogenicity and hepatotoxicity, these protocols could allow curation of the disease, thereby increasing the patients’ quality of life, and a reduction in both direct and indirect costs throughout the lifetime of patients suffering from chronic lifelong diseases [ 54 ].…”

Section: Discussionmentioning

confidence: 99%

“…Although further economic studies are required to address this dilemma, it would be misleading to conclude that gene therapy is inherently more costly [ 54 ]. A rigorous economic evaluation of the new therapies requires a careful comparison between the costs and benefits of gene therapy and the standard of care without adverse events throughout the patient’s lifetime, including the relevant adjustments for price distortions.…”

Section: Cost-effectiveness Of Gene Therapy For Hemophiliamentioning

confidence: 99%

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Gene Therapy in Hemophilia: Recent Advances

Rodríguez-Merchán

Pablo-Moreno

Liras

2021

IJMS

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Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. The palliative treatment of choice is based on the use of safe and effective recombinant clotting factors. Advanced therapies will be curative, ensuring stable and durable concentrations of the defective circulating factor. Results have so far been encouraging in terms of levels and times of expression using mainly adeno-associated vectors. However, these therapies are associated with immunogenicity and hepatotoxicity. Optimizing the vector serotypes and the transgene (variants) will boost clotting efficacy, thus increasing the viability of these protocols. It is essential that both physicians and patients be informed about the potential benefits and risks of the new therapies, and a register of gene therapy patients be kept with information of the efficacy and long-term adverse events associated with the treatments administered. In the context of hemophilia, gene therapy may result in (particularly indirect) cost savings and in a more equitable allocation of treatments. In the case of hemophilia A, further research is needed into how to effectively package the large factor VIII gene into the vector; and in the case of hemophilia B, the priority should be to optimize both the vector serotype, reducing its immunogenicity and hepatotoxicity, and the transgene, boosting its clotting efficacy so as to minimize the amount of vector administered and decrease the incidence of adverse events without compromising the efficacy of the protein expressed.

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Section: Discussionmentioning

confidence: 99%

Section: Cost-effectiveness Of Gene Therapy For Hemophiliamentioning

confidence: 99%

Gene Therapy in Hemophilia: Recent Advances

Rodríguez-Merchán

Pablo-Moreno

Liras

2021

IJMS

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“…32 Regardless of the selection or design of an APM, purchasers need to have a means of establishing price. For example, the annual cost of standard prophylaxis care for haemophilia A in high-income countries is in the approximate range of $.3 -.5 million, and as much as $1.0 million or more for patients with inhibitors, [32][33][34][35][36][37] although national tenders, confidential discounts, and rebates in some countries introduce variability at the lower end of this range. The relevance of the higher cost of prophylaxis for patients with inhibitors as a comparator will depend on whether any gene therapies become available that are indicated for such patients.…”

Section: Costs and Analytical Time Horizonmentioning

confidence: 99%

Alternative payment models for durable and potentially curative therapies: The case of gene therapy for haemophilia A

Goodman

Berntorp

Wong³

2022

Haemophilia

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Introduction:The emergence of durable and potentially curative cell and gene therapies (also known as advanced therapy medicinal products) with high price tags is challenging conventional health care payment systems. Among these are gene therapies in various phases of development for haemophilia A and B. The emergence of these therapies comes with clinical and economic uncertainties for payers, providers, patients, and manufacturers. These include uncertainties about expression of the intended physiological response, patient outcomes, and duration of treatment effect, along with potentially high upfront costs, variable additional costs, and uncertainties about uptake of the therapy among indicated patient populations. These clinical and economic uncertainties raise interest among payers, providers, and manufacturers in risk-sharing arrangements, including alternative payment models (APMs).Summary: APMs differ from traditional fee-for-service payment for health care. For example, some APMs are designed to reward clinicians and provider organizations for delivering high-quality and cost-effective care. The APMs described here, outcomesbased models (or value-or performance-based models) and finance-based models, are designed to mitigate or otherwise manage financial risks associated with health care payment, including instances in which patient outcomes for costly therapies are uncertain. We examine how such APMs may be applicable in the context of emerging gene therapies for haemophilia A, including considerations in determining whether any particular APM may be most suitable.

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“…Yet the first gene therapy approved in the USA, Luxturna, approved in 2017 to treat Leber's congenital amaurosis, costs ~$850,000 to treat both eyes (Darrow, 2019). Zolgensma, approved in the USA in 2019 to treat spinal muscular atrophy, costs ~$2,125,000 per treatment (Garrison et al, 2021). Such prices give pause as to whether gene editing treatments for the 7,000 rare diseases will realistically be accessible to those who urgently need them, or if the limited demand would justify therapeutic development at all.…”

Section: The Most Threatening Challengementioning

confidence: 99%

“…However, most gene therapies are not valued based on cost of goods sold; rather, they are valued based on calculations such as quality-adjustedlife-years gained by treatment, and how much less expensive the treatment appears compared to life-long drug or molecular therapy (Zimmermann et al, 2019;Dean et al, 2021). For example, the high cost of ~$15, 000, 000 for treating a hemophilia A patient with factor VIII over the course of their life has been used to justify the comparatively lower cost of Zolgensma (Garrison et al, 2021). Such valuations based on more expensive treatments or monetization of quality-adjusted-life-years are not likely to change due to a reduction in cost of goods.…”

Section: The Most Threatening Challengementioning

confidence: 99%

The promise of gene editing: so close and yet so perilously far

Segal

2022

Front. Genome Ed.

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and indels are produced only by the action of the nuclease, have been deregulated in many countries. An exception are those countries within the European Union, where, despite being the third largest producer of genetically engineered crops behind China and the USA, SDN1 crops remain subject to the stringent regulations for genetically modified organisms (GMOs). Such stringent regulations are considered to have a dampening effect on agriculture innovation in the EU, and are perhaps similar to the dampening effect of long regulatory delays on the genetic engineering of livestock animals (Van Eenennaam et al., 2021). Since the first report of genetic engineering in livestock animals in 1985, only a single food animal has been commercialized. This is in part due to the USA Food and Drug Administration and their EU counterparts classifying any intentional altered genomic DNA in animals as an investigational new animal drug (INAD) that is not generally recognized as safe. However, there is a growing realization that the current EU policy towards SDN1 crops needs to be updated (Dima et al., 2022), giving hope to the wider use of these directed editing methods that can dramatically accelerate the production of new varieties compared to traditional breeding techniques.Interestingly, regulations have not hindered innovation in the application of genetic engineering to human health. In fact, this area has been a significant driver of technological advances. Recent publications and scientific meetings, such as the Keystone Symposium on Precision Genome Engineering and the American Society for Gene and Cell Therapy Annual Meeting, highlight the rapid advances in genome editing tools, driven in large part by a sense that new treatments for human disease enabled by these tools are just around the corner. Indeed, by some estimates there are over 100 products using genome editors now in clinical trial (CRISPR Medicine News), led by companies, such as CRISPR Therapeutics, Intellia Therapeutics, Sangamo Therapeutics, Editas Medicine, Precision Biosciences, Caribou Biosciences, Locus Biosciences, and many others. In the academic sector, Phase 1 of the NIH Somatic Cell Genome Editing Consortium (Saha et al., 2021), which had focused primarily on developing new editors and delivery methods, has led to a Phase 2 that is primarily focused on using these tools to

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Gene therapy may not be as expensive as people think: challenges in assessing the value of single and short-term therapies

Cited by 19 publications

References 37 publications

Gene Therapy in Hemophilia: Recent Advances

Gene Therapy in Hemophilia: Recent Advances

Alternative payment models for durable and potentially curative therapies: The case of gene therapy for haemophilia A

The promise of gene editing: so close and yet so perilously far

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