Appeal type: Medical Necessity
Decision: Overturned

Summary Reviewer  
The patient is a 38-year-old female who presented to her provider on 4/20/22. The records noted 
that the patient had been diagnosed with adult onset Stills disease (AOSD) in 2017 following a 
hospitalization  due  to  fever,  sore  throat,  arthralgias,  myalgias,  and  rashes.  The  patient  has 
requested authorization and coverage for Actemra injection 162 mg/0.9 mL. The Health Insurer 
has denied this request and reported that the requested medication is not medically necessary for 
the treatment of this patient. This denial is the subject of this appeal and determination. The 
physician reviewer found that the submitted documentation supports the medical necessity of the 
requested medication. Adult onset Still's disease (AOSD) is a rare systemic inflammatory disorder 
of unknown etiology, characterized by quotidian or doublequotidian spiking fevers with an 
evanescent  rash,  arthritis,  and  multiorgan  involvement.  While  its  etiology  is  largely 
unknown/idiopathic, it has recently been suggested that alterations in cytokine production have an 
important pathophysiological role in AOSD. Treatment in AOSD has been exclusively empirical, 
with data on treatment efficacy extrapolated from case reports and small scale retrospective 
studies. High-dose glucocorticoids and nonsteroidal anti-inflammatory drugs (NSAIDs) are used 
in initial treatment and for flares. The U.S. Food and Drug administration notes that Actemra 
(generic name tocilizumab) is an interleukin-6 (IL-6) receptor antagonist indicated for treatment: 
rheumatoid arthritis in adult patients with moderately to severely active rheumatoid arthritis who 
have  had  an  inadequate  response  to  one  or  more  disease  modifying  anti-rheumatic  drugs 
(DMARDs); giant cell arteritis in adult patients; polyarticular juvenile idiopathic arthritis (PJIA) 
in patients two years of age and older with active PJIA; systemic juvenile idiopathic arthritis (SJIA) 
in patients two years of age and older with active SJIA; and cytokine release syndrome (CRS) in 
adults and pediatric patients two years of age and older with chimeric antigen receptor T cell-
induced  severe  or  life-threatening  CRS.  While  there  are  no  randomized  clinical  trials  to 
prospectively evaluate the efficacy/safety of tocilizumab in AOSD, multiple case series and 
retrospective case reports support its use for the resolution of disease activity, ability to taper 
steroids faster, and overall tolerability. Kr and colleagues report that tocilizumab is an effective 
and well-tolerated treatment option for treatment resistant AOSD. Tang and colleagues report that 
patients with AOSD refractory to high-dose corticosteroids and methotrexate may respond well to 
tocilizumab treatment with a steroid-sparing effect and an acceptable safety profile. Reihl Crnogaj 
and colleagues report that there is experience supporting subcutaneous tocilizumab as a promising 
option for treatment of refractory AOSD patients. In this case, given current medical literature and 
as the patient has been symptom-free, flare-free, and able to avoid taking glucocorticoids since 
starting Actemra in December 2017, the requested medication is medically indicated. Therefore, 
Actemra injection 162 mg/0.9 mL is medically necessary for the treatment of this patient.