NICE Opposes Adding Spinraza to UK Public Health System; SMA Groups Object

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by Grace Frank |

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The National Institute for Health and Care Excellence (NICE), the U.K. agency that decides on the cost-effectiveness of therapies for inclusion in the public health system of England and Wales, is recommending against Spinraza (nusinersen) being offered to treat people with spinal muscular atrophy (SMA).

This stance, released as “draft guidance” by a NICE committee, was met with disappointment by SMA groups across the U.K., which have supported  Spinraza being opened to all SMA patients at low- or no-cost under the subsidized National Health System.

Some groups, including Treat SMA, reacted with outrage, calling Aug. 14 “a black day for more than a thousand people affected by spinal muscular atrophy in our country” on its website.

Approved for use in patients with SMA types 1–4 across the European Union in June 2017 — and in the U.S. in December 2016 — Spinraza will still be available to U.K. patients with funding arrangements already in place for as long as they last, NICE said. This likely includes those being treated under the current NHS/Biogen Expanded Access Program (EAP), until the patient/family and the NHS treating physician decide to stop, the SMA Trust reported on its website.

NICE noted the considerable benefit of the Biogen‘s therapy even as it rejected covering it.

“Clinical trial evidence shows that nusinersen improves a range of outcomes that are important to patients” with SMA, a rare neurological disease with “an unmet need for effective treatment,” a guidance report by the appraisal committee states. However,  it continues, “there is no long-term evidence, so the long-term benefits are highly uncertain.”

That said, the treatment’s price appears to have been the ultimate decider.

At an estimated £450,000 (about $572,000) for a first year of treatment and £225,000 (about $286,000) for subsequent years, the NICE report states, “the cost of nusinersen is too high for it to be considered a cost-effective use of NHS resources. Because of this, nusinersen is not recommended.”

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Biogen is offering to create a managed access program for U.K. patients wanting Spinraza, NICE noted. But it cautioned that such a program, if established, would require further negotiations between NICE and other stakeholders, like patients, caregivers and physicians, and must include a fresh evaluation of cost effectiveness, or the treatment’s price in light of proven benefits.

Biogen’s proposed arrangements for such a program “were vague and currently insufficient for it [the advisory committee] to assess whether it could be an option for nusinersen.”

Public comment on the decision is being accepted through Sept. 5, and can be submitted directly to NICE using this link. A second appraisal committee meeting is set for Oct. 23, possibly in advance of NICE’s original decision date of Nov. 21, the SMA Trust reported. The group added that it is working with other patient associations on “providing responses.”

Spinraza, delivered as an injection into the spinal cord, acts to restore to working levels the survival motor neuron (SMN) protein that is largely absent or insufficient in SMA patients. This protein is essential for motor neuron health.

Clinical trials, including ENDEAR (NCT02193074) and CHERISH (NCT02292537), demonstrated its effectiveness and safety in patients, including infants with SMA type 1, the disease’s most severe form.