What compassionate use means for gene therapies

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

doi:10.1038/s41587-019-0081-7

Cited by 17 publications

(13 citation statements)

References 8 publications

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“…Similarly for the latter, where EA programmes were traditionally also meant exclusively for treatment, data collection and thereby research has become a reality. The changing position of EA patients from treatment subjects to (partly) research subjects, provides a challenge for bioethicists 11,12,33 …”

Section: Discussionmentioning

confidence: 99%

“…Historically, however, EA programmes were only deemed fit for treatment and not for research. Although the primary purpose of EA is treatment, scholars have argued that there is a moral obligation to collect outcome data in all cases where patients are treated with investigational medicine 11–13 . The debate on combining data collection and EA has substantially increased, 14–16 with FDA officials confirming, beginning 2018, their willingness to review data from EA programmes to support drug applications 11 …”

Section: Introductionmentioning

confidence: 99%

“…Although the primary purpose of EA is treatment, scholars have argued that there is a moral obligation to collect outcome data in all cases where patients are treated with investigational medicine. [11][12][13] The debate on combining data collection and EA has substantially increased, [14][15][16] with FDA officials confirming, beginning 2018, their willingness to review data from EA programmes to support drug applications. 11 Considering the increasing interest in both EA and RWD, the question arises of whether alternative routes of access to novel treatments can provide clinical information and impact regulatory decision making.…”

Section: Introductionmentioning

confidence: 99%

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Expanded Access as a source of real‐world data: An overview of FDA and EMA approvals

Polak

Rosmalen

Groot

2020

Brit J Clinical Pharma

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Aims To identify, characterize and compare all Food and Drug Administration (FDA) and European Medicines Agency (EMA) approvals that included real‐world data on efficacy from expanded access (EA) programmes. Methods Cross‐sectional study of FDA (1955–2018) and EMA (1995–2018) regulatory approval documentation. We automated searching for terms related to EA in 22,506 documents using machine learning techniques. We included all approvals where EA terms appeared in the regulatory documentation. Our main outcome was the inclusion of EA data as evidence of clinical efficacy. Characterization was based on approval date, disease area, orphan designation and whether the evidence was supportive or pivotal. Results EA terms appeared in 693 out of 22,506 (3.1%) documents, which referenced 187 approvals. For 39 approvals, data from EA programmes were used to inform on clinical efficacy. The yearly number of approvals with EA data increased from 1.25 for 1993–2013 to 4.6 from 2014–2018. In 13 cases, these programmes formed the main evidence for approval. Of these, patients in EA programmes formed over half (median 71%, interquartile range: 34–100) of the total patient population available for efficacy evaluation. Almost all (12/13) approvals were granted orphan designation. In 8/13, there were differences between regulators in approval status and valuation of evidence. Strikingly, 4 treatments were granted approval based solely on efficacy from EA. Conclusion Sponsors and regulators increasingly include real‐world data from EA programmes in the efficacy profile of a treatment. The indications of the approved treatments are characterized by orphan designation and high unmet medical need.

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Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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Expanded Access as a source of real‐world data: An overview of FDA and EMA approvals

Polak

Rosmalen

Groot

2020

Brit J Clinical Pharma

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“…Consequently, overall safety and efficacy of a therapy cannot be accurately determined. The broad application of EAPs might also detract from the resources needed to conduct standard clinical trials, including patients, drugs, money, and data [28,29]. The EUA does not carry terms that are required to provide important information on the efficacy and safety of a drug [26].…”

Section: Gaps and Challenges Associated With Eaps And Euamentioning

confidence: 99%

Expanded Access Programs, compassionate drug use, and Emergency Use Authorizations during the COVID-19 pandemic.

Rizk

Forthal

Kalantar-Zadeh

et al. 2021

Drug Discovery Today

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The US Food and Drug Administration (FDA) Expanded Access (EA) Program, which allows for compassionate uses of unapproved therapeutics and diagnostics outside of clinical trials, has gained significant traction during the Coronavirus 2019 (COVID-19) pandemic. While development of vaccines has been the major focus, uncertainties around new vaccine safety and effectiveness have spawned interest in other pharmacological options. Experimental drugs can also be approved under the FDA Emergency Use Authorization (EUA) program, designed to combat infectious disease and other threats. Here, we review the US experience in 2020 with pharmacological EA and EUA approvals during the pandemic. We also provide a description of, and clinical rationale for, each of the EA-or EUA-approved drugs (e.g. remdesivir, convalescent plasma, propofol 2%, hydroxychloroquine, ruxolitinib, bamlanivimab) during the pandemic and concluding reflections on the EA program and its potential future uses. Teaser: Several drugs have been authorized by the US FDA through the Expanded Access and/or the Emergency Use Authorization programs during the COVID-19 pandemic. How well did these programs function and what have we learned?Reviews POST SCREEN

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“…3 Finally, we agree with the authors' previous arguments that, at a minimum, acceptance of data from patients treated under expanded access in regulatory decision making may help incentivize companies to arrange for expanded access. 4,5 Given the certain high prices of gene therapies and the uncertain long-term cost effectiveness, aligning societal and industry perspectives on expanded access to gene therapies remains "a tough nut to crack." The authors deserve credit for their efforts to advance the field of the ethics of expanded access in general, and that of pediatric gene therapies in particular.…”

mentioning

confidence: 99%

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

Polak

Bunnik

2021

Molecular Therapy

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What compassionate use means for gene therapies

Cited by 17 publications

References 8 publications

Expanded Access as a source of real‐world data: An overview of FDA and EMA approvals

Expanded Access as a source of real‐world data: An overview of FDA and EMA approvals

Expanded Access Programs, compassionate drug use, and Emergency Use Authorizations during the COVID-19 pandemic.

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

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