NICE Reviews UK Spinraza Reimbursement for SMA Type 3 Patients Unable to Walk

England’s National Institute for Health and Care Excellence (NICE) has begun a review process to decide whether to include spinal muscular atrophy (SMA) type 3 patients who are unable to walk unaided in the country’s conditional reimbursement program for Spinraza (nusinersen).

This conditional reimbursement program is part of a managed access agreement (MAA) between NICE, the National Health Service (NHS) — the country’s publicly funded healthcare system — and Biogen, the therapy’s manufacturer.

Under the MAA, which began in July 2019, NHS will fund Spinraza to eligible patients for five years, while further data is collected to help address any clinical uncertainties identified during NICE’s original evaluation of Spinraza’s potential public funding.

Notably, Spinraza’s eligibility criteria in the final MAA included essentially all SMA patients, except those with type 3 disease who have lost their ability to walk. This presumably was due to a lack of strong evidence of the therapy’s benefits in these patients. Importantly, pediatric — but not adult — patients with SMA type 3 who lost their ability to walk in the previous 12 months are currently considered eligible for Spinraza’s reimbursement.

Now, Biogen, SMA U.K., and other patient groups and clinicians will have the chance to submit additional national and international evidence on Spinraza’s effectiveness that will help NICE determine if these SMA type 3 patients might benefit from the therapy and whether they should be included in the MAA treatment eligibility criteria. NICE’s final decision is expected in six months.

“There are people with SMA who are not able to access treatment with [Spinraza] under the terms of the MAA which began in July 2019,” Meindert Boysen, deputy chief executive and director of NICE’s Centre for Health Technology Assessment, said in a press release.

“At the time we said that we would not close the door to these people,” Boysen said, noting that “uniquely for this type of arrangement we made a commitment that we would review new evidence on the potential benefits of [Spinraza] for type [3] SMA patients who are not currently receiving it.”

“If that review shows those patients would benefit we will recommend they are included in the MAA to receive [Spinraza],” Boysen said.

Importantly, during the review process, a particular treatment-stopping criteria for children with type 3 disease will be suspended. Per the current MAA, type 3 children who lost their ability to walk unaided in the 12 months prior to the agreement’s implementation — on July 28, 2019 — and who could not regain that function after 12 months of treatment with Spinraza would cease to be eligible for reimbursement.

However, the NHS England and NHS Improvement agreed to suspend this criterion until NICE reaches a decision on the evidence review, which will allow these children to continue Spinraza treatment.

In 2017, Spinraza became the the first disease-modifying therapy approved for SMA in the European Union. However, its public reimbursement in the U.K. started only in July 2019, with the implementation of the MAA. NICE had initially recommended against the therapy’s reimbursement in 2018, due to concerns over Spinraza’s long-term effectiveness and high cost. At that time, based on its list price, NICE had said the treatment’s annual cost-effectiveness ranged between £400,000 and £600,000 ($508,000 to $763,000).

Toward the end of the MAA period, which is set for July 2024, NICE will review clinical and cost-effectiveness data collected and submitted through the MAA to make a final decision on whether Spinraza should continue to be funded on the NHS after MAA expiration.

According to a press release, SMA U.K. will start contacting people who have SMA type 3 and have lost their walking ability to find out if they would wish to start Spinraza, should they become eligible. This aims to assist NHS England and neuromuscular treatment centres with their planning in case NICE issues a positive recommendation.

“We are honouring our commitment to conduct a review and are engaging with the company and other stakeholders to ensure that every piece of relevant evidence can be considered as part of that process,” Boysen said.