Subject: Letters to the Editor: Orphan-Disease Patients' Desperation
Date: Published: 4/3/91 (67 lines)
Source: Wall Street Journal. Copyright Dow Jones & Co. Inc.

    Letters to the Editor:
    Orphan-Disease Patients' Desperation

    Since AIDS patients often feel neglected and desperate, it
 is understandable that AIDS activist James Driscoll
 (editorial page, March 6) reacts so strongly to Rep. Henry
 Waxman's (D., Calif.)  attempts to improve the Orphan Drug
 Act.  But in Mr. Driscoll's eagerness to clutch at any straw,
 he totally neglects the fact that AIDS is but one of 5,000
 so-called orphan diseases covered by this law and that there
 are some 20 million Americans who suffer from these diseases
 and feel equally, if not more, neglected and desperate.
    It is the total population of rare-disease patients,
 including those who have AIDS, that concerns Rep. Waxman.
 What motivates him is a genuine fear that the extraordinarily
 profitable orphan drugs threaten the very future of the
 Orphan Drug Act and thus jeopardize all the ailing people who
 look to it as their major hope.
    The purpose of the Orphan Drug Act is to encourage
 development of drugs that are "of little commercial value" to
 be used for diseases with small patient populations.  It was
 never envisioned as a shelter against competition for
 blockbuster drugs.  And now these drugs, which Congress has
 called "abuses," have weakened public confidence in the act
 and created a perception that it was written to benefit
 certain drug companies, not the patients.
    Mr. Driscoll would have us put up with price gouging in
 the belief that by turning a blind eye we are encouraging
 development of more treatments and cures.  He calls this being
 pragmatic, but I call it being naive because he ignores the
 real question -- and that is "who pays the bill."
    Shielded from competition, pentamidine costs AIDS patients
 four times as much in the U. S. as it does in Europe.  But
 maybe the real point to be made is that AIDS patients aren't
 paying for it.  Increasingly, the bill is being paid by
 Medicaid, just as Medicare pays more than $250 million
 annually for Erythropoetin (EPO), and insurance companies are
 paying most of the annual $240 million tab for human growth
 hormone.  But, in the end, we are all paying the bills, and as
 the revolt against rising health-care costs builds, we
 sincerely fear that the Orphan Drug Act may be thrown out
 with the bathwater because of these blockbusters.
    Mr. Driscoll therefore has it all wrong:  Mr. Waxman is not
 anti-profit, and he certainly is not anti-AIDS patients.  He
 is pro-profit:  the profit of those companies that use the law
 as it was intended, that is, to produce reasonable profits
 from drugs that are otherwise drugs of little commercial
 value.
    As in the human body, the diseased part of the Orphan Drug
 Act should be carefully and surgically removed, and as soon
 as possible, or it will spread with possibly fatal
 consequences.  That is the approach Rep. Waxman has taken, and
 NORD and millions of orphan-disease sufferers applaud him for
 it.
    Abbey S. Meyers
    Executive Director
    National Organization for Rare Disorders
    New Fairfield, Conn.

[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.]