Subject: Plan to Speed Drug Approvals Pits Small Companies Against Big Ones
Date: Published: 4/3/87 137 lines
Source:  Wall Street Journal.  Copyright Dow Jones & Co. Inc. 

   Plan to Speed Approval of Test Drugs
   Pits Small Companies Against Big Ones
    ---
   By Michael Waldholz
   Staff Reporter of The Wall Street Journal

   Food and Drug Administration proposals to speed
experimental medicine to the severely ill are driving a wedge
between large and small drug companies.
   The brewing controversy could lead to a dilution of the
plan or at least delay its implementation.
   The proposals, which were published last month and are now
reaching industry officials' desks for evaluation, would
speed the release of a host of experimental drugs for cancer
and other diseases to many gravely ill patients.  Under the
plan, certain drugs could be approved by the FDA for limited
use without the currently required proof that they are both
safe and effective.
   Experimental drugs would be divided into two categories,
those for ailments that would be fatal without treatment, and
those for ailments that are serious but not necessarily
fatal.  Drugs in the former category could be released if they
showed at least slight evidence of value and clearly weren't
toxic.  Drugs in the latter category would have to pass more
rigorous tests for effectiveness that still fall short of
present rules.
   The conflict arises over another feature of the proposal.
Under current rules, patients can receive, without charge,
some drugs in very advanced stages of research if their
doctors apply to the FDA. The new rules would let small drug
companies, such as the biotechnology firms, charge patients
in clinical studies for the drugs "if the company can show
that without charging it wouldn't be able to conduct a
study," an FDA official says.  And all companies could charge
for experimental drugs for which studies had been completed.
   Smaller drug companies expect that provision to free them
from the need to share their discoveries with larger,
established companies that can afford the expense of bringing
a drug to market.  The large companies, some industry
officials say, fear the competition and may try to delay or
dilute the new regulations.
   The affected drugs in both categories, mostly produced
through biotechnology, are expected to revolutionize the use
of experimental medicines.  Some drug makers and FDA officials
say that some prescription drugs in research, such as
interleukin-2, some interferons and laboratory-manufactured
antibodies, would be distributed widely now, two to four
years earlier than expected.
   Other drugs in research that may gain the limited but
immediate release include those for fighting infections that
accompany potent cancer treatments, several for use against
acquired immune deficiency syndrome, or AIDS, as well as
drugs for treating kidney disease, several types of cancer
and heart disease, multiple sclerosis and Alzheimer's
disease.
   "If they go through as written now, the proposed rules
will provide an extraordinary opportunity for the public and
the biotech industry," says James McCamant, editor of Medical
Technology Stock Letter, a San Francisco investment adviser.
   Indeed, the rules' release has set off a flurry of
activity, especially at smaller companies.  Nils Ribi,
chairman and chief executive officer of Ribi Immunochem
Research Inc., a tiny biotech company based in Hamilton,
Mont., spent part of this week lobbying to gain support for
the new rules.  His company's Detox, an experimental treatment
for severe skin cancer, is three or four years away from FDA
approval.  "I think under the new rules many skin cancer
patients could get the drug right away," he says.
   The new rules are the brainchild of the FDA's
commissioner, Frank E. Young, who says he has personally
worked on them for more than a year.  Part of the rules'
impetus was to get research drugs for AIDS to patients more
quickly, he says.  But he adds, "There are some very major
advances in pharmaceutical science taking place in which
drugs are being developed for diseases where there is no
other therapy.  We want to get those patients these drugs much
faster than current rules allow without in any way
jeopardizing them."
   Dr. Young is optimistic that the regulations will become
effective in mid-June following a 90-day period for public
and industry comment.  But many in the drug industry expect
the magnitude of the changes to spur a lengthy debate and
revisions that may take months, perhaps years, to
incorporate.
   Some FDA critics say relaxing the agency's current system
will risk releasing potentially poisonous or worthless drugs.
They also worry that making experimental drugs widely
available will jeopardize research since desperately-ill
patients won't be willing to take a placebo, or non-acting
drugs, required as a control in research studies.  And some
argue that, since insurers don't pay for experimental
medicines, the wealthy will benefit most.  There is also
concern that the FDA won't be able to regulate the charges.
   Moreover, some industry officials expect certain big drug
makers to lobby for dilution of the proposals.  "Current FDA
regulations act as a barrier to the market for the little
guys," says one biotech company executive.  Already, the
Pharmaceutical Manufacturers' Association has requested a
45-day extension to the comment period, saying it needs more
time for consideration.
   Small biotech firms say that while the new revenue they
hope to get under the proposal won't be large, it would be
important.  For small companies developing a new drug, "each
month is critical," says Gabriel Schmergel, president and
chief executive officer of Genetics Institute Inc.,
Cambridge, Mass.  The company says its studies of a natural
protein called GM-CSF that bolsters a cancer patient's
ability to fight infections would benefit.
   Others say the proposed rules would also trigger studies
to find wider uses for the drugs and expand production
capacity.  Cetus Corp., Emeryville, Calif., says it is
studying its interleukin-2 in 1,000 patients and won't do
more unless it can generate funds to increase output.
   Hubert Schoemaker, president of Centocor Inc., Malvern,
Pa., notes that wider availability would provide more
patients to evaluate a drug's usefulness, speeding final FDA
approvals.  He says his company would immediately seek
distribution of its three monoclonal antibody products for
fighting life-threatening infections, and pancreatic and
ovarian cancers.
   Cetus says it expects to seek the limited use of
interleukin-2 for treating cancers of the kidney, skin and
blood cells and Amgen of Thousand Oaks, Calif., said it hoped
to make available its EPO, a drug that would replace the
transfusions that kidney dialysis patients often require.
   "There is an amazing list of exciting products in the
pipeline at FDA," says Dr. Young, the agency commissioner.

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