There's a growing clamor for broader access to information on health care practices, policies and costs, and it extends to
data from clinical trials, postmarket safety reports, manufacturing records, and pharma financial arrangements and marketing
activities. Consumers now expect more detailed information on costs and benefits of competing health plans, including the
specifics on drug formularies and coverage. Recent disclosure of vast differences in prices charged by hospitals for routine
procedures, with little apparent relationship to quality or outcomes, shocked the public, and physicians are gearing for similar
postings on how much money they earn for treating Medicare patients. Research organizations such as ProPublica are publishing
extensive information on Medicare services and prices, including how doctors prescribe medications under Part D.
The latest transparency challenge for pharma marketers is the "Sunshine" initiative for disclosing payments to physicians
and teaching hospitals, ostensibly to uncover financial transactions that influence prescribing and treatment choices. Open
Payments is now poised for launch based on instructions posted in early February for how pharma and medical product companies
should register and file aggregate payment data for 2013 by the end of March. Then marketers will submit by Aug. 1 massive
detailed information on all covered transactions, along with legal attestation to the accuracy of the data.
The roll-out schedule aims to give providers time to review the reports before they are made public September 30. The Obama
administration hopes that this phased-in registration and data filing approach will help iron out any glitches and avoid the
system failures experienced by health exchanges, but that may be wishful thinking.
Disclosing clinical data
Meanwhile, the clinical trial transparency bandwagon is gathering steam, as pharma companies sign up for programs designed
to provide more open access to research results. The European Union is driving this train, looking to mandate disclosure of
clinical data from all European trials that support market applications.
In response, the European Federation of Pharmaceutical Industries & Associations (EFPIA) and the Pharmaceutical Research and
Manufacturers of America (PhRMA) launched a voluntary program in January to vet proposals from outside researchers for access
to clinical trial data, protocols and clinical study reports for new medicines approved in the US and EU. Johnson & Johnson
soon after announced a collaboration with Yale University's Open Data Access Project (YODA), a similar program to facilitate
distribution of proprietary research data, initiated last year by medical device maker Medtronic in response to pressure to
disclose product safety information. GlaxoSmithKline, Roche and other companies also have established independent boards to
review requests for confidential research information. Participating companies hope these efforts will bolster their image
as responsible and innovative research entities, while also retaining some control over who can access patient-level data
and some protection for trade secret information.
These initiatives and related issues are being analyzed by an Institute of Medicine (IOM) panel preparing a report on "Strategies
for Responsible Sharing of Clinical Data." The group released a "discussion framework" in January to solicit input on proposed
data-disclosure principles and activities, issues that were explored at a February workshop on the pros and cons of different
data sharing models.
Nowhere To Hide
Total open access, as backed by some academics and advocacy groups, may facilitate study reproducibility and analysis of failed
trials, with an eye to preventing repeat studies harmful to patients. However, this model runs the risk of compromising patient
privacy, raises difficult informed consent issues, and could lead to "rogue science" that produces biased and inaccurate analyses.
Controlled access, as provided by YODA and industry initiatives, allows release of some data while protecting patient privacy
and intellectual property. Yet, there are questions about the independence of third-party reviewers and about who will set
standards and bear the cost of building data platforms and infrastructure for responding to researcher queries. Administration
of data sharing programs can cost millions of dollars — equivalent to 15% of a total clinical research budget, according to
early analysis of these undertakings. And outlays would be even higher for retrospective study analysis, which can involve
tracking down participants to redo informed consent.
Conflicting legal regimes in different countries and regions, multiple ethical issues, and complex rules for protecting intellectual
property further complicate the data sharing landscape, explained attorney Mark Barnes of Ropes & Gray. Certain EU member
states place limits on patient data disclosure, while some African nations require special permission for secondary uses of
clinical trial data collected in that country. Current FDA informed consent policy, moreover, could block disclosure because
sponsors now have to assure participants that only a summary of the clinical trial — not de-identified data – will be made
available to outside parties.
By undermining patent protection and data exclusivity, disclosure of clinical trial data could kill a small biotech company,
warned Chimerix president Kenneth Moch. Roger Rosenblatt, chief medical officer of Merck, said that all parties should be
concerned about the "unintended consequences" of disclosure requirements, which can generate misleading data and erroneous
conclusions about drug efficacy and safety. "This is not a theoretical risk," Rosenblatt stated, noting that negative reports
often have dire consequences for vaccines.
The push for transparency has prompted FDA to propose making available de-identified and masked data submitted in market applications,
as described in a request for comments published last June (2013) on whether such disclosure is feasible and if it would lead
to new knowledge that promotes public health. Agency data disclosure is limited by laws protecting trade secret and confidential
commercial information on regulated products. Most comments from industry indicate that de-identifying massive amounts of
clinical data would be challenging and may not be worth the effort, and that the risk of patient re-identification is real.
Such concerns may modify current proposals for more data transparency, but probably won't end them entirely.
Jill Wechesler is Pharm Exec's Washington correspondaent. She came be reached at firstname.lastname@example.org