August 25, 1998
FDA Office of Orphan Product Development (HF-35)
5600 Fishers Lane
Rockville, MD 20857
Dear Ms. Donahoe,
When we spoke on July 29, 1998, you asked me why MAPS sought Orphan Drug designation for marijuana for the treatment of AIDS wasting. Your sincere question deserves a thorough answer, even though MAPS' motivations are a separate issue than whether marijuana qualifies as an Orphan Drug under the law. Hopefully this written response will explain more clearly than I was able to on the phone.
You already know that I think marijuana does qualify as an Orphan Drug for the treatment of AIDS wasting, just like several other drugs which have been designated as Orphan Drugs for AIDS wasting though by a somewhat different definition than the one MAPS proposed. You also already know that the medical use of marijuana is a controversial issue. Thousands of AIDS patients suffering from wasting report using marijuana medicinally, despite the inherent legal risks of this choice.
The primary reason MAPS is seeking Orphan Drug designation is that such designation would facilitate the process whereby this potential treatment could be evaluated scientifically. If marijuana is indeed proven safe and efficacious to rigorous FDA standards, patients would no longer need to worry about going to jail for using marijuana medicinally. If marijuana is proven unsafe or not efficacious, then these patients could turn to more appropriate treatments.
If marijuana is declared an Orphan Drug for AIDS wasting, the FDA, acting under direction from Congress, will provide special incentives to facilitate research to determine whether marijuana is indeed safe and efficacious for such use. As a result, it may become easier to obtain permission to conduct such research. What you may not be fully aware of is that marijuana is the only drug in the United States for which all privately-funded research is currently forbidden. This drastically limits opportunities for research to only those projects that the federal government is willing to fund. Is that scientific freedom regarding medical marijuana research?
NIDA retains a monopoly on the supply of marijuana legal for use in FDA-approved research. According to a policy established by NIDA Director Dr. Alan Leshner, NIDA will not allocate any of its marijuana, no matter how small an amount, to any study not funded by the NIH through the peer-reviewed grant application process. Both the NIH Expert Committee on the Medical Utility of Marijuana and the AMA House of Delegates have recommended that this policy be changed to one in which NIDA agrees to provide marijuana to all FDA-approved projects. However, no change has yet been made.
Incidentally, NIDA does not retain a monopoly on the supply of LSD, MDMA, ibogaine, DMT, psilocybin, and other Schedule 1 drugs. As a result, it is possible to conduct privately-funded research with these drugs as long as approval is obtained from the FDA, the local Institutional Review Boards, and, when the research takes place in California, from the California Research Advisory Panel. In addition, the researchers who will handle the Schedule 1 drug being studied are required to obtain licenses from the DEA.
If the FDA designates marijuana as an Orphan Drug for the study of marijuana in the treatment of AIDS wasting, it then becomes possible to submit protocols for evaluation by the peer-reviewed grant program established by the Office of Orphan Product Development. Your Office's peer-review process is still under the jurisdiction of NIH, operates much more rapidly than the traditional NIH grant review process, and might satisfy NIDA that certain protocols were well-designed, perhaps even regardless of whether NIH funds were allocated.
If NIDA changes its policy so that it becomes possible to conduct privately-funded research, Orphan Drug designation will make it much easier for MAPS to obtain funding for such research. MAPS is a 501 (c) (3) non-profit research and educational organization with one of its goals being to conduct research into the medical uses of marijuana. As I mentioned on the phone, MAPS relies on donations from individuals and foundations for its income. I have already spoken to several potential donors to MAPS who have indicated a willingness to support marijuana/AIDS wasting research if it becomes possible for them to do so. These donors particularly like the idea that Orphan Drug designation involves patent protection for seven years for the sale of marijuana for AIDS wasting, in the eventuality that the FDA determines that marijuana is indeed safe and efficacious for that clinical indication. Through this program offered by your office, MAPS might one day become legally licensed to sell marijuana for the treatment of AIDS wasting through the prescription drug system. If this becomes possible, MAPS might be able to raise funds for additional research into other possible medical uses of marijuana through profits on sales, instead of through further donations. This possibility motivates donors who like to help make non-profit organizations self-sufficient.
As you probably do not know, about $8 million has been donated in non-tax-deductible funds to support several state-wide medical marijuana initiative campaigns in this election cycle. There will definitely be initiatives supporting the medical use of marijuana on the ballot in Alaska, Washington, Oregon, Nevada, and Arizona, and perhaps in several other states. In my view, this is a tragic misallocation of resources. The medical use of marijuana is an issue that should be settled through scientific research, not at the ballot. However, funds are going towards politics and not science because it is currently impossible to obtain permission for any privately-funded research into any medical use of marijuana and it is exceedingly difficult to obtain government funding for such research. Dr. Donald Abrams' MAPS-supported efforts to obtain permission and NIH funding for his study of the medical use of marijuana in the treatment of AIDS wasting was successful only after almost six years of effort, and represents the first and only safety study of the medical use of marijuana in a patient population in fifteen years. Frustrated patients, physicians, activists and donors have largely abandoned research and focused their efforts instead on the political process. The FDA has lost what should be its leading role over the medical use of marijuana due to political decisions that make FDA approval of protocols insufficient to initiate research.
In the long-run, the key to expediting research into the use of marijuana in the treatment of AIDS wasting is to develop a non-governmental source of marijuana that would be acceptable to the FDA for use in human research. Unfortunately, the only facility licensed by the DEA to grow marijuana for research purposes, the farm at the U. of Mississippi under the direction of Dr. El Sohly, supplies all of its production to NIDA and will not enter into contract to provide marijuana to any non-governmental entity. At present, there is no foreign source I know of for FDA-approved marijuana available for import. The Home Office in England has just licensed Dr. Geoffrey Guy of GW Pharmaceuticals to establish a facility in England to produce marijuana extracts for research purposes, and MAPS is exploring whether it will become possible in the future to import research material from GW Pharmaceuticals.
If MAPS is granted Orphan Drug designation for the use of marijuana for AIDS wasting, it is my intention for MAPS to request a DEA license to grow marijuana for research purposes. Currently, the DEA refuses even to grant licenses for small, experimental farms seeking to grow non-psychoactive strains of marijuana called hemp for commercial uses. If marijuana is declared an Orphan Drug for the treatment of AIDS wasting, the DEA might be more willing to seriously consider an application to set up a marijuana production facility.
I hope this letter has answered your question about why MAPS is seeking Orphan Drug Designation. If you have any further questions, please let me know and I will try my best to address them. I look forward to hearing from you.
Public Policy Ph.D. candidate, Harvard's Kennedy School of Government