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High prices, murky financial relations, and a reluctance to disclose clinical data undermine public trust in industry and the research enterprise.
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.
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.
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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.
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 firstname.lastname@example.org.