1. Prof Alex Faulkner Centre for Global Health Policy
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

2. 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.

3. The product regulatory environment
Advanced Therapy Medicinal Products ATMPs
Tissues and Cells
HTA MHRA
Pharmaceuticals
Medical Devices

4. Key parameters Scientific and effectiveness uncertainty
Standard of care?
Data poverty
High cost
Curative?
Disruptive to NHS?

5. 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?
UK pharma ‘Specials’;
Unmet need;
‘Wait-and-see’ risk-sharing hybrid marketing authorisation; Rare diseases/orphans
→ ‘experimentalist’ adaptation of governance?

6. Medicines pathways - EU
‘Licensing flexibilities’ for ‘unmet need’ and public health emergency:
Conditional approval
Exceptional circumstances
Accelerated assessment
-> ‘Medicines Adaptive Pathways for Patients’ (MAPPS)
Mapps a non-legal category

7. ‘Compassionate use’ (Hyry et al, 2015)
Allowed by Pharmaceutical Directive 2004
50 programmes notified to EMA ,
- 40% relate to ‘Orphan’ drugs/’Rare disease’
- pre-approval, pre-reimbursement
(Hyry et al, 2015)
Once orphan gets MA, secured statutory monopoly for 10 years.
Reimbursement varies, in fact France has intro system where hosp pharmacies can pay full price (or + premium) for such treatments.

8. 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
Specials most used in UK, other euro countries using HE more widely, controversial cuz fear can distort market, and EMA has doubts about stem cell standard control

9. ‘Medical device’ pathway
e.g. closed system intra-operative cell processors
transplant e.g. for reconstructive surgery
multiple clinical uses
Subs manipulation/homologous use criteria

10. Flexible pathways to healthcare adoption/clinic - ?
‘Promising innovative medicine’/ ‘Early access to medicines’ (MHRA)
NICE valuation methodology and assessment decisions
NHS Executive commissioner schemes
Individual patient funding

11. 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’

12. First ‘PIM’ designation
-for a cell therapy product for the treatment of cancer on 8 September
-Cancer immunotherapy -
-DCVax-L (from NW Bio) covers all malignant gliomas, which including both Glioblastoma multiforme (the most severe grade) and less malignant grades
PIM opens way for EAM scheme

13. 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.’

14. 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 …
( report-on-approaches-to-assessing-innovative-regenerative- medicines) 29 March 2016

15. Payment methods/ uncertainty
Projected benefit high (10 QALYs) + v. high uncertainty:
-> ‘The lifetime leasing method of payment …
was viewed with considerable interest by the Panel. .. where a monthly fee is paid for the duration of treatment benefits (until death) greatly reduces the decision uncertainty… It is important to note that… the asset in question is the health gain delivered rather than the CAR T-cell therapy itself.’

16. NHS service commissioning

17. 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

18. Risk-sharing RM example
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?

19. Converging Pathways ‘Early access schemes’ ‘Managed Entry Schemes’
‘Conditional Authorisation’
‘Adaptive Pathways’
‘Risk-based approach’/’Risk-sharing’ payment
NICE/NHSE coordination
NICE/MHRA coordination
Proliferation of schemes…bridging the market authorisation/clinical application boundary

20. From Accelerated Access Review Final Report 2016

21. Summary – growth of provisional, converging pathways
Legal/market regulatory gatekeeping
Converging: legal easing; broadening criteria
Adoption gateways
ATMP Hospital Exemption
Adaptive licensing
‘Specials’ license (UK only)
Orphan designation
Exceptional Circumstances
Accelerated Assessment
Medical device
-Early Access Scheme/Promising Innovative Medicine (PIM) assessment
-Adaptive pathways
-Conditional approval
-Risk-sharing
-Innovative payment systems
‘Parallel review’ (Value-based assessment )
NHS Executive special clinical service schemes
NHSE Commissioning through Evaluation
Multiple individual patient funding requests

22. 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?
Generally not population-wide at the moment