Pharmaceutical Executive | Jill Wechsler
It seems like open season on
the pharmaceutical industry. Academics and consumer activists charge pharma
companies with hiding clinical trial information on medical product safety,
fueling the campaign to expand public access to confidential research
information. The federal government's "Sunshine" program for
disclosing financial ties between industry and physicians reflects a lengthy
campaign to curb marketing tactics perceived to boost inappropriate
prescribing.
Media reports regularly attack
high drug prices, both for life-saving specialty drugs and for widely used
treatments such as asthma inhalers. And recent disclosures raise questions
about too-close ties between pharma companies and Food and Drug Administration
officials.The well-known industry response to these and other charges is that prescription drugs account for only 10 percent of US spending on healthcare and that appropriate drug use saves money by keeping people out of hospitals and operating rooms. Developing new drugs, moreover, is enormously expensive and risky, warranting strong patent protection and a healthy return on investment.
Such arguments, sadly, fail to
generate public confidence in the biomedical research enterprise. Public
surveys give pharma companies poor ratings, citing high prices, low integrity,
and failure to disclose unfavorable safety information. There's a clamor for
valid data on drug effectiveness and comparative prices, and high hopes that
health reform initiatives will make such information more transparent.
Data disclosure
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.
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 in dossiers.
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.
Trust critical
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.