Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Thyroid Eye Disease
Treatment: Tepezza
The insurer denied Tepezza.
The determination is overturned.

This is a female patient with thyroid eye disease for whom coverage of Tepezza is the subject of review. Reportedly, the patient has severe active thyroid eye disease causing disabling pain, swelling, and eye protrusion not responding to other therapies. Coverage was previously denied as the request did not have enough information to determine if the service was medically necessary.
Chart notes document active thyroid eye disease with a Clinical Activity Score (CAS) of 8. A course of intravenous (IV) methylprednisolone was initiated, and the provider confirmed no improvement in symptoms after 5 weeks of such therapy. Thyroid function testing found triiodothyronine (T3) and thyroxine (T4) levels to be normal. However, policy criteria required labs confirming the patient as euthyroid be performed within 30 days. At issue is the medical necessity of Tepezza.

The requested Tepezza is medically necessary for this patient who has had active thyroid eye disease for about a year, had been confirmed to be euthyroid and has not responded to a reasonable course of IV corticosteroids. Such treatment is consistent with the standard of care and supported by current medical literature:
Smith et al conducted a multicenter, double-masked, randomized, placebo-controlled trial to determine the efficacy and safety of teprotumumab, a human monoclonal antibody inhibitor of insulin like growth factor-insulin receptor (IGF-IR), in patients with active, moderate-to-severe ophthalmopathy. A total of 88 patients were randomly assigned to receive placebo or active drug administered intravenously once every 3 weeks for a total of eight infusions. All patients met criteria for euthyroid status (within plus/minus () 50 percent (%) of the reference range). The primary end point was the response in the study eye. This response was defined as a reduction of 2 points or more in the Clinical Activity Score (scores range from 0 to 7, with a score of greater than or equal to (>=) 3 indicating active thyroid-associated ophthalmopathy) and a reduction of 2 mm or more in proptosis at week 24. Secondary end points, measured as continuous variables, included proptosis, the Clinical Activity Score, and results on the Graves' ophthalmopathy-specific quality-of-life questionnaire. Adverse events were assessed. In the intention-to-treat population, 29 of 42 patients who received teprotumumab (69%), as compared with 9 of 45 patients who received placebo (20%), had a response at week 24 (probability (P) less than (<) 0.001). Therapeutic effects were rapid; at week 6, a total of 18 of 42 patients in the teprotumumab group (43%) and 2 of 45 patients in the placebo group (4%) had a response (P< 0.001). Differences between the groups increased at subsequent time points. The only drug-related adverse event was hyperglycemia in patients with diabetes; this event was controlled by adjusting medication for diabetes. The authors concluded that, in patients with active ophthalmopathy, teprotumumab was more effective than placebo in reducing proptosis and the Clinical Activity Score.