The new year begins on a note of optimism. A major breakthrough in stem cell research promises to open the door to new biomedical
research opportunities. The drawn-out Congressional debate over user-fee reauthorization and drug safety regulation is over,
and most parties seem satisfied with resulting compromises. The vaccine industry is experiencing a resurgence after years
in the doldrums, with important new vaccines on the market and more under development. And unlike many previous years, the
US Food and Drug Administration (FDA) had a confirmed commissioner for all of 2007 and relatively stable leadership.
Manufacturers will be busy in coming months as they face continued scrutiny of safety signals, pressure to discover important
new treatments, and push back on high prices for medical products. Some of the leading policy issues on the table for 2008
• Implementing FDAAA. The legislative wrangling is over, and now the lawyers are in charge of deciphering what members of Congress really intended
for many complex and contentious portions of the FDA Amendments Act. There is considerable uncertainty about how and when
manufacturers will have to file Risk Evaluation & Mitigation Strategies (REMS), especially for high-risk drugs already on
the market. FDA has to clarify a process for tracking and enforcing compliance with Phase 4 study commitments. And new requirements
for registering a greatly expanded range of clinical trials and trial results present a substantial challenge for FDA and
the National Library of Medicine.
FDA Deputy Commissioner Janet Woodcock indicated that a REMS may not be required for all drugs, in discussing Food and Drug
Administration Amendments Act (FDAAA) at a Food and Drug Law Institute conference in November. The agency will use its REMS
authority "judiciously," she commented, noting that too many restricted drug distribution programs could increase confusion,
harm patients, and "decrease access to the therapy."
• Safety slowdown? While implementing new programs, FDA faces the challenge of demonstrating that it has not become overly gun-shy about approving
new drugs for market. New drug approvals were down last year, as were the number of new molecular entities coming to market.
Manufacturers complain that the agency is issuing more approvable letters that demand additional trials and testing before
final approval, and that many approvals carry narrower indications. Black box warnings seem to be proliferating, as well as
agency safety alerts and advisory committee reviews of safety issues for certain drugs and drug classes.
Delays in launching new products can make a serious dent in manufacturer business plans, while the emergence of postmarket
safety issues can be disastrous for marketed products. FDAAA provides FDA with additional tools for assessing product safety
before and after approval, but the political rhetoric about manufacturer malfeasance is sure to continue in this election
• Attack on drug pricing. The Medicare Part D drug benefit has significantly increased government payments for drugs, intensifying public scrutiny
of prices, especially for costly biotech treatments. For example, high expenditures for anemia-fighting erythropoietin sparked
Congressional investigations and led to a curb on Medicare reimbursement for the drug.
The desire to reduce the nation's prescription drug bill continues to fuel the campaign to grant the government authority
to negotiate drug prices for Medicare Part D plans, for curbs on drug advertising, for more disclosure of manufacturer prices,
and for greater access to low-cost generic drugs. To further this last goal, Congress will continue to seek a pathway for
FDA to approve follow-on biologics. The legislators decided to drop such a proposal from FDAAA, but generic makers have not
let up efforts to resolve the issue, and projections of large cost savings from biosimilars is likely to keep it on the front
burner in the coming year.
• Push for comparative effectiveness analysis. Efforts to determine appropriate prescribing and pricing of medical treatments stand to benefit from comparative effectiveness
reviews of medical procedures and products. Better information on what medical treatments work best for certain patients could
reduce inappropriate drug utilization, avoid unnecessary or harmful procedures, and support a more efficient and less costly
healthcare system. These possibilities are boosting support for a larger government role in assessing medical treatment effectiveness,
while health plans and payers also are demanding more evidence that medical products are cost-effective as well as safe and