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Specialised Services Stakeholder Surgery
Meeting Note
Time: 16:00 – 16:30
Date: 25 April 2017
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:
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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
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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:
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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:
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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
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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
expediting access.
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
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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.
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.
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.
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).
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.
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.
AM confirmed he will send regional data, although unpublished,
in support of symptomatic evidence which illustrates national
fairness of equity.
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
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found in the Methods document on NHS England’s website.
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.
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.
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.
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.
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.
JP concluded meeting confirming next steps.
Action: AM to provide data (pre-publication).
Action: FW to liaise with SMA Support UK re communication.
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.
Action: JR to investigate further evidence that could be
presented, ahead of publication.
Record of the meeting to be circulated to all for approval.
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