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The Scientist 18/03/04

FDA announces reform plans Agency hopes to reduce development costs by using
diagnostic and imaging technologies | By Paula Park

The Food and Drug Administration (FDA), citing a gap between investment in
pharmaceutical research and new drug approvals, announced plans Tuesday
(March 16) to reform the regulatory path to therapy development.

The plans represent the agency's attempt to reduce the cost of developing
drugs by using new diagnostic and imaging technologies to better design
trials. “We're not seeing the increases in new products that we expected
on all the advances in science,” said Janet Woodcock, the FDA's director of
cross-center initiatives. “The professional staff at the agency is also
concerned about how the new scientific knowledge is going to be worked up to
apply to
product development.”  

[Fred A. Baughman Jr., MD:
Fred Baughman: I can just visualized the
functional scan (SPECT, PET, MRI) picture of "clinical depression" going
quickly with the Zoloft or new anti-depressant under trial to the
functional concomitant of feeling "just great" even "elated" and this will
be the new proof, no more long-term trials of 3-6 weeks. And I can just see
the wonderful Dr. Daniel Amen as the new FDA director of
psychoneurobiological imaging--he of he 6 discrete subtypes of ADHD. We are
truly in for boom time at the FDA-Big Pharma, and all dove-tailing very
nicely with the GSK-Imperial College plans. ]

The FDA estimates that a new drug costs from $800,000 to $1.7 billion to
bring to market, but most drugs do not work on most people, Woodcock told
Scientist. According to the FDA report, despite a growth in government and
investment, the number of new drugs with novel chemical structures has
from roughly 70 in 1993 to less than 30 in 2003. New biologics applications
have fallen from just under 30 to just under 20 during the same time period.

“The big story, I think, here, is that if you were to write a description of
how drugs are developed in 2004, that really hasn't changed very much in the
last 30 years,” said Alastair J.J. Wood, assistant vice chancellor for
at Vanderbilt University. “This is an early attempt on the FDA's part to
the case that maybe we should at least start a conversation on how we should
change that,” said Wood, who was at one time considered a candidate for FDA

Reducing the cost of drug development, as proposed in the FDA's plan, could
help open opportunities for smaller biotechs daunted by the huge costs, Wood
told The Scientist. It would also give the pharmaceutical industry, under
tremendous pressure to develop new drugs, a needed boost. Officials from
sectors welcomed the proposed changes. “The proposed FDA initiative is
timely and
significant,” Merck spokeswoman Anita Larsen told The Scientist. “The FDA is
a unique position to identify new processes, technologies, and best

[Fred A. Baughman Jr., MD:
terms rapidly replacing the time-worn: "proof"]

and to spearhead this improvement initiative.”

Carl B. Feldman, president of the Biotechnology Industry Organization, in a
written statement, applauded what he called a “courageous statement…
recognizing the serious problems that are preventing new, innovative drugs
biologics from getting to the patients who need them.”

The FDA report outlines three phases in drug development—ensuring safety,
demonstrating efficacy, and manufacturing effective treatments—that may
reform. Tools for testing safety have changed little in the last few years,
report says. Trials to determine efficacy are a source of “innumerable
failures,” and problems in designing, characterizing, and scaling up
“routinely derail” or delay development programs and keep needed treatments

The report outlines no concrete plans for change, but lists “opportunities”
to use new techniques and technologies. For example, the report cites
“proteomic and toxico-genomic approaches” that could better test safety, and
they be used early in the drug-development process. The agency also
targeted toxicology research. “Take liver toxicity… it's one reason that
drugs fail,” Woodcock said. “It's very rare… why does one in 10,000 fail?”

The agency hopes that by improving safety testing tools, companies can
identify patients who will most likely experience liver toxicity, exclude
them from
trials, and inform them of the dangers of taking the drug once they are on
market. “We can't make it safer by testing it,” Woodcock said. “We can only
make it safer by getting information about who can take it.”

In streamlining drug development, FDA hopes to work in tandem with the
National Institutes of Health (NIH), and the report says the agency's work
complement the NIH Roadmap, a plan to invest an estimated $2.1 billion over
next 5 years to linking basic research with clinical benefits. The FDA also
invites industry and advocacy officials to help develop a list of “critical
opportunities,” a kind of a task list for change.

Woodcock said that she expects the first phase—gathering information for its
task list—to be complete in 6 months and that the agency has already started
internal “refocusing” to align its research with the new approach.

Jamie Love, director of the Consumer Project on Technology, said that the
most critical opportunity would be improving the financing of research and
development so that drugs could get to people who need them but who cannot
their high costs.

Those costs have also prompted a flurry of realignments and mergers in the
pharmaceutical industry. Merck's Larsen said that the company has already
developed alliances with diagnostic technology or biotechnology companies
that can
streamline the development process. The pharmaceutical giant signed 10 such
deals and alliances in 1996 and closed 46 of them in 2003, she said.

Wood called the FDA report “an opening salvo” in a long “conversation” that
could eventually be beneficial to both industry and patients. He stressed
that it is important for the discussion to take place outside the FDA,
industry officials tend to treat the agency as many Soviet officials once
regarded the Kremlin, trying to decode every word as if it was an important

Still, the agency's willingness to change is a good sign, he told The
Scientist: “The bottom line is that it's in everybody's interest to reduce
the cost
of drug development and to increase the likelihood of success.”

Links for this article
Challenge and Opportunity on the Critical Path to New Medical Products, Food
and Drug Administration report, March 2004.

NIH Roadmap: Accelerating Discovery to Improve Health

E. Russo, “NIH presents new research 'roadmap'” The Scientist, October 1,

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