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MAPS Succeeds in Securing
an Orphan Drug Designation for Marijuana
Rick Doblin
Two and a half years ago, MAPS submitted an application to the Food
and Drug Administration's Office of Orphan Drug Products requesting that
marijuana be designated an Orphan Drug for the treatment of AIDS wasting
syndrome.
The Orphan Drug program was created by Congress to facilitate development
of drugs for rare diseases, defined as fewer than 200,000 patients per years.
Drugs for such rare diseases have not been considered sufficiently profitable
for pharmaceutical companies and research has been minimal, leaving drugs for
rare diseases "orphans." Orphan Drug
designation provides a package of incentives for research and development,
culminating in seven years of patent protection (exclusive right to market)
should convincing data about safety and efficacy ever be submitted to the FDA and
the drug be approved for marketing. Marinol, the oral THC pill, was approved for
AIDS wasting under the Orphan Drug program.
On May 25, 1999, the FDA wrote a letter
announcing that MAPS' application had finally been accepted. The acceptance letter came after three letters of rejection from FDA, responses (referred to in correspondance as "amendments to the application") from MAPS to each rejection, and two additional amendments.
[Archive of this correspondance] This designation is a demonstration of good faith on the part of the FDA and represents
a major milestone in MAPS' efforts to support research
into the medical uses of marijuana.
As MAPS members and other readers of the
Bulletin probably know, MAPS began working in 1992 with
Dr. Donald Abrams, UC San Francisco, in an effort to
obtain permission for his study of the use of marijuana in
AIDS patients. Dr. Abrams' study was eventually
approved, received a $970,000 grant from NIDA, and treated its
first subject in 1998. Dr. Abrams' study is the first
FDA-approved investigation of the medical use of marijuana in
a patient population in 15 years. Dr. Abrams will
complete the dosing phase of the study in early 2000. MAPS'
successful effort to have marijuana declared an Orphan Drug
will make it easier to continue to research the medical use
of marijuana for AIDS wasting syndrome, if the data
gathered by Dr. Abrams demonstrates that marijuana can be
administered safely to AIDS patients.
Another benefit of having marijuana declared
an Orphan Drug by the FDA is that it will theoretically
be easier for MAPS to obtain a DEA license to establish
a domestic medical marijuana
production facility to produce high-quality marijuana for FDA-approved research. In
late July 1999, MAPS received a grant of $20,000 from Peter Lewis to
explore whether it really will be possible to obtain a DEA license to establish
a domestic medical marijuana production facility.
Read update on this effort
MAPS is the first organization to use the Orphan Drug program to
help facilitate research into any medical use of marijuana. The way the program
is structured, other entities can also seek Orphan Drug designation, either
for AIDS wasting or for other rare diseases that marijuana may be useful in
treating. Congress intended Orphan Drug designation to be the starting point
for scientific research that would culminate in an
informed decision regarding the potential safety and efficacy of
each designated drug for the treatment of the specific
indication so designated.
The growth of political support for scientific
research into the medical use of marijuana has resulted in
several for-profit firms initiating research into various forms
of marijuana extracts and isolated cannabinoids
administered in non-smoking delivery systems such as vaporizers
and aerosol sprays. MAPS supports all of these efforts to
develop needed medicines for patients. MAPS' work on behalf
of research into the plant itself is intended to ensure that
an accurate risk/benefit ratio can be determined for the use
of marijuana when smoked or used in a vaporizer.
Profit-making firms with obligations to shareholders
have powerful incentives to abandon efforts to study and
obtain approval for the plant itself, which is likely to be the
least expensive, least profitable dosage form and is certainly
the most politically controversial. As a non-profit
organization, MAPS does not face such pressure and will therefore
focus on facilitating research with the marijuana plant.
Orphan Drug designation is merely the first step in
a very long process. Whether any additional progress will
be made remains to be determined.
- Rick Doblin, MAPS President
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