Please find below the official press release from NICE.
NICE has announced that today (3 July 2019) it has published amended draft guidance, after having received a proposal for extending the terms of the managed access agreement (MAA) between NHS England and Biogen for funding Spinraza (also called nusinersen) for treating 5q spinal muscular atrophy. The company made the proposal taking into consideration further clinical evidencei. The MAA has also been refined in light of a number of requests for clarification.
The revised MAA has been approved by the 3 main patient groups (Treat SMA, Muscular Dystrophy UK, Spinal Muscular Atrophy UK), as well as by NICE, NHS England, Biogen and clinicians.
The most significant change to the MAA is that it will now include paediatric patients who have recently (in the previous 12 months) lost the ability to walk independently. This replaces* the previous criteria that patients, who had previously gained ambulation, should still be able to walk independently at the start of their treatment with Spinraza.
Another significant change to the MAA is that expert advice from a multidisciplinary team in the form of an expert clinical panel set up by NHS England on the feasibility of intrathecal administration of Spinraza will be available to the centres providing treatment. This will ensure that access to treatment will not be affected by variations in the ability of clinicians to administer it.
The MAA is a special arrangement between NHS England and Biogen to provide treatment with Spinraza and collect data on the impact of that treatment in groups where additional evidence is required to address the uncertainty. It is also designed to address the financial risk and challenges for implementation in the NHS. It is not routine commissioning of a treatment in line with its marketing authorisation.
Meindert Boysen, director of the Centre for Health Technology Assessment at NICE, said: “Having considered new evidence about the effectiveness of Spinraza in people who have lost the ability to walk, NHS England and Biogen have been able to find a way forward that all parties are happy with and that will ensure Spinraza is available to as many people with SMA as possible.”
“There will still be people with SMA who will not be able to access treatment with Spinraza under the terms of this agreement. But we are not closing the door to these people. Uniquely for this type of arrangement we have made a commitment that, during the 5-year course of the MAA, should evidence become available on the potential benefits of Spinraza for type III SMA patients that are currently not included in the MAA, we will review that evidence to see whether it would support a change in the MAA inclusion criteria.”
NICE will now update its draft guidance to reflect the new MAA criteria and this will be issued to consultees for appeal. If no appeals are received, final guidance is expected to be published at the end of July.
NHS England will provide information about the availability of nusinersen, under the terms of the MAA, to the youngest and most severely affected (SMA type 1) patients.
* To clarify, all patients who were eligible under the old MAA will continue to do so under the new criteria.