NICE Asks Biogen to Submit Spinraza for STA on Path Toward Potential NHS Funding

The U.K’s National Institute for Health and Care Excellence (NICE) has formally invited Biogen to submit Spinraza (nusinersen) for Single Technology Appraisal (STA) on its course toward possible NHS funding.

Spinraza is the first approved spinal muscular atrophy (SMA) treatment in the E.U., the United States, and other countries.

NICE and England’s National Health Service (NHS) are also developing a Managed Access Agreement, which reassures Biogen that there is a long-term reimbursement path for Spinraza in England.

Technology appraisals are recommendations based on clinical and economic evidence for the use of new and existing medicines and treatments within the NHS. An STA covers a single technology for a single indication.

Clinical evidence in the STA shows how well the medicine works; economic evidence shows how effectively the treatment represents value for the money, or how well it works in relation to how much it costs the NHS.

Spinraza was reviewed under the European Medicines Agency’s (EMA) accelerated assessment program and approved in June 2017.

However, its availability in the E.U. has varied from country to country depending on national healthcare systems. Biogen has been working with health systems and government agencies to help patients access the medication.

Spinraza is currently reimbursed in Italy and Sweden, with ongoing negotiations taking place in other European countries.

“Whilst we have been disappointed by the delays in the NICE process so far, we have been encouraged by a recent meeting with NICE and NHS England, where the need to respond to specific STA and access challenges in appraising nusinersen was expressly acknowledged, and an immediate plan for a Managed Access Agreement that can work alongside the STA process was agreed,” Terry O’Regan, vice president and managing director of Biogen UK and Ireland, said in a press release.

The Managed Access Agreement is being developed at the same time as the STA process so that urgently needed treatment can be provided to patients in England.

Biogen will submit the STA to NICE in March. The NICE appraisal committee will then outline its initial recommendation in June, after which feedback will be asked of SMA stakeholders such as clinicians and patient representatives.

In November 2018, NICE will provide its recommendation on whether NHS funding can be made available to SMA patients in England. The STA will be considered for all patients who may benefit from Spinraza.

Clinical trials, including ENDEAR (NCT02193074) and CHERISH (NCT02292537), demonstrated the clinically meaningful efficacy and favorable risk-benefit profile of Spinraza.

Since then, Biogen has opened expanded access programs as an interim measure for those with infantile-onset SMA (consistent with type 1). As a result, eligible children in the U.K. have been treated with Spinraza through a collaboration between Biogen and the NHS, with Biogen providing the medicine free of charge. The Biogen expanded access program remains open to all eligible children.

SMA is caused by mutations or loss of the SMN1 gene, leading to a deficit of the SMN protein. Spinraza is an antisense oligonucleotide that leads to an increase in production of full-length SMN protein.