Subject: Letters to the Editor: Parental Guidance for Orphan Drugs
Date: Published: 4/17/92 (89 lines)
Source: Wall Street Journal. Copyright Dow Jones & Co. Inc.

    Letters to the Editor:
    Parental Guidance for Orphan Drugs

    Suzanne Tregarthen's Counterpoint article ("Prescription
 to Stop Drug Companies' Profiteering," op-ed page, March 5)
 appears to confuse orphan-drug "designation" with orphan-drug
 "approval."  This leads her, in turn, to erroneous conclusions
 about the amendment of the Orphan Drug Act under
 consideration in Congress.
    A firm that "wins" orphan designation does not win tax
 credits and an exclusive seven-year right to market that
 product, as indicated by Ms. Tregarthen.  These incentives are
 granted only if the firm proceeds to test and develop the
 product fully and obtain marketing approval from the Food and
 Drug Administration.  That is a time-consuming, risky and
 costly process.
    Although the FDA has awarded 488 orphan designations, the
 only products actually to reap the benefits available under
 the Orphan Drug Act are the 60 orphan drugs that have been
 approved for marketing by the FDA.  
    Nor, as suggested by Ms. Tregarthen, does it matter under
 the terms of the Orphan Drug Act who discovered a substance.
 When a substance is not patentable, or is no longer protected
 by patent, the incentives in the act are designed to
 encourage companies to undertake the exhaustive testing for
 specific uses that is necessary to win FDA approval.  Ms. 
 Tregarthen's endorsement of Sen. Howard Metzenbaum's (D.,
 Ohio) amendment to revoke the Orphan Drug Act's benefits once
 an orphan drug's sales exceed a certain revenue level would
 chill the remarkable progress made possible by the act.
    There is no question that the act has worked.  The 60
 orphan drugs that have been approved will help only an
 estimated 2.4 million patients.  There are still 10 million to
 20 million Americans who suffer from the 5,000 known rare
 diseases for which no treatment has been approved.
    Gerald J. Mossinghoff
    President
    Pharmaceutical Manufacturers Association
    Washington
    ---
    The column contained several serious inaccuracies about
 the development of Retrovir brand zidovudine (AZT) and its
 designation as an orphan drug.  In 1985, when Burroughs
 Wellcome Co. applied for orphan-drug status for AZT, there
 were only a few thousand people who had been diagnosed with
 AIDS.  Given the circumstances, application for orphan status
 was entirely appropriate.
    The article overstates any benefits that may have accrued
 to Burroughs Wellcome through Retrovir's orphan-drug
 designation.  In reality, the tax credits were modest.  Such a
 credit is allowed only for expenses involved in research on
 humans conducted after the date of orphan-drug designation
 and before FDA marketing approval -- in this case, a period
 of about 21 months.
    The article also inflated the price of Retrovir by nearly
 300%.  The current price to wholesalers is $2,200 to $2,600
 annually, depending on a patient's prescribed dosage of
 therapy.  The drug is also made available free to patients who
 cannot afford it through the company's HIV Patient Assistance
 Program, as are all of our therapies related to the treatment
 of HIV disease.
    Retrovir was not developed by government scientists, as
 the author stated, and the research was not conducted under a
 Collaborative Research and Development Agreement.  Burroughs
 Wellcome scientists first conceived of the use of Retrovir
 for treating people infected with HIV.  Before that discovery,
 there was no treatment for HIV.  In fact, before any
 government scientists could conduct any human tests with the
 drug, they had to cross-reference the information contained
 in Burroughs Wellcome's Investigational New Drug application
 already filed with the FDA.  
    No effective treatment for AIDS was available until
 researchers at Burroughs Wellcome discovered the use of
 Retrovir, with or without orphan-drug designation.  Patents
 first granted in 1988, and not orphan-drug status, serve as
 the basis for the company's exclusivity in marketing of the
 product.
    J. R. Whitehead
    Vice President, Corporate Affairs
    Burroughs Wellcome Co. 
    Research Triangle Park, N. C. 

[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.]