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<p>The National Institute for Health and Care Excellence (NICE) is currently developing
recommendations for the National Health Service on the use of Spinraza for the treatment
of spinal muscular atrophy (SMA) through its technology appraisal programme. NICE
recommends technologies for use in the NHS when the evidence demonstrates that they
are clinically effective and represent a cost-effective use of NHS resources.</p><p>There
are several subtypes of SMA and, in the case of Spinraza, NICE has advised that it
was not presented with evidence in SMA type 0 and SMA type 4. Evidence presented for
the SMA type 3 population, who had lost the ability to walk, was limited and immature
and resulted in its independent Appraisal Committee concluding that the size and nature
of the benefits of Spinraza for this population were uncertain. Therefore, they were
not included in the managed access arrangement (MAA) agreed by NHS England and Biogen.</p><p>Following
publication of the initial MAA, the company presented further clinical evidence and
the MAA was extended to include paediatric patients, who have recently (in the previous
12 months) lost the ability to walk independently.</p><p>Uniquely for this type of
arrangement, during the five-year course of the MAA, should evidence become available
on the potential benefits of Spinraza for type 3 SMA patients that are currently not
included in the MAA, NICE will review that evidence to see whether it would support
a change in the MAA inclusion criteria.</p><p> </p>
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