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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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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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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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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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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