Food and Drug Administration
The Federal Food and Drug Administration (FDA) is tasked with protecting public health by ensuring the safety, efficacy and quality of drugs and medical products marketed to the public.
The process of approving a drug begins with reviewing information about the drug:
- The indication for which the drug has been shown to be effective at treating, including specific uses in children or the elderly, if any;
- What patients may benefit from its use, including information about whether the drug has been tested in children;
- What adverse effects have been reported for individuals taking the drug;
- How the drug should be taken (e.g., orally, intravenously);
- The dose of the drug that is recommended to be used; and
- How the drug is made (e.g., as a pill, liquid) and what is in the drug, including both active and inactive ingredients. 1
The FDA’s drug development paradigm includes the following steps:
- New Drug Application (NDA) – This FDA application is based upon clinical and non-clinical data from a new drug and presented for review.
- Investigational New Drug (IND) Application – This application presents a plan for testing the drug in humans, based upon initial testing data and the drug’s composition and manufacturing. It includes protocols for the clinical studies, researchers’ qualifications, and most importantly, safeguards protecting the rights, safety and welfare of the humans participating.
These clinical trials consider how the drug can be safely administered and at what doseage. Then the drug’s efficacy is studied. As success is experienced the clinical trials are expanded to include more subjects. A manufacturing process producing a high quality product must be proven. This is very difficult when the drug is derived from a botanical source such as marijuana.
“Botanicals include herbal products made from leaves, roots, stems, seeds, pollen or any other part of a plant. Botanical products pose challenges that are unique to this class of product, including lot-to-lot consistency. These unpurified products, which may be either from a single plant source or from a combination or undefined, making it difficult to determine if the product is causing the change in a patient’s condition, or the change is related to some other factor.” 1
The FDA approved two drugs which contain the active ingredients found or similar to those found in the marijuana plant, Marinol and Cesamet. These are prescribed for anorexia associated with AIDS and nausea associated with chemotherapy. Marinol contains the active ingredient, dronabinol, a synthetic THC, the psychoactive component. Cesamet contains synthetic cannabinoid nabilone as the active ingredient.
Future for Marijuana as a Medicine
“If there is any future for marijuana as a medicine, it lies in its isolated components, the cannabinoids and their synthetic derivatives. Isolated cannabinoids will provide more reliable effects than crude plant mixtures. Therefore, the purpose of clinical trials of smoked marijuana would not be to develop marijuana as a licensed drug but rather to serve as a first step toward the development of nonsmoked rapid-onset cannabinoid delivery systems.” 1
The FDA has four procedures in place to facilitate and expedite development and review of new drugs designed to treat unmet medical conditions, Fast Track, Accelerated Approval, Priority Review and Breakthrough Designation.
”… in April 2014, GW Pharmaceuticals announced that the FDA granted Fast-Track designation to its investigational drug product Sativex®, composed primarily of two
cannabinoids: CBD (cannabidiol) and THC, administered as a metered-dose oromucosal spray, for the treatment of pain in patient with advanced cancer, who experience inadequate analgesia during optimized chronic opioid therapy. Sativex is currently in Phase 3 clinical trials for this indication.” 1
“…on June 6, 2014, GW Pharmaceuticals announced that FDA granted Fast-Track designation to its investigational CBD product, Epidiolex®, in the treatment of Dravet syndrome, a rare and catastrophic treatment-resistant form of childhood epilepsy.” 1
“The expanded access program is being used in the area of marijuana. Epidiolex®, containing CBD, is being developed for the treatment of certain seizure disorders in children. GW Pharmaceuticals has announced that there are now 21 active expanded access INDs for Epidiolex treating approximately 300 patients with epilepsy syndromes. Approximately 95 percent of these INDs are for patients between 1 and 17 years of age.” 1
The FDA is also tasked with setting scientific standards for Controlled Substances. Their recommendations for the scheduling of drugs go to the Department of Health and Human Services, HHS, and the DEA.
Marijuana – Substance I Controlled Substance
“… marijuana is currently listed as a Schedule I controlled substance. Schedule I includes those substances that have a high potential for abuse, have no currently accepted medical use in treatment in the United States, and lack accepted safety for use under medical supervision. Nevertheless, Schedule I substances, including drugs that are derived from botanical sources such as marijuana, can be and are the subject of clinical trials under the FD&C Act …”
The FDA’s Center for Drug Evaluation and Research (CDER) established an “eight-factor analysis” as the basis for scheduling drugs. The “eight-factors” are:
- Its actual or relative potential for abuse;
- Scientific evidence of its pharmacological effect, if known;
- The state of current scientific knowledge regarding the drug or other substance;
- Its history or current pattern of abuse;
- The scope, duration, and significance of abuse;
- What, if any, risk there is to the public health;
- Its psychic or physiological dependence liability; and
- Whether the substance is an immediate precursor of a substance already controlled under the CSA (Controlled Substances Act).
At a Congressional hearing before the Subcommittee on Government Operations, Committee on Oversight and Government Reform, U.S. House of Representatives on June 20, 2014 Douglas C. Throckmorton, M.D. testified about the Administration’s policy on marijuana, Mixed Signals: The Administration’s Policy on Marijuana.
Dr. Throckmorton, the Deputy Director for Regulatory Programs, Center for Drug Evaluation and Research, FDA, explained the FDA’s ongoing efforts to meet unmet medical needs, their strict adherence to rigorous scientific research, and their “eight-factor analysis” basis for scheduling drugs as it pertained to marijuana.
1 Throckmorton, Douglas C. M.D., Deputy Director for Regulatory Programs, Center for Drug Evaluation and Research, Food and Drug Administration, Department of Health and Human Services. Before the Subcommittee on Government Operations, Committee on Oversight and Government Reform, U.S. House of Representatives, June 20, 2014.