Appeal type: Medical necessity
Decision: Overturned

Diagnosis: Idiopathic thrombocytopenia purpura. 
Treatment: Doptelet 20 milligrams (mg) OR (oral) tabs. 

The insurer denied coverage for Doptelet 20 milligrams (mg) OR tabs. 

The denial is overturned. 

A request for Doptelet was denied. The request was denied as the use of Doptelet in patients under the age of 18 was not approved by the United States Food and Drug Administration (FDA) product labeling. 

The provider requested reconsideration for the patient's Doptelet. The patient had been diagnosed with idiopathic thrombocytopenia purpura (ITP) which continued to be highly refractory. It had not been responsive to IVIG (intravenous immunoglobulin), WhinRho, mycophenolate mofetil, Sirolimus, Promacta, steroids, and Nplate. They had decided against Rituximab due to underlying immune deficiency. The patient's activities of daily living and quality of life had continued to be suboptimal due to her refractory condition. The provider recommended daily Doptelet. The provider submitted articles and support of the requested medication as a second line treatment. The patient weight was equivalent to a young adult weight. The most recent platelet count was 1000. The provider recommended initiation of Doptelet (avatrombopag) 20 mg daily. 

Doptelet is medically necessary for this patient for treatment of chronic idiopathic thrombocytopenia purpura. 

The Food and Drug Administration (FDA) states that Doptelet is indicated for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia who have had insufficient response to previous treatment. It is unknown if Doptelet is safe and effective in children. 

Per the cited literature, while many children with idiopathic thrombocytopenia can be safely observed, treatments are often needed for various reasons, including to decrease bleeding or to improve health-related quality of life. Management of the condition continues to evolve. Avatrombopag is attractive as a potential future option in children because it is oral and does not have the same dietary restrictions that accompany eltrombopag. Pediatric trials have begun. Studies have noted the efficacy and safety of other thrombopoietin receptor agonists in children with chronic immune thrombocytopenic purpura, specifically romiplostim and eltrombopag. 

In this case, the patient suffered from refractory idiopathic thrombocytopenia purpura. She had been treated extensively and continued to be symptomatic. The provider recommended Doptelet. The request does not meet the Food and Drug Administration guidelines as the guidelines indicate that it is unknown if the treatment is safe and effective in children. However, clinical studies are often lacking in pediatric patients, especially in conditions that are less common such as chronic idiopathic thrombocytopenia purpura. Thus, provider's must take into consideration not only clinical studies but expert opinions, the patient's unique clinical status, and trending treatment options that would best suit their patients. The patient is suffering from idiopathic thrombocytopenia purpura and has exhausted standard treatments. Literature notes that thrombopoietin receptor agonists have been found safe and effective for children for treatment of thrombocytopenia purpura. Given the paucity of treatments available to the patient, it is reasonable to consider a Doptelet trial. Lack of attempts at treatments will result in decompensation and is detrimental to the patient's health. Therefore, the Doptelet is medically necessary for the patient. As such, the denial of Doptelet is overturned. 

Based on the above, the insurer's denial must be overturned. The health care plan did not act reasonably and with sound medical judgment and in the best interest of the patient. The medical necessity for Doptelet 20 milligrams (mg) OR tabs services is substantiated.