Pre-Conference Workshop Day | October 23rd 2023
Pre-Conference Workshop Day
Monday 23rd October 2023
9:00 am – 11:00 amWorkshop A
Approaches & Opportunities to Bring Real World Data Threads Together to Meet Use Cases in Clinical Development
Synopsis
The sheer volume of existing RWD presents a large but overwhelming opportunity to leverage different sources to inform and innovate drug development programs. Alongside using data sources individually, the opportunity to bring multiple RWD threads together is a new and emerging strategy to take a holistic approach to utilizing RWE for developing a more efficient and effective clinical development program in the increasingly cost constrained payer and HTA environment.
Attend this workshop to learn:
• What the different RWD threads available to you are for informing early clinical development
• When is it appropriate to bring these data threads together / what are the use cases?
• What strategies are available for incorporating multiple data threads together to meet these use cases – e.g. linkage / tokenization / multiple coordinated analysis
• How can you optimize your ROI on your data investment?
11:30 am – 13:30 amWorkshop B
Current Challenges & Future Considerations – Leveraging the FDA’s External Controls Draft Guidance to Execute Clinical Trials with External Control Arms
Synopsis
Following the guidance on the use of RWD to develop RWE for regulatory decision making, in February the FDA expanded upon this to release the draft guidance ‘Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products’ or the External Controls Draft Guidance. However, although the guidance shines a light upon the potential to use a synthetic control group in clinical trials to demonstrate safety as well as substantial evidence of effectiveness for a new drug, there are still many uncertainties surrounding the applicability of external control arms outside of rare diseases and the ability to utilize regulatory grade data to ensure acceptance by the FDA.
Attend this workshop to learn:
- Statistical Considerations for the Design and Conduct of Externally Controlled Trials
- Highlighting international work currently being done to harmonise guidance and best practices regarding real world data (RWD)
- Reviewing the importance of randomisation as well as statistical assumptions like causal inference and assay sensitivity. Going over considerations for assessing quality and comparability of data, with a particular focus on RWD
- Exploring statistical analysis, such as bias, confounding, and missing data, and how these can affect the interpretation and generalisability of the study results.
- Focusing on the FDA Draft Guidance: “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” but will speak to other related guidance's as well.
14:30 am – 16:30 amWorkshop C
Exploring End-to-End Uses of Tokenization for Collecting, Linking & Analyzing Data to Generate RWE
Synopsis
Tokenization techniques provide clinical teams with the opportunity to save both time and money by removing the need to wait for sufficient product uptake in the real world and expensive open label extension trials to generate data. However, despite the ability of novel approaches like tokenization to provide payers with RWE on the safety and effectiveness of therapies, their uptake across the pharmaceutical industry is still lagging.
Attend this workshop to learn:
• About the concept of tokenization and how to develop informed consent, taking into consideration the privacy and legal implications
• How you can execute tokenization as part of your clinical trial and which partners you require
• How you can analyze tokenized data and what types of analyses are available