Thank you for the opportunity to speak today. My name is Dr. Stephanie Fox-Rawlings. I am a Senior Fellow at the National Center for Health Research. Our research center analyzes scientific and medical data to provide objective health information to patients, providers and policy makers. We do not accept funding from drug companies so I have no conflicts of interest.
Nocturia symptoms are caused by a wide range of underlying conditions. It is not surprising that SER120 does not create a clinically meaningful improvement when averaged across all of these conditions. SER120 may be effective for a subset of people with specific underlying conditions or other characteristics, but the sponsor has not identified that group of patients or their underlying conditions. To justify FDA approval, a drug should have a clinically meaningful improvement over placebo for the patients to whom it would be prescribed. A general indication for any patients with nocturia would not be appropriate because the drug clearly does not work well for a general population of nocturia patients.
You probably share my concern that the sponsor’s studies excluded patients with diseases/treatments that could reduce the safety of SER120. And yet, those same patients would consider the drug if it were approved for all adults with nocturia.
There are other safety concerns as well. The studies did not measure possible effects on the underlying conditions. Further studies should determine that treatment with desmopressin does not worsen any of the conditions that cause nocturia or that co-occur with it. In addition, about 11% of patients experienced mild hyponatremia and 1% experienced severe hyponatremia, which requires careful monitoring. For approval, the benefits of SER120 need to outweigh the risks for most patients, but to achieve that we need data on which patients are most likely to be harmed. And, that information needs to be widely available and mentioned in any advertising or promotional materials.
Patients’ age is also a concern. Although most patients with nocturia are over 50 years old, there are also many patients under 50, and the safety and effectiveness of the drug could be very different for younger adults. This may be especially true for premenopausal women.
Only a small number of premenopausal women were studied and they were not analyzed as separate subgroups, so it is impossible to know if the drug is appropriate for premenopausal women. Since 78% of patients were white, the risks and benefits may differ for other racial groups.
There are versions of desmopressin on the market already. It is not clear that this version has a better risk-benefit profile. Whether or not it is better, if approved, SER120 will be much more expensive. While cost is not FDA’s concern, the skyrocketing cost of older pharmaceuticals that are repurposed is a clear threat to Medicare, the affordability of health insurance, and to public health. For that reason, this Advisory Committee should make sure that it only approves a drug for an appropriate indication, and that the indication includes the ages and types of patients most likely to benefit.
Unfortunately, information in labels has little impact on prescribing behavior and DTC (direct to consumer) ads tend to minimize those details.
In conclusion, do not recommend this as the first drug approved for nocturia symptoms, unless there is a clinically meaningful benefit and sufficient safety profile for a clearly indicated population. An overly broad indication does not help patients and could harm them.
Thank you.