News Updates

Many developments have brought clinical trial data transparency to the forefront of our stakeholders’ minds – from the expanding implementation of electronic health records and electronic data submitting requirements to the new proposed rules from HHS on clinical trial data reporting. This year, OIG plans to review the extent to which clinical trialists comply with FDAAA clinical trial reporting requirements and to assess FDA’s efforts to ensure these requirements are being met.

On the legislative front, the 21st Century Cures Initiative discussion document included Title II, Subtitle F - Building a 21st Century Data Sharing Framework to “optimize patient care and collaboratively discover the next generation of patient-centered solutions.” And let’s not forget the looming PDUFA VI negotiations due to begin this year. For all of these reasons, it’s no surprise that DIA thought leaders have named “Clinical trial data transparency/data sharing” as the #2 trend to shape the world of medical product development in 2015.

The call for data sharing stretches back to FDA’s 2004 Critical Path Challenges and Opportunities Report. Leveraging existing clinical and pre-clinical data to explore new or modified biomarkers or trial endpoints and to evaluate the predictability of pre-clinical safety data was one of the opportunities identified in this report.

A recent example where FDA utilized existing data to answer a key development question was the reexamination of the primary endpoint – sustained virologic response at 24 weeks of follow-up – in chronic hepatitis C trials. FDA scientists analyzed data combined across five drug development programs and determined that the sustained virologic response measurements at 12 and 24 weeks of follow-up were concordant across viral genotypes, treatment regimens and treatment durations– all without the need for a new clinical study. The conclusion that a shortened timeframe for follow-up is sufficient to reliably demonstrate efficacy can now allow for hepatitis C virus treatment options to be available earlier for patients.

This work in the chronic hepatitis C space is just one example of FDA’s considerable expertise in analyzing individual patient level data, as well as aggregated clinical trial data. This expertise, compounded with the fact that FDA possesses the largest collection of clinical trial data in the world, could be the catalyst for major advances in the science of drug development in multiple therapeutic areas. However, FDA does not own these data, nor does it have the technical or human resources to fully realize their potential. In short, more experts need to become actively engaged in this research.

There are efforts underway to provide access to these data and greater transparency to FDA’s work. OpenFDA is a portal that enables easier public access to drug, device and food data related to adverse events, recalls and labeling information. In a more consumer-focused activity, CDER recently launched the Drug Trials Snapshots program to provide our constituents with demographic information that has supported FDA new drug approvals. In addition, most of our multi-stakeholder scientific consortia have data sharing elements built into their core programs.

As a broader approach to data sharing, FDA is exploring strategies to provide access to de-identified and masked data that have research value (e.g., data from the control arms of studies, which could contribute to natural history studies). Naturally, the data must be presented in a way that would both safeguard the privacy of patients enrolled in clinical trials and protect the commercial investments of sponsors. To gather more input from the public and further develop FDA strategies, a workshop will soon be held to help develop FDA’s plan for clinical trial data transparency. This workshop will complement over sixty comments received in response to the 2013 FR notice on the topic.

Analysis of multiple data sets provides an opportunity to advance the science of drug development and to improve therapy for specific diseases, thus more fully utilizing the contributions of trial participants. The strides that have been made in data sharing should be celebrated, but there is always more work to be done. The welfare of patients must remain the mainstay of our combined efforts to make better use of data.

To learn more, plan to attend Clinical Trial Disclosure & Data Transparency - The Expanding Global Environment.

This article originally appeared in the April 2015 Global Forum - read the complete issue.