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Record W3123183430 · doi:10.2196/14563

Investigating the Food and Drug Administration Biotherapeutics Review and Approval Process: Narrative Review

2021· review· en· W3123183430 on OpenAlexvenueno aff
Samuel Bonet Olivencia, Farzan Sasangohar

Bibliographic record

VenueJMIR Formative Research · 2021
Typereview
Languageen
FieldMathematics
TopicStatistical Methods in Clinical Trials
Canadian institutionsnot available
Fundersnot available
KeywordsNarrative reviewFood and drug administrationCLARITYMedicineScope (computer science)Process (computing)Drug approvalRisk analysis (engineering)Computer scienceDrugPharmacologyIntensive care medicine

Abstract

fetched live from OpenAlex

BACKGROUND: The development, review, and approval process of therapeutic biological products in the United States presents two primary challenges: time and cost. Advancing a biotherapeutic from concept to market may take an average of 12 years, with costs exceeding US $1 billion, and the product may still fail the US Food and Drug Administration (FDA) approval process. Despite the FDA's practices to expedite the approval of new therapies, seeking FDA approval remains a long, costly, and risky process. OBJECTIVE: The objective of this paper is to explore the factors and gaps related to the FDA review and approval process that contribute to process inefficiencies and complexities as well as proposed methods and solutions to address such gaps. This paper also aims to investigate the available modeling efforts for the FDA approval process of therapeutic biological products. METHODS: A narrative review of literature was conducted to understand the scope of published knowledge about challenges, opportunities, and specific methods to address the factors and gaps related to the review and approval of new drugs, including therapeutic biological products. Relevant peer-reviewed journal articles, conference proceedings, book chapters, official reports from public policy professional centers, and official reports and guidelines from the FDA were reviewed. RESULTS: Of the 23 articles identified in this narrative literature review, none modeled the current FDA review and approval process structure to address issues related to the robustness, reliability, and efficiency of its operations from an external point of view. Although several studies summarize the FDA approval process with clarity, in addition to bringing to light the problems and challenges faced by the regulatory agency, only a few attempts have been made to provide solutions for the problems and challenges identified. In addition, although several reform models have been discussed, these models lack the application of scientific methodologies and modeling techniques in understanding FDA as a complex sociotechnical system. Furthermore, tools and methods to assess the efficacy of the models before implementation are largely absent. CONCLUSIONS: The findings suggest the efficacy of model-based systems engineering approaches for identifying opportunities for significant improvements to the FDA review and approval process. Using this holistic approach will serve several investigative purposes: identify influential sources of variability that cause major delays, including individual, team, and organizational decision making; identify the human-system bottlenecks; identify areas of opportunity for design-driven improvements; study the effect of induced changes in the system; and assess the robustness of the structure of the FDA approval process in terms of enforcement and information symmetry.

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.022
metaresearch head score (Gemma)0.071
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMetaresearch, Meta-epidemiology (narrow), Research integrity
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Systematic review · Consensus signal: none
GenreCandidate signal: Review · Consensus signal: Review
Teacher disagreement score0.509
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0220.071
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0040.000
Bibliometrics0.0000.002
Science and technology studies0.0010.001
Scholarly communication0.0000.000
Open science0.0010.000
Research integrity0.0000.003
Insufficient payload (model declined to judge)0.0000.000

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.738
GPT teacher head0.686
Teacher spread0.052 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one teacher head, not a consensus.

Study designSystematic review
Domainnot available
GenreReview

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations6
Published2021
Admission routes1
Has abstractyes

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