Step by Step Guide to the FDA Approval Process For Cannabis Research

The landscape surrounding cannabis research is a complex and intricate one, primarily due to its classification as a scheduled substance by the U.S. Drug Enforcement Administration (DEA). While the therapeutic potential of cannabis has sparked immense interest, the federal classification presents significant challenges for scientists and researchers seeking to study its medicinal properties.

In this blog post, we will delve into the FDA approval process for cannabis research, shedding light on the intricacies involved and exploring how this regulatory framework impacts the advancement of scientific knowledge in the field.

The scheduling of cannabis as a controlled substance has imposed restrictions on its cultivation, distribution, and utilization. As a result, researchers face numerous hurdles when attempting to investigate its potential medical applications. However, the FDA plays a crucial role in overseeing the approval process for conducting cannabis-related studies, ensuring the safety and efficacy of any investigational drugs derived from cannabis compounds. Understanding this regulatory landscape is vital for researchers, healthcare professionals, and the public alike, as it can shape the future of cannabis-based medicine. In this blog post, we will provide an overview of the FDA's role in cannabis research and outline the various stages of the approval process for both marijuana and hemp.

FDA Approvals and Considerations: Understanding the Status of Cannabis-Derived Medications

The FDA has made significant strides in evaluating and approving specific cannabis-derived medications for therapeutic purposes. Among them is Epidiolex, a drug containing purified cannabidiol (CBD), which has received FDA approval for the treatment of seizures associated with Lennox-Gastaut syndrome and Dravet syndrome in patients aged two and older. This approval signifies that the FDA has deemed Epidiolex safe and effective for its intended use, providing patients with a regulated treatment option.

Marinol and Syndros are two other FDA-approved medications utilized in the United States. These medications, containing the active ingredient dronabinol, are indicated for combating nausea associated with cancer chemotherapy and for treating anorexia in AIDS patients. Dronabinol is a synthetic form of delta-9-tetrahydrocannabinol (THC), the psychoactive component responsible for the “high” associated with cannabis use. Additionally, Cesamet, another FDA-approved drug, contains nabilone, which closely resembles THC in its chemical structure. Cesamet is primarily used to alleviate nausea related to cancer chemotherapy.

FDA Approval Process For Cannabis Research

The FDA plays an important role in supporting scientific research into the medical uses of cannabis and its components. They provide guidance and information to researchers who want to study cannabis for medical purposes.

In 2016, the FDA updated its guidelines on developing botanical drugs, which include cannabis-derived drugs. They also offer support and assistance to investigators throughout the drug development process. Researchers who want to conduct clinical studies using cannabis need to work with the FDA and submit an application called an IND. This application outlines the proposed studies, the qualifications of the investigators, and measures to protect the rights and safety of the participants.

The FDA reviews these applications to ensure the studies are safe and ethical. For researchers studying cannabis as an animal drug, they would follow a similar process with the Center for Veterinary Medicine. The FDA's goal is to support researchers in developing safe and effective drugs derived from cannabis through a careful and regulated process.

Marijuana Studies

1. The sponsor contacts the FDA's review division to request a pre-IND meeting and obtains a pre-IND number. If it's for animal drug research, they engage with the Center for Veterinary Medicine (CVM) to establish an INAD file.
2. The sponsor contacts NIDA or another DEA-registered source to get information about available cannabis and cannabis-derived substances for their research. They need this information to include in their IND submission, which includes details about chemistry, manufacturing, controls, and botanical raw material.
3. If the selected substance's manufacturer has a Drug Master File (DMF), the sponsor needs to obtain a Letter of Authorization (LOA) to reference the necessary data. Otherwise, they must include all the required information in their IND submission to ensure the study drug's safety for human use.
4. The sponsor sends a copy of the IND, clinical protocol, and LOA (if applicable) to the FDA for review. They must wait for 30 days, unless notified earlier by the FDA, before initiating any clinical trials to ensure the submission is reviewed for safety.
5. If the FDA authorizes the IND as “safe to proceed,” the sponsor can submit their clinical protocol registration application to the DEA, along with the referenced IND number, to obtain the protocol registration. After receiving this registration, the sponsor contacts NIDA or a DEA-registered source to obtain the cannabis and/or cannabis-derived substances for the study and can begin the research.
6. For nonclinical research under an INAD file with CVM, prior authorization from the FDA is not required. Investigators can immediately proceed with the protocol registration application to the DEA, obtain investigator and study site licensure, and then obtain the Schedule I cannabis-derived study drug for their research.

Steps For Hemp Research

1. The sponsor requests a pre-IND meeting and obtains a pre-IND number from the FDA's CDER review division. If it's for new animal drug research, they can engage with CVM to establish an INAD file. A pre-IND meeting is optional and provides guidance from the FDA on research plans and IND submission requirements.
2. The sponsor includes all relevant chemistry, manufacturing, and controls (CMC) and botanical raw material (BRM) information in the IND for FDA review. This includes information about hemp cultivars, if applicable.
3. If the hemp manufacturer has a Drug Master File (DMF), the sponsor needs to obtain a Letter of Authorization (LOA) to reference the CMC and BRM information. Alternatively, the IND submission should contain all the necessary CMC data to ensure the study drug's safety for human use.
4. The sponsor sends a copy of the IND and clinical protocol, along with the LOA if applicable, to the FDA for review.
5. The FDA reviews the submitted IND. The sponsor must wait for 30 calendar days after submission before starting any clinical trials, unless notified otherwise by the FDA. During this time, the FDA assesses the submission for safety to protect research subjects from unreasonable risks.

What is the FDA’s Role in the Drug Approval Process?

The FDA plays a crucial role in regulating drugs, including cannabis and cannabis-derived products. This involves carefully reviewing applications to market drugs to determine their safety and effectiveness for their intended uses. The FDA's drug approval process relies on well-designed and conducted clinical trials that provide the necessary scientific data for decision-making. Without this review, the FDA cannot ensure the safety, effectiveness, and quality of a drug product. The lack of FDA approval and oversight on cannabis-based drugs poses potential risks to individuals using them.