1. Adaptive Licensing UK

2. Content What is adaptive licensing? Draft criteria for pilot candidate selection EMA Road Map 2015 and work programme 2012 UK perspective Summary of workshop agreements

3. What is adaptive licensing (AL)? Adaptive licensing is a prospectively planned customised approach to regulation based on iterative assessment of benefits, risks and uncertainties. Adapted from Eichler et al. Nature CPT, 91: 3, 426-437.

4. AL: Aims To increase the efficiency of the biomedical innovation paradigm by streamlining of development, regulation and market access for selected products. To balance timely access for patients with the need to provide adequate evolving information on benefits and risks.

5. AL: Key features Judicious authorisation based on early risk/benefit assessment in agreed restricted population. Followed by iterations of prompt label adaptations informed by data from monitoring of ‘real-life’ effectiveness and safety. All stakeholders contribute to a prospective development plan and input into the design of the confirmatory trials. Customised drug- and disease- specific licensing pathways that may utilise existing regulatory mechanisms. Managed market entry controlling patient-access with re- imbursement conditional on data collection on safety and efficacy. With the opportunity to inform value assessment.

6. Draft criteria for pilot candidate selection 1. A willing commercial participant. 2. A robust case for why existing regulatory pathways not suitable. For example unmet clinical need. 3. Anticipated acceptable risk/benefit profile in the target disease area with reasonable certainty. 4. Sufficient numbers of patients that can be recruited in the UK or opportunity for EU collaboration. 5. Appropriate data collection mechanisms. For example robust trial networks in the specific disease area with clinicians that are interested in participating in research. 6. If possible, existing patient support mechanisms. 7. AL route anticipated to reduce time to market and development costs.

9. The UK perspective Strategy for life sciences: A group of experts drawn from government, regulators, the NHS, industry, and the academic and third sector communities will meet quarterly to discuss healthcare regulation issues, including the development of new initiatives and innovations. The Ministerial (Biopharmaceutical) Industry Strategy Group Innovative Technology Forum will be responsible for ensuring the delivery of these initiatives, and will provide an annual report to DH and BIS ministers. This will set out measures of performance such as the use of conditional authorisation pathways, and uptake of the Early Access Scheme, alongside ‘next steps’ proposals for further regulatory innovation. UK stakeholder workshop 2 nd April 2012, Welcome Trust.

10. April 2012 Workshop agreement There are a number of issues with the current innovation ecosystem which may need different solutions for different medicines. Adaptive licensing is an interesting proposition that is worth further exploration. A working party should be established with membership drawn from key stakeholders. Their activity should be guided by an independent advisory group. The UK could be a potential site for an adaptive licensing pilot and an expression of interest should be submitted to the EMA. The timelines should be established and a project plan developed. Collaborative links to continue with the NEWDIGS consortium.