Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021

Monge, Andrea N.; Sigelman, Daniel W.; Chahal, Harinder Singh

doi:10.1001/jamanetworkopen.2022.39336

Cited by 18 publications

(15 citation statements)

References 21 publications

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“…1(p31) The FDA's subsequent approval of Relyvrio represents the agency's increasingly flexible approach, prioritizing earlier access over evidentiary certainty. 2,3 However, the FDA has not typically shown proportionate flexibility in exercising its postmarket authorities, taking a narrow approach to requiring postmarket efficacy studies to confirm benefit. 4 Moreover, when these studies have been required, the FDA has often not strictly enforced deadlines and has been hesitant to withdraw approval even after substantial delay or failure of completed studies to demonstrate benefit.…”

Section: Introductionmentioning

confidence: 99%

“…The FDA’s subsequent approval of Relyvrio represents the agency’s increasingly flexible approach, prioritizing earlier access over evidentiary certainty . However, the FDA has not typically shown proportionate flexibility in exercising its postmarket authorities, taking a narrow approach to requiring postmarket efficacy studies to confirm benefit .…”

Section: Introductionmentioning

confidence: 99%

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Extending the US Food and Drug Administration’s Postmarket Authorities

et al. 2023

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ImportanceThe US Food and Drug Administration (FDA) has expansive regulatory flexibility regarding the quality and quantity of evidence it deems sufficient to approve new drugs, which has been increasingly used to grant approval based on less certain evidence of benefit. However, the FDA’s regulatory flexibility with respect to standards for approval has not been matched by sufficient stringency in its exercise of postmarket safeguards, including the FDA’s authority and willingness to require confirmation of benefit through postmarket efficacy studies or to withdraw approval when benefit is not confirmed.ObjectiveTo identify and evaluate opportunities for the FDA to extend its authority to require postmarket efficacy studies and use expedited withdrawal procedures for drugs approved despite substantial residual uncertainty outside the accelerated approval pathway.EvidenceThe FDA’s current approaches to regulatory flexibility with respect to standards for drug approval; examples of shortcomings in the postmarket period; existing statutes and regulations governing the scope of the FDA’s authority to impose and enforce postmarket study requirements; and recent legislative reform and agency action regarding the accelerated approval pathway.FindingsDrawing on the broad language of the federal Food, Drug, and Cosmetic Act, the FDA could independently extend its core accelerated approval authorities—required postmarket efficacy studies and expedited withdrawal procedures—to any drug approved with substantial residual uncertainty regarding benefit, such as those supported by a single pivotal trial. To avoid exacerbating existing problems that have become evident during the past 3 decades of experience using the accelerated approval pathway, however, the FDA must ensure that postmarket studies are well designed and completed quickly, while compelling expedited withdrawal when needed.Conclusions and RelevanceUnder current FDA approaches to drug approval, patients, clinicians, and payers may be left with little confidence about a drug’s benefit not only when it first enters the market but also for an extended period thereafter. If policy makers continue to favor earlier market access over evidentiary certainty, flexible approvals must be matched by more expansive use of postmarket safeguards, an approach possible within the FDA’s existing legal authorities.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Extending the US Food and Drug Administration’s Postmarket Authorities

et al. 2023

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“…At the same time, the ability to deliver on unmet medical needs has driven the use of a variety of accelerated review options to deliver therapies to patients faster. A recent study on the use of various expedited drug development and review programs offered by the US Food and Drug Administration (FDA) between 2008 and 2021 revealed that 97 of 139 (70%) approved biologic products used one or more expedited programs (Monge et al, 2022). This is good news for patients but only if manufacturing can meet patient demand.…”

Section: Introduction—current Challengesmentioning

confidence: 99%

Driving adoption of new technologies in biopharmaceutical manufacturing

Schaefer¹,

Balchunas²,

Charlebois³

et al. 2023

Biotech & Bioengineering

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The challenge of introducing new technologies into established industries is not a problem unique to the biopharmaceutical industry. However, it may be critical to the long‐term competitiveness of individual manufacturers and, more importantly, the ability to deliver therapies to patients. This is especially true for new treatment modalities including cell and gene therapies. We review several barriers to technology adoption which have been identified in various public forums including business, regulatory, technology, and people‐driven concerns. We also summarize suitable enablers addressing one or more of these barriers along with suggestions for developing synergies or connections between innovation in product discovery and manufacturing or across the supplier, discovery, manufacturing, and regulatory arms of the holistic innovation engine.

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“…In the Original Investigation titled “Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021,” 1 published November 1, 2022, the numbers in parentheses on the x-axis were incorrect in Figure 1B, C, E, and F. This article has been corrected. 1 …”

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confidence: 99%

Errors in Figure 1

2023

JAMA Netw Open

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Use of US Food and Drug Administration Expedited Drug Development and Review Programs by Orphan and Nonorphan Novel Drugs Approved From 2008 to 2021

Cited by 18 publications

References 21 publications

Extending the US Food and Drug Administration’s Postmarket Authorities

Extending the US Food and Drug Administration’s Postmarket Authorities

Driving adoption of new technologies in biopharmaceutical manufacturing

Errors in Figure 1

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