Kaposi's Sarcoma: DOX-SL Approval Recommended



The FDA's Oncologic Drugs Advisory Committee voted eight to
zero to recommend that DOX-SL be given accelerated approval
for treatment of AIDS-related Kaposi's sarcoma (KS) in
patients who have failed conventional treatment. Under
accelerated approval, the company will be required to conduct
additional research, continuing after the drug is approved.
The committee had previously voted ten to zero against
approving DOX-SL without requiring the further research --
and some earlier critical votes were close. (Some newspapers
got the story wrong, and erroneously reported that the
committee had rejected the drug.)

AIDS TREATMENT NEWS was not at the February 14 hearing; the
best report we have seen was in BioCentury, a weekly faxed
newsletter published by BioCentury Publications Inc., San
Carlos, California. Apparently the committee was unhappy with
data analysis provided by the drug's developer, Liposome
Technology Inc., and based the approval recommendation on the
FDA's analysis.

Comment

Although this case ended well, it illustrates an ongoing
problem with the current drug-approval system. Each advisory
committee -- in this case consisting of cancer experts, not
AIDS experts -- sees many approval applications over the
years, and has developed its own "corporate culture" for
judging them. This corporate culture can include an
accumulation of in-group or academic criteria, each of which
may have been useful in making some decision in the past, and
which then become institutionalized.

When a major pharmaceutical company which is well-accustomed
to doing business with a particular committee brings a new
drug before it, this ingrown academic or committee culture is
not a problem; it may even be a benefit. Everyone knows the
rules, and the rules can improve over time.

But a smaller biotechnology company, which may have a single
product, is likely to be coming before the committee for the
first time, having seen only one new-drug application -- its
own. It is at a serious disadvantage because it has no
background in the nuances of the committee. And the committee
cannot easily compensate for this inexperience, because its
members have little background in the company, its people, or
(often) its technology. This can and does lead to the
mistaken rejection of important drugs which clearly benefit
patients.

The FDA saved the day in this case, as it has done in some
other cases before. The problem is that we should not have to
keep our fingers crossed for the system to work. Changes in
personnel, or just bad luck, could lead to the kinds of
delays which people with AIDS routinely suffered in the early
years of the epidemic.

It is easier to point to a problem than to suggest a
solution. Perhaps the FDA could develop a body of knowledge,
based on past experience with its committee system, to help
committee members avoid certain perennial mistakes.