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Does
the drug approval process work? That’s an important
question at any time, but especially now, because
Congress is considering legislation to reform the FDA
process. The FDA Prescription Drug User Fee Act
expires in 2007, and since the renewal is must-pass
legislation, it’s an obvious vehicle for FDA reform.
Senators Michael Enzi and Ted Kennedy are working on a
reform bill aimed at preventing future Vioxx-like
scandals, improving public disclosure of clinical
trials, and reducing conflicts of interest in FDA
advisory committees. At press time, the bill was still
in draft form.
Meanwhile,
a very different kind of reform is proposed in S.1956,
The Access, Compassion, Care and Ethics for
Seriously Ill Patients Act, short name The Access
Act, filed by Senators Brownback and Inhofe with
cosponsors Allen, Isakson, and Sessions. That bill
aims to make it easier for seriously ill patients,
such as those with terminal cancer, to receive drugs
that are not yet fully approved by the FDA. The bill
sets a tiered system for drug approvals corresponding
to the three phases of clinical trials: Companies
could market drugs after Phases I and II, with
suitable warnings and controls.
Each
bill addresses problems at opposite ends of the
drug-disease spectrum. The Access Act addresses the
needs of the relatively few seriously ill patients who
urgently need treatment and are willing to try drugs
that are not fully tested; the Enzi and Kennedy bill
addresses the needs of everyone else who can wait a
while and want more assurances about the drugs they
are taking.
The
Access Act follows up on a Federal appeals court
ruling in a lawsuit brought by the Abigail Alliance
for Better Access to Developmental Drugs. The appeals
court ruled that patients have a constitutional right
to drugs that have achieved Phase I approval, since
such approval implies that the FDA has found the drug
to be safe enough and promising enough to warrant use
by the relatively large number of patients needed for
a Phase II trial (1). The ruling left huge loopholes
for the FDA to reassert the status quo and may not
survive further court action. It will be years before
the matter is resolved in the courts.
The
Access Act has generated fierce opposition. The
Society for Clinical Trials (SCT) issued a position
statement opposing the bill (2) and published an
article from a patient advocate who also opposed the
bill (3). Donald Kennedy, editor-in-chief of Science,
described the bill as “Laetrile Redux” in a recent
editorial (4), and Science published a letter
to the editor summarizing the objections of the SCT
(5). Neither chose to publish opposing views.
I
found the SCT position paper to be poorly reasoned. It
is more of a knee-jerk response than a scholarly
analysis. To make the argument that “[t]he large
majority of drugs ultimately fail to reach the
market,” the authors cite the high failure rate of
preclinical projects. But experience with drugs after
Phases I and II is much more relevant here. They claim
the current drug approval system protects patients
from disappointment, offering as proof a few
well-known cases in which drugs that looked good
initially proved to be worthless or even harmful after
more rigorous trials. They neglect to mention cases in
which standard clinical trials gave the wrong answer.
Hormone replacement therapy, anyone? C’mon, guys: I
know Congress is an easy target, but if you’re going
to lambaste them for lack of rigor, you’d better get
your own arguments in better shape than this!
FDA
reform should not be a controversial issue. Everyone
has the same goal: get more good drugs to patients
more quickly, while keeping bad drugs away. Of course,
“good” and “bad” are not clear-cut, and there
are disagreements about risk-benefit analysis and
other important details. But the really bedrock
problem is the question I led with: Does the current
drug approval process work? No one really knows, and
even more importantly, no one knows how to make it
work better. As scientists and drug developers, this
is the FDA reform we should be addressing.
Duncan
Brown contributed to this article.
Read and respond to Nat Goodman’s column at www.Goodman.PharmaDD.com.
References
1.
See www.cov.com/publications/download/oid24148/606.pdf
for a legal analysis of the decision.
2. Clin
Trials 3, 154-7; 2006.
3. Clin
Trials 3, 149-53; 2006.
4. Science
312, 1105; 2006.
5. Science
312, 195; 2006.
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