One clear sign of the times is the success of the campaign for broader access to proprietary clinical data. The conversation
already has shifted from data "disclosure" to data "sharing," as sponsors seek strategies to retain some control over proprietary
information in the face of the European Medicines Agency (EMA) proposal to release patient-level clinical reports submitted
in regulatory dossiers beginning January 2014. FDA is weighing comments on its June 2013 query about making available masked
and de-identified data submitted in applications. And 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 that will assess the benefits and risks of data sharing
and opportunities for responsible disclosure.
Debating Tax Deductions
Pharma companies in the United States and Europe are pressing hard to modify or postpone the EMA data release plan so that
third-party programs and voluntary initiatives will gain time to demonstrate that they can enhance data transparency while
protecting patients and the research enterprise. There is general agreement that clinical data sharing can be beneficial in
improving the efficiency of clinical trials, validating regulatory decisions, and increasing public confidence in clinical
research. But sponsors are leery about who controls access to data, the purposes of disclosure, and the adequacy of safeguards
to protect all parties.
A related fear is that releasing full regulatory dossiers could expose proprietary formulation and manufacturing data and
information on product development and future indications. And public access to clinical data 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 Pharmaceutical Research and Manufacturers of America (PhRMA) briefing in October. He warned
that if the EMA proceeds with its plan for full release of regulatory submissions, companies may rethink their development
and registration strategies, possibly by delaying submissions for approval in Europe or limiting confidential commercial information
To head off the EMA proposal, sponsors are rolling out voluntary data sharing initiatives, as outlined in a "principles" document
issued in July by PhRMA and the European Federation of Pharmaceutical Industries and Associations (EFPIA). Companies are forming
independent scientific review boards to evaluate outside data requests and procedures to protect patient privacy. But there's
a lot of skepticism about how comprehensive and impartial these programs will be.
Paying for access
Data sharing is integral to multiple FDA-industry partnerships formed to evaluate and validate innovative research methods
to accelerate testing of new drugs and medical products. Yet such initiatives frequently draw fire as opportunities for industry
to influence regulatory decision-making. FDA's increased reliance on user fees, as well as its interest in accelerating the
development and approval of breakthrough drugs, prompt critics to question the objectivity and completeness of the agency's
evaluation of new, risky medicines.
FDA is examining whether its policies for managing public-private partnerships are sufficiently transparent and ethical following
a report that industry "paid to play" in collaborative efforts to improve the development and testing of new opioids and other
pain medications. A headlined article in the Washington Post (Oct. 10, 2013) claimed that to participate, pharma companies had to pay a $25,000 sponsor fee to the meeting organizers.
Although the cited activities are based on 10-year-old e-mail communications and have been superseded by other FDA initiatives
and policies, Congress may investigate the case, partly in light of strong public concerns about the marketing and distribution
of illegal opioids.
Low credibility with the public and the medical community makes it difficult for pharma companies to make their case on these
thorny policy issues. The current level of trust in industry-funded research and study results "is extremely low," observe
Lisa Egbuonu-David, director of ROI-Squared, and Tanisha Carino, senior vice president at Avalere Health, in a commentary
published by Health Affairs in September. This "trust conundrum," they note, makes it more challenging for sponsors to produce
credible evidence of the value of new drugs. And such evidence is key to justifying coverage by payers and pharmacy benefit
managers, particularly for costly but critical specialty drugs.
The authors emphasize that it is important to restore trust in industry-sponsored research and to develop innovative models
for obtaining evidence of real-world effectiveness, in a world with increased transparency in the cost of hospital procedures,
medical care, and out-of-pocket spending on medical products that makes consumers more conscious of perceived excessive charges
for medical care. Pharmaceutical companies need to conduct real-world studies on products and be able to discuss results with
key decision-makers. A comprehensive, consistent approach to measuring the clinical value of medical products is central to
a framework that encourages industry funding of scientifically valid research for all stakeholders.
Jill Wechsler is Pharm Exec's Washington correspondent. She can be reached at email@example.com