Appeal type: Medical necessity
Decision: Overturned

Diagnosis:  Active Moderate to Severe Thyroid Eye Disease (TED).
Treatment:  Injection (J3241) Teprotumumab-trbw (Tepezza), 10 mg (milligrams), 1150 units.

The insurer denied Injection (J3241) Teprotumumab-trbw (Tepezza), 10 mg, 1150 units.  The denial is overturned.

The patient is an adult male with thyroid eye disease for whom coverage of Tepezza is the subject of review. Coverage was previously denied by the health plan, which does not allow for more than 8 lifetime infusions. Upon appeal, it is identified that the patient started Tepezza treatment 3 years ago, but his course was interrupted when manufacturing stopped during COVID. He did go on to complete all 8 infusions and had a good response to Tepezza, evidenced by nearly resolved symptoms after treatment. However, the patient has experienced significant recurrence with active thyroid eye disease and a Clinical Activity Score (CAS) of 7. 

The requested second course of Tepezza is medically necessary.

Douglas et al. evaluated the safety and efficacy of teprotumumab in patients with TED (thyroid eye disease) who were nonresponsive or who experienced a disease flare. The Treatment of Graves' Orbitopathy to Reduce Proptosis with Teprotumumab Infusions in an Open-Label Clinical Extension Study (OPTIC-X) was a teprotumumab treatment and re-treatment trial following the placebo-controlled teprotumumab Phase 3 Treatment of Graves' Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study (OPTIC) trial. OPTIC nonresponders or those who flared (2-millimeter increase in proptosis, 2-point increase in clinical activity score [CAS], or both) during follow-up were treated for the first time (previous placebo patients) or re-treated with teprotumumab in OPTIC-X with 8 infusions over 24 weeks.

Main outcome measures included proptosis response and safety. Secondary outcomes included proptosis, CAS (clinical activity score), subjective diplopia, and quality-of-life. Thirty-three of 37 placebo-treated OPTIC patients (89.2%) became proptosis responders (mean  standard deviation, -3.5  1.7 mm) when treated with teprotumumab in OPTIC-X. The responses were equivalent to the OPTIC study. In these responders, proptosis, CAS (clinical activity score) of 0 or 1, and diplopia responses were maintained in 29 of 32 patients (90.6%), 20 of 21 patients (95.2%), and 12 of 14 patients (85.7%), respectively, at follow-up week 48. The median TED (thyroid eye disease) duration was 12.9 months versus 6.3 months in those treated with teprotumumab in the OPTIC study.
The authors concluded that patients with TED (thyroid eye disease) of longer disease duration responded similarly to those treated earlier in the disease course. Patients with an insufficient initial response or flare may benefit from additional teprotumumab therapy.

Therefore, as there is a growing body of evidence for the efficacy of retreatment with Tepezza in patients with chronic TED (thyroid eye disease) with low CAS (clinical activity score) as well as those experiencing flare following prior improvement with Tepezza treatment, medical necessity is established in this case.