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


  • 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


  • 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


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


  • 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 trials 

12:15 pm Levelling up Patient Enrolment and Retention


  • 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

  • Melody Dai Senior Director, Regulatory Affairs Gene Therapy, Adverum Biotechnologies
  • Rocio Beneitez Regional Regulatory Affairs Head of New Product Strategy, Novartis


  • 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 Trials 


  • 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?