Gene therapy companies have an ethical obligation to develop expanded access policies

Kearns, Lisa S.; Chapman, C. P.; Moch, Kenneth I.; Caplan, Arthur L.; Watson, Tom; McFadyen, Andrew; Furlong, Pat; Bateman-House, Alison

doi:10.1016/j.ymthe.2021.03.008

Cited by 12 publications

(9 citation statements)

References 6 publications

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“…The findings also suggest that accessing experimental products from smaller and newer companies may be more challenging for patients and clinicians, given these companies are less likely to have publicly available EA policies. This finding is consistent with prior articles on EA, which described several practical, legal, and ethical challenges associated with implementing expanded access 11–15 . In particular, drug company participation in EA may be limited due to administrative burden, as EA can require significant human resources 12 .…”

Section: Discussionsupporting

confidence: 88%

“…This finding is consistent with prior articles on EA, which described several practical, legal, and ethical challenges associated with implementing expanded access. [11][12][13][14][15] In particular, drug company participation in EA may be limited due to administrative burden, as EA can require significant human resources. 12 There is a need to improve awareness about processes and procedures for seeking EA [16][17][18] and better enforce Cures Act EA policy requirements.…”

Section: Articlementioning

confidence: 99%

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Implementation of 21st Century Cures Act Expanded Access Policies Requirements

Kang

Chang

Ross

et al. 2021

Clin Pharma and Therapeutics

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The US Food and Drug Administration (FDA) expanded access pathway allows patients with life-threatening or serious conditions to access investigational drugs outside of trials, under certain conditions. The 21st Century Cures Act ("Cures Act") requires certain drug companies to publicly disclose their expanded access policies. We characterized the proportion of applicable US biopharmaceutical companies, with an oncology related drug, implementing Cures Act requirements for expanded access policies and whether available policies contain the information described in the Act. We found about one-third of applicable biopharmaceutical companies (32%, 140/423) implemented the Cures Act requirement to have a public expanded access policy. Less than one-third of public policies contained all described information (31%, 44/140). Larger companies and those with at least one drug receiving an FDA expedited designation (59% vs. 21%; P < 0.001), or at least one FDA-approved drug (57% vs. 28%; P < 0.001) were more likely to have a public policy. Our results suggest the Cures Act may be having a limited impact on its goals of supporting timely medical decisions and closing informational gaps for patients and doctors around expanded access to investigational oncology therapies, especially for products sponsored by smaller and newer companies.The US Food and Drug Administration (FDA) expanded access (EA) pathway, sometimes also called compassionate use, is a route for patients with immediately life-threatening or serious diseases or conditions to potentially access experimental medicines outside of clinical trials. To qualify, patients must be unable to enroll in a relevant clinical trial, have no comparable satisfactory alternative FDA approved therapy options, and be deemed more likely to experience benefit than risk from the unapproved intervention. Additionally, expanded access provisions should not threaten a product's development program (e.g., trial enrollment). Formalized in 1987, in the context of the AIDS epidemic, the EA pathway has been amended multiple times, most recently in 2016 through section 561 of the Food, Drug, and Cosmetics Act (FDCA), as a part of the 21st Century Cures Act ("Cures Act").The Cures Act aimed to help increase awareness among patients and clinicians about the processes and procedures for accessing

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Section: Discussionsupporting

confidence: 88%

Section: Articlementioning

confidence: 99%

Implementation of 21st Century Cures Act Expanded Access Policies Requirements

Kang

Chang

Ross

et al. 2021

Clin Pharma and Therapeutics

View full text Add to dashboard Cite

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“…We hope that their work and the above considerations may positively impact manufacturers' decisions to provide expanded access. Either way, we fully agree with Kearns et al 1 that expanded access policy should be transparently explained and publicly accessible.…”

supporting

confidence: 74%

“…We wholeheartedly support the message by Kearns et al 1 that "gene therapy companies have an ethical obligation to develop expanded access policies." The authors discuss several important factors that companies should consider when developing expanded access policies: clinical trial design, potential for data collection, risk-benefit analysis, and impact on program feasibility and profitability.…”

mentioning

confidence: 56%

Financial considerations in expanded access policy for gene therapies: A tough nut to crack?

Polak

Bunnik

2021

Molecular Therapy

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“…In this context, we propose to broaden current WHO registration data elements specifically for HGE trials by including ICF, as well as expanded access information, which should follow the appropriate FDA regulations (21 CFR 312) [ 51 ]. Despite the value of data collected during the compassionate use of conventional drugs is mostly considered as limited [ 52 ], non-trial preapproval use might provide important information on outcomes and AEs related to this unique class of therapeutics, mostly intended for patient populations that may be small, and whose effects in most cases cannot be reversed [ 53 ]. Other steps required to improve the transparency of HGE trials are more demanding, since the WHO ICTRP platform gathers trial registration data sets provided by different primary registries, still not completely complying with the WHO TRDS [ 54 ].…”

Section: Discussionmentioning

confidence: 99%

Inadequate reporting quality of registered genome editing trials: an observational study

Jurić

Zlatin

Marušić

2022

BMC Med Res Methodol

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Background To assess registration completeness and safety data of trials on human genome editing (HGE) reported in primary registries and published in journals, as HGE has safety and ethical problems, including the risk of undesirable and unpredictable outcomes. Registration transparency has not been evaluated for clinical trials using these novel and revolutionary techniques in human participants. Methods Observational study of trials involving engineered site-specific nucleases and long-term follow-up observations, identified from the WHO ICTRP HGE Registry in November 2020 and two comprehensive reviews published in the same year. Registration and adverse events (AEs) information were collected from public registries and matching publications. Published data were extracted in May 2021. Results Among 81 eligible trials, most were recruiting (51.9%) phase 1 trials (45.7%). Five trials were withdrawn. Most trials investigated CAR T cells therapies (45.7%) and used CRISPR/Cas9 (35.8%) ex vivo (88.9%). Among 12 trials with protocols both registered and published, eligibility criteria, sample size, and secondary outcome measures were consistently reported for less than a half. Three trials posted results in ClinicalTrials.gov, and one reported serious AEs. Conclusions Incomplete registration and published data give emphasis to the need to increase the transparency of HGE trials. Further improvements in registration requirements, including phase 1 trials, and a more controlled publication procedure, are needed to augment the implementation of this promising technology.

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Gene therapy companies have an ethical obligation to develop expanded access policies

Cited by 12 publications

References 6 publications

Implementation of 21st Century Cures Act Expanded Access Policies Requirements

Implementation of 21st Century Cures Act Expanded Access Policies Requirements

Financial considerations in expanded access policy for gene therapies: A tough nut to crack?

Inadequate reporting quality of registered genome editing trials: an observational study

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