More people to be eligible for nusinersen following review of Managed Access Agreement
The National Institute for Health and Care Excellence (NICE) announced on the 4 May 2021 that more people the rare genetic disorder spinal muscular atrophy (SMA) will be able to benefit from nusinersen (also called Spinraqza, made by Biogen) following a review of collected data as part of the Managed Access Agreement (MAA). This review involved Biogen, patient groups, clinicians, SMA REACH UK and NHS England and NHS Improvement assessing whether new evidence that had become available would support a change in the MAA treatment eligibility criteria. It specifically looked at whether people with type III SMA who are unable to walk could benefit from nusinsersen and therefore should be included in the MAA.
The MAA is a special arrangement between NHS England and NHS Improvement and Biogen. It allows patients to access treatment with nusinersen while at the same time collecting data on its impact in groups where additional evidence is required to address the uncertainties identified by the independent NICE appraisal committee in its original assessment of nusinersen. On top of this, the MAA also there to address the financial risk and challenges for implementation in the NHS.
The deputy chief executive and director of the Centre for Health technology Assessment at NICE, Meindert Boysen, said: ‘There are people with SMA who are not able to access treatment with nusinersen under the terms of the MAA, which began in July 2019. At the time we made a commitment that we would review new evidence on the potential benefits of nusinersen for type III SMA patients who are not currently receiving it. We are therefore pleased that the review has concluded that it is appropriate to extend the clinical eligibility criteria to allow access to nusinersen for type III SMA patients who aren't able to walk. It will also allow the removal of the rule which meant that patients who had lost the ability to walk needed to regain that ability within 12 months of treatment in order to be eligible for further treatment’.
Thousands of patients now to be eligible for bempedoic acid with ezetimibe following final NICE guidance
Around 70 000 adults in England who have primary hypercholesterolaemia or mixed dyslipidaemia, also known as high cholesterol, will now be eligible for treatment with bempedoic acid with ezetimibe, if statins cannot be taken, or have not worked for them.
The current standard treatment for those with high cholesterol included dietary changes and statins for lowering low-density lipoprotein cholesterol (LDL-C) levels. Patients could also be treated with ezetimibe and either alirocumab or evolocumab when their cholesterol levels are not lowered enough with the maximally tolerated dose of statins.
New NICE guidance means bempedoic acid with ezetimibe will be offered as an option for people who are unable to use statins and whose high cholesterol is not well-controlled with ezetimibe alone.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said: ‘high cholesterol, if left untreated, can lead to a range of serious health conditions. Although statins and other treatments are used successfully by a large portion of the population, some people may require other options to control their cholesterol. We are pleased to be able to recommend bempedoic acid with ezetimibe as a new treatment option for these individuals.
Bempedoic acid with ezetimibe are both taken once daily in tablet form. They can be used as separate tablets (Nilemdo by Daiichi Sankyo, plus ezetimibe) or in a fixed-dose combination (Nustendi by Daiichi Sankyo).