FDA Reform in Congress: AIDS Community Absent



Far-reaching FDA reforms are now being proposed by
Congressional Republicans, who may have the votes to force
major changes in how FDA operates. The goal of many of these
changes is faster and less expensive approval of important
new drugs; but some proposals could also increase the risk of
ineffective drugs being approved (the advocates insist that
standards for safety will not be affected). As FDA reform
moves with unexpected speed into prominence in the Republican
Congressional agenda, it is important that those concerned
about AIDS be involved in the process and make our views
known. But so far the AIDS community is largely ignorant and
unengaged in what is happening with this issue.

A number of proposed reforms will be discussed next week in
hearings by Senator Nancy Kassebaum (Republican, Kansas) of
the Senate Labor and Human Resources Committee -- who,
incidentally, is regarded as an important friend of people
with AIDS, due to her work on reauthorization of the Ryan
White Care Act. At the hearings, scheduled for April 5 and 6,
legislation to amend the Food, Drug, and Cosmetic Act is
expected to be introduced. As we go to press we do not know
what the legislation will contain, but the following
proposals have been discussed:

* Accelerated Approval. Under current law, a drug cannot be
approved without "adequate and well-controlled trials"
(generally interpreted as more than one trial). A proposed
change would require the FDA to approve a drug if the risk of
the drug is no greater than the risk of the condition, and
the drug is reasonably likely to be effective in a
significant number of patients. This judgment could be based
on expert opinion; under the proposal, phase III efficacy
trials would not necessarily have to be done before approval.
FDA's refusal could be taken to court, and conceivably the
courts could order the FDA to approve the drug.

* Phase I/II Testing. Another proposal would allow IRBs
(institutional review boards) to approve a phase I or phase
II test of a new drug; the FDA would have to be notified, but
its approval would not be required before the trial could
proceed. The FDA would still have to approve phase III
trials; and sponsors could submit phase I and phase II
protocols to the FDA, instead of to an IRB, if they wished.

[Comment: This change could be important in reducing the
great difficulty of getting the earliest human data on new
treatment ideas. Current law gives the FDA 30 days to reply
to an application for an IND; if it does not reply, the trial
can go forward. But sometimes, just before the deadline, the
researchers receive a long list of objections which would be
almost impossible to satisfy. Some experts believe this
happens because the FDA does not have the staff to meet the
30-day requirement, and is controlling its workload by
effectively eliminating some research proposals. In any case
the impact may be greatest on creative ideas that do not
already have widespread support -- and may in part be
responsible for the lack of creative ideas in AIDS treatment
research. Allowing IRBs to approve early trials could bypass
this bottleneck.]

* Off-Label Information. Currently the FDA greatly restricts
the ability of companies to distribute information about
unapproved uses of approved drugs -- even though some
unapproved uses become the standard of care in widespread use
by physicians. A proposed change would allow companies
limited distribution of information about unapproved uses,
through a special section in advertisements or by giving
peer-reviewed scientific publications to physicians or
others. This could help physicians be more educated about de
facto new uses of drugs when those uses have not been
approved by the FDA.

These are only a few examples of some of the proposed
legislative changes which would affect the FDA. Other
proposals are intended to speed the advisory-committee
process, allow easier export of experimental drugs when legal
in the receiving countries, allow minor manufacturing changes
without prior FDA approval, and preventing the RAC
(Recombinant Advisory Committee) at the U.S. National
Institutes of Health from having separate jurisdiction over
clinical trials, in order to focus this jurisdiction in a
single agency, the FDA.