Day Two - Thursday August 21

8:55 am Chair’s Opening Remarks

Integrating Methods for Early Patient Participation to Optimize for a Patient-Therapy Fit

9:00 am Fireside Chat: Addressing the Practical Needs of Patients During Long-Term Follow Ups to Ensure Patient Retention

  • Pat Furlong Founding President & Chief Executive Officer, Parent Project Muscular Dystrophy
  • Desiree Wienand National Director - Corporate Engagement, Lupus Foundation of America

Synopsis

  • Learning from patient organization leaders on overlooked challenges for consistent regular check-ups to highlight areas of improvement in the industry’s follow-up strategy.
  • Suggesting new and improved strategies that incorporates the needs of patients within reason to bring alignment to patient organizations and patient engagement.
  • Showcase experiences of good-practice support during long-term follow-ups to confirm the applicability of these new strategies.

Investigating New Strategies Using Patient Voices to Increase Therapeutic Access

10:00 am Parent Interview Session: Exploring How Patients Decide on Joining Cell & Gene Clinical Trials to Optimize for Better Decision-Making Tools

Synopsis

  • Understanding a parent’s thought-process when deciding whether her son should join a clinical trial to identify where industry can provide valuable support
  • Critiquing the industry’s weakness within there shared decision-making strategy to elevate the standards of support to patients considering clinical trials
  • Suggesting patient focused feedback for closer patient partnership to help elevate the anxiety of joining a clinical trial.

10:30 am Morning Refreshment Break & Networking

11:00 am Collaborating with Patient Groups to Tell the Right Story to Payers & Insurance Companies

Synopsis

  • Showcasing benefits of early patient engagement to increase accessibility
  • Developing improved strategies to integrate patient caregivers’ circumstances into payer discussions
  • Introducing new health economic approaches to showcase the holistic importance of access 

11:30 am Learning How to Create a Coalition to Cultivate State Policy Change

  • Erin Frey Senior Director, State Government Affairs, Ultragenyx Pharmaceutical Inc.

Synopsis

  • Exploring the issues being considered by state legislators and the value of a rare stakeholder coalition to impact access through state legislation
  • Evaluating the right people and characteristics to identify coalition members
  • Discussing the best practice of creating a cohesive team to create an efficiently working coalition

12:00 pm Lunch Break & Networking

1:00 pm Fireside Chat: Exploring the Value of Patients Voices to Inform the Development Process & Future Probability of Therapeutic Access

  • Michael Sherman Chief Executive Officer, Sherman Health Systems
  • Wendy Erler Senior Vice President - Patient Affairs, Sarepta Therapeutics

Synopsis

  • Leveraging the experience of patient advocacy group experiences to optimize therapy access
  • Aligning the needs of payers with the experiences of patient groups to satisfy payer high criteria for access
  • Understanding the common pitfalls of unsuccessful access requests caused by the lack of patient centricity to avoid poor practice of patient engagement

Evaluating Best Practice for Patient Engagement & Advocacy for Effective & Patient Centric Collaboration

2:00 pm Afternoon Refreshment Break & Networking

2:30 pm Audience Workshop: Spearheading New Approaches in Quantifying Cell & Gene Therapy Patient Engagement to Showcase Patient Engagement’s Critical Value

  • Adam Clark Director of Foundation Operations, Emily Whitehead Foundation

Synopsis

What is an Audience Workshop? Our brand-new approach to advancing meaningful measurement of cell & gene therapy patient engagement. Join Adam Clark (Emily Whitehead Foundation) as he explores unconventional ROI metrics, followed by a collaborative discussion on the current efforts and future opportunities.

  • Identifying the unique challenges of quantifying cell and gene therapy specific patient engagement
  • Contrast the different data collection methods between early phase and later phase
  • Propose new metrics and ways of presenting data to stakeholders to showcase business impact of patient engagement

3:30 pm Understanding How to Optimize the Collaboration Between Patient Engagement & Clinical Operations to Recruit & Retain Ideal Cohort

  • Mary Rohrer Director - Patient Recruitment & Retention, BridgeBio Pharma, Inc.

Synopsis

  • Dealing with the challenge of patient engagement and clinical operations professionals operating in different locations to explore potential resolutions
  • Aligning skillset between these two professionals to maximize patient recruitment match and retention
  • Collaborating patient engagement and clinical operation professionals to operationalize the task of patient recruitment efficiently

4:00 pm End of Conference Day Two