JEAN CAMPBELL is nationally recognized as one of the leading rare disease patient advocates with an impressive record of success in fundraising, patient advocacy and corporate relations. Jean served nearly two decades with the National Organization for Rare Disorders (NORD) in senior management positions including Vice President of Development. A well-respected resource in the rare disease community, Jean formed JF Campbell Consultants LLC, in 2010. Her vast expertise is valuable to all size companies and organizations. Jean’s consultancy has focused on the importance of creating, maintaining and advancing the patient advocacy function within the industry setting. Sharing the patient advocacy ‘tools of the trade’ which includes how to build effective relationships, delving into organizational landscapes and conducting patient advisory boards are just some of the ways that this function’s roles and responsibilities bring value and collaboration to a company’s internal goals. Jean is a co-founder and board member of the Professional Patient Advocates in Life Sciences (PPALS), which was created to meet the ever-growing needs of the ever-evolving patient advocacy function within industry. She coordinates PPALS programs as well as its annual Patient Advocacy Certificate Training (PACT) held in partnership with Sanford Research. Jean is a member of the Rare Collective and serves on the Erdheim-Chester Disease Global Alliance Board of Directors. Jean enjoys serving on MedunikCanada’s Advisory Board, Exceptional Parent Editorial Board, Sanford CoRDS’ External Advisory Board, and Our Odyssey’s Board of Directors. In May 2021, Jean was named a Patient Advocate Hero by Exceptional Parent Magazine during its 50th Anniversary celebration.

As the Global and US Lead for Multiple Myeloma Patient Advocacy & Engagement at Takeda Oncology, Scott embodies the role of a true Patient Advocate – a trusted liaison between patient organizations and industry, as well as other stakeholders who shape the patient experience. From research & development to access and support services, Scott champions the voice of the patient at every step of the process of delivering potentially life-transforming therapies for patients with the greatest unmet needs. He uses real-world patient insights and patient experience data to design impactful education programs and help shape decisions in the industry. Scott also leads the US field-based team of Patient Advocacy Liaisons, a program aimed to uncover real, first-hand insights from the multiple myeloma community and create meaningful educational engagements for cancer patients and their loved ones. Scott is driven by enthusiasm, empathy, and urgency in his role as the Director of Patient Advocacy & Engagement at Takeda Oncology. He strives to be actively present with the multiple myeloma community, whether that is from the office, attending patient education programs, or by participating in advocacy events like the Moving Mountains for Multiple Myeloma hike to raise funds for cancer research. Scott has been recognized by the McGivney Global Advisors and The Lynx Group in being named “40 under 40 in Cancer” as well as in the PM360 ELITE Awards for his part in leading the Takeda Oncology Patient Leadership Council. His passion for helping patients is reflected in his own words: “Who’s not inspired by the opportunity to make a cancer diagnosis even the slightest bit better?”

Jennifer Helfer is working within the advocacy community to positively disrupt the healthcare system to create access, transparency, and education so that gene therapy can become available to all those who can benefit. Jen joined bluebird bio in December of 2015. As part of the Patient Advocacy team, Jen serves as a conduit to patient advocates and advocacy organizations. The Patient Advocacy team is responsible for building and sustaining trusting relationships and collaborations with key stakeholders around shared goals to address patients’ and their families’ needs, as well as facilitate the integration of patient and caregiver perspective throughout the nest to inform decisions made at bluebird bio. Before joining bluebird bio, Jennifer was Associate Director of Scientific Operations at the National Brain Tumor Society, the largest nonprofit organization dedicated to the brain tumor community in the United States. Prior to finding her passion in advocacy, Jennifer engaged in basic research at Boston University Medical Center. Jennifer received a B.A. in Psychology from the University of North Carolina Wilmington, a M.A. in Behavioral Neuroscience from the University of Delaware, and a PhD in Biology, Neuroscience, from the University of Victoria.

Shortly after I was diagnosed with Systemic Juvenile Idiopathic Arthritis (SJIA) at age 11, my mother was told by our family’s health insurance plan that coverage for my treatment was denied because arthritis “was not on their list of approved childhood conditions.” From this point on, my childhood was marked by similar barriers to effective treatments - the consequences of which ultimately shaped my access to educational and professional opportunities into adulthood. Today, I’m the head of strategy and development for a health economics and policy research center driving a research agenda that elevates patient engagement in value assessment in healthcare - working to ensure that the barriers to treatments that I’ve experienced are addressed on a system level. I combine my expertise as a patient with my experience in program and project management to consult with healthcare start-ups, pharmaceutical companies, and other organizations offering a collaborative and integrated approach to incorporating patient values into healthcare design, technology, and policy reform.

Carrie has over 20 years of clinical drug development experience, including roles of increased responsibilities at small biotech as well as large pharmaceutical companies. Prior to joining X4, Carrie was Vice President Global Clinical Operations at TESARO and subsequently Vice President Global Clinical Sciences and Delivery, Oncology, after the acquisition of TESARO by GSK. Prior to joining TESARO, Carrie held positions at Kura, EMD Serono, Millennium: The Takeda Oncology Company, and Pfizer. Through her tenure, she has held various leadership positions responsible for worldwide clinical development, and has previously built development operations functions from scratch. She has led groups of 125+ associates spanning clinical operations, data management, biostats and programming, vendor management, site management and monitoring, contracting, and TMF management. Carrie has an MBA from Boston University and a BS, with Highest Distinction, in Nursing from the University of Rhode Island. Fun Facts about Carrie: She is a die-hard NE Patriots fan, a huge basketball fan – both professional and college basketball, and last, but not least, she is the biggest U2 fan you’ll ever meet. She has met Bono and The Edge when she was in high school and still has the Bono signed concert t-shirt to prove it! She is happily married (to David Melvin) and is the mother of two; her son, Jack, is in his sophomore year at WPI and is busy studying Biomedical Engineering and playing basketball as part of the WPI basketball team; her daughter, Haley, is in her Senior year of High School and is a Captain of the Varsity basketball team. She also has two rescue dogs – Thunder (male) and Lightning (female) who both keep the family busy and entertained.

Dr. Edward "Ed" Neilan is the Chief Medical and Scientific Officer of the National Organization for Rare Disorders (NORD). Ed is a physician-scientist and rare disease expert and he joined NORD in 2021 to lead its medical and research programs. Ed seeks to encourage and enable institutions and companies to develop innovative approaches and new treatments to help rare disease patients. He is an expert in clinical trial design and drug development, has contributed data that helped support the FDA and global regulatory approvals of five new rare disease therapies, and has authored or co-authored multiple clinical trial protocols and safety and regulatory reports to global health authorities. Prior to joining NORD, Ed worked at Sanofi Genzyme, a major biopharma company, where he led global medical affairs strategy and execution for the rare neurological diseases portfolio and contributed medical expertise to clinical development efforts across multiple programs. Prior to that, he served as the President of the Medical Staff at Boston Children's Hospital. As a staff physician, clinical geneticist, and the Director of Quality Improvement in the metabolism program at Boston Children's, Ed directly cared for and studied patients with many genetic diseases. After completing BS and MS degrees in Biology at Yale University, Ed earned his M.D. and Ph.D degrees at Stanford University. He completed residency and fellowship training at Harvard Medical School, where he subsequently served as a faculty member for more than 12 years. Ed is triple board-certified in pediatrics, clinical genetics, and clinical biochemical genetics. He is a fellow of both the American Academy of Pediatrics and the American College of Medical Genetics and Genomics.

As a member of the rare disease and cancer patient communities for over a decade, Michele Rhee, MBA, MPH, has dedicated her career to getting treatments to the patients who need them. Michele is a founding board member of Costs of Care and volunteers as a Sambassador for the Samfund. She received her MBA from the Yale School of Management and her MPH from the Yale School of Public Health. She is the incoming Vice President of Patient Affairs and Advocacy at X4 Pharmaceuticals.

As Head of Patient Advocacy, Stuart is responsible for advocacy strategy and community engagement globally across the Chiesi rare disease portfolio. He is committed to empowering those impacted by rare diseases by building trusted partnerships with them and their communities. Stuart has over 25 years of global advocacy experience, including roles as Global Director of Public Affairs & Patient Advocacy for rare blood disorders at Sanofi Genzyme and leading Global Patient Advocacy for Bioverativ Therapeutics’ precommercial rare disease pipeline. In addition, Stuart is the Cofounder and Director of the Sanfilippo Research Foundation—a nonprofit dedicated to research—in Sanfilippo Syndrome/MPS III, an ultra-rare inherited lysosomal storage disorder that affected his son Benjamin. Stuart is no stranger to the personal impact rare diseases can have on individuals and their loved ones. That is why he also serves on advisory boards and is an active member of numerous NGO, industry, and governmental rare disease groups and associations.

Jamie Sullivan recently joined the EveryLife Foundation for Rare Diseases as the Director of Public Policy after a spending 15 years with the Alpha-1 and COPD Foundations. Jamie’s work has focused on achieving patient-centered federal, state and regulatory policy changes in the areas of health appropriations, public health infrastructure and access to care. In her role at the COPD Foundation, Jamie also focused on building programs to engage and train patient advocates as well as advocating for the robust inclusion of patient and caregiver perspectives in all aspects of treatment development and review. Prior to joining the COPD Foundation, Jamie supported the research and clinical programs of the Alpha-Foundation. She obtained her master’s in public health policy and management at the Florida International University.

While playing football for Penn State in a game against rival, Ohio State, Adam Taliaferro made a routine helmet-to-helmet tackle that would change the course of his life. He had broken his neck—a similar injury suffered by the late, Christopher Reeve. Given only a 3% chance of ever walking again, Adam fought insurmountable odds to not only walk, but to walk well. Though playing football is no longer in Adam's future, many around the nation have followed his inspirational tale of recovery leading authors, Scott Brown and Sam Carchidi, to chronicle his remarkable story in their 2001 book Miracle in the Making. Adam currently leads State Advocacy & Alliance Development for Bristol Myers Squibb and holds office as a member of the New Jersey General Assembly. Adam also has his own foundation, “The Adam Taliaferro Foundation,” that provides financial assistance to individuals affected by spinal cord injuries of which has raised over $1 million to date. Through his foundation and his passion for motivational speaking, Adam continues to provide hope for many people who may feel that there is no reason to hope at all.

For more than 20 years, I have worked as a writer and provided editorial support and guidance and strategic consultation to medical nonprofits and pharmaceutical and biotechnology companies. I have extensive experience writing about rare disorders for both patients (in lay terminology) and physicians (in medical terminology). At one time, I oversaw the development and expansion of the National Organization for Rare Disorder’s Rare Disease Database. I understand the unique needs of patients in the rare disease community and work to foster better communication and understanding between these diverse groups and the industry representatives that work with them. My projects have included helping to start and guide a nonprofit organization, running a competitive grants program, and creating the content and format for a lecture series on rare disorders and identifying and engaing the physician experts. Professional Patient Advocates in Life Sciences (PPALS) I am part of the founding faculty for PPALS and work to build recognition of this vital role in life sciences and to foster better relationships between PAO leadership and the Patient Advocacy professionals who serve them. WHY RARE DISEASES? There are over 7,000 rare diseases. Many are genetic, many affect children. Most of these disorders do not have an FDA-approved treatment. I realized that my efforts could make a difference - however small - in a community that has been long ignored.

Rogerio Vivaldi, MD, MBA is the President and Chief Executive Officer of Sigilon Therapeutics. During his more than 25 years as a physician and an industry executive, Dr. Vivaldi has developed a reputation as a devoted advocate for patients.