Pre-Conference Workshops
Workshop A: Beginning Patient Engagement in Early Stage Gene Therapy Development
Tuesday, March 23 | 8.00am - 11.00am EST | 5.00am-8.00am PST
When patient engagement is mentioned in the context of gene therapy, often clinical development is what first comes to mind. However, it’s becoming increasingly evident that as more companies gear up for clinical trials, patient engagement beginning several years prior to the first in-human dosing provides companies not only with an excellent understanding of the patient community, but also equips them with the tools they need to build and implement a successful trial. This workshop will delve into how the patient voice can be incorporated prior to clinical trial recruitment to set your program up for success.
Attend this workshop to learn about:
• The importance of patient engagement at research and development stages
• Exploring strategies to effectively gather patient input right at the beginning of gene therapy development
• Managing expectations on early stage development
• Understanding how patients live with their disease
Workshop Leaders:
Erin Frey
Senior Director of Advocacy
CureDuchenne
Susan Walther
Director of Patient Engagement
Friedreich’s Ataxia Research
Alliance
(FARA)
Nan Doyle
Associate Director, R&D Patient
Engagement – Rare Genetic and
Hematology TAU & PDT
Takeda
Workshop B: Delivering Quantifiable Metrics in Patient Advocacy for Gene Therapy
Tuesday, March 23 | 11.30am - 2.30pm EST | 8.30am - 11.30am PST
Determining ‘success’ of a patient advocacy team in gene therapy is challenging, as patient advocacy does not align well with a ‘Return on Investment’ way of thinking. Determining what is working well (as well as what is not) ultimately decides where to focus both time and investment in the future, so being able to effectively assess this in patient advocacy is key for any gene therapy company. This workshop will discuss what ‘success’ means in the context of engaging patients in gene therapy development and develop tools that can be
implemented in your own gene therapy patient engagement strategies.
Attend this workshop to learn about:
• Altering thinking from ‘Return on Investment’ to ‘Return on Partnerships’
• Identifying at what stages metrics can be introduced
• Exploring different tools which effectively determine performance
• The role of qualitative vs. quantitative data
Workshop Leader:
Fernanda Copeland
Executive Director,Global Head Patient
Advocacy & Engagement
AVROBIO
Workshop C: Making the Clinical Trial Participation Process Accessible for Patients
Tuesday, March 23 | 3:00 - 6:00pm EST | 12:00pm - 3:00pm PST
The phrase ‘reducing the patient burden’ is something the gene therapy community are familiar with but is not always easy to effectively implement from a company perspective. Making the patient journey easier throughout their clinical trial participation is important for patient
wellbeing, as well as actively demonstrating that a company really does put the patients at the center of what they do. This workshop will discuss the key ways that gene therapy companies can positively influence the patient experience throughout their clinical trial journey.
Attend this workshop to learn about:
• Aligning on expectations from the outset
• Supporting logistical considerations, including travel, accommodation, and any other requirements
• Speaking with the patient community to understand what specific needs they will require while at the trial site
• Developing local/at-home follow-up assessments wherever possible
Workshop Leader:
Sarah Friedhoff
Senior Manager, Patient Advocacy and
Clinical Affairs
Abeona Therapeutics
