Drug pricing, safety, access to dominate agenda in the year ahead

Key Points

The global economic downturn, the contentious debate over national health reform, and important changes on the regulatory front have taken a toll on biomedical research and new drug development. The new year brings optimism for clarity on healthcare coverage and regulatory requirements. National healthcare reform legislation either will become law in some form, or die a painful death.

The new team heading up FDA will be firmly established and eager to implement new policies and programs. Payers, plans, and formulary committees will be watching key trends that will shape drug development and coverage in 2010:

PRICES AND PATHWAYS

Such developments will continue to build support for access to generic drugs, including follow-on biologics. Congress is poised to establish a regulatory pathway for approving biosimilars for market, based on agreement among generics makers and innovator firms on FDA regulatory standards and clinical testing requirements. But a 12-year market exclusivity for innovator products sought by biopharma companies raises concerns for payers and could limit generic drug company investment in follow-on products.

DRUG SAFETY CHALLENGES

Pressure to prevent exposure to adulterated medicines and to improve detection of drug safety problems is prompting multiple initiatives at FDA. The FDA Amendments Act of 2007 (FDAAA) authorized post-approval label changes when new safety issues arise and penalties for manufacturers that fail to conduct agreed-on post-marketing studies.

There are mandates for more extensive listing of clinical trials and study results on http://www.clinicaltrials.gov/ and for Risk Evaluation and Mitigation Strategies (REMS) governing post-market prescribing and surveillance. FDA has approved REMS programs for almost 90 products and is formulating a REMS for the entire class of extended-relief opioid medicines; a more coordinated risk management strategy aims to ensure continued access to these medications for patients suffering from chronic pain, while also curbing inappropriate prescribing, unintentional overdosing and intentional abuse.

Researchers, health professionals, and patient groups will be watching closely to see how well these programs detect and prevent drug safety problems and whether these efforts support more FDA flexibility in approving somewhat risky new products for market. Internally, FDA has launched a Safety First program in the Center for Drug Evaluation and Research (CDER) to clarify responsibilities for pre- and post-approval drug safety assessment and to strengthen safety-related policies and procedures. The agency's Sentinel Initiative aims to monitor medical product safety more effectively through an electronic system linked to healthcare data bases. Starting with several pilot projects, the system eventually will be able to detect emerging safety problems in real time and identify patient subgroups experiencing adverse events.

And FDA's Safe Use Initiative, which Commissioner Margaret 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 partner with other government agencies and with healthcare providers and plans to address situations that lead to medication errors and inappropriate prescribing.

IT ADOPTION

The Sentinel System requires access to electronic health information systems, a prominent goal of national health reform efforts. Earlier economic stimulus legislation jump-started this effort by providing $19 billion to support use of electronic health records by doctors and hospitals and to develop standards and systems supporting electronic health information exchange. Healthcare 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. This raises concerns that privacy issues could limit access to patient information needed for pre-clinical and post-approval studies and for ensuring appropriate prescribing and treatment.

PERSONALIZED MEDICINE, COMPARATIVE RESEARCH

Electronic health information systems also are critical for the development of 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 identify those individuals who should-or should not-receive a treatment based on specific genotype.

Researchers also will be tapping health system data bases to assess 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 comparisons, but more CER will inevitably highlight comparative prices as well as health benefits.

GLOBAL VACCINES, TREATMENTS

The rising importance of overseas markets for drugs and medical products is prompting more investment by pharmaceutical companies in new treatments for malaria, tuberculosis and neglected tropical diseases. Global funding of a vaccine to combat the H1N1 influenza pandemic also has spurred research and construction of vaccine manufacturing facilities in the United States and abroad.

New vaccines to combat malaria and TB are in clinical trials, and scientists remain optimistic about moving forward with the long-sought vaccine to prevent human immunodeficiency virus infection. Novel antigens are in the works; an anti-smoking vaccine is moving into late-stage testing; and scientists continue to seek therapeutic vaccines for cancer and other conditions.

FDA is encouraging such efforts, and is likely to do more under Commissioner Hamburg, who has a strong background in public health and infectious disease. The agency is supporting development of new tuberculosis treatments to replace ineffective old therapies. To better monitor the growing volume of pharmaceutical imports and foreign clinical trials, FDA also is establishing overseas offices and doubling the number of foreign inspections it conducts each year.

MORE DISCLOSURE

"Transparency" is a recurrent theme in health reform and regulatory matters. Efforts to lower drug prices are likely to involve broader disclosure of manufacturer rebates and discounts negotiated by payers and health plans. Congress further seeks to shed "sunshine" on drug company payments to physicians as a way 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 is examining whether FDA should be able to disclose more information about new drug applications filed with the agency; what kind of emerging safety information should be disclosed and when; and whether early communication about ongoing product 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.

DIGESTING HEALTH REFORM

However Congress resolves the great health reform debate of 2009, there will be lasting effects on pharmaceutical research, costs and coverage. The discussion has brought to the fore a range of proposals for improving the Medicare Part D drug benefit, for reducing the cost of drugs to state Medicaid programs, and for stimulating more comparative effectiveness research on prescription drugs, and these issues are not likely to disappear. Policymakers will continue to seek ways to close the Part D coverage gap, increase manufacturer rebates, and impose additional fees and disclosure requirements on healthcare providers and products. Closer links are likely between FDA approval and Medicare coverage decisions. These developments will affect private plans and payers and broader access to medicines.

Ms Wechsler is a Washington-based reporter specializing in federal and state healthcare issues.