1. Qualification of Innovative Development Methods/Tools Themes from Breakout Discussion 13 February 2013

2. Participating Group Members Richard Meibach Martha BrumfieldCarolyn Compton ShaAvhree BuckmanAlan Morrison Nathalie SeigneuretBernard Munos Carol MimuraSharon Olmstead Lynn HudsonHubert Leufkens Nancy Cutter Foster Ruth Mcnerney Mike ward

3. Premise of Qualification Need to define Context of Use – Defines specific purpose, method of use Biomarkers need careful scrutiny – are population specific – Predictive, prognostic, etc – How to garner interest with those using “accepted biomarkers” to fully “validate” them for qualification processes

4. Harmonize or not? Meaning of FDA qualification and EMA Scientific Advice Opinion are not equivalent – FDA prefers some degree of predictability – EMA prefers a more principles based approach Level of evidence varies because of the above Legal structure with fees, timelines are not the same

5. CTD or not? Willingness of FDA and EMA to consider commonalities where there are similarities with parts of dossier being customized for what that Agency requires Discussed possibility of conducting a pilot with C-Path and FDA/EMA

6. Prioritization Priority to whom? Perhaps best to start with gap analysis – Where is the greatest need – for diseases with lack of treatments; with industry being successful?? With regulatory review? – Vet information with all stakeholders

7. Performance Indicators Why do this? – To learn from successes and failures Who should own the collection and reporting? – Publication of information should be a goal Agreement that it is needed but not sure how to go about it

8. Performance Indicators MAAs in EU will include information about how Scientific Advice was addressed – including any use of biomarkers……so the information could likely be extracted NDAs/BLAs in US do not necessarily require this specificity Industry will need to step up to assist with collecting this information

9. Transparency Theme Better understanding of what “qualification” means with FDA, with EMA, with PMDA Better communications of decisions Ideal to be able to share lessons learned – Rejected Letters of Intent ?? – Idea to consider a publication of Regulator’s and Industry’s perspective

10. Incentive to Qualify Encourage researchers and industry to bring more tools forth for Incentive of a “priority review voucher” concept – Faster review or waiver of fees???