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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.

The Institute just released a 113 page Draft Evidence Report assessing the comparative clinical effectiveness and value of exagamglogene autotemcel (“exa-cel”, Vertex Pharmaceuticals and CRISPR Therapeutics) and lovotibeglogene autotemcel (“lovo-cel”, bluebird bio) for sickle cell disease. This preliminary draft marks the midpoint of ICER’s eight-month process of assessing these treatments, and the findings within this document should not be interpreted to be ICER’s final conclusions.

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.

Sickle cell disease can affect nearly every organ system in the body, and severe sickle cell disease affects nearly every aspect of a person’s life,” said ICER’s Chief Medical Officer, David Rind, MD. “From the earliest days of gene therapy, patients, families, and clinicians have imagined that someday it might be possible to address the underlying genetics of sickle cell to achieve a cure. These first two genetic therapies, using different technologies and altering different genetic targets may mean that day has nearly arrived.”

The prevalence of sickle cell disease is unknown, but CDC estimates place it at about 100,000 cases in the U.S. It is a blood disease that can spur chronic complications among all organs, and annual healthcare costs rack up to $2.98 billion, the ICER said.

The model developed to generate cost-effectiveness findings in ICER’s draft report used cutting-edge evidence from academic researchers and was informed by what matters to patients.  

Through collaborative input from patients, clinicians, health economists, payers, and manufacturers, ICER’s draft model not only includes the projected health benefits and cost offsets from reducing the acute events as measured in the clinical studies but also includes the projected benefits from eliminating the fear of future acute events, reductions in chronic events and mortality, health equity considerations, and reductions in lost productivity and caregiver burden.

Two proposed gene therapies for sickle cell disease (SCD) are each worth up to $1.9 million, according to an April 12 draft evidence report from the Institute for Clinical and Economic Review.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.

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.

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.