High prices, murky financial relations, and a reluctance to disclose clinical data are undermining public trust in industry and the research enterprise, writes 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.
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.
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.
To head off the EMA proposal, sponsors are rolling out voluntary data sharing initiatives, as outlined in a “principles” document issued in July by the Pharmaceutical Research and Manufacturers of America (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.