Discussion
Despite the potential benefits of initiating HEMT at an earlier age, obtaining insurance coverage for “off label” use of a medication can be difficult. Based on these results, we found a relatively high overall approval rate for early coverage of ELX/TEZ/IVA for CF in children 6 to 11 years of age. Early insurance coverage was achieved regardless of insurance type.
Most cases required at least one appeal to obtain approval. Additionally, many care centers were required to submit supporting patient data and evidence of clinical need. The increased workload required for obtaining approval may be reduced through collaboration between CF care centers on successful appeal letters.
While ELX/TEZ/IVA was approved by the FDA in June 2021 for children 6 to 11 years of age, the results of this study may still be applicable for patients looking for earlier initiation of ELX/TEZ/IVA or patients seeking off label use based on their genetic mutation.
One limitation of this study was that not all CFF-accredited care centers provided information. Although this study found a high overall approval rate regardless of payer type, certain CF care centers may find more difficulty depending on the predominant insurance providers in their geographical region.
References
  1. Zemanick ET, et al. A Phase 3 Open-Label Study of ELX/TEZ/IVA in Children 6 Through 11 Years of Age With CF and at Least One F508del Allele. Am J Respir Crit Care Med 2021. PMID: 33734030
  2. Nichols AL, Davies JC, Jones D, Carr SB. Restoration of exocrine pancreatic function in older children with cystic fibrosis on ivacaftor. Paediatr Respir Rev. 2020 Sep;35:99-102.
  3. Munce D, Lim M, Akong K. Persistent recovery of pancreatic function in patients with cystic fibrosis after ivacaftor. Pediatr Pulmonol. 2020 Dec;55(12):3381-3383.
  4. Aoyama BC, Mogayzel PJ. Ivacaftor for the treatment of cystic fibrosis in children under six years of age. Expert Rev Respir Med. 2020 Jun;14(6):547-557.