[This is off-the-cuff live blogging, so apologies for any errors, typos, etc]
First up is Mark Lange from Eli Lilly (who notes that he is here in his personal capacity only!), speaking about “Data Transparency and the Role of the FDA.”
He prefaces his talk by noting that when he refers to “data,” he means raw, patient-level data from clinical trials. Most calls for the transparency of such data, he says, reflect a common theme about lack of trust in the pharmaceutical industry. So we might wonder: why doesn’t the pharmaceutical industry simply accede to that request and make their data available?
Mark notes that industry has several concerns. One important one pertains to data exclusivity. In several (if not all) markets, data exclusivity rights are premised on keeping the relevant data confidential, and posting it publicly would be deemed a waiver of those rights. In addition, data exclusivity prevents generic competitors from free riding, and publishing data could allow them to circumvent the very point of data exclusivity.
Moving to privacy concerns, Mark notes that research subjects’ understanding is that their data will be used for particular purposes and shared with regulators, but not be publicly posted on the Internet for anyone to do with whatever they want. Relatedly, there is the potential for interpretation of public data to be biased; research results may be over-interpreted and analyses may be flawed or even erroneous. Competitors might look for fairly trivial flaws the the data and try to use them to their advantage rather than sincerely trying to advance scientific progress and transparency.
Mark suggests, however, the choice between privacy and transparency is a false one. A better alternative is available — namely, for objective, expert regulators such as the FDA to receive and vet data in ways that address both audiences and both sets of concerns. The FDA is in fact already experienced in doing this. For example, it determines whether research demonstrates that a drug is safe and effective for a particular use through its marketing application approval mechanism, and it determines the accuracy and adequacy of the portrayal of research results in product labeling and product advertisements. And late last year, it was given responsibility for overseeing clinicaltrials.gov, which includes results from all pre-specified primary and secondary outcomes measures from nearly all clinical trials either conducted in the U.S. or intended to be used in support of an application for marketing approval in the U.S. This new responsibility, Mark suggests, could be a powerful tool, depending on how the FDA uses it. For instance, the FDA could exercise authority to monitor and enforce the absence of required results and the inclusion of false or misleading results data.
In concluding, Mark stresses that, when faced with requests for public access to patient-level trial data, we should consider the important role of regulators as trusted intermediaries who can balance competing concerns. Read More