Specialised Services Stakeholder Surgery

Meeting Note

Date: 25 April 2017Time:16:00 – 16:30

Location: Skipton House, 80 London Road, London SE1 6LH

Company name and address: / Muscular Dystrophy UK, Biogen, SMA Support UK
Company attendee(s)/contact detail(s): / Dr Adnan Manzur. Consultant Paediatric Neurologist, Great Ormond Street Hospital.
Jonathan Randell. Associate Director, Market Access, Biogen
Liz Ryburn. Support Services Manager at SMA Support UK
Robert Meadowcroft. Chief Executive at Muscular Dystrophy UK
NHS England attendees: / Mr James Palmer, Medical Director Specialised Services;
Malcolm Qualie, Pharmacy Lead, Specialised Services;
Edmund Jessop, Public Health Advisor, Highly Specialised Services
Anthony Prudhoe, Senior Programme Manager (Women & Children);
Fraser Woodward, Head of Communications, Engagement and Partnerships (Specialised Services);
Kate Bedford, Business Coordinator.
Jessica McAllister Observer
Initial Request for Meeting: / December 2016
Product: / Nusinersen
Disease area: / Spinal muscular atrophy (SMA)
Developmental status: / On accelerated approval programme; CHMP opinion issued, waiting EMA authorisation. Phase III trials waiting publication.
England HTA status: / Pending NICE Appraisal
Relevant PoC/CRG: / Paediatric Neurosciences
Key question:
What support and advice can be given to centres delivering the Expanded Access Programme for nusinersen to treat infants with spinal muscular atrophy Type 1? These centres currently have limited capacity to deliver the treatment to all eligible patients. This may limit or prevent the administering of the drug.
Key issues:
  • SMA Type 1 is a severe, infant onset condition. For 95% of children living with the condition, life expectancy is less than 18 months
  • Nusinersen has been shown in clinical trial to have a clinical significant impact upon motor function in patients with SMA Types 1 and 2
  • The company, Biogen, has announced an Expanded Access Programme for the drug, which covers patients in the UK affected by SMA Type 1
  • However, capacity restrictions mean that without the right support administering centres may not be able to provide the drug to patients with SMA Type 1
Briefing
Nusinersen is an investigational, potentially disease-modifying therapy for the treatment of SMA. It is manufactured by the company Biogen. Nusinersen has been trialled in infants with SMA Type 1. The trial results have shown a statistically significant improvement in the achievement of motor milestones in this group of patients compared to those on placebo. Biogen has also recently published positive clinical trial results for patients with SMA Type 2.
Expanded Access Programme
Because of the promise of the clinical trial results, in Autumn 2016, Biogen took the decision to launch an Expanded Access Programme (EAP) for the drug. This programme included the UK.
A positive CHMP has been issued and expect a EMA decision within 60 days as nusinersen is on the accelerated approval program. There is also then further time needed for NICE, NHS England or other bodies to determine whether the drug will receive approval in the UK. An EAP therefore recognises the severity of SMA Type 1 and the need for this drug to reach patients as quickly as possible.
The EAP currently apply to infants with SMA Type 1, who have had clinical symptoms and signs earlier than six months of age (consistent with an SMA Type 1 diagnosis).
The EAP operates as follows:
  • The first step for Biogen will be to ensure that all patients from their current ENDEAR study had access to Nusinersen. This process is known as transferring to an extension study.
  • This has now been completed at the two trial sites, Great Ormond St & Newcastle and patients have begun to be enrolled at other centres.
  • In England, the EAP is now running from GOSH and Newcastle, the original clinical trial sites. Treatment is also being administered from Oxford and Evelina Hospitals. Other centres are in the process of applying to Biogen and their Trusts for EAP status. To date a total of 12 children are being treated via the EAP across the UK.
Centres need to ensure:
  • Patients have an initial screening evaluation
  • Patients are called in for doses at days 1, 15, 30, 60, 180, 300 and then at days 420, 540 and 660
  • Pharmacy orders in all doses four weeks prior to injections
Whilst clinicians are taking all steps they can to move this process forward, administering the drug is intensive– and lack of capacity may limit or prevent the delivery of the drug to all patients eligible under the EAP criteria. SMA Type 1 is exceptionally severe, and it is essential that any capacity shortages are addressed without delay.
These centres and Patient Advocacy Groups representing the SMA community are also receiving frequent requests from families all over the UK who are understandably desperate for their infant child to access the therapy. Additionally, it is known that nine families with infants with SMA Type 1 who have been unable to access treatment in the UK are travelling to Paris where they are being treated via the EAP there.
Attendees:
James Palmer
Malcolm Qualie
Edmund Jessop
Kate Bedford
Adnan Manzur
Jonathan Randell
Liz Ryburn
Robert Meadowcroft /
  1. James Palmer (JP) opened the surgery by explaining how NHS England Specialised Commissioning operates and its governance.
  2. Jonathan Randell (JR), who was invited by MDUK to attend,gave an overview on behalf of Biogen in relation to their objective when requesting the meeting and what they hoped to achieve, in particular what support and advice can be given to centres delivering the Expanded Access Programme (EAP) for Nusinersen to treat infants with spinal muscular atrophy (SMA) Type 1. Concern around centres currently having limited capacity to deliver the treatment to all eligible patients was highlighted.
  3. It was highlighted that there are three types of SMA, Type 1, 2 and 3. For the purposes of this stakeholder surgery Type 1 was discussed and is the most severe. It was discussed that 95% of children living with the condition survive less than 18 months.
  4. JR confirmed that although discussions have taken place with NHS England previously, SMA Support UK and MDUK wished to discuss with more clarity what the next steps would be in order for NHS England to consider taking a stronger view on supporting this treatment with a consideration towards expeditingaccess.
  5. The drug is currently being provided free of charge by Biogen under the EAP but there are other treatment costs for administering the drug as patients receive the drug by intrathecal injection.
  6. JR confirmed that a provisional scoping exercise with the National Institute for Health and Care Excellence (NICE) had taken place in January 2017. It was expected that in June 2017 NICE would announce whether the technology will be appraised by Technology Appraisal (TA) or Highly Specialised Technology (HST). The topic had not yet been formally referred by ministers to NICE. It was highlighted that it would probably be a HST but final decision had yet to be taken.
  7. JP confirmed that if the topic was in the NICE work programme, NHS England would not include in the work programme for formation of clinical policy. It was confirmed that the NHS England Specialised Commissioning team cannot expedite the NICE decision-making process. It was highlighted that NICE has the ability to consider unpublished data such as the European Medicine Agency EPAR but that NHS England in deciding on clinical policy require peer reviewed published evidence.
  8. JR highlighted that major regulators such as the European Medicines Agency’s Committee on Human Medicinal Products had given a positive opinion, and the Food and Drug Administration in the United States had issued a marketing authorisation.
  9. JR confirmed Phase III data hasn’t been published but further data was presented at AAN. The interim data from ENDEAR phase 3 study in infantile onset SMA has been presented at the BPNA meeting in January 2017. The ENDEAR end of study data has been presented at the AAN meeting in April 2017. The interim data from the CS3A phase 2 open label study in infantile onset SMA was published in the Lancet in December 2016 (Finkel et al).
  10. Adnan Manzur (AM) highlighted the urgency with which the treatment needs administering. Typically there is a four week period between the infant demonstrating symptoms to start of respiratory failure.
  11. AM believed that there are currently about 60 patients in England with Type 1 SMA. A consensus on prioritising patients and when to review ongoing access is needed.
  12. AM confirmed he will send regional data, although unpublished, in support of symptomatic evidence which illustrates national fairness of equity.
  13. It was explained an interim NHS England policy (before a NICE appraisal) can be considered where the evidence base and benefit is substantial. Such a proposal was considered by the NHS England Clinical Panel on 19 April. The Panel appraised the 3 top published papers submitted by the clinical lead. Only one of the 3 related to the treatment population (Type 1 SMA), this was a pharmacokinetic study. The policy process can be found in the Methods document on NHS England’s website.
  14. The clinical panel also were made aware of recent unpublished data presented on early analysis of the Endear study. The panel discussed the promise in this study. The panel was unable to recommend an interim ‘routine’ commissioning position at this stage due to the early nature of the evidence publication.
  15. JR confirmed he will check publication data re: evidence and feedback to Anthony Prudhoe (AP). The panel will be able to review the commissioning position when the evidence has been accepted for publication or earlier if possible in confidence so that a policy position can be confirmed as close to publication date. JR confirmed the evidence, once published, will support short term impact only and that long term impacts are unknown at this point.
  16. As a result NHS England is unable to confirm the funding of the treatment costs of the intervention as part of the EAP. There are therefore two options: either the company includes the cost of the treatment costs in the EAP; or the providers make a decision to include treatment as part of their current service contracts.
  17. Discussion was had about the need to assist with the prioritisation of patients accessing the EAP.Edmund Jessop (EJ) will explore whether NHS England could help to facilitate a national scheme for deciding which patients should receive priority for pre-market access under the scheme organised by Biogen.
  18. Communication was discussed and it was confirmed Fraser Woodward (FW) would liaise with SMA Support UK to work together on appropriate wording of the current treatment access position.
  19. JP concluded meeting confirming next steps.
  20. Action: AM to provide data (pre-publication).
  21. Action: FW to liaise with SMA Support UK re communication.
  22. Action: EJ to explore national scheme facilitation regarding patient prioritising under the scheme organised by Biogen, liaising initially with Biogen and patient advocacy group SMA Support UK.
  23. Action: JR to investigate further evidence that could be presented, ahead of publication.
  24. Record of the meeting to be circulated to all for approval.

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