BOSTON – May 3, 2018 – The Institute for Clinical and Economic Review (ICER) today 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, 2018. 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,” noted Dan Ollendorf, PhD, ICER’s Chief Scientific Officer. “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% 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.”
A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report reflects changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.
Key Report Findings
ICER evaluated the therapies in three distinct populations, based on a number of different types of genetic mutations involved in CF. The indicated therapies were compared to best supportive care in each of the key populations. For individuals with gating and residual function mutations, evidence on ivacaftor provides high certainty of a substantial net health benefit. For patients homozygous for the F508del mutation, there is high certainty that lumacaftor/ivacaftor provides a small net health benefit, and moderate certainty that tezacaftor/ivacaftor provides a small-to-substantial net health benefit. For patients heterozygous for the F508del mutation with an additional approved residual function mutation, there is moderate certainty that tezacaftor/ivacaftor provides a small-to-substantial net health benefit.
ICER’s economic analyses found that, in all populations considered, the cost of the drugs far exceeded commonly accepted thresholds for cost-effectiveness of $100,000-$150,000 per quality-adjusted life year (QALY) gained. To align the costs of the drugs with the clinical benefit provided to patients, net prices would need to be reduced by 71%-77%.
ICER’s report notes that decision-makers often give special considerations to therapies for ultra-rare diseases such as CF that may lead to coverage and funding decisions at higher thresholds for cost-effectiveness.
During the May public meeting, pre-registered stakeholders will provide brief oral comments on the report and its findings, the Midwest CEPAC will vote on key questions raised in the report, and a roundtable of experts will discuss recommendations for applying the evidence to policy and practice. Requests to make an oral comment were accepted during the public comment period on the Draft Evidence Report and are now closed.
The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.