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Budding Growth: Gottlieb Expanded GI Drug Approvals, Fostered Medical Cannabis Development
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In the fall of 2016, I was selected to take part in the ACG-FDA fellowship where I was embedded with the Division of Gastroenterology and Inborn Errors Products in the Center for Drug Evaluation and Research at the FDA for one month.
While there, I had the opportunity to audit the medical officer training curriculum, and attend many meetings with FDA experts, in addition to meetings with industry, learning about how the two sides interact. Through these experiences, I learned a great deal about the regulatory pathway to drug and device approval and gained a greater understanding of the gravity with which the FDA takes their role as protectors of public health and safety by working as closely as possible with industry and academia to approve drugs that are both safe and effective.
Last month, Scott Gottlieb, MD, announced that he was resigning as commissioner of the FDA effective April 5, 2019. During his tenure, several positive steps were taken at the FDA regarding the field of gastroenterology and hepatology and drug pricing. Additionally, actions were taken to initiate policies that will affect the development of cannabis-derived compounds.
Several new GI drugs were approved during Gottlieb’s tenure as FDA commissioner. Tofacitinib was approved, which is not only the first Janus kinase (JAK) inhibitor approved for patients with inflammatory bowel disease (IBD) but is also the first oral therapy approved for patients with moderate to severe ulcerative colitis. The approval of this drug was exciting and potentially life changing for many of our patients who do not want to undergo injections or drug infusions. Additionally, plecanatide and prucalopride – therapies for chronic idiopathic constipation and irritable bowel syndrome (IBS) with constipation – were approved during his tenure. The approval of prucalopride was especially exciting as many gastroenterologists anxiously awaited that approval, considering it has been an approved therapy in other countries for several years, and we were hoping it would eventually get approved in the U.S.
Under Gottlieb’s tenure, the FDA was developing policy to take on exorbitant drug pricing to increase access to drugs for patients by fostering competition in the generic drug market. Our GI patients would certainly benefit from lower prices and increased access to many of these expensive drugs.
Things That Need Improving
Our options for pain control are quite limited, especially for our GI patients. Not only do GI patients often have pain as a strong component of GI disease (eg, IBS, IBD, and chronic pancreatitis), but traditional pain medications used for GI and non-GI pain often causes consequences in the GI tract, such as opioid-induced motility disorders. The FDA should, and I think they are beginning to, work on fostering development of alternative pain control medications.
Another area where drug development is needed is non-alcoholic fatty live disease (NAFLD), given the high prevalence of the disease, quickly making it the most common indication for liver transplant. Currently there is only one treatment option for NAFLD, adjuvant vitamin E. There are several drugs in development for NAFLD currently, and these should be a priority for the FDA.
Growth of Cannabis
Cannabis may hold potential therapeutic benefits for many GI disorders, and not just for abdominal pain, although that is the most common indication for which people would likely be prescribed cannabis. For example, the use of cannabis to improve inflammation associated with IBD is currently being investigated.
During Gottlieb’s tenure, Epidiolex – known as cannabidiol – was approved in June 2018 for the treatment of seizures associated with rare epilepsy syndromes. While unrelated to GI disease specifically, this was the first cannabidiol (CBD)-derived medication approved by the FDA, and the approval was based on adequate and well controlled clinical studies. Further, Congress passed the Agriculture Improvement Act in 2018 that made hemp-compounds legal. As a result, hemp compounds were removed from the Controlled Substances Act and the Drug Enforcement Agency (DEA) moved cannabidiol compounds with very low levels of tetrahydrocannabinol (THC < 0.1%) from schedule I to schedule V drugs as long as they are approved by the FDA. This is the first time the DEA has moved any cannabis-derived compound from schedule I. This may improve access to patients, and hopefully improve access for researchers in obtaining these low levels of THC. Prior to his resignation, Gottlieb released several statements regarding new steps the FDA is taking to evaluate and advance regulatory pathways for cannabis-containing and cannabis-derived products. These include a public hearing scheduled on May 31 for stakeholders to share their knowledge and experience, particularly regarding safety, with these compounds, and to help develop lawful pathways by which these products can be developed and marketed in an efficient and predictable manner. Additionally, he has formed a high-level internal working group at the FDA to explore regulatory and legislative pathways for the lawful marketing of CBD-containing foods and dietary supplements. Currently, it is illegal to market foods or supplements containing CBD with health claims, as CBD is an FDA approved and regulated substance. As part of Gottlieb’s statements, he released news of several compliance actions including FDA warning letters issued to multiple companies marketing CBD-containing compounds with egregious health claims.
The steps taken by the FDA are small, but I believe will eventually lead to larger changes to cannabis policy that will hopefully benefit those who want to use cannabis-derived or cannabis-containing compounds for medical research and for the development of cannabis-derived therapies. My hope is that the new commissioner holds the planned public hearing on development of cannabis-derived compounds and continues to develop guidance to foster the appropriate development of cannabis-derived drugs that Gottlieb began under his tenure. Additionally, the new commissioner should continue to develop policy toward reducing drug prices to increase access for our patients.
Kendall Beck, MD
Assistant Professor of Medicine
University of California San Francisco
Disclosure: Beck reports receiving research funding from AbbVie.