Dive Brief:

• Substantial price cuts would need to take place for Vertex Pharmaceuticals' cystic fibrosis drugs to become cost effective, according to a new report from the Institute for Clinical and Economic Review, an independent group that evaluates the economics of prescription medications.
• The report determined the list prices of Vertex's Kalydeco, Orkambi and Symdeko would have to come down 71% to 77% to meet common cost-effectiveness thresholds. The list prices of these drugs sit at $311,719, $272,886 and $292,258, respectively.
• ICER noted that decision makers are often more willing to shell out big bucks on treatments for rare diseases like CF. Even accounting for that, the group concluded the price tags on Vertex's drugs don't match up to the quality adjusted life years (QALYs) they provide. "We evaluated thresholds up to $500,000 per QALY and still found that drug prices would need to be reduced by about 40% to be considered cost effective at this threshold."

Dive Insight:

Drug pricing is arguably the most taboo topic in biopharma right now. That's given added weight to analyses like the ones ICER does, in turn creating some animus between the group and the industry. So far, ICER has flagged the costs associated with many promising new therapies, such as CGRP migraine medications, GlaxoSmithKline's asthma drug Nucala (mepolizumab) and Spark Therapeutics' gene therapy Luxturna (voretigene neparvovec).

More treatments are sure to catch criticism too, as ICER plans to expand its investigations through $14 million in funding it received at the end of 2017.

In its latest report, the group took a look at the three treatments on the market that modulate the cystic fibrosis transmembrane conductance regulator (CTFR). The treatments are sold under the brand names Kalydeco (ivacaftor), Orkambi (lumacaftor/ivacaftor) and Symdeko (tezacaftor/ivacaftor).

Kalydeco first gained Food and Drug Administration approval in 2012, and has since racked up several label expansions. It's indicated for CF patients with certain mutations that cause residual CFTR protein function. Orkambi and Symdeko are for patients with two copies of the F508del mutation, though Symdeko is also OK'd for patients who have at least one mutation responsive to it.

In assessing the clinical evidence supporting each drug, ICER determined with high certainty that Kalydeco provides the patients it's approved to treat with a substantial net health benefit. The group was also highly certain about Orkambi, but concluded it provides a small net health benefit. There was moderate certainty that Symdeko provides a small-to-substantial net health benefit.

ICER did acknowledge, however, that more investigations would have to take place to obtain a clearer picture of the health benefits of these treatments.

"CFTR modulator data is unfolding, with the evidence base for some regimens limited to a few published studies. Outcomes of interest, particularly related to weight changes and pulmonary exacerbations, are not consistently reported across studies. Thus, conclusions on individual outcomes are based mostly on one or two trials," the report said.

Vertex fired back at the report in a May 3 letter, calling the ICER review process a "sham" and reliant on "flawed scientific methodology." Additionally, Vertex claimed ICER and others were using the findings to restrict patient access to transformative CF therapies.

"From a scientific standpoint, your analyses utilized a series of arbitrary modeling choices that fail to capture the full benefits of our medicines, thereby intentionally leading to worse outcomes for these transformative medicines. Omission of such evidence for medicines that are the first to treat the underlying cause of CF and fundamentally change the course of disease progression for patients is particularly egregious," the biotech said in the letter.