As the founder of the nonprofit Bioethics International and the Good Pharma Scorecard, I have been working on developing standards for clinical trial transparency and benchmarking the performance of new drugs and drug companies against those standards. Our goal is to improve ethics, trustworthiness, and patient-centricity/engagement in the development and commercialization of medical products and healthcare innovation. Our initial area of focus is clinical trial transparency.
At BEI, transparency means that trials are registered, have summary results accurately reported in a public databank, are submitted for publication in the medical literature, and comply with US disclosure requirements defined in the Food and Drug Administration Amendments Act of 2007 (FDAAA). We also support the sharing of patient-level data and the returning of lay summary results to trial participants.
In the Good Pharma Scorecard, we assess transparency for 3 different groups of trials: all trials in an FDA-approved New Drug Application (NDA), patient trials (trials conducted in patients, excluding trials in healthy volunteers), and FDAAA-applicable trials (trials legally required to be reported in ClinicalTrials.gov).1
We benchmark transparency using these 3 different sets of trials, because we found that clinical trial transparency means different things to different people, groups, and institutions. Thus, we try to provide key stakeholders with the performance metrics that are meaningful and useful to them.
“…increasing levels of transparency have opened up other ethics conversations. For example, thanks to enhanced transparency we now know more details about clinical trial designs and enrollments.”
We regularly convene stakeholders such as patient groups, physician groups, ethicists, academics, and companies to understand what clinical trial transparency and ethical innovation means to them. In turn, we help catalyze stronger consensus and operationalization of best practices. We also try to connect with regulators and other groups to develop guidance and opportunities to align our work with theirs.
As part of this work, my team at BEI reviewed the data sharing statement from the International Committee of Medical Journal Editors (ICMJE), which advises researchers to publicize their data-sharing policy each time they publish clinical trial results in one of their member journals. We applaud this step forward, but the ICMJE was largely silent on the contents of those policies. Technically, an author could have a policy that states they won’t share any data.
To avoid this, BEI proposes researchers and trial sponsors commit to sharing analyzable datasets and Clinical Study Reports (CSRs) for all phase 2 and 3 trials by the latter of 6 months after regulatory approval of a new drug or 18 months after the trial’s completion. These proposed timelines are similar to those of the Institute of Medicine (IOM)-National Academy of Medicine (NAM).
Better transparency definitions and implementation are pillars for trustworthiness in healthcare innovation, but it’s also important to note that increasing levels of transparency have opened up other ethics conversations. For example, thanks to enhanced transparency we now know more details about clinical trial designs and enrollments. Stakeholders have noticed that drugs are often tested on younger, whiter, and healthier populations than the patients who ultimately will use a drug after FDA approval.2 Typical patients are often older, sicker, and more ethnically diverse than trial participants. As a result, stakeholders now want to improve trial designs, improve inclusivity in trial enrollment, and use more real-world evidence in drug development.
So, it is tempting to worry that the spouting whale will get harpooned, meaning if we disclose more information, it will expose all of us to more complaints and criticism.
However, criticism isn’t all negative; it actually represents hope. The fact that people are complaining about problems means they recognize that things can be better and the importance of the work being done in drug development. I am confident that BEI and other groups can work together to define what best practices look like and what metrics can be used to track progress over time. After all, what gets measured often gets done. And, implementation and recognition of best practices will be a cause for celebration—no harpoons needed.