Clinical trial results shape how physicians treat patients and how new therapies enter the market. But recent experts argue that what appears in academic journals may not always align with the findings regulators uncover during their independent reviews. A new JAMA Oncology article highlights why the U.S. Food and Drug Administration (FDA) and other regulatory authorities should increase the visibility of their analyses of primary clinical trial data.
Why It Matters
Peer-reviewed journals are the traditional source clinicians rely on for trial evidence. Yet reviewers often only have access to summary or secondary data provided by sponsors, which may emphasize positive outcomes. The FDA’s regulatory reviews, by contrast, are based on the raw data submitted during the approval process. These reviews frequently identify risks, limitations, or methodological flaws not evident in published papers.
The authors warn that without greater visibility into FDA assessments, physicians may not have the full picture when making treatment decisions—especially for complex oncology products where risks can be significant.
Case Studies Highlight the Gap
The article compared FDA reviews with published data for three cancer drugs recently discussed by the Oncologic Drugs Advisory Committee (ODAC):
- Olaparib – FDA found concerning signals in patients with prostate cancer who lacked BRCA variations, leading the agency to restrict the drug’s approval. These risks were not evident in the published journal article.
- Sotorasib – FDA flagged systemic trial biases, including early crossover, imaging irregularities, and inconsistent progression assessments. While dropout rates were noted in publications, these deeper issues were not disclosed.
- Ciltacabtagene autoleucel – FDA identified higher rates of treatment-related mortality and a decline in overall survival in the early months post-treatment, findings not clearly presented in journal reports.
Together, these examples reveal how pivotal trials may meet endpoints while still raising red flags in FDA’s independent analysis.
Expert Opinions
Some experts see publishing FDA’s reviews as the logical next step in transparency. “Publishing FDA analyses in academic platforms would make them far more accessible to clinicians,” said Aaron Kesselheim of Harvard Medical School, though he acknowledged resource limitations as the agency cuts staff and advisory committees.
Others, like Joseph Ross of Yale School of Medicine, emphasize the importance of making FDA’s independent reviews more visible through platforms such as ClinicalTrials.gov or indexing them in the National Library of Medicine. While concerns about data-sharing agreements with sponsors remain, both agree that regulatory insights provide critical context for clinicians.
Implications for Industry
For drug developers, this conversation signals growing expectations around transparency and accountability. Regulatory reviews increasingly shape perceptions of a therapy’s risk-benefit profile, even after approval. Companies should anticipate closer scrutiny not only of their trial endpoints but also of trial conduct, patient heterogeneity, and real-world applicability.
EMMA International’s Perspective
At EMMA International, we recognize the importance of bridging the gap between regulatory review and clinical practice. By helping sponsors design transparent, robust trial programs and prepare for regulatory scrutiny, we ensure that the data tells the complete story. Our expertise in regulatory strategy, risk management, and real-world evidence integration helps companies align with evolving expectations—supporting both approval success and physician trust.
For more information on how EMMA International can assist, visit www.emmainternational.com. Contact us at (248) 987-4497 or info@emmainternational.com.
Reference:
Craven, J. (2025, September 22). Experts recommend increased visibility for regulatory analysis of primary clinical trial data. Regulatory Affairs Professionals Society.
For more information on how EMMA International can assist, visit www.emmainternational.com. Contact EMMA International at (248) 987-4497 or by email at info@emmainternational.com to learn more.
