Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis: Ulcerative Colitis. 
Treatment: Stelara 90mg/mL Syringe. 

The insurer denied the Stelara 90mg/mL Syringe. 
The denial is upheld. 

The patient is a male who has the diagnosis of Crohn's disease. The patient was maintained on Stelara every eight weeks. As the patient has evidenced symptoms on this dosing regimen, the provider now requests the provision of Stelara every four weeks. The provider supplied two medical articles for review. 

No, the health plan should not cover the proposed treatment. 

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 four weeks in Crohn's disease other than anecdotal reports ,e.g. as follows: 

Per Greenup A. et al, in the absence of therapeutic drug monitoring, empirical dose escalation has been considered as a strategy to optimize response in patients with either primary or secondary non-response. Efficacy and safety of UST (ustekinumab) 90 milligrams (mg) subcutaneous (SC) every four weeks is not known. 
A retrospective, observational study of compassionate use of UST (ustekinumab) in CD (Crohn's disease) was conducted at a Canadian tertiary center. 

Results indicate that real-life experience of UST (ustekinumab) use in patients with CD (Chron's disease), maintenance dose escalation to 90mg every 4 weeks had a modest benefit in achieving a clinical response. This may be suggestive of the mechanism of loss of response to UST (ustekinumab) being driven by factors other than low bioavailability due to processes such as rapid clearance. Further assessment in larger cohorts as well as the use of therapeutic drug monitoring will be important to evaluate the usefulness of dose escalation for patients on UST (ustekinumab). 

Reports such as the above are anecdotal and involve small numbers of patients. Therefore 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 medically necessary. 

Despite the member'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 yet no cogent 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, are as effective and without more adverse side effects than the requested non-formulary dosing schedule. 

No, the requested health service is not more beneficial than any standard treatment for the insured's condition or disease. 

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 other than anecdotal reports ,e.g. as follows: 

Per Greenup A. et al, in the absence of therapeutic drug monitoring, empirical dose escalation has been considered as a strategy to optimize response in patients with either primary or secondary non-response. Efficacy and safety of ustekinumab (UST) 90 milligrams subcutaneous (SC) every four weeks is not known.

A retrospective, observational study of compassionate use of ustekinumab (UST) in Crohn's disease (CD) was conducted at a Canadian tertiary center. 

Results indicate that real-life experience of ustekinumab (UST) use in patients with Crohn's disease (CD), maintenance dose escalation to 90 milligrams every four weeks had a modest benefit in achieving a clinical response. This may be suggestive of the mechanism of loss of response to ustekinumab (UST) being driven by factors other than low bioavailability due to processes such as rapid clearance. Further assessment in larger cohorts as well as the use of therapeutic drug monitoring will be important to evaluate the usefulness of dose escalation for patients on ustekinumab (UST). 

Reports such as the above are anecdotal and involve small numbers of patients. Therefore, 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 medically warranted. 

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 four 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 eight weeks for maintenance, is as effective and without more adverse side effects than the requested standard treatments and dosing.