Rob is a former All-American punter and has lived the rare disease journey. He has a steadfast commitment and connection to the Rare Disease Community as a rare brain cancer survivor. As a former star college athlete on a path to the NFL, his passion and drive to advance the mission of Uplifting Athletes is inspirational. Gain encouragement from Rob’s success in building innovative programs that seek to advance rare disease research and advocacy while uplifting patients and families.

• Build the next generation of rare disease advocates
• Provide unique experiences for individuals and families impacted by rare diseases
• Encourage and celebrate researchers of rare diseases

A patient-centric approach to clinical development is no longer enough. To truly engage with patients, the industry needs to adopt a patient-first culture. By listening to the needs of patients’ and caregivers’ and putting them at the forefront when designing and operationalizing clinical trials, we make trials more accessible and improve the patient experience. This session will discuss:

• The evolution to a patients-first culture
• How to reinforce this culture both internally and externally
• Tangible actions you can take to develop a patients first approach in your organization.

Build relationships with the chief executive leaders of our industry to ensure patient and corporate goals are aligned. Be prepared to raise priority issues when you have the opportunity. A panel of cross-functional executive leaders share insight into what they see in patient advocacy and why they are champions.

• Discuss the value that advocacy brings to the organization
• Learn what steps to take when more executive involvement is needed
• Assess approaches to conversations about funding
• Consider ways to build bridges

The Inflation Reduction Act and recent advances in FDA’s patient focused drug development create important new challenges and opportunities. Gain an understanding of how the implementation of policy and regulatory changes impact patient advocacy.

• Unravel the Inflation Reduction Act and what it means for patients
• Get an update on patient focused drug development and FDA’s recent initiatives
• Implement FDA guidance on clinical trial diversity
• Examine what’s on the horizon for patient generated data
• What is the ethical role of advocacy in FDA discussions

Increasing diverse representation in clinical trials involves overcoming several barriers – such as education, access, awareness, and equity. Diverse representation in clinical trials is imperative so all patients can be treated most effectively. The biggest challenge to overcome is patient mistrust and misunderstanding. Credible patient and community-centered health education is a powerful means to break through this challenge and build trusting relationships between providers, researchers, and under-supported populations. In this timely discussion, expert panelists will share:

• The importance of patient voice and diverse representation in health education and clinical trial experience
• Success stories from recent digital education programs centered around diversity in clinical trials
• Insights gained through patient and provider educational programs and the ability to activate diverse communities to learn more and engage in the clinical trial process.

• Obtain the latest information on ICER’s value assessment process
• Hear what ICER recognizes in patient reported outcomes and the patient journey
• Help advocates begin to prepare to participate in value assessments
• Hear lessons learned from engaging in past ICER value assessments

See a comprehensive view of touchpoints for patients by our patient access teams to ensure you have the complete picture. Pinpoint compliance risks in patient interactions while the focus on patient centered programs increases.

• Visualize what patient engagement through Patient Ambassador Programs looks like and whether it overlaps with patient advocacy
• Describe ways to address health equity and identify missed opportunities
• Explain the role of HUBs in helping patients gain access to needed medicine
• Hear about OIG’s recent scrutiny on patient interactions and how this impacts you
• Conduct materials review

Clinical trials for gene therapies are on the increase and require additional education awareness and thus collaboration of multiple stakeholders. The decision to participate in a clinical trial is enormous and requires significant commitment for patients and families. As advocates, in addition to providing education we are working to improve the clinical trial experience by listening to patients and taking action to implement their feedback.

• Discuss the method of multiple stakeholder engagement to ensure clinical trial success
• Understand the patient community in the geographies where they exist and communicate that landscape to clinical research teams
• Work with patient advocacy organizations worldwide to raise awareness and potential opportunity to participate in a clinical trial
• Review the design protocol and give feedback to clinical research
• Measure how much of patient feedback on clinical trials is being put to use
• Outline ethical considerations and conflicts of interest for patient advocacy and clinical trial recruitment

Choose among 6 dynamic, concurrent working groups. The interactive working groups are designed to optimize peer-to-peer learning by crowdsourcing solutions to common challenges and brainstorm new, innovative initiatives. The connections you make will become a valuable takeaway.

Realize Self-Care for the Patient Advocate

Working with patient communities requires enormous empathy. You are often facing difficult questions requiring complex answers and you are so invested in your work that it may be draining. Here is your reminder that self-care is crucial to your success professionally and for life in general.

• How to spot fatigue
• Recognize that your energy isn’t endless
• Acknowledge both the possibilities and potential limitations in particular initiatives
• Reverse the danger of burnout and rediscover the commitment to the important work you do

The goal is to design a framework that brings value to the patient community, the patient organizations and internal collaborative partners. Whether you are starting or scaling a patient advocacy function, you’ll benefit from a strategic discussion on modeling.

• Start with the end in mind and understand what’s important to the patient community as well as internal stakeholders
• Scaling up – when to hire for what or leverage consultants
• Keep cross functional communication and support fine-tuned

Incorporate Patient Voice in Pre-Clinical Research

Improve working with patients in pre-clinical research. Learn more about what early engagement looks like and how to begin.

• Learn best approaches for getting started early
• Assess missed opportunities for when advocacy comes later to the party
• Tips for identifying important stakeholders in pre-clinical

Best Practices for Relationship Building with Patient Advocacy Organizations

Discover the results of a survey of patient advocacy organizations about how they would like to engage with industry

• Identify what patient communities need and what their parameters are
• Gain tips on how to listen first and come to the table to co-create
• Discuss some ideas for managing difficult discussions

Working with Grassroots & Community-Based Organizations

As many patient advocacy groups are shifting from a primarily national focus to more local and community-based initiatives, you’ll learn how to:

• Expand your advocacy function to meet local and regional needs
• Convene functions across your organization to collaborate at the local level
• Build trust with marginalized & underserved communities to advance equitable health outcomes

Expand Capabilities in Health Literacy

After an introduction of health literacy and BMS’ Universal Patient Language, this group will break into subsets and work through questions to improve patient communication across the organization.

• Gain insight on how to strategize health literacy initiatives
• Hear about how and why the Universal Patient Language capability at BMS was built
• See how UPL helps with the informed consent process

Amy Dockser Marcus is a health and science reporter for The Wall Street Journal. Amy's articles depict how scientific advances are transforming society and raising bioethical questions. In 2005, Amy won a Pulitzer Prize for a series of stories about cancer survivors and the social, economic, and health challenges they face living with the disease.

She is also the author of the 2023 book, We the Scientists: How A Daring Team of Parents and Doctors Forged A New Path for Medicine, which chronicles an innovative partnership between parents of children with a rare and fatal genetic disease, scientists, and doctors, who all work together in the search for a cure. Their collaboration, where everyone is recognized as an expert, offers a model for accelerating research and drug development in rare diseases and wrestles with ethical and social questions that must be addressed in order for science to move forward.

Gain a roadmap following the molecule from start to finish and how it changes from positive data through phase 2, 3, and preparing for launch. Hear directly from patients and caregivers on what they need from BioPharma through every step of the process.

• Breakdown silos between functions
• Best practice sharing early through commercial
• Following patients and therapies from start to finish
• How to change patient engagement throughout the R&D cycle for the benefit of patients, caregivers, and the business

Now more than ever the teamwork between commercial and advocacy teams is critical. Throwing projects over the wall will not work and early engagement of commercial partners is key.

• Get involved early with brand teams to achieve unified brand messaging
• Leverage opportunities to hear and communicate better
• Examples of successful relationships

Patient advocacy professionals share a mission to do what is best and what is right for patients. But patient advocacy departments in industry are often small and patient-focused drug development and community engagement strategies can be deprioritized in the larger organization. Committing to patient centric research requires buy-in from leadership and cultivation of advocates in every function. In this session gain tips to:

• Unite cross-functional teams in defining minimum expectations for patient input
• Establish patient advocacy as a strategic partner in the development and execution of patient input and engagement strategies
• Create internal tools and guidance to facilitate input and patient-centric research
• Communicate expectations and lessons-learned within an organization

The biopharma/medtech industry undertakes numerous projects and initiatives to amplify patient awareness. Hear about how to get a good idea off the ground.

• Case example of Sanofi’s TORCH Awards
• Take risks on ideas
• Prepare strategy, launch and test of ideas that break out of the box
• Examine benefits, risks, costs, human resources and reasons why innovative programs are worth it

Lilly Stairs, 3x autoimmune patient turned advocate will tell her inspirational story from the hospital bed to the boardroom. She will harness her insights from working across the healthcare ecosystem to help attendees unlock novel ways to empower and educate the patient community.

Her keynote will address:

• Tangible examples of effective programming that goes beyond patient panels
• Patient advocacy's critical role in integrating technology and innovation into treatment and care plans
• Ways to quantify the value of patient advocacy and translate metrics into conversations across functions in your organization

Understanding and managing the nuances of funding and finances are an integral part of your role.

• Strategizing for both the short and long term
• Honorarium, fair market value, consulting services, and more when working within the patient community
• Making the case to growing the team
• Optimizing activities on a shoestring budget

Evaluate ways to improve the impact of clinical trial awareness and education within diverse patient populations.

• Discuss how companies are putting forth diversity action plans
• Review the most urgent challenges in specific disease areas
• Create your strategy for reaching underserved patient populations
• Discuss FDA Guidelines
• Hear about multistakeholder initiatives to increase diversity in clinical trial

Generating real-world data is one method used to collect Patient Experience Data. What is the future for how we look at patient experience data and engage patients in the generation and the use of real-world data?

• Identify steps for engaging patients in the co-design and contextualization of data
• Assess how patient experience data may offer a bridge between patient advocacy and other functions
• Discuss the potential for greater emphasis on having patient engagement and patient experience data in future regulatory submissions