Sharing of clinical trial data and results reporting practices among large pharmaceutical companies: cross sectional descriptive study and pilot of a tool to improve company practicesBMJ 2019; 366 doi: https://doi.org/10.1136/bmj.l4217 (Published 10 July 2019) Cite this as: BMJ 2019;366:l4217
- Jennifer Miller, assistant professor12,
- Joseph S Ross, associate professor13,
- Marc Wilenzick, lawyer24,
- Michelle M Mello, doctor of jurisprudence, professor56
- 1Department of Internal Medicine, Yale School of Medicine, Yale University, New Haven, CT, USA
- 2Bioethics International, New York, NY, USA
- 4Taro Pharmaceuticals, USA, Hawthorne, NY, USA
- 3Department of Health Policy and Management, Yale School of Public Health, Center for Outcomes Research and Evaluation, Yale University, New Haven, CT, USA
- 5Stanford Law School, Stanford University, Stanford, CA, USA
- 6Department of Health Research and Policy, Stanford University School of Medicine, Stanford University, Stanford, CA, USA
- Correspondence to: J Miller
- Accepted 21 May 2019
Objectives To develop and pilot a tool to measure and improve pharmaceutical companies’ clinical trial data sharing policies and practices.
Design Cross sectional descriptive analysis.
Setting Large pharmaceutical companies with novel drugs approved by the US Food and Drug Administration in 2015.
Data sources Data sharing measures were adapted from 10 prominent data sharing guidelines from expert bodies and refined through a multi-stakeholder deliberative process engaging patients, industry, academics, regulators, and others. Data sharing practices and policies were assessed using data from ClinicalTrials.gov, Drugs@FDA, corporate websites, data sharing platforms and registries (eg, the Yale Open Data Access (YODA) Project and Clinical Study Data Request (CSDR)), and personal communication with drug companies.
Main outcome measures Company level, multicomponent measure of accessibility of participant level clinical trial data (eg, analysis ready dataset and metadata); drug and trial level measures of registration, results reporting, and publication; company level overall transparency rankings; and feasibility of the measures and ranking tool to improve company data sharing policies and practices.
Results Only 25% of large pharmaceutical companies fully met the data sharing measure. The median company data sharing score was 63% (interquartile range 58-85%). Given feedback and a chance to improve their policies to meet this measure, three companies made amendments, raising the percentage of companies in full compliance to 33% and the median company data sharing score to 80% (73-100%). The most common reasons companies did not initially satisfy the data sharing measure were failure to share data by the specified deadline (75%) and failure to report the number and outcome of their data requests. Across new drug applications, a median of 100% (interquartile range 91-100%) of trials in patients were registered, 65% (36-96%) reported results, 45% (30-84%) were published, and 95% (69-100%) were publicly available in some form by six months after FDA drug approval. When examining results on the drug level, less than half (42%) of reviewed drugs had results for all their new drug applications trials in patients publicly available in some form by six months after FDA approval.
Conclusions It was feasible to develop a tool to measure data sharing policies and practices among large companies and have an impact in improving company practices. Among large companies, 25% made participant level trial data accessible to external investigators for new drug approvals in accordance with the current study’s measures; this proportion improved to 33% after applying the ranking tool. Other measures of trial transparency were higher. Some companies, however, have substantial room for improvement on transparency and data sharing of clinical trials.
Contributors: JEM, JSR, and MMM conceived and designed the study. JEM and research assistants extracted and analyzed data related to the trial transparency measures. JEM, JSR, MW, and MMM, analyzed data for the data sharing measurements and results. All authors drafted the manuscript, interpreted the data, critically revised the manuscript for important intellectual content, and approved the final manuscript. JEM is the first author and guarantor. The corresponding author attests that all listed authors meet authorship criteria and that no others meeting the criteria have been omitted
Funding: This work was conducted as part of the Good Pharma Scorecard, at Bioethics International, funded by a grant from the Laura and John Arnold Foundation, which supports JEM, MMM, JSR, and MW. This funder did not design the study, analyze or interpret findings, or draft the manuscript and did not review or approval the manuscript before submission. The authors assume full responsibility for the accuracy and completeness of the ideas presented.
Competing interests: All authors have completed the ICMJE uniform disclosure form at www.icmje.org/coi_disclosure.pdf and declare: In addition to the grant support for this project, the authors report the following relevant financial relationships: MW is employed as an attorney advising pharmaceutical companies on compliance with the Food and Drug Administration and has received compensation from private and public organizations conducting clinical research, including Taro Pharmaceuticals. JSR receives support through Yale University from Johnson & Johnson to develop methods of clinical trial data sharing, from Medtronic and the FDA to develop methods for post-market surveillance of medical devices, from the FDA to establish the Yale-Mayo Center for Excellence in Regulatory Science and Innovation, from the Blue Cross Blue Shield Association to better understand medical technology evaluation, from the Centers of Medicare and Medicaid Services 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 at Yale and the Good Pharma Scorecard. Raw data may be requested for this paper and will be posted on the Bioethics International website, Good Pharma Scorecard page.
Ethical approval: Not required.
Data sharing: The dataset will be made available on Bioethics International’s website for the Good Pharma Scorecard (see www.bioethicsinternational.org/good-pharma-scorecard/scorecard-methodology).
Transparency: The lead author (JEM) affirms that the manuscript is an honest, accurate, and transparent account of the study being reported; that no important aspects of the study have been omitted; and that any discrepancies from the study as planned (and, if relevant, registered) have been explained.
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