GlaxoSmithKline’s gene therapy for the so-called “bubble boy” disease was approved by Britain’s healthcare cost watchdog NICE, despite a price tag of almost 600,000 euros ($700,000).
(Reuters) – GlaxoSmithKline&rsquo-s gene therapy for the so-called &ldquo-bubble boy&rdquo- disease was approved by Britain&rsquo-s healthcare cost watchdog NICE, despite a price tag of almost 600,000 euros ($700,000).
Gene therapy is designed to deliver a one-off cure for the patient and drugmakers are typically asking a hefty price that is comparable to the combined costs of alternative life-long treatment.
Britain&rsquo-s National Institute for Health and Care Excellence (NICE) said in draft guidance published on Friday that Strimvelis gene therapy used against adenosine deaminase deficiency, or ADA-SCID, improves overall survival compared with standard stem cell transplant therapy.
The inherited condition disables the immune system and without treatment, children with ADA-SCID need to be kept in isolation to avoid infections – hence it has become known as the &ldquo-bubble baby&rdquo- or &ldquo-bubble boy&rdquo- syndrome.
&ldquo-Strimvelis represents an important development in the treatment of ADA-SCID, offering the potential to cure the immune aspects of the condition and avoid some of the disadvantages of current treatments,&rdquo- NICE said.
&ldquo-Costing 594,000 euros, the treatment is usually given once only and the effects are thought to be life-long,&rdquo- it added.
The draft guidance marks the first time NICE has applied its new cost effectiveness limits for treatments for very rare conditions.
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