Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Thyroid eye disease
Treatment: Tepezza
The insurer denied the Tepezza.
The determination is overturned.

The patient has thyroid eye disease and a second course of Tepezza is the subject of review. Coverage was previously denied per policy which considers continued use of Tepezza beyond an initial full course of Tepezza unproven and not medically necessary. Reportedly, the patient was treated with a first course of Tepezza. Due to manufacturer issues during coronavirus disease (COVID), the patient only received 1 of the 8 planned doses. The remaining doses were scheduled as the supply chain allowed, and she then resumed therapy. The patient is identified has having refractory disease at that time and had not responded well to conventional interventions. The provider cites literature which shows a repeat course can be indicated and helpful in refractory cases. An exam dated  documents vision pinholing to 20/30 right eye (OD) and 20/150 left eye (OS). Exophthalmos with lower periorbital edema and erythema was noted both eyes (OU). Hertels measured 24 millimeters (mm) OD and 26 mm OS. Bilateral upper eyelid retraction was noted with lagophthalmos OS greater than (>) OD. Conjunctival injections was noted OU. It is noted that the patient did previously have significant subjective improvement with regards to dry eyes, injection and diplopia after prior treatment with Tepezza. The patient restarted oral steroids. However, with worsening visual acuity, her prednisone dosage has been increased.

At issue is the medical necessity of the requested Tepezza.

The requested second course of Tepezza is medically necessary for this patient as supported by peer reviewed medical literature.

Douglas et al. evaluated the safety and efficacy of teprotumumab in patients with thyroid eye disease (TED) 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-mm 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, subjective diplopia, and quality-of-life. Thirty-three of 37 placebo-treated OPTIC patients (89.2%) became proptosis responders (mean plus/minus ( ) 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 of 0 or 1, and diplopia responses were maintained in 29 of 32 patients (90.6 percent [%]), 20 of 21 patients (95.2%), and 12 of 14 patients (85.7%), respectively, at follow-up week 48. The median TED duration was 12.9 months versus 6.3 months in those treated with teprotumumab in the OPTIC study. Of the 5 OPTIC teprotumumab nonresponders re-treated in OPTIC-X, 2 responded, 1 showed a proptosis reduction of 1.5 mm from OPTIC baseline, and 2 discontinued treatment early. Of the OPTIC teprotumumab responders who experienced flare, 5 of 8 patients (62.5%) responded when re-treated (mean proptosis reduction, 1.9  1.2 mm from OPTIC-X baseline and 3.3  0.7 mm from OPTIC baseline). Compared with published double-masked trials and their integrated follow-up, no new safety signals were identified. Mild hearing impairment was reported; 4 events occurred during first course of treatment, and 2 events reoccurred after re-treatment. The authors concluded that patients with TED 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 with low CAS as well as those experiencing flare following prior Tepezza treatment, and as this patient is experiencing recurrent symptoms previously improved with Tepezza, even when such treatment was interrupted, the medical necessity of a second course of Tepezza is established.