Wednesday, January 4, 2012

Public Reporting of Patient Recruitment?

A few years back, I was working with a small biotech companies as they were ramping up to begin their first-ever pivotal trial. One of the team leads had just produced a timeline for enrollment in the trial, which was being circulated for feedback. Seeing as they had never conducted a trial of this size before, I was curious about how he had arrived at his estimate. My bigger clients had data from prior trials (both their own and their CRO’s) to use, but as far as I could tell, this client had absolutely nothing.

He proudly shared with me the secret of his methodology: he had looked up some comparable studies on, counted the number of listed sites, and then compared that to the sample size and start/end dates to arrive at an enrollment rate for each study. He’d then used the average of all those rates to determine how long his study would take to complete.

If you’ve ever used in your work, you can immediately determine the multiple, fatal flaws in that line of reasoning. The data simply doesn’t work like that. And to be fair, it wasn’t designed to work like that: the registry is intended to provide public access to what research is being done, not provide competitive intelligence on patient recruitment.

I’m therefore sympathetic, but skeptical, of a recent article in PLoS Medicine, Disclosure of Investigators' Recruitment Performance in Multicenter Clinical Trials: A Further Step for Research Transparency, that proposes to make reporting of enrollment a mandatory part of the trial registry. The authors would like to see not only actual randomized patients for each principal investigator, but also how that compares to their “recruitment target”.

The entire article is thought-provoking and worth a read. The authors’ main arguments in favor of mandatory recruitment reporting can be boiled down to:

  • Recruitment is many trials is poor, and public disclosure of recruitment performance will improve it
  • Sponsors, patient groups, and other stakeholders will be interested in the information
  • The data “could prompt queries” from other investigators

The first point is certainly the most compelling – improving enrollment in trials is at or near the top of everyone’s priority list – but the least supported by evidence. It is not clear to me that public scrutiny will lead to faster enrollment, and in fact in many cases it could quite conceivably lead to good investigators opting to not conduct a trial if they felt they risked being listed as “underperforming”. After all, there are many factors that will influence the total number of randomized patients at each site, and many of these are not under the PI’s control.

The other two points are true, in their way, but mandating that currently-proprietary information be given away to all competitors will certainly be resisted by industry. There are oceans of data that would be of interest to competitors, patient groups, and other investigators – that simply cannot be enough to justify mandating full public release.

Image: Philip Johnson's Glass House from Staib via Wikimedia Commons.

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