According to a report from Cystic Fibrosis News Today, Arkansas Medicaid has settled a federal lawsuit brought by patients denied the cystic fibrosis (CF) drug Kalydeco due its high cost. Kalydeco is an orally administered drug engineered and manufactured by Vertex Pharmaceuticals, a drug maker based in Cambridge, Mass.
The drug treats a statistically small subset of CF patients whose disease is caused by a specific mutation in the CF transmembrane conductance regulator (CFTR) gene called G551D.
The Cystic Fibrosis News Today report notes that Kalydeco is an expensive drug, and the main reason is that it is exclusively indicated for the treatment of a very small number of potential end-users. The proportion of CF cases positive for the G551D mutation in the CFTR gene is low, with only a subset of about 3% of CF patients in North America.
The three patients who brought the lawsuit, Catherine Kiger, Elizabeth West and Chloe Jones, filed in 2014 contending that their civil rights had been violated by the state for 2 years in refusing to pay for the drug. Kate Luck, spokesperson for the Department of Human Services, says the settlement included no monetary awards. However, the state has agreed not to alter the eligibility criteria for Kalydeco for 2 years.
The Cystic Fibrosis News Today report notes that if the state denies the drug to a Medicaid patient, it is required to provide justification to the court as to why, though the courts have no jurisdiction over the decision and can only ask Arkansas Medicaid to outline the reasons for denial.
Also, under the new policy, patients will have to show improvement in only one of three previously mandated measures (weight gain, fewer hospitalizations, and better lung function) or provide other evidence of clinical benefit.
According to Cystic Fibrosis News Today, the state no longer requires patients to use standard CF therapy for 12 months before being considered for Kalydeco or to show evidence of failure for the standard therapy. Arkansas has covered the CF drug since 2012 and approved its first patient for coverage in 2013.
Source: Cystic Fibrosis News Today