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Committee Papers PDF 5.4 MB NATIONAL INSTITUTE FOR HEALTH AND CARE EXCELLENCE SINGLE TECHNOLOGY APPRAISAL Nusinersen for treating spinal muscular atrophy [ID1069] The following documents are made available to the consultees and commentators: 1. Response to consultee, commentator and public comments on the Appraisal Consultation Document (ACD) – (to follow) 2. Additional question circulated to consultees and commentators post ACD release 3. Consultee and commentator comments on the ACD and additional question circulated post ACD release from: Biogen Muscular Dystrophy UK Spinal Muscular Atrophy Support UK & SMA Trust (joint response) TreatSMA British Paediatric Neurology Association SMA Reach A “no comments” response was received from the Department of health and Social Care 4. Comments on the ACD and additional question circulated post ACD release from experts: Dr Adnan Manzur, Consultant Neuromuscular Neurologist – Clinical Expert, nominated by Biogen and SMA Reach Elizabeth Lockley, Patient Expert – nominated by SMA Trust 5. Comments on the Appraisal Consultation Document received through the NICE website 6. Biogen Appendix 1: Additional Clinical Data 7. Biogen Appendix 2: Cost-effectiveness model revisions and updated results 8. Evidence Review Group (ERG) commentary on company’s ACD response – Addendum 9. Company additional evidence from Biogen © National Institute for Health and Care Excellence 2019. All rights reserved. See Notice of Rights. The content in this publication is owned by multiple parties and may not be re-used without the permission of the relevant copyright owner. 10. NICE clarification questions to the company on their additional evidence ERG request to the company on sub-models Company response to ERG request on sub-models ERG additional clarification questions Company response to ERG additional clarification questions 11. Evidence Review Group critique company additional evidence Any information supplied to NICE which has been marked as confidential, has been redacted. All personal information has also been redacted. © National Institute for Health and Care Excellence 2019. All rights reserved. See Notice of Rights. The content in this publication is owned by multiple parties and may not be re-used without the permission of the relevant copyright owner. Nusinersen for treating spinal muscular atrophy Single/Multiple Technology Appraisal Response to consultee, commentator and public comments on the Appraisal Consultation Document (ACD) Type of stakeholder: Consultees – Organisations that accept an invitation to participate in the appraisal including the companies, national professional organisations, national patient organisations, the Department of Health and the Welsh Government and relevant NHS organisations in England. Consultees can make a submission and participate in the consultation on the appraisal consultation document (ACD; if produced). All non-company consultees can nominate clinical experts and/or patient experts to verbally present their personal views to the Appraisal Committee. Company consultees can also nominate clinical experts. Representatives from NHS England and clinical commissioning groups invited to participate in the appraisal may also attend the Appraisal Committee as NHS commissioning experts. All consultees have the opportunity to consider an appeal against the final recommendations, or report any factual errors, within the final appraisal determination (FAD). Clinical and patient experts and NHS commissioning experts – The Chair of the Appraisal Committee and the NICE project team select clinical experts and patient experts from nominations by consultees and commentators. They attend the Appraisal Committee meeting as individuals to answer questions to help clarify issues about the submitted evidence and to provide their views and experiences of the technology and/or condition. Before they attend the meeting, all experts must either submit a written statement (using a template) or indicate they agree with the submission made by their nominating organisation. Commentators – Commentators can participate in the consultation on the ACD (if produced), but NICE does not ask them to make any submission for the appraisal. Non-company commentator organisations can nominate clinical experts and patient experts to verbally present their personal views to the Appraisal Committee. Commentator organisations representing relevant comparator technology companies can also nominate clinical experts. These organisations receive the FAD and have opportunity to report any factual errors. These organisations include comparator technology companies, Healthcare Improvement Scotland any relevant National Collaborating Centre (a group commissioned by NICE to develop clinical guidelines), other related research groups where appropriate (for example, the Medical Research Council and National Cancer Research Institute); other groups such as the NHS Confederation, the NHS Commercial Medicines Unit, the Scottish Medicines Consortium, the Medicines and Healthcare Products Regulatory Agency, the Department of Health, Social Services and Public Safety for Northern Ireland). Public – Members of the public have the opportunity to comment on the ACD when it is posted on the Institute’s web site 5 days after it is sent to consultees and commentators. These comments are usually presented to the appraisal committee in full, but NICE reserves the right to summarise and edit comments received during consultations, or not to publish them at all, where in the reasonable opinion of NICE, the comments are voluminous, publication would be unlawful or publication would be otherwise inappropriate. Please note: Comments received in the course of consultations carried out by NICE are published in the interests of openness and transparency, and to promote understanding of how recommendations are developed. The comments are published as a record of the submissions that NICE has received, and are not endorsed by NICE, its officers or advisory committees. Com NICE Response ment Type of Organisation Stakeholder comment Please respond num stakeholder name Please insert each new comment in a new row to each comment ber 1. C onsultee Biogen Idec Biogen appreciate the opportunity to provide comments on the NICE Appraisal Consultation Document (ACD) for nusinersen Thank you for (company) Ltd. for treating spinal muscular atrophy (SMA) [ID1069]. your comment. The Biogen are disappointed that the Appraisal Committee was unable to recommend nusinersen in the ACD, however, we are recommendation committed to collaboratively finding solutions that address the remaining uncertainties, mitigate risk to the NHS and ensure that s in the Final access to nusinersen is managed appropriately without further undue delay. Appraisal Document (FAD) In addition, Biogen believe that several conclusions in the ACD are not an accurate interpretation of the evidence and have changed. encourage the Appraisal Committee to reconsider its conclusions, as described in the following comments. Nusinersen is now To support the comments, Biogen have submitted an appendix containing clinical data that explain the long-term model recommended for assumptions. The clinical data also clarify the health benefits beyond the primary trial endpoints of survival and motor function. pre-symptomatic Furthermore, Biogen have put forward a revised commercial offer for consideration. The revisions reduce the ICERs within and types 1, 2, ranges that may be deemed cost-effective pending further data collection. In parallel, Biogen are continuing to develop a and 3 SMA in the proposal for a managed access agreement (MAA) for consideration by the committee in October. context of a managed access agreement (MAA). Responses to comments on specific sections are made below, alongside the respective comments. 2. C onsultee Biogen Idec Paragraph 1.2 notes that there is an unmet need for effective treatments that could slow disease progression. We are Thank you for (company) Ltd. concerned that this recommendation may imply that the unmet need is similar across infantile onset (type I) and later onset your comment. (types II and III) SMA. Patients with infantile onset SMA fail to develop any motor milestones and rarely survive to their second The section referred to is birthday without permanent ventilation.(1,2) Standard of care only helps to prolong survival of infantile onset patients through intended to highly invasive tracheotomy or permanent ventilation, and it does not slow or prevent the decline of motor and respiratory briefly summarise function.(3) Therefore, given the rapid functional decline in patients with infantile onset SMA, there is an urgent unmet need for the decision and an effective treatment that significantly extends life without permanent ventilation, and allows a child to develop any motor rationale which is 2 of 105 Com NICE Response ment Type of Organisation Stakeholder comment Please respond num stakeholder name Please insert each new comment in a new row to each comment ber functions that are not possible with the current standard of care. covered in more detail in section In contrast, patients with later onset SMA are expected to survive until adults, albeit with a progressive loss of motor function 3.3 (now section 3.4) of the FAD. and independence.(4–7) Standard of care is aimed at relieving the symptoms, but it does not slow or prevent the decline of See also motor and respiratory function.(3) For these patients, the progressive decline and loss of ability can have
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