Jill Wechsler
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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 are satisfied with the resulting compromises. The Medicare drug benefit moved into its third year showing
signs of maturity and acceptance. The vaccine industry is experiencing a resurgence after years in the doldrums, with important
new vaccines on the market and more under development. Unlike many previous years, the US Food and Drug Administration had
a confirmed commissioner for all of 2007, making for relatively stable leadership.
Manufacturers will be busy in the coming months dealing with continued scrutiny of safety signals, pressure to discover important
new treatments, and resistance to high prices for medical products. This column will examine 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
in many complex and contentious portions of the FDA Amendments Act (FDAAA). Considerable uncertainty remains about how and
when manufacturers will have to file Risk Evaluation and Mitigation Strategies (REMS), especially for high-risk drugs already
on the market. FDA must define a process for tracking and enforcing compliance with Phase IV 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.
In Washington This Month
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FDA Deputy Commissioner Janet Woodcock indicated that a REMS may not be required for all drugs when she discussed FDAAA at
a Food and Drug Law Institute conference in November 2007. 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."
Woodcock is temporarily heading the Center for Drug Evaluation and Research (CDER), following the departure of former CDER
director Steven Galson. She is thus responsible for issuing guidances, determining the need for revised regulations, and establishing
the many policies FDAAA requires under tight timeframes.
A serious complication when Pharmaceutical Technology went to press last month was that FDA was not yet able to use the added revenues authorized under the Prescription Drug User
Fee Act section of FDAAA. Although FDA could start collecting the higher fees, it cannot spend the money until Congress approves
its 2008 appropriations bill, which has been delayed by partisan wrangling between Democrats and the Bush administration over
all federal government expenditures. Because of these circumstances, efforts to modernize FDA's adverse-event reporting system
and to expand access to outside safety information to fulfill new requirements may be slow to get off the ground, as required
by FDAAA.
Slowing down for safety
While implementing new programs, FDA faces the challenge of demonstrating that it has not become gun-shy about approving new
drugs for market. New drug approvals were down last year, including the number of new molecular entities (NMEs) 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.
In just the last few months, for example, FDA has been examining safety issues related to asthma drugs, attention deficit
hyperactivity disorder products, and cold remedies for children. The agency recently issued an alert that a smoking-cessation
treatment may promote behavioral changes, and that an immunosuppressant drug carries added risks for pregnant women. The possibility
that osteoporosis treatments may raise cardiac risks is under review.
