Costs for approved cystic fibrosis (CF) treatments and new approvals — including therapies extended to more age groups or targeting new mutations — could double in price, according to pharmacy benefit manager (PBM) Prime Therapeutics. The cost increases could result in higher premiums for Prime members.
According to the company, the forecast comes as a result of the advancements in the development of so-called cystic fibrosis transmembrane conductance regulator (CFTR) modulator treatments.
The U.S. Food and Drug Administration (FDA) has approved three CFTR modulator treatments that “correct” the functions of the CFTR protein, which is defective in CF patients. The approved therapies — Kalydeco (ivacaftor), Orkambi lumacaftor/ivacaftor), and Symdeko (tezacaftor/ivacaftor, all developed by Vertex Pharmaceuticals — treat about 60 percent of CF mutations at an annual average cost of $300,000 per Prime member.
Also, the company expects further advancements by the end of 2019, including broadening CFTR modulator usage, and the approval of a combination-treatment with three CFTR modulator therapies.
An analysis performed by Prime researchers showed that of the company’s 15 million members, 1,288 had CF and were eligible to receive a CFTR modulator therapy. However, only about half (54%) of those members have medical claims for a modulator. The limited number of claims at this point suggests the use of modulators will increase.
Previous increases in modulator usage climbed from $0.08 per member, per month in January 2015 to $0.96 in November 2018.
Based on these numbers, researchers estimated that the monthly member-cost could reach $1.21 by December 2019. According to their calculations, if the usage of CFTR modulators increases to 100 percent among eligible members, the cost could rise to $2.07 by the end of this year.
“With the use of cystic fibrosis treatments continuing to rise and new products coming in the class, it is becoming increasingly more critical to evaluate if they are priced appropriately according to the value they provide,” Catherine Starner, PharmD, said in a press release. Starner is senior principal health outcomes consultant at Prime Therapeutics.
“Plan sponsors should use this research to assess if providing clinical programs to closely manage appropriate use can help and whether negotiating value-based contracts with pharmaceutical manufacturers can help recoup costs if the treatments don’t improve health outcomes,” Starner added.
Researchers involved in the analysis were schedule to present the results at the Academy of Managed Care Pharmacy’s (AMCP) Managed Care & Specialty Pharmacy 31st Annual Meeting in San Diego in March 25-28.