FEDERAL PANEL SEEKS DRUG-APPROVAL REFORMS

Two years ago, then Vice-President George Bush asked the
President's Cancer Panel to study the Federal role in new-drug
development and approval of treatments for cancer and AIDS, and
to suggest changes to speed research, improve patient access to
new therapies, and facilitate their transfer to standard medical
practice. The President's Cancer Panel appointed the National
Committee to Review Current Procedures for Approval of Drugs for
Cancer and AIDS -- usually called the Lasagna Committee, after
its chair, Louis Lasagna, M. D. The Lasagna Committee held ten
hearings between January 1989 and April 1990; on August 15 it
issued its final report, a 25-page document with 20
recommendations for improving the drug- approval process.
Recommendations include:

* Adopting "a national policy...to foster the development of
new drugs for AIDS and cancer." A permanent oversight committee,
appointed by and reporting to the Secretary of Health and Human
Services, would monitor the needs and performance of the FDA in
drug regulation. (Note: the importance of this recommendation
is that today there is no such national policy, and no one
responsible when the system as a whole is not working. Each
office just does its own job, even when it is clear that disaster
will result.)

* Severe staff and equipment shortage at the FDA can be
addressed not only by providing more resources, but also by
allowing institutional review boards, and outside contractors
approved by the FDA, to do some of the reviews now done by staff.
The Committee recommended that phase I trials could optionally
be approved by a qualified institutional review board (IRB)
instead of by the FDA. Also, pharmaceutical companies could
optionally pay the FDA to rapidly review an NDA (marketing)
application by contracting the work to outside experts. These
changes are necessary because "Congress has placed many new
responsibilities on the FDA in the last decade, while the number
of employees in the agency has decreased... In the short term no
quick solution is apparent to these inadequate resources."

* FDA advisory committees should have a much larger role,
with their own staffs, and responsibility for their own agendas.

* Medicare, Medicaid, and private insurance should pay for
experimental drugs and unlabeled uses of approved drugs, and all
associated medical care, "if the use has been approved by expert
government agencies, in authoritative medical compendia, or by a
committee established by the Secretary of Health and Human
Services." Coverage should be the same under Medicare, Medicaid,
and all private insurance, and should not vary between geographic
areas. Individual insurance companies "should have no discretion
with respect to such matters."

* Earlier marketing approval for AIDS and cancer drugs
should occur as soon as there is credible evidence of efficacy,
with other research being completed after approval. The
Committee noted that the large phase III trials, which commonly
take years, have little practical impact on approval, since 90
percent of drugs which pass phase II also pass phase III.

In addition, "It is only after initial NDA approval of the
drug as a single entity that its full potential is realized,
because physicians are then free to use it in combination with
other drugs in accordance with their best clinical judgment.
While still under investigation, such combination uses occur only
infrequently and with little opportunity for full clinical
exploration. For all of these reasons, large phase III studies
have been, and should continue to be, conducted in the post-
approval setting."

* Other recommendations include support for community-based
trials, for parallel track if it does not delay trials, and for
the FDA's responsiveness to patient advocacy groups. It urged
improved relationships between FDA and pharmaceutical- company
personnel, with more open communication.

To obtain a copy of the report, contact the Committee's
executive secretary: Dr. Elliott H. Stonehill, National Cancer
Institute, Bethesda, MD 20892, 301/496-1148.