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Approval
of Velcade for multiple myeloma
On
May 13th the FDA approved the novel cancer drug Velcade,
made by Millennium Pharmaceuticals Inc. (MLNM), as a
treatment for multiple myeloma. Velcade is the first
of a new class of medicines called proteasome inhibitors
which blocks the proteolytic activity of the proteasome.
By blocking the proteasome, Velcade disrupts numerous
biologic pathways, including the failure to degrade
IkB, which thus remains bound to NF-kB instead of being
degraded. By disruption of the signaling action of NF-kB
a multitude of genes needed for the proliferation and
adhesion of cancer cells are downregulated. Furthermore,
it is believed that in solid tumors this mechanism would
inhibit angiogenesis (new blood vessel formation). Velcade
is currently in trials in several solid tumor types.
Time
from first in human to approval
The
time to approval was extremely rapid. The approval was
4.5 years from first in human to market release. In
fact, as I understand it, the approval was granted by
looking at response rates and not survival time. It
makes one wonder in the post approval monitoring if
there turns out to be minimal survival benefit if it
will be recalled. It has been stated that MLNM worked
hand in hand with the FDA and even the expedited approval
process took two months less than expected. It is unclear
what effect this quick approval will have on current
drug development, as some are advocating the expansion
of the FDA's subpart H program, which allows for the
accelerated approval of drugs that address life-threatening
diseases.
Velcade
vs. normal FDA course
The
normal course of FDA approval is that it takes 7-10
years from IND filing to approval of a new drug and
at a cost of $500 million to $800 million. This researcher
has personal knowledge of phase III trials which the
FDA required extensive monitoring that pushed the price
tag close to $500 million for the phase III alone. This
major price tag shuts out all but the most well funded
companies from carrying their own products to approval.
A main reason drugs are so expensive in this country
is the increasing regulations that come with approval.
Before prescription drugs can become cheaper, there
must be reforms at the FDA. Let's hope that the expedited
approval of Velcade in the beginning of that reform
process.
Why
is this the exception?
Many
websites such as Consumers Against High Drug Prices
(http://www.stopfda.org/)
put forth that the clinical trial process and the FDA
is to blame for the high cost of drugs in the United
States vs. other countries. They make a lot of valid
points about how small companies cannot develop drugs
effectively because the massive amounts of money it
takes to successfully navigate the approval process.
They go further than that the FDA and major drug companies
work together to construct the system to prevent small
innovative companies from growing and challenging their
market share (I don't share thier view).
The
solution proposed by much with those who believe as
they do is that the FDA regulations should be relaxed
or eliminated and allow a "free market" to
emerge where companies that make good drugs will survive
and companies that make bad drugs will go out of business.
In that type of world there would be no investment in
biotech or pharma by venture capital due to an unacceptable
risk profile and drug development would suffer dramatically.
There are no statistics on adverse drug reactions before
the implementation of the current clinical trial process,
but one can assume that there were considerably more
cases of ADRs.
We
don't need to scrap the clinical trial process as some
would advocate, but it certainly needs a significant
overhaul. When doing this overhaul it should be considered
that less extensive clinical testing would certainly
shift the burden to the patient and physician to better
understand of the risks and benefits of medication and
would require more extensive post-approval monitoring
of drug performance. A start would be to take a hard
look at the success in implementation of the subpart
H program...
theLabRat
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