Appeal type: Experimental/Investigational
Decision: Overturned

Diagnosis:  Crohn's Disease.
Treatment:  Stelara 90mg every 4 weeks.

The insurer denied the Stelara 90mg every 4 weeks.  The determination is overturned.

The patient has a history of Crohn`s disease.  He has tried and failed infliximab, methotrexate and corticosteroids.  He was then stated on Stelara standard dose (90 milligrams (mg) every 8 weeks).  He had some improvement on Stelara; however, he becomes symptomatic before the next Stelara dose.  He gets increased stool frequency and fatigue the week before the next dose.  He was found to have an undetectable Stelara level.

The requested service is Stelara dose escalation to 90 mg every 4 weeks in order to improve the drug level and the clinical response.  At issue is whether the disease for which treatment is requested is a rare disease, whether there is a standard treatment that is likely to be more beneficial than the proposed treatment, and whether the treatment is likely to benefit the enrollee in the treatment of the enrollee's rare disease and whether such benefit outweighs the risks of such health service or procedure.

Crohn`s disease is not considered a rare disease.  Per the Crohn`s and Colitis foundation of America (CCFA), approximately 780,000 Americans currently have Crohn's disease.  There is adequate knowledge about Crohn`s disease in addition to many published practice guidelines.

Therefore, Crohn`s disease is not considered a rare disease.

This patient has a case of severe Crohn`s disease.  He has already tried and failed numerous therapies including Remicade, methotrexate and steroids.  He also failed to adequately respond to the standard dose of Stelara.

The available treatments for Pediatric Crohn`s disease are very limited.  This patient has tried and failed most of them.  Humira is another biologic treatment that is approved for pediatric Crohn`s disease.  The patient has not tried Humira, however, he is unlikely to respond to it because he has already tried and failed a similar medication (Remicade). He has improved on Stelara, but does not have adequate drug levels.

Therefore, Stelara dose escalation is likely to be more beneficial than the available alternatives.

Stelara is Food and Drug Administration (FDA) approved for the treatment of Crohn`s disease (CD) and ulcerative colitis in adults.  Despite the lack of FDA approval for pediatric patients, there are many peer reviewed publications supporting the efficacy and safety of Stelara in pediatric patients with inflammatory bowel disease.  Chavannes M et al. reported a multi-centered cohort study supporting the safety and efficacy of Stelara in pediatric patients with Crohn`s disease.  A recent report by Dayan JR et al. showed that ustekinumab (Stelara) is efficacious and safe in pediatric patients with inflammatory bowel disease (IBD).  J Rosh, et al. recently published a multi-center, double-blind, induction dose-ranging study in pediatric patients with Crohn`s disease.  The study supported the safety and efficacy of Stelara in this patient population.  Therefore, the use of Stelara in pediatric patients is consistent with the generally accepted standards of care.

It is very common for patients with inflammatory bowel disease to lose response to their biologic over time or to require higher than standard dosing regimen approved by the FDA.  It is common practice in the management of inflammatory bowel disease to try to rescue the treatment by increasing the dose frequency (dose escalation).  This practice is very commonly used in biologics in general including Stelara.  There are many peer reviewed literature reports showing that low Stelara levels were associated with poor clinical response. (Battat, Robert, et al).

Dose escalation is proven to be beneficial.  Per Ollech, Jacob E., et al., "Shortening the ustekinumab 90 milligram (mg) dose interval to 4 weeks for patients with CD who did not respond to doses every 8 weeks improved clinical and biological indices of disease activity."

This patient showed an incomplete response to standard dose of Stelara and has a documented low drug level.  This patient is expected to experience serious disease complications if this request is denied.  Therefore, the requested dose escalation is likely to benefit the member.