New Approaches to Drugs Value Demonstration in the Age of Big Data: Anatomy of a Paradigm Shift
“In God we trust. All others must bring data”
With the development of advanced medical therapies, for targeted or orphan diseases, the need for data generation has never been more important. Payers, HTA bodies and Decision Makers increasingly need evidence beyond Randomized Clinical trials (RCT), an artificial if rigorous way to demonstrate causality. Hence the efforts to collect data from registries, pragmatic trials, claims databases - to provide real world evidence (RWE) once confined to anecdotal evidence or nice-to-have complementary data.
The real questions are: what is like to bring the evidence needed? Can RWE be label enabling? How to implement this approach in the lifecycle of a product? Recently, regulatory bodies have indicated their interest for non-RCT data, not only for safety purposes but also for establishing effectiveness and the risk-benefit of a medical intervention. One other key aspect, especially for rare diseases that may not have a broad evidence basis at the point of registration is to understand and define the role of real-world evidence data in developing new reimbursement systems that allow or coverage with future evidence development.
In the pharmaceutical industry we have witnessed the remarkable advent of new methodologies and statistical approaches to extract data from apparently confusing datasets and to run more sound and reliable comparative analysis. From network meta-analysis to propensity score matching, from disease area models to discrete choice experiments, we have never been more equipped to capture signals from a background noise. Therefore, the need for harmonization, new and better data capturing and analytical technologies as well as more dynamic reimbursement systems that take the evolving evidence base from real life patient experience into consideration, is more and more important as the 21st century medicine is now based on big data rather than only RCTs.
Recent advances and perspectives in this field will be shared during the meeting, with an emphasis on practical aspects and how-tos. Payers, health economists, pharmaceutical physicians, trialists and regulators will share their knowledge with practical examples triggering engaging discussions and guiding participants’ efforts in the jungle of methods, designs and analyses that are now available. The objective is to help you finding solutions to the many challenges that a cross-functional development team must solve to maximize the new opportunities ahead of us.
If you’d like to know what is the EMA strategy on registries, what’s the role of big data in approval decisions or what are hybrid designs, this event is for you!
See you in Barcelona
Data will be provided Mr. Deming!
The EPI Forum Scientific Committee
Christian Agboton, Sr Global Brand Medical Director at Takeda
Michele Intorcia, Health Economics Outcomes Research (HEOR) CoE Head at Vifor Pharma
Ulf Staginnus, SVP Global Market Access & Pricing at Ipsen Pharmaceuticals
Who should attend?
The event is addressed to professionals with a deep interest in drug development, healthcare decision making, academia and healthcare research, belonging to department such as:
• Medical Affairs
• HTA/ Market access
• Medical Informatics
• Clinical Operations
• Regulatory Affairs
• Biostatistics and Data Management
from Pharmaceutical, Biotechnology and Medical Device companies, CROs, Universities/Hospitals, Academic Research, Patient Associations and Healthcare Organizations.