Policies to widen access to patient level data from clinical trials are gaining traction, despite strong opposition from research
sponsors that such initiatives will undermine patient privacy and incentives for new drug development. There is general agreement
that clinical data sharing can improve the efficiency of clinical trials, validate regulatory decisions, and increase public
confidence in clinical research. But sponsors are concerned about who controls access to data, the purposes of disclosure,
and safeguards to protect all parties.
To head off a plan by EMA for public release, or "sharing," of study data in registration dossiers, sponsors are rolling out
voluntary data sharing programs, as outlined in a "principles" document issued in July by the Pharmaceutical Research and
Manufacturers of America (PhRMA) and the European Federation of Pharmaceutical Industries and Associations. Companies are
advised to establish independent scientific review boards to evaluate outside data requests, plus procedures to protect patient
privacy. The aim is to delay full data disclosure by EMA in January 2014, so that standards, third-party programs, and voluntary
initiatives have more time to demonstrate their ability to enhance research transparency while protecting patients and research
Industry objects that the EMA plan fails to limit access only to bona fide researchers seeking to address valid scientific
questions. And the prospect of releasing full regulatory dossiers could expose proprietary formulation and manufacturing data
and information on product development and future indications. Broad release of clinical data, moreover, could undermine product
exclusivity in countries such as Australia, Brazil, China, and Korea that link exclusivity to data confidentiality, explained
Pfizer Senior Vice President Justin McCarthy at a PhRMA briefing in October. He warned that companies may rethink their development
and registration strategies if the EMA proceeds with its plan for full release of regulatory submissions. Sponsors may decide
to delay submissions for approval in Europe or seek to eliminate or redact more confidential commercial information in dossiers.
Sponsors also are concerned about a June 2013 proposal from FDA to make available masked and de-identified data submitted
in applications, possibly limited to information aggregated across drug classes. FDA reopened comments to this proposal in
October, seeking additional information on what factors to consider in masking study data and if there are ways to identify
patients after removing names, birth dates, death dates, and geographic information.
Industry is more comfortable with FDA's more narrowly focused data sharing plan, although still wary that outside researchers
with advanced computer systems and access to extensive genomic information will be able to re-identify patients, particularly
those involved in studies on rare diseases or cancers that affect small patient cohorts. And permitting non-experts to gain
access to data, comments PhRMA, could result in "unjustified second-guessing of FDA's regulatory decisions" that raises alarm
among patients and providers and discourages appropriate treatment.
These controversies are spurring efforts to reach consensus on achieving data transparency while enhancing biopharmaceutical
innovation. An Institute of Medicine committee began deliberations last month on "Responsible Sharing of Clinical Trial Data,"
with the intent of issuing guiding principles and a framework for such initiatives. An interim report is due in January, and
a final report in December 2014 to assess the benefits and risks of data sharing and opportunities for responsible disclosure.
While more pharma companies will launch data sharing programs in January, these voluntary initiatives have limitations. Firms
don't plan to hand over clinical data sets to outsiders, but to run their queries through in-house data systems and provide
results. And there are concerns about the real independence of a company-appointed scientific review board.
Patient representatives at the PhRMA briefing noted problems with informed consent if clinical data is to be used by outside
parties for other purposes. FDA requires sponsors to advise clinical trial participants as part of the consent process that
a summary of data from the study will be posted on http://clinicaltrials.gov/, but that this information will not identify specific patients, attorney Mark Barnes pointed out at the October Clinical
Trial Disclosure conference sponsored by the Drug Information Association. Weakened privacy safeguards, however, could invalidate
Sponsors insist that they should decide how to share research information—not government regulators. And patient advocates
are fearful of questionable secondary statistical analyses that challenge regulatory decisions and sponsor assessments. But
there's optimism that the disclosure movement will spur data interoperability standards and open sourcing models, all key
to transforming clinical research.