The Push for Transparency: Risks, Costs, and Concerns

Transparency initiatives are multiplying, affecting research, marketing and compliance, writes Jill Wechsler.

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.

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

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.

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.