Appeal type: Experimental/Investigational
Decision: Overturned

Reviewer #2:

Diagnosis:  Crohn's disease.
Treatment:  Stelara 90 milligram/milliliter (mg/ml) syringe every 4 weeks.

The insurer denied coverage for Stelara 90 milligram/milliliter (mg/ml) syringe every 4 weeks.  The denial is upheld.

The patient is a young adult who has Crohn's disease. At issue is a denial of coverage for Stelara 90 milligram/milliliter (mg/ml) syringe every 4 weeks which the provider had ordered due to increasing symptoms. The plan declined coverage as this request exceeds the plan maximum allowance for this condition for this disease state.

The scientific evidence in peer-reviewed literature does not support a result of improvement in health outcome.

The provider has requested Stelara at a dosing not approved by the Food and Drug Administration (FDA) or the plan guidelines. A review of the literature indicates that there have been no significant studies to date demonstrating the safety and efficacy of Stelara dose escalation to every 4 weeks in Crohn's disease. The reports are anecdotal and involve small numbers of patients. As such, the usefulness, efficacy and safety of dose escalation of Stelara in patients with refractory Crohn's disease requires further study and would not be considered effective. Despite the patient's presentation and history, there is no cogent medical literature or society guidelines to recommend dosing Stelara at an interval of every 4 weeks for maintenance. There is no medical literature to show that this dosing frequency has a positive effect on healthcare outcomes. The formulary medications, including Stelara at a dosing of every 8 weeks for maintenance, is as effective and without more adverse side effects than the requested non-formulary medication. Therefore, the request for Stelara 90 milligram/milliliter (mg/ml) syringe every 4 weeks is not considered more beneficial for this patient.

"A subset of patients with Crohn's disease (CD) do not respond to ustekinumab (Stelara) at the standard dose of 90 milligrams (mg) every 8 weeks. Little is known about the efficacy of shortening the interval between doses." (Ollech, et al., 2021).

"Crohn's disease is an immune-mediated disease that results in panenteric chronic inflammation in genetically predisposed individuals exposed to an appropriate environment. The past two decades have witnessed the emergence of an important class of drugs known as anti-tumour necrosis factor (TNF) agents in the treatment of Crohn's disease. Unfortunately, the utility of these agents have been hampered by primary and secondary nonresponse in a significant proportion of patients. Ustekinumab, a monoclonal antibody to the p40 subunit of interleukin (IL) 12 and 23, is a novel pharmacotherapy for this patient cohort that offers an out-of-class option. It is approved for use in psoriasis and psoriatic arthritis, and has now been evaluated in phase II trials for moderate-to-severe Crohn's disease." (Simon, et al., 2016).

Stelara 90 milligram/milliliter (mg/ml) syringe every 4 weeks does not have final approval from the appropriate regulatory bodies for this diagnosis.

This patient is not a good candidate for Stelara 90 milligram/milliliter (mg/ml) syringe every 4 weeks.

Stelara 90 milligram/milliliter (mg/ml) syringe every 4 weeks is not the best available treatment for this patient at this time.

Adverse risks are increased over standard treatment/services as this has not been studied.

The requested service is not likely to be more beneficial than any of the standard treatments/procedures for this patient. The requested dosing is not more beneficial than standard dosing

In summary, the denial of Stelara 90 milligram/milliliter (mg/ml) syringe every 4 weeks
 and coverage for the requested service/treatment should be upheld. This treatment is not likely to be more beneficial than any standard treatments for this patient's condition and the adverse risks are not known over standard treatments.