Pre-Conference Workshop Day

9:00 – 11:00 am
Workshop A

11:00 am Exploring the Pillars of Good Patient Communication with Gene Therapies

  • Nan Doyle Associate Director, Patient Insights - Rare Diseases Drug Discovery Unit, Takeda
  • Sara Johnson Davis Senior Director, Patient Advocacy & Government Affairs, CRISPR Therapeutics


Our global healthcare system was designed for chronic treatment options. As such, there is an inherent knowledge gap for patient communities to bridge between traditional prescription products and the complexities of novel gene therapies. Informing patients on the methods of action, risks and benefits; managing and setting expectations, and tailoring educational pieces to different health literacies are complex challenges patient engagement departments must navigate for successful communication strategies.

Attend this workshop to learn about:

• Developing a wide variety of educational materials for different patient groups

• Empowering the patient community to identify, set and lead on their care priorities

• Addressing and creating an open dialogue around key patient concerns

• Experiences with common patient perceptions around gene therapies, and how to tackle these

• Forging relationships with patient advocacy groups to leverage their patient insights

• Working effectively with communication service providers to convey complicated information is an easy-to-understand manne

11:30 – 1:30 pm
Workshop B

11:30 am Understanding the Role of Patient Input for Patient Identification & Clinical Trial Recruitment

  • Craig Martin President & Chief Engagement Officer, Rithm Health


With the gene therapy space geared towards rare disease, inherent challenges emerge in finding, recruiting and retaining patients for clinical trials. Given this, how can drug sponsors engage with patient communities to understand the patient decision drivers and incentives for enrolling, as well as finding patients and mapping their healthcare journeys.

Attend this workshop to learn about:

• Collaborating effectively with patient groups on finding patients

• Strategies for patient journey mapping to locate patients

• Making use of patient input to streamline recruitment processes, and understand what patients need and want to make an informed decision on enrollment

• Exploring the role of industry in progressing genetic testing and newborn screening

• Engaging with patients to determine support packages and incentives for a more accessible enrollment process

2:30 – 4:30 pm
Workshop C

2:30 pm Finetuning the Informed Consent Procedure

  • Jennifer Helfer Most Recently Head Patient Advocacy & Engagement, Encoded Therapeutics


A hallmark of an ethical gene therapy program is in the informed consent process. While the potential ramifications of introducing new genetic materials require a patient to have comprehensive understanding of risks involved, numerous factors must be considered, and approaches tailored around. Language barriers, differing healthcare literacies age, and decision drivers are just a few of many points to consider to assume consent is truly fully informed.

Attend this workshop to learn about:

• Understanding patient decision drivers to participate where no other treatment options exist

• Conveying all potential risks in both a comprehensive and comprehensible manner

• Engaging with patient communities to design consent forms

• In what ways can we supplement traditional informed consent forms?

• Where applicable, how can we tailor approaches to obtaining assent of children?