After considering the feedback from consultation, the Appraisal Committee has prepared a Final Appraisal Document (FAD) on risdiplam for treating spinal muscular atrophy in children and adults and submitted it to NICE.
The FAD has been sent to consultees for this appraisal who have 15 working days to consider whether they wish to appeal against it and/or notify us of any factual errors. The FAD has been sent to commentators for information, who can consider notifying us of any factual errors only. Subject to any appeal by consultees, the FAD may be used as the basis for NICE's guidance on the use of the appraised technology in the NHS in England.
Please note that the appeal period for this appraisal will close at 5pm, Friday 2 December 2021.
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Final appraisal document Managed Access Agreement (PDF 270 KB)
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Final appraisal document Data Collection Arrangement (PDF 246 KB)
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Final appraisal document Appendix A - Using other treatments after onasemnogene (PDF 129 KB)
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Final appraisal document Appendix B - Managed Access Patient Agreement form (PDF 110 KB)
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