Personalized medicine & comparative research

Electronic health-information systems also are critical for the development of personalized, or individualized, medicine and the necessary diagnostics to determine who may benefit or experience harm from certain therapies. The shift to more targeted treatment requires access to personal health data to recruit research subjects who meet increasingly specific genetic-based enrollment criteria and to identify those individuals who should—or should not—receive a treatment based on specific genotype.

Researchers also will be tapping health-system databases 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. Industry seeks limits on federal funding of 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.

Whatever the outcome of healthcare reform legislation, prescription drug prices will remain in the spotlight. Broader coverage of pharmacy benefits could reduce public complaints about high drug prices, but consumer groups and healthcare providers will continue to publicize how costly medicines block patient access to treatment. Recent reports from Wall Street analysts as well as patient advocates document record increases in wholesale drug prices this past year, at a time when inflation has declined notably. The studies exposed manufacturers to charges of jacking up revenues in anticipation of tighter controls on pharmacy spending.

Such developments will continue to build support for greater access to new generic drugs, including follow-on biologics (FOBs). Congress is poised to establish a regulatory pathway for approving biosimilars for market, as there is agreement between generic-drug manufacturers and innovator firms on FDA regulatory approaches and clinical-testing requirements.

But there's still strong disagreement on data exclusivity issues, as pharmaceutical and biotechnology companies successfully pushed for 12-years of exclusivity on FOBs. Manufacturers on all sides will be assessing how the various requirements and protections will shape the development of biosimilar products.

More transparency

Transparency is a recurrent theme in healthcare reform and regulatory matters. Efforts to lower drug prices will seek broader disclosure of manufacturer rebates and discounts negotiated by payers and health plans. Congress further aims to shed "sunshine" on drug company payments to physicians as a way to reduce industry influence on prescribers.

Manufacturers also face more transparency in FDA regulatory decisions and pending drug applications. Commissioner Hamburg established a high-level FDA transparency task force last June to identify ways to better inform the public about FDA operations, a move designed to enhance agency credibility.

Under the leadership of FDA Deputy Commissioner Joshua Sharfstein, the task force is examining whether it should be able to disclose when an application is filed, withdrawn, or turned down by the agency; to discuss data contained in product applications that are abandoned or withdrawn; and to reveal the contents of complete response letters sent to sponsors.

The panel is examining what kind of emerging safety information should be disclosed and when, and whether early communication about ongoing safety reviews of foods and medical products would be beneficial or raise unnecessary concerns about a product. 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 would require new regulations or legislation.

Assessing healthcare reform

However Congress resolves the great health-reform debate of 2009, there will be lasting effects on pharmaceutical manufacturers. The discussion has brought to the fore a range of proposals for improving the Medicare Part D drug benefit, reducing the cost of drugs to state Medicaid programs, and stimulating more CER on prescription drugs. These issues are not likely to disappear.

Congressional leaders have made it clear that pharmaceutical companies should accept lower drug prices in exchange for policies to boost drug coverage to some 30 million potential customers. Cost-cutting strategies include discounts on drugs provided to Medicare beneficiaries caught in the Part D coverage gap, added rebates on drugs provided to low-income Medicare beneficiaries, higher rebates on drugs dispensed by state Medicaid programs, and various fees and disclosure requirements.

Congress is anxious to gain any and all savings from the pharmaceutical industry because reform legislation does very little to "bend the cost curve" on the nation's healthcare system. Proposals to curb spending by cutting fraud and waste, establishing electronic medical records, assessing the effectiveness of medical treatments, and bolstering preventive care may improve healthcare quality, but won't make any real dent in federal expenditures. The choice for members of Congress is to adopt admittedly imperfect reform legislation, or to admit failure and abandon the entire exercise. Either way, the outcome will be crucial to pharmaceutical manufacturers as we move into 2010.

Jill Wechsler is Pharmaceutical Technology's Washington editor, 7715 Rocton Ave., Chevy Chase, MD 20815, tel. 301.656.4634, jwechsler@advanstar.com