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 events.

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 prescribing.

IT Adoption

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 health benefits.

More Disclosure

"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 jwechsler@advanstar.com