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Drug price watchdog ICER, the Institute for Clinical and Economic Review, Posted a draft report on organic blue bird‘s betibeglogene autotemcel gene therapy for beta-thalassemia. Despite the proposed price of $2.1 million, the not yet finalized ICER report supports the cost-effectiveness of the therapy. This is good news for the recent besieged the society.

Gene therapies are usually designed to “cure” a disease by replacing or repairing a damaged gene. Bluebird’s therapy, which is listed under the brand name Zynteglo, had been approved in Europe and the UK, where it is priced at around $1.7 million (US). However, the company pulled the therapy from the market in Europe due to what it called a hostile pricing and reimbursement environment.

April 5, bluebird bio announcement it was beginning a comprehensive restructuring in hopes of cutting costs by $160 million over the next two years. He planned to refocus on near-term catalysts, which include Zynteglo in the US, gene therapy for cerebral adrenoleukodystrophy (eli-cel), and a potential biolicense application (BLA) for the gene therapy lovotibeglogene autotemcel ( lovo-cel) for sickle cell disease. cellular disease. The BLA application is scheduled for 2023, while US regulatory decisions are expected this year. The PDUFA date for Zynteglo is August 19, 2022 and September 16, 2022 for eli-cel.

As part of the restructuring, the company is reducing its workforce by around 30%.

ICER’s recommendations are not binding, but they do have an impact. If ICER says a drug is too expensive, it provides ammunition for payers, such as Medicare and insurers, to push back on proposed prices.

Gene therapies are very expensive. For example, Novartis‘ Zolgensma, the unique onasemnogene abeparvovec gene therapy for spinal muscular atrophy (SMA), is generally seen as the most expensive drug with a price tag of $2.1 million. On the other hand, as an apparent “cure” for a disease that kills children at the age of two, it is very rare. The argument in favor of these therapies, besides their curative potential for otherwise incurable diseases, is that they are cost-effective throughout the patient’s lifetime.

Novartis and Therapeutic SparkLuxturna gene therapy (voretigene neparvovec) short approximately $850,000 per patient in the United States. The treatment concerns hereditary retinal dystrophy with RPE65 mutations. It is usually diagnosed in childhood and eventually causes near total blindness, and therapy is essentially a cure.

Beta-thalassemia is a genetic disease that impairs the ability of red blood cells to make hemoglobin, the body’s molecule that carries oxygen. There are approximately 40,000 newly diagnosed cases in children each year worldwide. People with the most severe form develop life-threatening anemia around four to six months of age and must receive monthly blood transfusions and other treatments, such as iron-chelating drugs. The only other potential cure is hematopoietic stem cell transplantation (HSCT), but requires a donor with a matching human leukocyte antigen (HLA) profile in the appropriate age range.

Bluebird’s Zynteglo appears to be another healing option, although the duration of the therapy’s effects remains an open question. The ICER report noted the uncertainties, but concluded that “evidence suggests that beti-cel provides clear health benefits to patients with TDT.”

The ICER report indicated, for Managed Health Frameworkthat “patients could be treated without reaching the potential budget impact threshold at three prices (approximately $1.85 million, $2.11 million, and $2.38 million per treatment cycle). This analysis was performed at multiple prices to document the percentage of patients that could be treated without crossing a potential budget impact threshold aligned with the overall growth of the US economy.

In phase III trials, 89% of patients who received treatment became transfusion independent, and in phase I/II and III trials, these patients remained transfusion free for at least 42 months. In general, side effects were mild and no deaths were reported. In December 2021, the blue bird data presented to American Society of Hematology meeting of a long-term study (LTF-303) which showed that adult and pediatric patients with beta-thalassemia who required regular transfusions of red blood cells can produce normal or near-normal levels of total hemoglobin and remain without transfusion with stable iron markers at seven years after receiving beti-cel.

A 2017 study published in Blood found that, on average, patients with beta-thalassemia required 17 transfusions per year, 23 days apart. The average total health care costs for patients were $128,062, plus or minus $62,260 per year. Total costs were mainly related to chelation and transfusion costs.

Although the severity of the disease varies, a 2009 study found that people with beta-thalassemia major “often die of cardiac complications from iron overload before the age of 30,” making bluebird’s new therapy, if successful, vital for these patients.