Thank you for the opportunity to speak today. My name is Dr. Megan Polanin. I am a psychologist previously at Johns Hopkins and currently a Senior Fellow at the National Center for Health Research. Our research center analyzes scientific and medical data and provides objective health information to patients, providers, and policymakers. We do not accept funding from industry, so I have no conflicts of interest.
We have concerns about the efficacy of this drug. Because the sponsor did not analyze whether there were differences in efficacy for different doses, we don’t have specific information about the proposed doses. While the data demonstrated non-inferiority to the comparator drug, unfortunately only 10.8% of people taking this drug reached 100% abstinence based on negative urine samples and self-reported opioid use. As you know, self-reported opioid use is often inaccurate.
We expect that the drug will perform even worse in the real-world. And this abstinence rate is worse than the treatment you reviewed yesterday, even though this time abstinence was based on only 12 weeks instead of 24.
Based on the sponsor’s definition of responding to this drug, a key question is: If individuals are not found to have used opioids more than once during the final weeks of a 12-week clinical trial, is that good enough to keep them off drugs long-term? We do not know the answer to this question.
We’re all left wondering about this drug’s long-term effects. With less than 11% achieving so-called abstinence in 12 weeks, how long will patients need to take this drug to either achieve or maintain abstinence? We don’t think data for 12 weeks is long enough to justify approval for opioid use disorder – an addiction that is very difficult to treat successfully.
We know little about the safety of this drug. First, there are too few patients in the safety studies for various proposed doses to generalize safety information. Next, the sponsor did not collect important data about the drug’s safety in people with liver or kidney impairment, the interaction of the drug with other drugs, or the risks of IV administration. Accidental or intentional IV administration of this drug has the potential to be deadly. Finally, this drug is packaged with a prefilled syringe with a needle, which would make it easy to abuse — a grave safety risk. There are no data on the potentially dangerous effects of accidental or intentional IV administration of this drug at any of the doses studied.
Due to risks of self-injection, potential abuse and misuse, and accidental pediatric exposure, if this product is approved, it should only be administered by a trained healthcare provider in a controlled clinical setting. This is the only way the drug was studied, and there are no data on take-home use or self-administration of the product by patients.
In conclusion, we all want more options to reduce opioid addiction, but if the FDA approves treatments that are less safe and less effective than those already available, we are worried that this will not help. Scientific methods used to assess treatments must be sound. Specific doses need to be evaluated for safety and effectiveness based on reasonable sample sizes for at least 24 weeks. Given potentially serious risks that outweigh this drug’s minimal benefits, we cannot recommend approval of this drug based on current data.
If, after different doses are evaluated for safety and efficacy, there is clear evidence of one or more safe and effective doses, we agree with the FDA that there should be REMS and ETASU to mitigate potential adverse consequences due to IV self-administration, including limiting dispensing to certified healthcare settings that have a DATA-waived prescriber or are DEA registrants.
Thank you for the opportunity to share our views.
The Psychopharmacologic Drugs Advisory Committee and the Drug Safety and Risk Management Advisory Committee voted 17-3 in favor of approving some doses of CAM2038 weekly and monthly buprenorphine depot. Read more about the meeting here.