The FDA’s Position
Last June the U.S. Food and Drug Administration announced a historic milestone, that it had made Epidiolex the first “FDA-approved drug that contains a purified drug substance derived from marijuana.” On that same day, FDA Commissioner Dr. Scott Gottlieb also issued a statement.
The approval of Epidiolex, he wrote, was an important medical advance. It demonstrated “that advancing sound scientific research to investigate ingredients derived from marijuana can lead to important therapies. This new treatment provides new options for patients.”
But Gottlieb also cautioned that the FDA’s approval of Epidiolex did not mean its approval of marijuana “or of all its components,” and that the agency had only approved one specific CBD (cannabidiol) medication for a specific treatment.
That approval, he added, “was based on well-controlled clinical trials evaluating the use of this compound in the treatment of a specific condition. Moreover, this is a purified form of CBD … delivered to patients in a reliable dosage form and through a reproducible route of delivery to ensure that patients derive the anticipated benefits. This is how sound medical science is advanced.”
There was another indication of the FDA’s stance towards medicinal cannabis this past December when Congress federally legalized hemp. Like marijuana, hemp is a form of the cannabis plant. But hemp contains only traces of tetrahydrocannabinol (THC), the intoxicating compound found in marijuana.
At that time, Dr. Gottlieb issued another statement. He acknowledged the public’s growing interest in cannabis and in particular in CBD, which can also be derived from hemp, but he warned against the way some cannabis products are being advertised to consumers.
“We continue to be concerned at the number of drug claims being made about products not approved by the FDA that claim to contain CBD or other cannabis-derived compounds,” he said.
“Cannabis and cannabis-derived products claiming in their marketing and promotional materials that they’re intended for use in the diagnosis, cure, mitigation, treatment or prevention of diseases (such as cancer, Alzheimer’s disease, psychiatric disorders and diabetes) are considered new drugs or new animal drugs and must go through the FDA drug approval process for human or animal use before they are marketed in the U.S.”
Such deceptive marketing of unproven treatments, he added, can create public health concerns — and might even keep some patients from getting “appropriate, recognized therapies to treat serious and even fatal diseases.”
The advocacy organization Public Citizen was critical of Gottlieb, saying his time in charge of the FDA was marked by decisions “that tilted further in favor of industry’s financial interests rather than the interests of public health.”
When DOPE contacted the FDA for some clarification on how the agency evaluates marijuana-based drugs, an FDA spokesman pointed to a page on their website that breaks down the FDA’s development and approval process for new drugs.
Getting FDA Approval
The FDA department charged with that mission is the Center for Drug Evaluation and Research (CDER). According to the website, the center’s evaluation of new drugs before they reach the market “not only prevents quackery, but also provides doctors and patients the information they need to use medicines wisely.”
In order to get federal approval, the FDA says there are “well-established paths” that a drug or a medical device needs to travel.
Here’s the basic five-step process:
- Discovery/Concept – Research for a new drug or device begins in the laboratory.
- Preclinical Research – Drugs and devices undergo laboratory and animal testing to answer basic questions about safety.
- Clinical Research – Drugs and devices are tested on people to make sure they are safe and effective.
- FDA Review – FDA review teams thoroughly examine all of the submitted data related to the drug or device and make a decision whether or not to approve it.
- Post-Market Safety Monitoring – FDA monitors all drug and device safety once products are available for use by the public.
But if a drug or device is considered to be a significant medical breakthrough — that treats not only serious diseases but also has advantages over existing treatments — then the FDA has alternative designations to make those drugs or devices available to the public as rapidly as possible. Those pathways are:
- Fast Track – This process is designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
- Breakthrough Therapy – A process designed to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy.
- Accelerated Approval – These regulations allow drugs for serious conditions that fill an unmet medical need to be approved based on a surrogate endpoint.
- Priority Review – A Priority Review designation means the FDA’s goal it to take action on an application within six months.
But the FDA approval process is far from fool-proof and has recently come under some intense scrutiny and criticism.
Last June PBS News Hour reported that the FDA reviews and approves drugs faster than any similar regulatory agency in the world. It also said the agency “is increasingly green-lighting expensive drugs despite dangerous or little-known side effects and inconclusive evidence that they curb or cure disease.”
As an example, PBS highlighted the case of Nuplazid, the first FDA-approved antipsychotic medication that treats the hallucinations and delusions associated with Parkinson’s disease.
According to a CNN report, the FDA put Nuplazid on its fast track approval process. The drug went on the market in April of 2016 at a pre-insurance cost of around $100 per day per patient.
CNN also noted that Nuplazid has been cited as a “‘suspect’ medication in hundreds of deaths voluntarily reported to the agency.” But last September, after “a thorough review,” the FDA announced its conclusion that the drug’s benefits outweighed its risks.
Cannabis and Parkinson’s
Parkinson’s disease affects dopamine receptors in the brain. Over the course of years, as its symptoms progress, it can affect a patient’s mental health as well as cause tremors, painful rigidity in the limbs, balance issues and other serious physical ailments.
More than 10 million people worldwide are thought to be living with Parkinson’s disease (PD). In the United States about one million people have PD, with more than 50,000 new cases diagnosed annually.
Parkinson’s has no known cure, and its treatment options can vary, from a variety of medications to surgery. But a surprising number of Parkinson’s patients and their doctors are considering medical cannabis as a way to cope with PD symptoms.
According to a recent survey by the Parkinson’s Foundation and Northwestern University, 80 percent of PD patients have used cannabis, while 95 percent of neurologists who work with PD patients say their patients have asked them to prescribe cannabis. And of the doctors who work with PD patients, 23 percent have received formal education on medical marijuana.
That said, the survey also reports that only 10 percent of physicians have recommended medical cannabis to their PD patients.
One of the biggest issues for PD patients and their healthcare providers, according to James Beck, chief scientific officer of the Parkinson’s Foundation, is that few clinical studies are investigating the effects of cannabis on PD symptoms.
“What hasn’t caught up … is the research to really understand what it’s good for and what it’s not good for,” he tells DOPE Magazine.
And because of marijuana’s federal illegality and a lack of certified medical studies, much of the mainstream medical community worries about repercussions and the potential loss of their medical licenses should they recommend or prescribe medical cannabis.
Beck says another important issue is the lack of practical information available on medical marijuana for PD physicians and others in the healthcare sector.
The challenge, he details, is getting the mainstream research and medical community “to awake from anecdotes to evidence, in order to improve the quality and the outcome of the people they care for. And we see great benefit from that. We’re moving from a period where [medical cannabis] has been offensive and kept in the dark, to where more light is shining on it.”
Another challenge: getting healthcare providers to help their PD patients understand both the benefits and risks of medical cannabis.
For example, cannabis might help some PD patients with appetite and pain issues, but it also can lower blood pressure. Falls are a leading cause of death in PD patients, so there can be issues with them using cannabis, which can affect a person’s motor skills and coordination.
Beck says there is no evidence to date that cannabis can stop the progression of Parkinson’s. “But is it a potentially effective way to handle some of the really troubling and disabling symptoms? Yes,” he adds.
And while there’s not enough evidence to prove medical cannabis can help treat PD symptoms, “suggestive and anecdotal reporting could lead us into that direction,” he says. “We haven’t done careful studies in which to do that.”
Once cannabis is recognized as a drug with the potential to treat Parkinson’s symptoms, Beck expects it will also “undergo the same type of scrutiny that any other medication that’s prescribed for individuals would go under.”