Appeal type: Prescription Drug
Decision: Favorable

You asked the Plan to cover Ruconest 2100 unit vial. The Plan did not grant this request. We received this case to conduct an independent review.We asked our Physician Reviewer to determine whether the Part D Plan was correct in denying the request for Ruconest 2100 unit vial. Ruconest is requested for ongoing treatment of acute attacks of hereditary angioedema. The prescriber has discussed the enrollee's clinical history/evaluation and has described the benefit of the requested drug in treating the enrollee's frequent episodic abdominal and facial swelling related to the noted diagnosis. The Plan denied coverage of the requested medication because the enrollee has normal C1 inhibitor levels. For coverage under the prescription drug benefit, Medicare requires that the drug prescribed be used for a medically accepted indication.Our Physician Reviewer found that the Medicare-approved resources do contain citations to support the use of the requested drug for treatment of the noted condition. Although the majority of cases of hereditary angioedema are associated with low C1 inhibitor levels or low C1 esterase inhibitor functional levels, there are rarer forms of the condition where there is no evidence of deficiency or dysfunction of C1 inhibitor. In the places in therapy section for the requested drug, it is noted to See Drug Consult reference: Hereditary Angioedema - Pharmacologic Prophylaxis and Treatment (Adults and Pediatric) with a link to the notedreference. The introductory paragraph of the noted monograph states: Hereditary angioedema (HEA) is a autosomal dominant disease associated with episodic unpredictable swelling, typically affecting the extremities, bowel mucosa, genitals, face, and upper airway. The swelling attacks do not respond to epinephrine, glucocorticoids, or antihistamines. There are 3 types of HEA: Type 1 has low levels of C1 inhibitors; Type 2 has normal levels of C1 inhibitors but with impaired C1 inhibitor function; the third type of HAE is very rare and has normal levels and function of C1 inhibitors. Other medical resources are consistent regarding the presence of the noted diagnosis with no evidence of deficiency/dysfunction of C1 inhibitor. The documentation supports the stated diagnosis, and the requested drug is indicated to treat the stated diagnosis. Therefore, the requested drug is being prescribed for a medically accepted indication.The enrollee does not satisfy the Plan's coverage criteria. However, the prescriber has indicated why all alternatives required by the Plan would likely not be as effective as the requested drug for the enrollee, and/or would likely cause adverse reaction(s) or other harm to the enrollee. Therefore, an exception to the Plan's coverage criteria is medically necessary. Our Physician Reviewer's opinion is that prior authorization exception criteria are met.From Up to Date - to lend support that HAE can exist with normal C1 esterase levels and function. The citation is not listed to support MAI, just the diagnosis. The best characterized forms of HAE arise from deficiency or dysfunction of C1 inhibitor (C1INH). However, there are other forms of HAE in which C1INH is normal. A small but growing number of suspected pathogenic variants in genes for other proteins have been identified to explain the disease in some families, while the pathogenesis in other families remains unclear.Medicare rules allow a Part D Plan to manage its formulary by requiring prior approval for certain drugs. A Plan must grant an exception to prior approval rules if it determines that the requested drug is medically necessary, consistent with the prescriber's statement. Based on the available documentation, our Physician Reviewer's opinion is that an exception to prior approval criteria is met. Therefore, our decision is the Plan is required to cover Ruconest 2100 unit vial.Please note that if the Plan does not agree with our decision, they can ask us to open a case again. We only open a case again if we believe there was a mistake or if there is new information to review. The health plan has to show us the mistake and/or send us the new information. This does not happen often. If we decide to open the case again, we will notify you in writing that the case is being reviewed again and the outcome of the reopening.