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September 24, 1997
Dr. Marlene Haffner
Director, Office of Orphan Drug Products Development (HF-35) FDA
5600 Fishers Lane
Re: Application #97-1053
Dear Dr. Marlene Haffner,
I'm writing in regard to the April 24, 1997 application for Orphan Drug Designation (#97-1053) submitted by the Multidisciplinary Association for Psychedelic Studies (MAPS) for the use of marijuana in the treatment of HIV-related wasting syndrome.
This letter is a response to your correspondence of June 27, 1997 requesting that MAPS submit statements from experts commenting on the prevalence of wasting when defined as, "involuntary weight loss of > 5% of baseline body weight in the presence of HIV infection." This information was requested because MAPS used 5% weight loss in its definition of HIV-related wasting yet based its estimated annual patient population on statistics from the Centers for Disease Control (CDC), which defined HIV-related wasting as occurring when there had been "involuntary weight loss of > 10% of baseline body weight in the presence of HIV infection."
Specifically, you suggested that the letters might come from the two experts cited in MAPS' original application, Dr Richard Selik, CDC, and Dr. Kathleen Mulligan, UC San Francisco, as well as one additional expert.
Dr. Richard Selik, CDC, declined to submit any estimates for prevalence of HIV-related wasting with the 5% lower limit since the CDC does not have any data on which to base such an estimate. Unfortunately, neither at the date of MAPS' original submission in April 1997 nor five months later when this letter is being written is there any published report estimating the prevalence of wasting of >5% weight loss.
Enclosed are letters from Dr. Donald Abrams and Dr. Kathleen Mulligan, both indicating that the authors believe that the prevalence of HIV-related wasting, when defined as "involuntary weight loss of > 5% of baseline body weight," is well below 200,000 people per year, the upper limit for an orphan disease. Dr. Abrams cites preliminary unpublished data in his letter in support of his estimate. Dr. Mulligan reports that her estimate is based on the reductions in the incidence of all cases of HIV-related wasting that she has seen as a result of the recently expanded use of protease inhibitors. In addition, she explains why the researchers and clinicians in the field have switched to the use of the 5% weight loss cut-off instead of retaining the 10% cut-off used by the CDC.
I am also submitting revised calculations for the prevalence of HIV-related wasting of >5% as of April 30, 1997. These new calculations are based on data released September 19, 1997 by the CDC about rates of death from AIDS for 1996, new cases of AIDS in 1996, and the number of people living with AIDS at the end of 1996. My previous estimate of 275,000 people living with AIDS as of April 30, 1997 has proven to be an overestimate, as I had intended would be the case. A more accurate estimate is 250,000 people living with AIDS as of April 30, 1997. The new data from the CDC, combined with the preliminary data reported by Dr. Abrams and the clinical impressions of Dr. Mulligan, suggests that it is virtually certain that as of April 30,1997, the total number of people with AIDS wasting of >5% was less than 200,000. The largest number of people living with AIDS as of April 30, 1997 with >5% involuntary weight loss that was produced by the various estimating approaches was 42% of 250,000 people, or 105,000.
Of note is that Dr. Donald Abrams was notified on September 18, 1997 that his NIH-grant application to conduct a clinical trial into the use of smoked marijuana, oral THC (Marinol), and placebo in AIDS patients taking the protease inhibitor, Indinavir, was approved and funded for the full amount of $978,000. This NIH funding decision marks the turning point in Dr. Abrams' five and a half year struggle to obtain permission to begin this line of research. Dr. Abrams' study is primarily a safety study in AIDS patients who are not suffering from HIV-related wasting. One purpose of the study is to gather information necessary for the design of a subsequent study of the use of smoked marijuana in AIDS patients with wasting of >5% involuntary weight loss, should the safety data gathered in the initial trial suggest that such a study would be warranted.
I look forward to hearing from you regarding this recent submission.
Public Policy Ph.D. candidate, Harvard's Kennedy School of Government