MRC Stratified Medicine Initiative Jonathan Pearce Medical Research Council, Translational Programme Manager University of Glasgow Industry Day: 24 September 2015

Presentation/Phenotype Stratified medicine has the potential to deliver improved diagnoses and therapies Input Tools Outputs Value Affected population Disease Strata Diagnostics to better predict disease state, prognosis, response Mechanism of disease leading to new therapies Clinical Presentation/Phenotype Genetic / Molecular Therapeutic Response Capturing this potential requires true partnership between basic and clinical researchers, industry (BioPharma and Diagnostics) and patients. No one group has all the necessary skills/resources 1

Programme Coordination … and partnership between funders, regulators, evaluators and providers Stratified Medicine Innovation Platform Programme Coordination Group 2

MRC Stratified Medicine Initiative Set up in 2010/11 and represented a new way of funding from the MRC Builds on the MRC/ABPI Inflammation and Immunology (I&I) Initiative £60m initiative to develop disease-specific research consortia, involving industry partners Consortia exploring predictors of response and mechanisms underpinning disease stratification, where there is evidence that therapeutically relevant strata exist 3

MRC Stratified Medicine Consortia 13 internationally competitive stratified medicine discovery engines Build on NIHR and DA clinical research funding Total investment c. £57m 3 charity co-funders (CRUK, ARUK, BHF) 32 academic and 51 commercial partners RASP-UK UKPBC RAMAP-II PSORT MATURA-II COPDMAP MASTERPLANS GAUCHERITE MATURA-I Auto-Immune Respiratory Cancer Infections Mental Health Cardiovascular Diabetes Congenital S-CORT AIM HY MASTERMIND STOP-HCV RAMAP-I STRATA

STOP-HCV – 21 Partners; 12 Academic, 8 Industrial, 1 Governmental and Patient

Stratified medicine relies on an ability to integrate and interrogate multi-level data In last 2 years (2012-2014) MRC has invested over £100m in informatics infrastructure and capabilities CIPHER London HeRC Scotland 24 academic institutions 2 MRC Units Medical Bioinformatics Imperial Oxford UCL/Crick Leeds Uganda Warwick/Swansea 6

Molecular pathology review If the UK is to capture the stratified medicine opportunity, we need to be able to translate both its therapeutic and diagnostic outputs to patient and economic benefit While much consideration has been given to the challenges faced by those developing new therapies, less work has focused on the needs of diagnostics MRC has undertaken a review focused on these needs 7

Review recommendations Path - Produce clear unified guidance setting out the critical path and required evidence for the discovery, development, approval and evaluation of tests. Address the gaps in the UK’s regulatory, evaluation, adoption and delivery system Proximity - Establish joint research/clinical service ‘nodes’ aligned with industry and complementing NIHR, TSB and other RC and partner investments People - Train next generation of research leaders in molecular pathology, potential merit of guaranteed follow through clinical lectureships Further development of UK capacity in statistics, bioinformatics and health economics Undergraduate medical curriculum to include molecular pathology, to aid adoption and interpretation 8

MRC and EPSRC molecular pathology nodes call In an initial response to recommendations, the MRC and EPSRC have launched a joint call to support up to eight molecular pathology nodes Up to £17.5m (£15m from MRC and £2.5m from EPSRC) Each node will be a multidisciplinary centre of innovative molecular diagnostic test discovery and development bringing together: the research base pathology/genetic services and industry 9

MRC Stratified Medicine Consortia and MRC EPSRC Molecular Pathology Nodes 13 internationally competitive stratified medicine discovery engines Total c. £60m 3 charity co-funders (CRUK, ARUK, BHF) 32 academic and 51 commercial partners 6 centres of innovative molecular diagnostic test development Total c. £16m 8 academic and 21 commercial partners Edinburgh/ St Andrews Glasgow RASP-UK Newcastle UKPBC RAMAP-II PSORT Nottingham MATURA-II COPDMAP Manchester Leicester/ Loughborough MASTERPLANS GAUCHERITE Auto-Immune Mental Health MATURA-I Respiratory Cardiovascular S-CORT Cancer Congenital AIM HY MASTERMIND Infections Diabetes STOP-HCV RAMAP-I STRATA Node

The nodes will work as a network and complement partner investments Discovery Science/ Early TRL Mid TRL Late TRL Science base MRC Stratified Medicine Consortia Precision Medicine Catapult NHS NIHR DECs Nodes EPSRC Analytical Sciences RC Centres MHRA NICE NIHR BRCs/BRUs NHS Innovation GEL Discovery Validation Regulation Evaluation Adoption