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
- 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
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