He visto en Fiercepharma que dice: Amid Zolgensma crisis, Novartis gets a gene therapy boost as NICE embraces Luxturna . En la web de NICE: NICE recommends novel gene therapy treatment for rare inherited eye disorder y dice entre otras cosas: " The list price of voretigene neparvovec is £613,410 per patient. This technology breached the Budget Impact Test, meaning it would have cost the NHS more than £20 million in one of the first three years after being introduced. However, the company has a commercial arrangement with NHS England making the treatment available to the NHS at a discount, the size of which is commercial in confidence." Por lo que no se publicita el precio The draft guidance is now with consultees, including the company, healthcare professionals and patient/carer organisations who have the opportunity to appeal against the draft recommendations. If no appeals are received NICE expects to publish final guidance next month.
Blog de Medicamentos de terapias avanzadas (MTA): de medicamentos de terapias génicas y de medicamentos de terapias celulares somáticas autorizadas por la EMA y la FDA, principalmente.