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Ethics
Research
Measuring clinical trial transparency: an empirical analysis of newly approved drugs and large pharmaceutical companies
  1. Jennifer E Miller1,2,
  2. Marc Wilenzick3,
  3. Nolan Ritcey2,
  4. Joseph S Ross4,5,6,
  5. Michelle M Mello7
  1. 1 Division of Medical Ethics, Department of Population Health, NYU School of Medicine, New York, USA
  2. 2 Bioethics International, New York, USA
  3. 3 International Aids Vaccine Initiative, New York, USA
  4. 4 Section of General Internal Medicine and Robert Wood Johnson Foundation Clinical Scholars Program, Department of Medicine, Yale School of Medicine, New Haven, Connecticut, USA
  5. 5 Department of Health Policy and Management, Yale School of Public Health, New Haven, Connecticut, USA
  6. 6 Center for Outcomes Research and Evaluation, Yale-New Haven Health, New Haven, Connecticut, USA
  7. 7 Stanford Law School and the Department of Health Research and Policy, Stanford University Stanford Law School, Stanford, California, USA
  1. Correspondence to Dr Jennifer E Miller; jennifer.miller{at}nyumc.org

Abstract

Objectives To define a series of clinical trial transparency measures and apply them to large pharmaceutical and biotechnology companies and their 2014 FDA-approved drugs.

Design Cross-sectional descriptive analysis of all clinical trials supporting 2014 Food and Drugs Administration (FDA)-approved new drug applications (NDAs) for novel drugs sponsored by large companies.

Data sources Data from over 45 sources, including Drugs@FDA.gov, ClinicalTrials.gov, corporate and international registries; PubMed, Google Scholar, EMBASE, corporate press releases, Securities and Exchange Commission (SEC) filings and personal communications with drug manufacturers.

Outcome measures Trial registration, results reporting, clinical study report (CSR) synopsis sharing, biomedical journal publication, and FDA Amendments Acts (FDAAA) compliance, analysed on the drug level.

Results The FDA approved 19 novel new drugs, sponsored by 11 large companies, involving 553 trials, in 2014. We analysed 505 relevant trials. Per drug, a median of 100% (IQR 86%–100%) of trials in patients were registered, 71% (IQR 57%–100%) reported results or shared a CSR synopsis, 80% (70%–100%) were published and 96% (80%–100%) were publicly available in some form by 13 months after FDA approval. Disclosure rates were lower at FDA approval (65%) and improved significantly by 6 months post FDA approval. Per drug, a median of 100% (IQR 75%–100%) of FDAAA-applicable trials were compliant. Half of reviewed drugs had publicly disclosed results for all trials in patients in our sample. One trial was uniquely registered in a corporate registry, and not ClinicalTrials.gov; 0 trials were uniquely registered in international registries.

Conclusions Among large pharmaceutical companies and new drugs, clinical trial transparency is high based on several standards, although opportunities for improvement remain. Transparency is markedly higher for trials in patients than among all trials supporting drug approval, including trials in healthy volunteers. Ongoing efforts to publicly track companies’ transparency records and recognise exemplary companies may encourage further progress.

  • clinical pharmacology
  • health Policy
  • law (see medical law)
  • medical ethics
  • public health
  • bioethics
  • pharma ethics
  • clinical trial transparency
  • good pharma scorecard

This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/

https://doi.org/10.1136/bmjopen-2017-017917

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    Footnotes

    • Contributors JM: conceived the study, supervised the collection and coding of data, led the data analysis and wrote the initial manuscript draft. NR: contributed to data collection, coding, and analysis. MMM, JSR and MW: provided guidance on the analytical approach, contributed to the interpretation of results, and drafted and revised the manuscript for critical intellectual content.

    • Funding This work was funded by a grant from the Laura and John Arnold Foundation. JSR: receives support through Yale University from Johnson and Johnson to develop methods of clinical trial data sharing, from Medtronic, Inc and the Food and Drug Administration (FDA) to develop methods for postmarket surveillance of medical devices, from the FDA to establish the Yale-Mayo Center for Excellence in Regulatory Science and Innovation (CERSI), from the Blue Cross Blue Shield Association to better understand medical technology evaluation, from the Centers of Medicare and Medicaid Services (CMS) to develop and maintain performance measures that are used for public reporting, and from the Laura and John Arnold Foundation to support the Collaboration on Research Integrity and Transparency (CRIT) at Yale.

    • Disclaimer The views expressed in the report are the authors’ and do not necessarily reflect those of the foundation.

    • Competing interests None declared.

    • Provenance and peer review Not commissioned; externally peer reviewed.

    • Data sharing statement Study data will be posted at bioethicsinternational.org upon publication of this article and can also be obtained from the lead author.