Post Conference Discussion Day
Clinical Operations Discussion Day
Thursday 23rd March 2023
9:20 am Chair’s Opening Remarks
Setting a Clinical Site up for Success
9:30 am 9:30am Key Considerations During the Site Selection Process for Rare Disease Trials
Synopsis
- Establishing whether a site will be able to handle the burdens of a clinical trial
- When conducting a clinical trial across numerous sites, how can sponsors maximise awareness of different regulatory timelines?
- Incorporating into decision making the country import/export licenses and abilities to handle equipment needs
10:00 am Practical Considerations For Clinical Site Preparedness: Perspectives From Pharmacy
Synopsis
- Defining risk groups and biosafety levels for gene therapy handling and containment
- Providing a tool for assessing the risk in handling gene therapies
- Discussing elements of the practical considerations involved in sponsors effectively preparing gene therapy pharmacy teams
10:30 am Morning Refreshments
New Models for Clinical Trial Recruitment and Retention
11:00 am Mastermind: Choosing a CRO for Clinical Trials
Synopsis
This mastermind session gives different groups the same set of questions to answer on designing clinical trials to support expedited approvals of gene therapies, exploring in-depth the below considerations. Following this, insights from different groups will be collated into a single document and circulated to the entire group.
Topics to be discussed include:
- Developing a checklist of key criteria to weigh up when presented with numerous potential CROs to partner with
- Key research considerations when doing your due diligence: how to get the selection right the first time
- How to ensure your relationship with a CRO is bilaterally commercially viable
11:45 am Developing Novel Approaches to Patient Recruitment for Rare Disease Trials
Synopsis
- Leveraging aggregated genetic testing data from natural history studies to increase patient recruitment numbers
- Addressing public perceptions issues surrounding this approach and highlighting an ethical, regulation-abiding path forward
- Insights from a case study utilising genetic testing to successfully recruit patients for clinical trialsÂ
12:15 pm Levelling up Patient Enrolment and Retention
Synopsis
- Showcasing success in scaling up patient recruitment
- Exploring various incentives to boost long-term patient retention in light of their gene therapy treatment showing initial success
- Redefining the future of rare and ultra-rare disease trials through the use of home clinical trial support and e-recruitment tools
- Analyzing the patient community viewpoints on trial recruitment: a patient advocacy perspective
12:45 pm Lunch & Networking
Removing Barriers to Successful Clinical Trials
1:45 pm Panel Discussion: Clarifying Key National Regulatory Requirements to Begin Clinical Trials
Synopsis
- Amassing an inventory of key process requirements for successful clinical trial applications across a select number of countries popular for rare disease trials
- A high-level viewpoint of different timescales, process, and key filing differences for these countries
2:15 pm Impact, Challenges and Differences in Establishing Pre- Approval Patient Registries and Natural History Studies for Rare Disease Drug Development Clinical TrialsÂ
Synopsis
- Registries vs natural history studies – the differences and how each contributes to the drug development process
- Understanding the associated benefits in better describing the natural history of a rare disease to inform the drug development process
- How to effectively incorporate multiple stakeholder perspectives when planning and designing natural history data collection efforts
- Lessons learned from the approval of a gene therapy where a retrospective natural history study was the basis of the approval
2:45 pm Chair’s Closing Remarks
Want to View Conference Day One & Two?
