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Safeguarding public health
Expert Group on Innovation in the Regulation of Healthcare products
Adaptive Licencing workshop at the Wellcome TrustOctober 15, 2012
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Slide 2Oct 15, 2012
Adaptive Licencing Workshop at the Wellcome Trust
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Expert Group work programme
• Early Access work
• International/comparative experience
• Clinical trial activity in the UK
• Defining the challenge in regulatory innovation - including Adaptive Licensing and developments in PV
• Mapping projects and initiatives - UK and US (Newdigs)
• Related initiatives- Advanced manufacturing regulation
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Slide 3Oct 15, 2012
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Developments and Action
• Information on flexibilities in existing licensing routes - On internet and with representative bodies- MHRA Paper
• Early access consultation
• EU proposal on Clinical Trials- Wide range of other work under the Growth Review
• Adaptive licensing- Initiatives and thinking on adaptive licensing - Key questions and scoping- A pilot programme of activity on adaptive licensing by Dec
2012
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Safeguarding public health
Early Access
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Early Access
Scope of the scheme- New medicines in areas of unmet need- Benefit:risk profile of the medicine must be positive- Availability restricted to predefined patient population
Characteristics of the scheme- MHRA provides scientific opinion on the medicine- Medicine could be available for around 1 yr before licence
grantedThe medicine remains unlicensed
- No advertising permitted
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Early Access
Eligible patients- Patients of any age (including children), no CT participants
Eligible drugs- Restricted to any therapeutic area with un-met need
Safety monitoring requirements- ADR reporting requirements will apply, additional
monitoring tbd on a case by case basis
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Early Access
Pricing- Freedom of pricing for producers of EA drugs but- NHS Trusts decide on purchase of medicine (lower cost
encourages NHS uptake)
Patient consent and liability issues- ‘Active’ patient consent- Effective information dissemination- Manufacturer remains liable for supplying defective product
Additional criteria- Benefit to UK economy- NHS funding for the medicines must be cost effective
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Safeguarding public health
Adaptive Licencing
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Adaptive Licencing: EU / UK? Existing Legislation or not?
If a UK scheme- Unlicensed use under the professional/legal responsibility
of prescriber- Product for un-met need- Essentially a variant of Early Access
If EU scheme- Centralised product; EU regulation applies- EMA, not UK coordinates licencing decision- European Commission will be the licensing authority
Existing regulation or not?- Essentially a matter of time-frame
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Adaptive Licencing
3 levels of activity / tripartite structure- Level 1: EU (EMA, CHMP discussions) - Level 2: HMG/MHRA- Level 3: Industry / third sector
Critical role for EU- EU licensing process- European Commission will be the licensing authority
Considerable opportunities for action under current rules- Alternative means long timeframes
Industry must come up with a product/asset- None identified so far – on either side of the Atlantic
Merit in both retrospective and prospective work
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Adaptive Licencing
Critical issues in regulatory decision making (risk/benefit calculation)
- Evidential levels to apply- Role of RCTs – and use and interpretation of observational data
Industry policy / public health policy- Industry financial/business risks / interests?
Patient / professional / media understanding
Wider system challenges: - Pricing / VBP - Company decisions / management of their development
pipelines and processes
Pilot project by end 2012
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An “asset” or a product
Likely characteristics:
- break/-through product for as yet un-met need- Perhaps not as “open and shut” as Glivec, but not far off- Likely to be for small(er) patient population
· but not necessarily- For some of the AL ideas, possibly a product with multiple
potential uses- [Probably] something with an identified patient group
· Informed consent etc
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Adaptive Licencing - Forward Look
EU / EMA
- Management Board discussion: March 2013
- CHMP and other EMA committees
- Commission
HMG/MHRA
- Defining the pilot, and the problem(s) it is to solve
- Licensing routes available- Toolbox of options- Structures and roles- Governance framework
needed- Relation and link with
industry/third sector support project
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Safeguarding public health
Conclusions of Expert Group meeting
October 9 2012:
focused on Adaptive Licensing
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Conclusions of EG meeting, Oct 9
• AL to be positioned at EU level within current legislative framework• Tripartite project structure makes sense
• Practical programme of activity by the end of the year• Need to be clear about what questions we are
trying to solve
• Regulation, Clinical Trials, purchasing and R&D strategies within companies all relevant
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Work and steps ahead
• Commissioned papers:- Pharmacovigilance
• Further consideration: - EMA level work on regulatory challenge of AL- Capacity of CPRD wrt AL
• Pilot project for the end of the year- Retrospective and prospective activity- Identification of potential products