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 goal is to create a surveillance system with access to product usage data on 100 million people in the US. Access to the
data is not an issue; sources include claims data, transactional data, and billing data, as well as the government's own sources
such as Medicare and the VA.
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
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