US group suggests way to boost access to trial data while preventing misuseBMJ 2013; 347 doi: http://dx.doi.org/10.1136/bmj.f6389 (Published 22 October 2013) Cite this as: BMJ 2013;347:f6389
A working group convened by the Multiregional Clinical Trials Center at Harvard University has recommended a new approach to ensuring that clinical trial sponsors make participant level data publicly available while protecting against inappropriate use of the data.1
It said that one way to achieve this might be with an independent board that oversaw data requests and use. Anyone using the data would have to follow a scientifically valid analytical plan.
Michelle Mello, professor of law and public health at the Harvard School of Public Health and the report’s lead author, said, “Our experiences with Vioxx [rofecoxib], Avandia [rosiglitazone], and other widely prescribed drugs that were revealed to have serious safety risks show how important it is to give independent scientists access to clinical trial data.
“The question is: how can we achieve the powerful public health benefits of data sharing while protecting research participants’ privacy, avoiding ‘junk science,’ and minimizing burdens on trial sponsors?”
Participant level data are those recorded for each participant in a study, in contrast to aggregate data averaged across all study participants. Growing demand for access to these “raw” data has led to some drug companies allowing access to participant level data from trials of approved products. And the European Medicines Agency said in a recent draft policy that it would provide public access to some data, including at the participant level, in files that drug companies submitted for approval of new drugs.2
The working group, including academics from the center, which receives funding from drug companies and non-profit organizations, together with experts in data sharing from the drug industry, reviewed current initiatives, potential risks and benefits, and legal and regulatory implications.
They found that analysis of individual data by independent researchers could detect important information on products’ safety and effectiveness, “serving as a check on a sponsor or investigator’s characterization of the risks and benefits.” Such analysis could also explore public health questions not answered in individual trials and could help trial sponsors to refine future trial design, they said. “The benefits in terms of unleashing a very powerful source of information about health are overwhelming,” said Mello.
But the group cautioned that the risk of revealing participants’ identities meant that they must understand the potential ramifications of data sharing. Inappropriate statistical methods and analysis that followed “strong private agendas” could lead to flawed and misleading analyses.
The working group recommended that any system that is ultimately adopted for expanded access to participant level data should provide sufficiently broad access so as to benefit scientific innovation and public health. “At a minimum, it should prospectively apply to trials of all approved prescription drugs, medical devices, and biologics,” it advised.
Because of the potential harm to public health from poor analyses, the group called for measures to ensure responsible use of data, as well as protecting study participants’ privacy. “Data requesters should commit to safeguarding the privacy of participants and to conducting analyses that meet accepted standards of scientific rigor and integrity,” members advised.
They suggested one way to achieve this might be with an independent review board that evaluated data requests and enforced conditions on use. Mello considered this approach would best balance the benefits of public access to data with ensuring responsible use.
The group cautioned that current proposals by the European Medicines Agency and US Food and Drug Administration lacked some attributes of an optimal system, including general applicability to all data generators and assessment of data requesters’ scientific expertise and analytic plans.
Their recommendations will be presented to a meeting of the Institute of Medicine later this week.
In an editorial accompanying the report Hans-Georg Eichler, senior medical officer at the European Medicines Agency, and colleagues support the benefits of access to patient level data, particularly in improving the efficiency of drug development, but recognize the risks of inappropriate secondary data analysis.3
“Two way transparency is crucial to address this risk,” they agreed. “Strong safeguards must be put in place to ensure that the clinical investments and intellectual property of innovators are not jeopardized by ‘free riders.’”
They suggested a “managed release environment” that allowed sharing of patient level data while ensuring privacy.
Cite this as: BMJ 2013;347:f6389
bmj.com News: Unpublished studies hold twice as much data on drugs as those in public domain, study finds (BMJ 2013;347:f6179, doi:10.1136/bmj.f6179); Feature: Medical data—does patient privacy trump access for research? (BMJ 2013;347:f5516, doi:10.1136/bmj.f5516)
See more BMJ articles on open access to trial data at bmj.com/open-data.