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, with most
parties satisfied with 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. And unlike many previous years, the Food and Drug Administration had a confirmed
commissioner for all of 2007, lending stability to the agency.
But this does not signal any slowdown in Congressional hearings and investigations. As the 2008 presidential campaign heats
up, the pundits and politicians will have much more to say about the hot topics on this year's agenda:
FDAAA Implemention 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 [see "Whither FDA 'Revitalization'?]. There is considerable
uncertainty 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 has to clarify 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.
FDA Deputy Commissioner Janet Woodcock indicated that a REMS may not be required for all drugs, in discussing 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 and decrease access to the therapy.
One serious complication when Pharm Exec went to press last month was that FDA could not yet use the added revenues authorized under the Prescription Drug User Fee
Act (PDUFA) section of FDAAA. The agency collected the higher fees, but could not spend the money until Congress approved
its 2008 appropriation bill, which was caught in the wrangling between Democrats and the Bush administration over federal
government spending. Efforts to modernize FDA's IT system and to expand access to outside safety information, among other
new projects, may get off the ground very slowly.
Approval slowdown While wrestling with new programs, FDA is under pressure to demonstrate that it has not become overly gun-shy about approving
new drugs for market. New drug approvals were down last year, as well as the number of new molecular entities (NMEs). Manufacturers
complain that the agency is issuing more "approvable" letters, which demand additional trials and testing before final approval
and often lead to narrow 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.
FDA has launched a pilot program to reassess the risks and benefits of typical NMEs 18 months after approval. The agency hopes
that it can speed up approvals and reduce postmarket crises by identifying ways to detect safety issues earlier in the review
process. FDAAA provides FDA with additional tools for assessing product safety before and after approval, but that may not
quell the political rhetoric about manufacturer malfeasance and FDA ineptitude.
Imports scrutiny Drug safety issues, along with the concerns generated by contaminated Chinese pet food and toys, have focused public attention
on how well FDA and other government agencies can detect and halt the import of dangerous products. The Bush administration
issued an Import Action Safety Plan in November that offered few new ideas or funding specifics but provides a basis for Congressional
action. Legislators already are raising questions about the quality of foreign drug products and how well FDA monitors the
growing volume of drugs and drug substances from China, India, and other countries. Investigators cite inadequate FDA computer
systems for tracking foreign manufacturers and for inspecting overseas plants in a timely and efficient manner. There are
proposals to authorize FDA curbs on inadequately documented imports and to collect user fees on imports to expand foreign
inspections; those revenues also could support proposed FDA satellite offices in China and India.