1. 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

2. 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

3. ....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

4. 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

5. 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.

6. 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.

7. 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

8. Thank you! EFPIA Brussels Office
Leopold Plaza Building * Rue du Trône 108
B-1050 Brussels * Belgium
Tel: + 32 (0) *