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Biogen’s Spinraza to go before NICE

Published on 22/01/18 at 11:31am

Biogen received marketing approval from the European Commission for Spinraza (nusinersen), its treatment for spinal muscular atrophy (SMA), in May of last year but has struggled to generate access across Europe.

One step towards improving patients’ access to the treatment was announced with Biogen announcing that NICE had agreed to evaluate the treatment through its Single Technology Appraisal (STA) route.

Biogen’s frustration at the delay in deciding how to determine the value of its treatment was tempered somewhat by NICE’s willingness to begin the development of a Managed Access Agreement immediately. This pathway allows patients to receive treatments whilst data is still being gathered, with the treatments being acquired at a discounted rate prior to any decision on the product.

Terry O'Regan, Vice President and Managing Director of Biogen UK and Ireland, comments, “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. Now is the time for all stakeholders to work together to ensure as many patients as possible get rapid access to this life saving medicine.”

Clinical trials have demonstrated the efficacy of Spinraza, with a 47% reduction in the risk of death or permanent ventilation observed in one trial and improvement to motor function in another. The sticking point, as is often the case for rare disease treatments, is cost.

The list price of Spinraza is an eye-watering £83,500 per dose, with treatment schedules usually involving four ‘loading doses’ over a period of a month and a half. Treatment is then tapered down to maintenance doses that need to be administered every four months for the rest of the patient’s lives.

This goes some way to explaining why only Sweden and Italy have advanced plans to reimburse the product, whilst negotiations continue across other member states.

Biogen plans to submit its STA application in March, before NICE notes its initial recommendation in June. The final decision will be reached in November 2018.

Ben Hargreaves

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