UK Rare Disease Drug Review Reform Urged As Spinraza Finally Gets NICE OK

The National Institute for Health and Care Excellence's decision to give the go-ahead at last to Biogen's spinal muscular atrophy (SMA) therapy Spinraza has been met with a mixture of joy and relief by patient groups in the UK who are calling for regulators to change the way rare diseases are evaluated by the cost watchdog.

NICE has recommended funding on the National Health Service for Spinraza (nusinersen) for the treatment of infants, children and adults with 5q SMA