Tuesday 8th November 2016, Jubilee Room, House of Commons
Regulating and paying for
regenerative medicine products
in the NHS –
challenges and opportunities
Prof Alex Faulkner
Centre for Global Health Policy
Outline
• Flexibilities in regulation that are part of attempts to
accelerate access to market of new products
-potential impact on NHS innovation and patients
• Debates about how to pay for such products when the
evidence base is necessarily still being developed.
The product regulatory environment
Tissues and Cells
Advanced Therapy
Medicinal Products
ATMPs
HTA
MHRA
Medical Devices
Pharmaceuticals
Key parameters
 Scientific and
effectiveness
uncertainty
 Standard of care?
 Data poverty
 High cost
 Curative?
 Disruptive to
NHS?
Pathways to market- centralised
EU systems and data
requirements
- Pharmaceutical
- Advanced Therapy Medicinal Product
- cell therapy; gene therapy; tissue-engineered product
- Medical device
-HOW MUCH FLEXIBILITY?
Medicines pathways - EU
‘Licensing flexibilities’ for ‘unmet need’ and public
health emergency:
-
Conditional approval
-
Exceptional circumstances
-
Accelerated assessment
-> ‘Medicines Adaptive Pathways for Patients’
(MAPPS)
‘Compassionate use’
Allowed by Pharmaceutical Directive 2004
50 programmes notified to EMA 2006-15,
- 40% relate to ‘Orphan’ drugs/’Rare disease’
- pre-approval, pre-reimbursement
(Hyry et al, 2015)
Hospital-based pathways
One-off or personalised nonroutine
applications
- ‘Hospital exemption’ (ATMP), or ‘Special’ license
(Medicines) , allows treatment and -> data for clinical
trial
- ? in-house medical device exemption
‘Medical device’ pathway
e.g. closed system intra-operative cell
processors
- transplant e.g. for reconstructive surgery
- multiple clinical uses
Flexible pathways to healthcare
adoption/clinic - ?
1. ‘Promising innovative medicine’/ ‘Early
access to medicines’ (MHRA)
2. NICE valuation methodology and
assessment decisions
3. NHS Executive commissioner schemes
4. Individual patient funding
Early Access to Medicines EAMs
Government Strategy for Life Sciences.
‘aims to give patients with life threatening or
seriously debilitating conditions access to
medicines that do not yet have a marketing
authorisation when there is a clear unmet medical
need’
- Designation as a PIM: ‘Promising Innovative
Medicine’
First ‘PIM’ designation
-for a cell therapy product for the
treatment of cancer on 8 September
2014.
-Cancer immunotherapy -DCVax-L (from NW Bio) covers all malignant gliomas,
which including both Glioblastoma multiforme (the most
severe grade) and less malignant grades
NICE exploration of
applicability of methods to RM
“…demonstrate the versatility and adaptability of
NICE’s approach to appraising health technologies to
enable appropriate consideration of these innovative
treatments.”
…recommends that NICE continues to further develop
the ways in which uncertainty can be quantified and
presented to its decision-making committees.’
furthermore…
‘Where there is a combination of great uncertainty with
potentially very substantial patient benefits,
innovative payment methods may have a key
role to play in managing financial risk …
(https://www.nice.org.uk/News/Press-and-Media/nice-publishesreport-on-approaches-to-assessing-innovative-regenerativemedicines) 29 March 2016
NHS service commissioning
NHS England commissioning
Alternative, conditional gateways to adoption:
• ‘Commissioning through Evaluation’ scheme
• ‘Risk-sharing’ schemes explored
• ‘Specialised services’ (NB ‘funding experimental and
unproven treatments’ part of Specialised Commissioning
public consultation Oct 2016)
• NHS Tariff innovations?
• CCGs
Risk-sharing RM example
Risk sharing
currently being used in Italy for Strimvelis (GSK)
(for ADA-SCID , severe immunodeficiency; EMA license as
gene therapy medicinal product/ ATMP May 2016)
Italy has a good history in risk sharing. Currently about 90
therapies reimbursed in this way. Medical tourism?
Converging Pathways
• ‘Early access schemes’
• ‘Managed Entry Schemes’
• ‘Conditional Authorisation’
• ‘Adaptive Pathways’
• ‘Risk-based approach’/’Risk-sharing’ payment
• NICE/NHSE coordination
• NICE/MHRA coordination
From Accelerated Access Review Final Report 2016
Summary – growth of provisional,
converging pathways
l
Legal/market
regulatory
gatekeeping
ATMP Hospital
Exemption
Adaptive licensing
‘Specials’ license (UK
only)
Orphan designation
Exceptional
Circumstances
Accelerated
Assessment
Medical device
Converging: legal
easing; broadening
criteria
-Early Access
Scheme/Promising
Innovative Medicine
(PIM) assessment
-Adaptive pathways
-Conditional approval
-Risk-sharing
-Innovative payment
systems
‘Parallel review’
(Value-based
assessment )
Adoption gateways
NHS Executive special
clinical service
schemes
NHSE Commissioning
through Evaluation
Multiple individual
patient funding
requests
Issues: To what extent is patient
access and public health promoted?
- Rare disease applications
- Exceptional ‘compassionate’ use route
favoured by SMEs
- ‘Unmet need’
- ‘Special’ and exempt patient access routes
- Provisional reimbursement
- Needs-driven?