Zynteglo Funding Rejection Sets "Dangerous Precedent" For Gene Therapies In UK
Bluebird Bio Rails Against Negative Reimbursement Decision
bluebird is "shocked and disappointed" at the draft negative reimbursement decision on Zynteglo in UK but measures outlined in the NICE methods review could change fortunes of gene and cell therapies if taken forward.
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The UK Thalassaemia Society says that HTA body NICE fails to properly incorporate patient experiences into its decision making and that its patient engagement processes need reform. The marketing of Zynteglo, the gene therapy from bluebird bio for which the group is advocating, has just been temporarily suspended.
Proposals extend the health technology appraisal body's focus beyond final guidance to support for early development and real world performance.
Following a Brexit-induced delay over the summer, England's HTA body NICE has come up with a concrete set of proposals that it says will help it “robustly and efficiently” evaluate innovative technologies such as advanced therapies, histology-independent cancer treatments, and technologies for rare diseases. Industry bodies have welcomed the plans, which have just been put out for consultation.