Seeking views on SMA treatment options

Published Date
Clare Lucas

NICE has started its appraisal of the new drug treatment onasemnogene abeparvovec (also known as Zolgensma) for infants who have SMA Type 1.

The treatment is being looked at under the highly specialized technology (HST) route and we have been asked to make a submission as part of the appraisal process.

As part of the appraisal NICE wants to know what people think about current treatments and care. As such, SMA UK have set up a survey to gather the thoughts of the SMA community on current treatments. You can take the survey here. 

To help people completing the survey we have worked with SMA UK to summarise the relevant information about Zolgensma and Spinraza. People can refer to this whilst completing the survey. You can view the table here.

Although the survey asks only about access for infants who have SMA Type 1, the views of the wider SMA Community are welcomed.

SMA UK will collate all the survey responses, summarise what they have been told and share the results with us and TreatSMA. The anonymised statistical results of the survey will then be used to inform our Patient Group submissions to NICE.

The survey should take around 20 minutes to complete, depending on how much detail you wish to include in your comments. You will need to complete it in one go as the survey doesn’t allow for breaks. 

More than one person from any family is welcome to submit their views – please would each person complete a separate survey.

The closing date for this survey is 11pm Sunday 21 July 2019.

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