Post Conference Discussion Day

Clinical Operations Discussion Day

Thursday 23rd March 2023

9:15 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 trials 

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

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

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 and key filing differences for these countries
  • How to navigate different requirements across multiple domestic internal review boards to avoid significant delays.

2:15 pm Impact and Challenges in Establishing Patient Registries for Rare Disease Clinical Trials

Synopsis

  • 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 patient registries
  • Top tips for incorporating patient registry data to inform and develop more patient-centred clinical trials

2:45 pm Chair’s Closing Remarks

Want to View Conference Day One & Two?