The FDA intends to accelerate the review process this year forboth drugs to treat fatal and debilitating diseases and for less-critical drugs.
Several measures are under way at FDA. The user feelegislation, passed late last year, will provide the funds to hireabout 600 additional drug reviewers to alleviate the agency'schronic short-handedness. But how quickly this will beimplemented is still up in the air, said FDA spokeswomanMonica Revelle. Although the agency has been studying howother agencies handle user fees, money for the programs hasnot yet been appropriated.
For the treatment of fatal and debilitating diseases such asAIDS, Alzheimer's and cancer, FDA will allow products to bemarketed based on surrogate end points -- physiologicalmeasure of safety and effectiveness -- instead of survival.Companies will be required to confirm the surrogate data withhuman trials, and the agency will require physicians to certifythat patients need such drugs.
A streamlined withdrawal process will allow FDA to quicklypull such drugs from the market if they begin to prove unsafeor ineffective of if the company fails to perform the humantrials.
For biologics, the agency has reorganized the Center forBiologics, Evaluation and Research (CBER), and will be addingabout 50 reviewers to hasten the review process there.
The reorganization involves creating special offices forresearching and reviewing blood, therapeutics and vaccination.Each of these offices will have final authority for productapproval, eliminating the need to forward decision making tothe director.
Reactions to the measures are varied. One former higher-up inFDA expected nothing but business as usual from the agency."All the things people are talking about have been tried in thepast, as early as 1950," the source said. "You don't needregulations, you need a change of attitude among the reviewersto get drugs out faster. In my judgment, it's improbable thatwill happen."
As for increasing the number of reviewers, "you give someoneplenty of free time to look at an NDA, and they may ask 10times more questions," the source said.
What more reviewers will help most, said Abbey Meyers,executive director of the National Organization for RareDisorders, is the "me-too" drugs.
But Martin Delaney, founding director of Project Inform, anAIDS information group, said, "There is no question the agencyis grossly understaffed. More reviewers should help." And theextra people "can be used in these crunches we put the agencythrough from time to time, the urgent, accelerated approvals,"so that "the rest of the agency need not be put on hold."
Meyers expressed concern that using surrogate markers maybackfire. "Very often drugs that work on animals don't work onhumans."
But, said Delaney, "long-term survival outcomes are notpractical when you are fighting a life-threatening illness."
Generally, said Meyers, earlier approval is based on whichdisease group makes the most noise, not pain and suffering."They (FDA) are looking at AIDS and cancer, period."
However, William Small, executive director of the Associationof Biotechnology Companies, said he is optimistic that the userfee law will work well. But "we will be closely monitoring theirefforts, and we will hold their feet to the fire if things don'tspeed up."
Jeffrey Trewhitt, director of media services at thePharmaceutical Manufacturers Association, was even moreoptimistic. "The user fee will give (FDA) the added money toreally streamline the drug approval process." But don't look fortoo much change too quickly, he said. "The agency has only justbegun the reform process, and they have a long way to go."
He was also optimistic about the reorganization of CBER. "Thereis a change of attitude in that office that we think is veryconducive to improving the system. (CBER Director) Kathy(Zoon) is not going to take any guff."
-- David C. Holzman Special to BioWorld
(c) 1997 American Health Consultants. All rights reserved.