THE HARBINGER

After three years of exhaustive hearings and debates, President Bush signed the FDAAA into law in September 2007. Dubbed the "drug safety law," the legislation grants additional authority to FDA and strengthens its focus on drug safety and efficacy. It grants FDA $25 million per year through 2012 to develop methods for obtaining observational, post-market patient-level information through public–private collaborations.

THE CHANGE

With this provision, postmarketing data collection and analysis will move into the public domain. The resulting landscape will not only be more regulated but also more transparent as the information becomes more widely available to third parties—including academic researchers, patient advocacy groups, the media, and the general public.

FDA has signaled that it will not control these data sources, but will rely upon a distributed data network. It will also coordinate adverse event reporting with the NIH so that a single submission will serve both agencies.

The real issues would seem to be around managing, rather than acquiring, the data. As stated by Janet Woodcock, director of the Center for Drug Evaluation and Research at FDA, "The science is not developed yet.... We get all this data, but what does it mean?"

THE IMPLICATIONS

There remain valid questions about the potential applications of this patient-level information. Given the goals of FDA, expect the information to be used for:

• Proactively identifying patterns of adverse events and safety issues
• Pharmacovigilance, including detection, evaluation, and prediction of safety issues
• Risk management planning and implementation
• Wider and easier access to information for meta-analysis
• Tying postmarketing data to clinical trials information
• Identifying patterns of disease incidence and treatment outcomes
• Comparing effectiveness of treatment alternatives

In the US, a public–private partnership, the Observational Medical Outcomes Partnership (OMOP), may lead the way in determining the value of using observational data to identify the safety and benefits of prescription drugs. This partnership will be governed by FDA, led by the NIH, and funded by PhRMA.

With the growing involvement of NIH, the pre- and post-regulatory environment has the potential to become more complex. Increased transparency could increase risk for manufacturers, insurers, academic centers, and employers, who will all have access—and different economic motivations and goals. FDA has estimated that just one of 10 adverse events are actually reported. Will this new system lead to a big increase in the number reported? If so, what will that mean in terms of damage to marketed products and pharma's overall image?

Companies will need to scan the data-bases themselves for adverse events so that they can head off negative FDA and public reaction to "false positives." They also will need a plan for working with FDA to respond in a timely and appropriate manner to real safety issues.

With the passage of the FDAAA, active surveillance may become "the future of healthcare," says Woodcock. This could bring forth a new phase of drug regulation, with the focus moving from clinical trials to performance in a real-world setting.

IMS Editorial Board: MURRAY AITKEN, Senior Vice President, Healthcare Insight; DIANA CONMY, Corporate Director, Market Insights; EVA EDERY, Senior Principal, Thought Leadership, Europe; GRAHAM LEWIS, Vice President, Global Pharma Strategy; JOHN MACCARTHY, Vice President, Thought Leadership, Europe; JOHN MORAN, Center of Excellence Leader, Marketing Optimization; SARAH RICKWOOD, Senior Principal, Thought Leadership, Europe