8:55 am Chair’s Opening Remarks

INCORPORATING THE PATIENT VOICE INTO CLINICAL TRIAL DESIGN AND CONDUCT

9:00 am Interactive Workshop: GPS for Advocacy in Gene Therapy

  • David Rintell Head of Patient Advocacy, BridgeBio
  • Mandy Rohrig Associate Director, Patient Advocacy BridgeBio Gene Therapy, BridgeBio

Synopsis

  • Identify shared goals between industry and disease communities at every stage of drug development
  • Develop ideas for patient engagement opportunities from initial engagement through commercialization
  • Problem solve common barriers and obstacles that can challenge patient engagement opportunities and community relationships at different stages of drug development

9:30 am Streamlining the Informed Consent Form Development Process

Synopsis

• Strategies for incorporating patient, caregiver, patient organization and healthcare professional input into the ICF development process

• Ensuring a balance is struck between the consent process being both comprehensive and comprehensible

• Optimizing patient friendliness throughout an ICF, equitable with all health literacies

10:00 am Morning Break & Networking

THE IMPORTANCE OF EARLY PATIENT ENGAGEMENT

10:45 am Ensuring Patient Experience Data is Accepted by the Regulatory Agencies

Synopsis

• Understanding the importance of early patient engagement to strengthen the case

• Highlighting best practices for PFDD meetings

• Exploring how patient engagement can be successfully demonstrated in various regulatory submissions

11:15 am Mastermind: Creating an Open Dialogue Around Key Patient Concerns

Synopsis

This mastermind will give each group the same set of questions surrounding this idea, and then answers will be collated at the end of the session and distributed to all attendees post-conference to gain insights into current perspectives in the field, and suggestions and ideas of relevant actions we can take as a collective to navigate the informed consent process in gene therapy.

Topics to cover include:

• Practical insights into establishing early on what the key patient concerns are for your target population

• How can we ensure patients are well informed on the immunogenicity risk of gene therapies before enrolling on a trial?

• In turn, how can industry make meaningful decisions based on established patient concerns?

12:15 pm Lunch & Networking

DEVELOPING A PATIENT-CENTRED STRATEGY TO ENTER THE CLINIC

1:15 pm Panel Discussion: Effective Clinical Trial Design for Optimizing Patient Enrollment

Synopsis

• Key considerations for disease modalities where alternative treatment options exist

• Is there a way to incorporate into your early clinical studies, sham and placebo control arms, without dissuading prospective trial participants?

• Strategies for navigating apathy towards enrolling in gene therapy dose ranging studies

• Methods for improving patient adherence to long-term follow-up studies

2:00 pm Working Together on the Design of Clinical Trials

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

Synopsis

• Learning from natural history and registry data

• Working with various stakeholders and means to collecting insights

• Aligning trial design across regulators, researchers, investigators, and patient communities

2:30 pm Ensuring Effective Representation in Gene Therapy Clinical Trials

Synopsis

• Highlighting the practicalities of engaging with the patient community early to establish what ‘diverse’ looks like in your specific patient group

• Strategies to incorporate diversity into patient recruitment for rare disease clinical trials where patient numbers are inherently low already

• How can biopharma (re)build trust when engaging with historically marginalized patient communities?

3:00 pm Afternoon Refreshments & Networking

OUTLINING THE ROLE OF PATIENT ADVOCACY IN COMMERCIAL CONVERSATIONS

3:30 pm Investigating the Market Access Dynamics of Gene Therapies & Resulting Communication With Patient Groups

  • Thomas Bols Head of Government Affairs & Public Policy, PTC Therapeutics

Synopsis

• Understanding patient concerns with respect to ease of access to gene therapies

• Which strategies are best to ensure the patient communities understand how gene therapies will be accessed?

• Bringing the patient voice into financing models

• What role do industry have in sharing and conveying this information?

4:00 pm A 101 in Patient Communication When First to Market

Synopsis

• Holding educational conversations with the patient community over tackling prior authorizations and potential coverage denials

• Outlining the opportunities for a community to engage with payors

• Developing well-designed patient services and associated infrastructure ahead of commercialization

4:30 pm Chair’s Closing Remarks

4:40 pm End of Conference