Appeal type: Drugs
Decision: Unfavorable

We decided that the Plan does not have to pre-approve Botox.

You asked the Plan to pre-approve Botox. You say that the requested botox is medically necessary. The Plan
denied your request. The Plan says the enrollee does not meet criteria for the requested botox.

The Plan must follow Medicare rules. The rules say that Medicare may pay for unlabeled uses for FDA approved
drugs if they are medically accepted as safe and effective or appropriate for the particular use.

We asked our physician reviewer to carefully review the case file and apply the relevant Medicare rules to
determine if the Plan was correct in denying the request to pre-approve Botox. Our reviewer says that the
enrollee does not meet criteria for the requested botox. Our physician reviewer found pertinent to this review,
the enrollee carries the diagnoses of GERD and gastroparesis. Per 10/1/2020 charting, the enrollee "describes
increased regurgitation and GERD symptoms while taking BID PPI. Had good success with EGD with botox to
the pylorus." An EGD report from 10/16/2018 documents "normal esophagus, normal stomach, normal
pylorus, normal duodenum" --despite which the pylorus was balloon-dilated to 15mm and intrapyloric botox
injection was performed. No objective documentation of gastroparesis was provided.

Though trials evaluating the effect of intrapyloric botulinum toxin injection on gastroparesis have been
reported, there is no agreement whether botulinum toxin can effectively relieve the symptoms and improve the
results of gastric emptying study in patients with gastroparesis.

In Bai's systemic review of 15 reports, "only 2 randomized controlled trials were available. Almost all the nonrandomized
trials reported significant improvement in subjective symptoms and objective gastric emptying
study after botulinum toxin injection. While the 2 randomized controlled trials did not confirm the efficacy of
botulinum toxin injection, none of the individual trials showed that there was statistically significant subjective
and objective improvement in the active patients when compared with patients receiving placebo. There is no
evidence to recommend botulinum toxin injection for the treatment of gastroparesis."

The above sentiment is echoed by Tack: "In refractory cases, especially when there is weight loss, invasive
therapeutics such as insertion of feeding tubes, intrapyloric injection of botulinum toxin, implantable gastric
electrical stimulation, or surgical (partial) gastrectomy are occasionally considered, but there is little evidence
of efficacy, and these are not devoid of potentially major complications." The American College of
Gastroenterology opines: "Second-line approaches include venting gastrostomy or feeding jejunostomy;
intrapyloric botulinum toxin injection was not effective in randomized controlled trials."

In the enrollee's case, her 2018 EGD findings were normal including an absence of pyloric narrowing. Further,
available records do not provide documentation of gastroparesis -- by gastric emptying scan or other methods.

We looked at this physician's review, the file, and Medicare rules. Based on this information, we decided that
Medicare rules for coverage of Botox have not been met. Therefore, we decided that the Plan does not have to
pre-approve Botox.