Evidence map›Paper›PMID 40413363›Full record

ReviewTherapeutic innovation & regulatory science2025

Keeping the End in Mind: Reviewing U.S. FDA Inspections of Submissions including Real-World Data.

Cheryl Grandinetti, Donna R Rivera, Lee Pai-Scherf, Anna Choe, Paul G Kluetz, Stefanie Kraus, Gabriel K Innes, Kassa Ayalew

Abstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Therapeutic innovation & regulatory science, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

7 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Quality of real-world registry studies.Perspectives in clinical research
    Article
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

8 authors.

Cheryl GrandinettiDivision of Clinical Compliance Evaluation, Office of Scientific Investigations (OSI), Office of Compliance, Center for Drug Evaluation and Research (CDER), US Food and Drug Administration (FDA), Silver Spring, MD, USA. Cheryl.Grandinetti@fda.hhs.gov.
Donna R RiveraOffice of Oncologic Diseases, Center for Drug Evaluation and Research and Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD, 20993, USA.
Lee Pai-ScherfDivision of Clinical Compliance Evaluation, Office of Scientific Investigations (OSI), Office of Compliance, Center for Drug Evaluation and Research (CDER), US Food and Drug Administration (FDA), Silver Spring, MD, USA.
Anna ChoeDivision of Rare Diseases and Medical Genetics, Office of Rare Diseases, Pediatrics, Urology, and Reproductive Medicine, Office of New Drugs, Center for Drug Evaluation and Research (CDER), US Food and Drug Administration (FDA), Silver Spring, MD, USA.
Paul G KluetzOffice of Oncologic Diseases, Center for Drug Evaluation and Research and Oncology Center of Excellence, US Food and Drug Administration, Silver Spring, MD, 20993, USA.
Stefanie KrausOffice of Regulatory Policy, Center for Drug Evaluation and Research (CDER), US Food and Drug Administration (FDA), Silver Spring, MD, USA.
Gabriel K InnesOffice of Medical Policy, Center for Drug Evaluation and Research (CDER), US Food and Drug Administration (FDA), Silver Spring, MD, USA.
Kassa AyalewDivision of Clinical Compliance Evaluation, Office of Scientific Investigations (OSI), Office of Compliance, Center for Drug Evaluation and Research (CDER), US Food and Drug Administration (FDA), Silver Spring, MD, USA.

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

The increasing use of real-world data (RWD) to generate real-world evidence (RWE) presents unique opportunities and challenges for drug development and regulatory decision-making, particularly in the area of good clinical practice inspections. FDA typically focuses their application review-based inspections on pivotal studies that generate evidence submitted to support new drug and biological product applications. This focus applies regardless of the data sources used in those studies. In this article, we discuss the fundamental role of good clinical practice inspections in verifying the quality, integrity, and reliability of RWD used in regulatory submissions. Through case examples, we highlight specific challenges related to accessing RWD source records, assessing data quality, and evaluating processes for data curation, transformation, and analysis. Our experience underscores the importance of early engagement with regulatory agencies as well as the implementation of robust quality management practices throughout the study lifecycle. As RWD continues to shape the regulatory landscape, these case examples provide insights in navigating the complexities associated with submissions utilizing RWE for drug approval.

Indexed as

Drug ApprovalUnited States Food and Drug AdministrationHumansUnited StatesData quality and integrityDrug approval submissionsGood clinical practice (GCP)Real-world data (RWD)Real-world evidence (RWE)Regulatory inspections

Identifiers

PMID40413363
PMCPMC12446128

What OpenQuestion holds

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LicenceCC BY
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Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.