Sponsors, health professionals, and patient groups will be watching closely to see how well these programs prevent drug safety
problems, and whether these efforts support more FDA flexibility in approving somewhat risky new products. Internally, FDA
is bolstering its safety assessment capabilities. A Safety First program in the Center for Drug Evaluation and Research (CDER)
seeks to clarify responsibilities for pre- and postapproval drug safety assessments. The agency's Sentinel Initiative aims
to detect medical product safety problems more effectively through an electronic monitoring system linked to healthcare databases.
Sentinel eventually should detect emerging safety problems in real time and identify patient subgroups experiencing adverse
FDA's Safe Use initiative, which commissioner Hamburg announced in November, aims to build partnerships with other components
of the nation's healthcare system to ensure that medicines are used safely and appropriately. FDA plans to collaborate with
other government agencies and healthcare providers to address situations that lead to medication errors and inappropriate
These programs require access to electronic health information systems, a prominent goal of national health reform. Earlier
economic stimulus legislation aimed to spur e-health development by providing $19 billion to support adoption of electronic
health records by doctors and hospitals and to develop standards and systems supporting electronic health information exchange,
including e-prescribing. Electronic databases promise to accelerate detection of drug adverse events and to streamline data
collection from clinical trials and population studies.
At the same time, broader access to personal health information has generated demands for stronger protections against unauthorized
disclosure of patient records. The concern for the biomedical research community is that privacy issues could limit access
to patient information needed for pre-clinical and postapproval studies. Research experts are exploring alternative models
for health surveillance and assessment based on the data-sharing capabilities of electronic information systems, approaches
that may gain more attention in coming months.
Personalized Medicine and CER
E-health systems also are critical for developing personalized, or individualized, therapies and the diagnostics needed to
determine who may benefit or experience harm from more targeted treatments. The shift to personalized medicine requires access
to personal health data to recruit research subjects who meet genetics-based enrollment criteria, and to identify those individuals
who should—or should not—receive a treatment based on specific genotype. Long-promised FDA guidance on developing personalized
medicine and diagnostics could give the field a welcome push.
Researchers also will be tapping health system databases to assess health outcomes and to compare the risks and benefits of
medical products. Federal agencies are funding a number of comparative effectiveness research (CER) projects with an eye to
assessing a broad range of medical treatments and strategies. Pharma companies seek limits on government-funded studies that
compare one drug to another and focus on cost issues, but more CER will inevitably highlight comparative prices as well as
"Transparency" is a recurrent theme in health reform and regulatory matters. Efforts to lower drug prices could require broader
disclosure of manufacturer rebates and discounts negotiated by payers and health plans. Congress further seeks to shed more
"sunshine" on drug company payments to physicians to reduce industry influence on prescribers.
More transparency in FDA regulatory decisions also is on the horizon. Commissioner Hamburg established a high-level transparency
task force last June to identify ways to better inform the public about FDA operations—and enhance agency credibility in the
process. The panel, headed by deputy commissioner Joshua Sharfstein, is examining whether FDA should be able to disclose more
information about drug applications filed with the agency; what kind of emerging safety information should be disclosed and
when; and whether early communication about ongoing safety reviews would be beneficial or raise needless concerns. The panel
will issue a report in the coming year on what information FDA should be able to release on its own, and what changes require
new regulations or legislation.
Jill Wechsler is Pharmaceutical Executive's Washington correspondent. She can be reached at firstname.lastname@example.org