Subject: Letters to the Editor:  FDA Rule Changes on Breakthrough Drugs
Date: Published: 10/29/87 108 lines
Source:  WALL STREET JOURNAL. Copyright Dow Jones & Co. Inc. 

    Letters to the Editor:  
    FDA Rule Changes on Breakthrough Drugs

    I read with great interest the Oct. 2 news article and
 Oct. 5 front-page article on Food and Drug Administration
 rule changes that may enhance the marketing of new,
 especially breakthrough, drugs.  The direction is very
 appropriate and a desirable one.  Such changes aren't without
 their risks, but we must proceed beyond the post-thalidomide
 period and put that tragedy in clear perspective and not have
 it remain the controlling factor in our drug-approval policy.
 Postponing the approval of a new drug is as significant a
 decision as approving one.
    It seems indefensible to me that new drugs, which have
 been created by the pharmaceutical industry in the U. S. and
 are being used in Western Europe, Canada or Japan remain
 unavailable in this country.  We have prevented patients,
 together with their physicians, from making an informed
 choice and assuming the risk for that decision.  Every drug
 has the potentiality for producing undesired side effects in
 a certain percentage of the population and that includes the
 use of aspirin and certain vitamins.  However, the decision to
 use these substances now resides with the individual.
 Similarly, the decision on the new breakthrough drugs for
 AIDS, cancer and vascular problems must be determined by an
 informed patient together with his or her physician.
    Drugs must, of course, be therapeutically effective.  The
 FDA must not relinquish its responsibility in this regard,
 but adult patients with their physicians must not be
 precluded from making decisions on the use of such
 medications.  We cannot have a riskless society, for with
 decision making comes full responsibility.  The issue must not
 be clouded by the financial potential for the pharmaceutical
 companies.  It is clearly a question of whether the individual
 together with his or her physician can determine the
 medication to be used based upon various therapies available.
    The FDA has the major role to play in providing the most
 up-to-date information available to all, but it should not
 arrogate for itself totally decision making regarding the use
 of such medications for adults.  A free and mature society
 must allow significant latitude for its citizens in
 conjunction with physicians and pharmacists in medication
 decision making provided the drugs are therapeutically
 effective and appropriate for the condition.
    Albert H. Soloway
    Dean, College of Pharmacy
    Ohio State University
    Columbus, Ohio
    ---
    Your Oct. 5 article was a good overview of the impact of
 the new FDA regulations on drug development.  However, it says
 that the immuno-supportive drug Imreg-1 is "in dispute."
    New developments in science are frequently accorded a high
 degree of skepticism.  That skepticism is a natural product of
 the scientific process, and is generally beneficial in the
 long term, if for no other reason than that it serves to
 sharpen the scientific arguments of the proponents.  Moreover,
 the development of immuno-supportive drugs is a new form of
 medical therapy for which there isn't an extensive data base
 of prior experience.
    Therefore, it isn't unexpected that questions will be
 raised about new drugs, their mode of action and results.
 Those who are developing new drugs have the full burden of
 proof as to efficacy and justification for their use by the
 medical community.  This is done by pursuing a rigorous,
 systematic demonstration of the effects of a new drug, a
 process that is not only intellectually challenging but
 costly as well.
    Those who are watching these developments should base
 their judgments on all of the pertinent information, when it
 becomes available, and should avoid the dangers of
 prejudgment.  Well-informed and well-intentioned criticism and
 skepticism will serve to benefit drug development.  Careless
 judgments at any stage will slow achievement of this goal.
    A. Arthur Gottlieb
    President, Imreg Inc. 
    New Orleans
    ---
    We applaud the FDA's recognition of the needs of seriously
 ill patients.  The "investigative new drug for treatment"
 (IND) rules may allow these patients greater access to new
 technologies and investigational drugs.  They shouldn't also
 be the "victims" of a cumbersome drug-development process.
    The Oct. 5 article states, however, that even our most
 advanced approach (interleukin-2 and activated killer cells)
 doesn't "benefit the majority of patients" and "has many
 toxic side effects."  This approach has shown promise in about
 30% of patients who have no standard treatment option.  Should
 these patients be denied this approach?  Also, we have
 developed and published a clinical and laboratory protocol
 that significantly reduces toxicity.
    Finally, health insurance does often pick up costs related
 to our programs.  Our experience has been that about 90% of
 patients' clinical charges and about 30% of patients'
 laboratory research costs are reimbursed.
    Louis P. Berneman
    President, Biotherapeutics Inc. 
    Franklin, Tenn.

[This article is made available here by Dow Jones Co. for the 
personal and non-commercial use of callers to this bbs, in the 
hope that it will be of some help to those who are suffering 
from the disease and others who are seeking to help them.]