Document 1117
 DOCN  M94A1117
 TI    Clinical decision-making and trial design: a review and critique of
       recent trials.
 DT    9412
 AU    Link D; Gay Men's Health Crisis, New York City, NY 10011.
 SO    Int Conf AIDS. 1994 Aug 7-12;10(2):205 (abstract no. PB0834). Unique
       Identifier : AIDSLINE ICA10/94371458
 AB    OBJECTIVE: To review and critique drug development strategies for the
       next group of antiretroviral compounds in phase III studies, including
       stavudine (d4T), and the Merck and Roche protease inhibitors. METHODS:
       Planned and on-going efficacy studies for these drugs will be
       comprehensively reviewed. The methodology and design of these studies
       will be examined and evaluated for their ability to answer
       clinically-meaningful questions for front-line physicians and patients.
       Leading statisticians and methodologists will be interviewed for their
       perspectives on how best to obtain clinical efficacy data on these
       compounds. RESULTS: Several important methodological weaknesses are
       evident in the drug development strategies for these compounds, which
       may limit their clinical application: 1.) treatment effect is
       over-estimated and sample sizes are too small. 2.) the clinical efficacy
       of control arms is unknown 3.) virologic and immunologic surrogate
       markers are unvalidated 4.) survival endpoints are not measured and the
       disease progression endpoint must be better characterized by ranking
       clinical events by prognosis and severity. DISCUSSION: This report will
       emphasize the need for clinical efficacy data on new antiretrovirals by
       front-line physicians and patients. The report will describe alternate
       trial designs and methodologies, which may help answer clinical efficacy
       questions.
 DE    Acquired Immunodeficiency Syndrome/*DRUG THERAPY  Antiviral
       Agents/*THERAPEUTIC USE  *Clinical Trials/METHODS  Decision Making
       Human  HIV Protease Inhibitors/THERAPEUTIC USE  *Research Design
       Stavudine/THERAPEUTIC USE  MEETING ABSTRACT

       SOURCE: National Library of Medicine.  NOTICE: This material may be
       protected by Copyright Law (Title 17, U.S.Code).