Dear Dr. Joby Varghese:
On behalf of people in New Jersey living with cystic fibrosis, we write to urge Amerigroup to update elexacaftor/tezacaftor/ivacaftor’s (Trikafta®) eligibility criteria to reflect the recent label expansion to people with cystic fibrosis age 2 years and older who have at least one copy of the F508del mutation or another eligible mutation in the CFTR gene per the Food and Drug Administration’s (FDA) label. For these individuals, elexacaftor/tezacaftor/ivacaftor should be available immediately as it targets the underlying cause of cystic fibrosis and may provide previously inconceivable improvements in their health. While we appreciate Amerigroup’s case-by-case approval of elexacaftor/tezacaftor/ivacaftor for eligible people with CF in this age group in the interim prior, it is important to update the formulary criteria as soon as possible so all eligible individuals can access therapy without delay.
About Cystic Fibrosis and the Cystic Fibrosis Foundation
Cystic fibrosis is a life-threatening genetic disease that affects nearly 40,000 children and adults in the United States, including approximately 750 people in New Jersey of which around 180 depend on Medicaid for some or all of their coverage. CF causes the body to produce thick, sticky mucus that clogs the lungs and digestive system, which can lead to life-threatening infections. Cystic fibrosis is both serious and progressive; lung damage caused by infection is irreversible and can have a lasting impact on length and quality of life. There is no cure for CF today. As a complex, multi-system condition, CF requires targeted, specialized treatment and medications.
As the world’s leader in the search for a cure for CF and an organization dedicated to ensuring access to high quality, specialized CF care, the Cystic Fibrosis Foundation supports the development of CF clinical practice guidelines and accredits 130 care centers and 55 affiliate programs nationally — including 8 CF programs in New Jersey.
About Elexacaftor/Tezacaftor/Ivacaftor
For eligible patients, elexacaftor/tezacaftor/ivacaftor represents the most transformative therapeutic advancement in CF. This combination therapy corrects the CFTR protein malfunction in individuals with at least one copy of the F508del mutation or other eligible mutation in the CFTR gene; thereby restoring function to the protein.
Elexacaftor/tezacaftor/ivacaftor presents an opportunity to preserve health and lung function, slow the progression of the disease, and keep patients from experiencing costly hospitalizations, declining health status, deteriorating quality of life, and ultimately premature death. The impact of this therapy may differ between people with CF who initiate treatment at an early age and those who begin the treatment later, after already experiencing irreversible lung damage. Phase three clinical trial data demonstrates that elexacaftor/tezacaftor/ivacaftor was generally well tolerated amongst those aged two to less than six and the safety data were consistent with those observed in previous studies. Elexacaftor/tezacaftor/ivacaftor cannot reverse damage that has already occurred and should therefore be initiated as soon as patients and their physicians determine it is medically necessary and appropriate to begin therapy.
Policy Recommendations
Recognizing Amerigroup’s case-by-case approval of elexacaftor/tezacaftor/ivacaftor for people with CF aged 2-5, the CF Foundation urges Amerigroup to update their eligibility criteria to make this transformative therapy available to all eligible CF patients in accordance with the FDA label.
We stand ready to answer any questions about elexacaftor/tezacaftor/ivacaftor or other CF treatments.