Better Science, Better Health: New Healthcare Models New Data Models: Managing Uncertainty and Leveraging New Sources of Evidence Author: Sabine Atzor * Date: 27 Oct 2015 * Version: Final New Data Models www.efpia.eu

From paradigm shifts for clinical evidence generation ... The way we are collecting data is changing radically. Biomarker-driven research has opened the door to an unprecendented knowledge of many diseases, in particular in the oncology sector. With that knowledge we have more opportunities on how we conduct clincila trials beyond the classical approach which is based on defined patient population with a defined disease is randomised to a new medicine or a control therapy. Two scenarios: Basket trial based on similar tumor mutation via Biomarker different organs same drug Umbrella Trial tumor in same organ Profiling with different biomarkers Different drug therapies Companies and regulators are increasingly applying these new approaches and have started a new learning path on what these new designs mean for evidence generation and managing uncertainties. “Basket Trials” “Umbrella Trials” AACR Cancer Progress Report 2014

....to a new era of data generation Genomic Sequencing/ NGS Classification of diseases Unprecendented knowledge of the disease From «Tissue is the issue» to Liquid biopsies multiplex assays and monitoring will allow continuous monitoring and data generation New technologies Wearables everywhere ______ Many of the new trends for data generation have been mentioned already during the course of the day. I would like to highlight some particular trends which will be strong drivers for developing regulatory concepts in the future. The arrival of NGS is going to revolutionise diagnosis and treatment. Our knowledge of the disease has been increasing substantially and for many cancers biomarkers have been established which allow a more precise classification of the disease allowing an unprecendented knowledge of the disease. New combination therapies. We have access to our genome and the potential of this is currently being explored through some government initiatives.: Next generation sequencing of the genes will allow a more precise classification of diseases and therapy needs. In combination with other assays, such as imaging this will open the door for continuous monitoring with so-called «liquid biopsies». Not «tissue is the issue» any more in diagnostics. Sensors and wearables are everywhere and allow a better monitoring of the disease and progress by patients and healthcare professionals. Reference: Healthcare IT News, Wireless Technology in Healthcare Survey, November 2010

Medicines Adaptive Pathways to Patients MAPPs prospectively planned, adaptive approach to bringing drugs to market starting from an authorised (eventually “niche”) indication iterative phases of evidence gathering and progressive licensing adaptations balancing timely access for patients with the need to provide adequate evolving information on benefits and harms. Transition from… To… Magic moment Prediction RCT only Big populations Focus on licensing Regulators/ industry Open utilisation Life-span management Monitoring Toolkit for evidence generation Small populations Focus on patient access Regulators, patients, industry, HTA bodies, payers Managed utilisation

MAPPs – Connecting Tools and Stakeholders Europe has numerous, maybe all tools in place to make MAPPs it happen. Old tools, such as conditional marketing authorisation and accelerated assessment, are reviewed and improved in their application to address evolution of science. A new tool is on the horizon to address the need for more frequent dialogue and foster development – PRIME. More stakeholders are now brought around the table: regulators, industry, patients, health technology assessment bodies, payers.

MAPPs – Connecting the Dots To make adaptive pathways happen we need to connect the dots in different areas which are currently under scientific or regulatory development. In Europe on a number of these different and interlinked topics initiatives have been launched. The pace of the parallel developments is ever increasing – . Tremendous opportunities for multi stakeholder collaboration at the interface of different responsibilities at European and national level – for the first time. But this increases complexity. And – at the same time global cooperation is key.

Public Private Partnership – IMI Address Bottlenecks of Innovation EU Comission In-kind contribution FTEs + cash In kind translated into € EU Comission matches the industry contribution Total project budget Industry consortium Value of companies' contribution EU funding available for public partners = IMI office = Neutral broker Process owner How can the need for more an multi-stakeholder interaction on this journey managed? IMI is the European role model for a public Private Partnership. IMI office as a neutral broker and with funding from the innovative industry through in kind contributions and cash which is matched by the EU Commission. Project include multi-stakeholder platforms – such as for MAPPS Research project – such as «Big data for better outcomes» Get Real – Real world data Methodologies for patient preference elicitation in development and assessment

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