The Institute for Clinical and Economic Review (ICER) on Thursday released an Evidence Report assessing the comparative clinical effectiveness and value of cystic fibrosis transmembrane conductance regulator (CFTR) modulators, including combination therapies tezacaftor/ivacaftor (Symdeko, Vertex Pharmaceuticals) and lumacaftor/ivacaftor (Orkambi, Vertex Pharmaceuticals), as well as the monotherapy ivacaftor (Kalydeco, Vertex Pharmaceuticals), for the treatment of cystic fibrosis (CF).
This Evidence Report will be the subject of an upcoming public meeting of the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC) in St. Louis, MO on May 17.
The Midwest CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.
“Our report summarizes the evidence demonstrating that CFTR modulator therapies, paired with best supportive care, provide clinical benefit for patients who have not been treated previously with disease-modifying therapy,” ICER Chief Science Officer Dan Ollendorf said. “The current prices for these drugs, however, produce overall costs that are far in excess of those needed to reach commonly cited cost-effectiveness thresholds. Our analysis suggested that discounts of up to 77 percent would be needed to bring the prices into alignment with their clinical value to patients. Our public meeting will bring stakeholders together to discuss strategies to achieve sustainable patient access to these therapies while supporting the innovation that will bring further progress in treating diseases like CF.”
To read the Evidence Report, click here.