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FDA omits numbers on drug to reduce night-time visits to the bathroom; placebo was nearly as good

FDA approves first treatment for frequent urination at night due to overproduction of urine

Our Review Summary

This news release heralds the FDA’s first approved treatment for adults who get up at least twice nightly to urinate due to a condition known as nocturnal polyuria, or overproduction of urine at night. The news release does a good job of cautioning that this treatment isn’t for everybody who suffers from frequent nighttime urination, but it falls short in describing the size of the benefit, which is a small improvement over a placebo. It also downplays the fact that this is an old drug — desmopressin — being adapted for a new use. While it’s good to see old drugs re-purposed in ways that might be beneficial for patients, the flip side is that companies typically use this as an opportunity to raise the price of the medication.

 

Why This Matters

Frequent urination at night is a common annoyance, especially in older people. In other words, it’s prime click bait material. So it didn’t surprise us to see at least one media outlet exaggerate the benefits of this product. Men’s Health ran with this misleading story: “Here’s How You Can Stop Waking Up Every Night to Pee.” But in this case you can’t blame the news release which did a good job of explaining that this treatment is designed to treat only a specific subset of cases, known as nocturnal polyuria, in which the body produces a disproportionate amount of urine at night. It’s not for the entire 20 percent or so of men who suffer from frequent nighttime bathroom runs as Men’s Health implies. It would have been helpful if the release had shared the estimated prevalence of this condition.

This appears to be an attempt to expand the market for an existing drug. If the pricing trend holds, this approval of an older drug for a new indication may result in a significant price increase.

Criteria

Does the news release adequately discuss the costs of the intervention?

Not Applicable

The FDA evaluates drugs without considering the cost, which is why we give the release a pass for not mentioning the price tag. We hope the FDA could one day also comment on the projected cost of drugs and their cost-effectiveness.

How much will this re-purposed drug cost? We found a 5-milliliter bottle of generic desmopressin acetate on the market for $69.95. However, the manufacturer that won approval for this new use might charge significantly more when it markets the drug under the name Noctiva. The company had no comment on pricing, according to a recent report in FiercePharma.

Does the news release adequately quantify the benefits of the treatment/test/product/procedure?

Not Satisfactory

The news release is vague on benefits, stating that although trials “showed a small reduction in the average number of night-time urinations with Noctiva compared to placebo, more patients treated with Noctiva were able to at least halve their number of night-time urinations, and patients treated with Noctiva had more nights with one or fewer night-time urinations.”

The benefit of being able to “halve the number of night-time urinations” is uninterpretable.

In fact, data submitted to the FDA shows patients using the drug went from an average of more than three urinations per night to about two, but the placebo was almost as effective. By our calculation, those using a placebo got up an average of one extra time every three nights compared with those who took the real drug. In other words, not a huge difference. The percentage of patients who experienced one or fewer nighttime urinations was 40 to 45 percent for those using Noctiva versus 30 to 34 percent for those taking a placebo. Again, a modest benefit.

Does the news release adequately explain/quantify the harms of the intervention?

Satisfactory

The news release does well describing harms, providing extensive details on adverse effects, particularly warning that Noctiva can cause a potentially life-threatening drop in blood sodium and shouldn’t be used by those at risk for that condition. The release says the drug will come with a boxed warning and a Medication Guide to draw attention to the risk. It also lists the most common side effects: nasal discomfort, cold symptoms, nasal congestion, sneezing, high or increased blood pressure, back pain, nose bleeds, bronchitis and dizziness.

It cautions against use in pregnant women and children as well as patients with symptomatic congestive heart failure or uncontrolled hypertension and patients with certain nasal conditions such as colds or allergies.

We would have liked to see the news release mention that 4 percent of patients in the trials stopped therapy due to adverse events. Also, an FDA document expressed concern that the drug could mask symptoms of underlying conditions, which was only alluded to in the news release with an advisory that doctors treat underlying conditions that could cause frequent urination.

Does the news release seem to grasp the quality of the evidence?

Satisfactory

The news release provides information about the strength of the research with this statement: “Noctiva’s efficacy was established in two 12-week, randomized, placebo-controlled trials in 1,045 patients 50 years of age and older with nocturia due to nocturnal polyuria.”

Evidence derived from two relatively short-term studies on a daily drug that could potentially be used for years doesn’t seem particularly strong. Do we know if the benefits are durable or whether the slight benefit over placebo might dwindle to nothing after 6 months or a year of use? The drug can have serious side effects but nocturnal polyuria is not a life-threatening condition. Patients and physicians would be well-served with longer term safety data.

And as stated above, placebo was nearly as effective as the drug in reducing the number of night-time visits to the bathroom.

Does the news release commit disease-mongering?

Satisfactory

The news release doesn’t engage in disease mongering. It provides useful context about what the condition is, but not how common it is. That would have been a useful addition.

Does the news release identify funding sources & disclose conflicts of interest?

Not Satisfactory

The news release doesn’t mention that the efficacy trials for desmopressin to treat nocturnal poluria were sponsored by the manufacturer, Serenity Pharmaceuticals.

Does the news release compare the new approach with existing alternatives?

Not Satisfactory

There’s no mention of other treatments for nocturnal polyuria including behavioral strategies such as fluid restriction and medications including the generic form of desmopressin (see the section on novelty).

Does the news release establish the availability of the treatment/test/product/procedure?

Satisfactory

The news release mentions that this drug is already approved for other uses and that the newly approved therapy is manufactured by Renaissance Lakewood for Serenity Pharmaceuticals.

Does the news release establish the true novelty of the approach?

Not Satisfactory

The news release downplays the fact that this is not a new drug. It does acknowledge that there are other FDA-approved medications that also contain desmopressin, the active ingredient in this new product, but we would have liked to see this fact higher up in the release. In fact, desmopressin has been approved for use in the U.S. market since 1978 and has been used to treat a variety of conditions including diabetes and hemophilia A. Physicians have also been prescribing desmopressin to treat nocturia, although it wasn’t officially approved for that use until now.

Total Score: 5 of 9 Satisfactory

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