Sickle cell disease gene therapies from Vertex, bluebird can be cost-effective at $1.9M: ICER

The world’s first CRISPR-based gene-editing therapy appears to be nearing the market. And an influential drug cost watchdog has an early idea of how the treatment should be priced to be considered cost-effective in sickle cell disease (SCD).

Vertex and CRISPR Therapeutics’ gene editing-based exa-cel—and bluebird bio’s gene replacement therapy lovo-cel—can be priced up to $1.93 million to be cost-effective, the Institute for Clinical and Economic Review said in a draft report (PDF) published Wednesday. The figure accounts for the drugs’ net prices after discounts and rebates.

The report comes shortly after Vertex and CRISPR last week said they had completed their rolling FDA applications for exa-cel in SCD and beta thalassemia with a request for priority review. If approved, exa-cel would become the first therapy based on the Nobel-winning CRISPR technology.

It’s not immediately clear how bluebird or Vertex-CRISPR will price their therapies. A Vertex spokesperson said the company is still reviewing the report. In a separate statement, a bluebird spokesperson said the company hasn’t set a price for lovo-cel and is currently focused on completing its FDA application.

During an interview with Fierce Pharma, ICER’s chief medical officer, David Rind, M.D., stressed that the cost-effectiveness analyses are preliminary and may change. ICER is gathering public comments until May 9 and pushed back its final report publish date to July 13 in anticipation of more data on exa-cel.

The novelty of the CRISPR technology doesn’t factor into ICER’s cost assessment other than to add some uncertainties about safety, Rind said. But “that is not in any way to detract from how incredibly exciting it is to have a CRISPR therapy and a more traditional gene therapy for this genetic disease that we’ve all been wanting to cure forever,” he said.

In ICER’s draft report, the organization said exa-cel and lovo-cel should be treated similarly because both have very limited clinical data. But it also suggested that future research may identify differences between the two therapies.

In lovo-cel’s registrational trial, 90% of 31 participants were free of any vaso-occlusive events between six months and 18 months after a one-time infusion, and 30 patients didn’t experience severe crises. In exa-cel’s case, only seven participants had 12 months of follow-up, and all of them were free of severe vaso-occlusive events, ICER noted.

ICER put exa-cel in a lower confidence rating in its comparison with standard of care because of a smaller dataset and the limited experience with the CRISPR technology.

In a Thursday note, analysts at Evercore ISI said that exa-cel’s data pool is larger now. The team estimated that Vertex and CRISPR had 23 to 31 patients with 12 months of data when they completed the FDA submission.

Given that both gene therapies offer the promise of a potential cure, ICER compared them with standard of care over a lifetime.

The health economics reviewers figured the two therapies could cost between $1.58 million to $1.72 million under commonly used cost-effectiveness thresholds that only look at benefits within the healthcare system.

The range goes up to between $1.79 million to $1.93 million when considering broader societal value.

ICER is only offering an economic analysis to help people make their own decisions about value, Rind said. Pointing to how SCD disproportionally affects Black people, Rind said that it’s possible that some of ICER’s voting experts might suggest a therapy has good value under a higher range of cost-effectiveness because of historical discrimination and inequities in the U.S.

Under an even higher possible threshold, the drugs could be fairly priced at up to $2.32 million, according to ICER’s calculations.

Gene therapies typically cost multiple millions of dollars per treatment. Bluebird recently launched Zynteglo, a sister med to lovo-cel, at $2.8 million. Zynteglo is approved for beta thalassemia, a rare blood disorder that affects about 1 in 100,000 individuals.

Bluebird makes pricing decisions for its gene therapies “based on measures of positive clinical outcomes, quality-of-life improvements, and the potential societal impact of patients and families living their lives more fully, engaging in meaningful work, and using the health care system less,” the company spokesperson said.

SCD is more prevalent, affecting about 100,000 Americans, or 1 out of about 400 Black people at birth. By Vertex’s esimtate, treatment costs for severe SCD patients can reach around $4 million to $6 million over a lifetime.

Editor's Note: The story has been updated with additional comments from a bluebird spokesperson.