Sponsorship and Funding for Gene Therapy Trials in the United States

Kassir, Zachary; Sarpatwari, Ameet; Kocak, Brian R.; Kuza, Courtney C.; Gellad, Walid F.

doi:10.1001/jama.2019.22214

Cited by 10 publications

(5 citation statements)

References 6 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…24 A 2020 study shows that in the United States, 36% of gene therapy trials were funded solely by industry. 44 However, one often overlooked fact is that the composition of “industry” or “startups” is increasingly fluid. They may consist of new entrants who have limited professional training or experience in either the life sciences or in business, or both.…”

Section: Commoning the Science At Largementioning

confidence: 99%

Commoning genomic solidarity to improve global health equality

Zhang

2023

Cell Genomics

View full text Add to dashboard Cite

Section: Commoning the Science At Largementioning

confidence: 99%

Commoning genomic solidarity to improve global health equality

Zhang

2023

Cell Genomics

View full text Add to dashboard Cite

“…Concerning research and development, it is important to highlight that far from all costs associated with clinical programs of gene therapies are carried by pharmaceutical companies. A recent review of sponsorship and funding for gene therapy trials in the US [ 30 ] revealed that 10% of all trials were sponsored by the National Institutes of Health (NIH), 40% by the industry, 25% by hospitals, and 25% by universities. In total, 36% of all trials were funded solely by pharmaceutical companies, and 50% by academia or the NIH; however, the industry sponsored all phase III trials [ 30 ].…”

Section: Gene Therapymentioning

confidence: 99%

“…A recent review of sponsorship and funding for gene therapy trials in the US [ 30 ] revealed that 10% of all trials were sponsored by the National Institutes of Health (NIH), 40% by the industry, 25% by hospitals, and 25% by universities. In total, 36% of all trials were funded solely by pharmaceutical companies, and 50% by academia or the NIH; however, the industry sponsored all phase III trials [ 30 ]. Accordingly, total research and development costs are commonly shared across multiple stakeholders, which should be considered in relation to decisions on commercial price points.…”

Section: Gene Therapymentioning

confidence: 99%

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

Landfeldt

2022

JND

View full text Add to dashboard Cite

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.

show abstract

“…Academia remains very active in the early phases of clinical trials designed to evaluate innovative GTMPs as potential complements, substitutes, or bridges to historical forms of haematopoietic cell transplants. One recent study calculated that even now, when industry interest in these therapies has increased significantly in the last 5-6 years, over 50% of CAR-T cell trials in the USA are still sponsored by academia (Kassir et al 2020). Many public institutions have invested significant resources to upgrade their processing facilities to GMP-compliant levels, thus allowing for small-scale manufacturing of experimental medicinal products to support phase I and possibly phase II studies.…”

Section: Role Of Academiamentioning

confidence: 99%

The Regulatory Framework for CAR-T Cells in Europe: Current Status and Foreseeable Changes AND Centre Qualification by Competent Authorities and Manufacturers

McGrath¹,

Machalik

2022

The EBMT/EHA CAR-T Cell Handbook

View full text Add to dashboard Cite

Under current European Union regulations, CAR-T cell therapies fall under the advanced therapy medicinal products (ATMPs) framework. ATMPs represent a category of medicinal products defined in EU Regulation 1394/2007 and subdivided into four categories, of which autologous or allogeneic CAR-T cells, among other therapies, are considered gene therapy medicinal products (GTMPs). ATMPs are subject to a centralized evaluation framework whereby one authorization is valid for all countries in the EU led by the European Medicines Agency’s Committee for Advanced Therapies (CAT). The framework includes different regulatory pathways for bringing ATMPs from clinical trials to market authorization, and the regulatory pathway taken will depend on a product’s characteristics and the target patient population. In 2018, two chimeric antigen receptor (CAR) T cell therapies, Yescarta and Kymriah, completed their authorization process via the priority medicines PRIME scheme to Marketing Authorization (Detela and Lodge 2019).

show abstract

Sponsorship and Funding for Gene Therapy Trials in the United States

Cited by 10 publications

References 6 publications

Commoning genomic solidarity to improve global health equality

Commoning genomic solidarity to improve global health equality

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

The Regulatory Framework for CAR-T Cells in Europe: Current Status and Foreseeable Changes AND Centre Qualification by Competent Authorities and Manufacturers

Contact Info

Product

Resources

About