Jean 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. Under Jean’s leadership, there was significant expansion of several of NORD’s individual and organization membership programs along with the creation of NORD’s restricted research, medical meeting, organization mentor and clinical broadcast programs. Jean played a key role in the establishment of the NORD Corporate Council, and was instrumental in the creation and supervision of the disease specific Patient/KOL Regional meetings. A well-respected resource in the rare disease community, Jean formed JF Campbell Consultants LLC, in 2010. Her expertise in patient advocacy, organizational governance, corporate relations, patient assistance programs and program management is valuable to all size organizations. Her clients include non-profit health organizations, pharmaceutical and biotech industries. Jean’s goal is to increase a client’s visibility in the health industry as well as create productive partnerships, effective strategic planning, and optimal program development. In addition, Jean provides valuable mentorship to start-up and growing patient organizations helping to strengthen their infrastructure in order to better serve their mission, members and communities.
Jean is a co-founder and board member of the Professional Patient Advocates in Life Sciences (PPALS), a member of the Rare Collective and an Erdheim-Chester Disease Global Alliance Board Member. Jean also serves on MedunikCanada’s Advisory Board, Exceptional Parent Editorial Board, Sanford CoRDS’ External Advisory Board, National Caregiving Council Advisory Board, and Atlantic Research Group’s Scientific Advisory Board. Jean has served as a grant reviewer for Global Genes’ Innovation Grants, the United Way of Western Connecticut and the City of Danbury, CT. Somehow she manages to find time to spend with her two granddaughters, Madeline and Juliette, her daughters and son-in-law, Meredith and Aaron and Liz, family, and friends. Rumor has it that she attends ballroom dance classes and is an avid movie-goer!

Katie is a seasoned communicator and patient advocate, applying her expertise to facilitate productive relationships with patient communities that advance business goals. As Executive Vice President, Managing Director at SmithSolve, she is responsible for creating and executing patient advocacy, education, engagement, and communication programs across clinical- and commercial-stage biopharmaceutical companies. She graduated with a B.A. in public relations from the S.I. Newhouse School of Public Communications at Syracuse University and received her Master of Business Administration degree from Montclair State University.

Tim has a background in nonprofit work having formerly run a nonprofit concert venue in CT. During his tenure he was responsible for repairing and restructuring the finances and operations at the Ives Center for the Performing Arts in Danbury CT, moving them from the brink of dissolution to a healthy operation. He currently lives in NYC where he works as consultant for pet related businesses. When he's not working he is spending time with the joys of his life, his two wonderful dogs Rebecca & Jacob.

David J. Eckstein, Ph.D., is the Program Director for Strategic Partnerships and Policy Development in the NIH Office of Clinical Research. He is responsible for oversight of the Bench to Bedside and Clinical Center Collaborative Research U01 Programs and is continuing his longstanding work on the Common Fund program, the Undiagnosed Diseases Network. Dr. Eckstein is also working to develop partnerships between the Clinical Center and nearby academic research centers and has helped develop policy documents around scientific review and resource prioritization for NIH clinical studies. Prior to joining the Office of the Director, he was a Health Scientist Administrator in the Office of Rare Diseases Research. During this time Dr. Eckstein initiated the first "Rare Disease Day at NIH" event in 2011 and led the planning for the first 7 annual events. He helped launch the NIH Undiagnosed Diseases Program and served as the point person for rare disease patient advocacy groups. Dr. Eckstein came to NIH in 1996 as a Technical Information Specialist in the Indexing Section of the National Library of Medicine. From 2000 to 2003, he served as a Program Analyst at the National Institute of Neurological Disorders and Stroke where, in addition to his analyst duties, he managed a portfolio of neuro-AIDS and prion diseases grants and contracts. After spending 6 months as a Congressional Fellow, working in a House member's personal office, Dr. Eckstein returned to NIH to serve as a Program Director in the Cancer Training Branch of the National Cancer Institute, managing a large portfolio of career development awards and individual and institutional training grants. During his time at NIH, Dr. Eckstein also has been selected to be an Excellence in Government fellow. Dr. Eckstein received a B.S. in zoology and a B.A. in anthropology from the University of Maryland, College Park, and a Ph.D. in cell biology/anatomy from the University of Texas Graduate School of Biomedical Sciences in Houston.

Dakota received her BA in biology from Bryn Mawr College in 2011 and a Familial Adenomatous Polyposis (F.A.P.) diagnosis shortly after. Between her F.A.P. and desmoid tumor diagnoses, statistically speaking, she's four in a million. Patient advocacy fills the void left by her cancerous colon and her job title. As Global Patient Advocacy Associate Director at BioCryst Pharmaceuticals, she ensures that the voices of rare patients and the non-profits that serve them are heard. She’s the co-founder of Young Adult Cancer Connection which hosts Cancervention, Philadelphia’s first young adult cancer conference, and the creator of F.A.P.ulousTV, the first YouTube Channel dedicated to discussing F.A.P.

Ben is Program Director, Strategic Partnerships & the CoRDS Registry Sanford Research. He has worked in the field of biomedical research since 2009. He spent nearly seven years working in a laboratory that focused on sensitive antioxidant biochemical pathways and worked on a project applying that knowledge to the rare disease, Friedreich's ataxia. Currently, Ben works to build and strengthen business relationships between the research scientists and physicians at Sanford Health and external groups in industry and academia. Ben also serves as a member of the CoRDS Registry team and works to make an impact in the rare disease community by connecting patients with researchers and gathering valuable patient data.

Jayne joined Amicus Therapeutics in 2006 and serves as Chief Patient Advocate, responsible for developing and executing the global strategies that ensure people living with rare diseases and their families remain at the core of all company operations. A long-time patient advocacy professional, Jayne leads the company’s highly regarded Global Patient & Professional Advocacy department, a function that ensures extraordinary patient dedication as the bedrock of Amicus since its earliest days as an R&D organization. Jayne founded the company’s Patient Advisory Boards program, which helps give voice to the concerns of patients, families, and caregivers, and leads Amicus’ Public Policy work to advocate for policies that satisfy unmet needs among those living with rare diseases, and initiated Healing Beyond Disease™, an Amicus initiative to further serve the rare disease community in extraordinary ways. She is a member of the company’s Executive Committee, reporting directly to the CEO, and sits on the board of directors. In February of 2019, Jayne received the second annual “Heart of BioNJ” Award in honor of her selflessness and dedication to patients in the rare disease community, and her renown role as a trailblazer in Patient Advocacy. She is a 2018 PharmaVOICE 100 honoree, recognized for providing inspiration and innovation in the life sciences industry. She is a member of the Boards of Trustees of BioNJ and of the Healthcare Institute of New Jersey and is a co-founder and vice chair of the board of Professional Patient Advocates in Life Sciences (PPALS). Jayne is the former executive director of National Tay-Sachs & Allied Diseases Association. A native of Medford, Massachusetts, Jayne graduated from Syracuse University with a dual degree in Newspaper Journalism and English Literature and studied marketing management at Radcliffe College. She and her husband, Bruce, reside in Lambertville, NJ; they have two adult children.

Polly Gill is the Associate Project Manager for the Coordination of Rare Diseases at Sanford (CoRDS). She has been working in the healthcare system for almost 10 years in various roles at Sanford. Polly works with advocacy groups/partners to build questionnaires and also works closely with the participants in CoRDS.

I discovered my passion for health care, patients and cancer care at a young age. My career started in 6th grade when I was a volunteer candy striper and transitioned into nursing and care of cancer and rare disease patients. Over the years my focus has been to work in a variety of roles on the healthcare team including the clinical setting, drug development for industry, facilitating health information and being a strong advocate for patients. These experiences have afforded me a broad and deep view into the health care delivery system’s capabilities as well as the unmet patient needs relative to life changing, chronic illnesses. Using my nursing background as a foundation, I have held positions and provided leadership in clinical nursing, clinical research and drug development, medical affairs, health information, and patient and professional education. While attending a post graduate program in health coaching at Georgetown University I learned to communicate using motivational interviewing, a style of communication which is less authoritarian, and focuses on a more self-directed approach. Using open, non-judgmental communications, this technique empowers the patient to make positive choices resulting in beneficial life changing behaviors. This method of communicating opened my mind to a less directive and more personalized approach to patients, all health care stakeholders and even my personal life! I get the greatest joy from my family and friends. I am married to a medical oncologist, have 2 children, 2 adopted dogs, a horse and a cat, and spend my free time horseback riding, organic gardening and cooking. I believe as Mahatma Gandhi said, “The best way to find yourself is to lose yourself in the service of others.”

Following a 23-year tenure as vice president of patient services at the National Organization for Rare Disorders (NORD) where she created a track record of success building, implementing, and managing over forty sustainable Patient Access Programs for Copay Assistance, Insurance Premium relief, and assistance with Travel expenses experienced by patient and families participating in clinical trials; as well as advising pharma/biotech industries in developing sound Expanded/Early Access programs for experimental therapies. Maria continues to work in the rare disease arena as Senior Advisor with JF Campbell Consultants offering valuable services and expertise in understanding OIG Oversight Compliance, Medicare/Medicaid changes, Co-pay Accumulators and other current issues to the pharma/biotech industries involved in developing therapies for orphan diseases; and to non-profit health organization that can assist in fulfilling their individual missions, making good governance decisions, and finding appropriate connections on which to build relationships with the biotech and pharmaceutical industries. She has advised and served on the faculty of Professional Patient Advocates in Life Sciences (PPALS) since its inception in 2016.

Nancy is a nationally recognized patient advocate with more than three decades of experience creating, leading, and directing patient organizations focused on rare diseases. Nancy views patient advocacy from many perspectives. She has been actively involved in many national organizations as a board member, an advisor and a leader at NORD, Global Genes, Child Neurology Foundation, WE MOVE , and the Best Pharmaceutical for Children's Act committee. She has been a consultant to the pharmaceutical industry for over 20 years but most importantly, she was the mother of a child with a devastating undiagnosed neurological disorder. Nancy brings a unique combination of empowerment and empathy to her work with patients, their families and caregivers, and the scientific communities. Her goals range from creating awareness and education, to assisting patient support programs and promoting the overall strategy to offer the best possible patient care. Nancy resides in California where she is a peer counselor, working with people in all areas of transition.

Nita is a registered nurse with experience in clinical research, obstetrics and genetic nursing. She has been working at Amicus since September, 2011. Her expertise in lysosomal diseases was gained from managing a lysosomal treatment center for approximately 15 years at St. Peter’s University Hospital Institute For Genetic Medicine located in New Brunswick, NJ. Nita has also provided training and education for patients in renal failure receiving peritoneal dialysis, pre-natal teratology counseling and testing. Nita has also monitored clinical studies in lysosomal disorders; her passion is to advocate for patients and represent their voice to industry and drug development. Nita has been an Ambassador for Needy Meds; she continues to help patients with their needs to access the medical care they deserve. Since 2016, Nita has been a faculty member for Patient Advocates in the life Sciences (PPALS), she lectures on Patient Advocacy and “Return on Investment”.
Nita has completed midwifery training in London, UK; received Genetic Nurse Certification, Obstetric Nurse certification and Clinical Research certification.

Publications:
Understand Epidermolysis Bullosa from patient perspective: Bruckner A1, Murrell D2, Wisk J3, Losow M3, Patel N3, Reha A3, Lagast H3, Gault J4, Cantor E3, Gershkowitz J3

The Patient and Clinician Point of View: Living With Late-onset Pompe Disease: Patel N,1 Kishnani P,2 Sathe S,1 Dietze D,3 Jain V,1 Viereck C,1 Barth JA,1 Sitaraman S1

Regulatory strategies for rare diseases under current global regulatory statutes: a discussion with stakeholders
Andrew E. Mulberg; Christina Bucci-Rechtweg; Joseph Giuliano; David Jacoby; Franklin K. Johnson; Qing Liu; Deborah Marsden; Scott McGoohan; Robert Nelson; Nita Patel; Klaus Romero; Vikram Sinha; Sheela Sitaraman; John Spaltro; Vivian Kessler

Orphanet Journal of Rare Diseases

David Pearce is President of Innovation, Research, & World Clinic for Sanford Health. He completed his undergraduate Bachelor of Science Degree with honors in biological sciences at Wolverhampton Polytechnic in 1986. He gained his PhD in 1990 at the University of Bath, UK, and did postdoctoral training at the University of Rochester, U.S., and Oxford University, UK.

Dr. Pearce has been researching Neuronal Ceroid Lipofuscinosis (Batten disease) since 1997. His research has led to the first clinical trial for Juvenile Batten disease. He has published over 100 research papers on Batten disease. He also oversees a national registry for rare diseases known as the Coordination of Rare Diseases at Sanford (CoRDS). He has served on numerous NIH review committees, has organized rare disease workshops for the National Institute for Neurological Disorders and Stroke (NINDS) arm of the National Institutes of Health (NIH) and is currently the chair of the consortium assembly for the International Rare Diseases Research Consortium (IRDiRC).

As President of Innovation, Research, & World Clinic at Sanford, he oversees the development of research programs, including more than 450 researchers, eight research centers and more than 300 ongoing clinical trials.

Heather Ramirez is an experienced life sciences attorney who counsels pharmaceutical and biotechnology companies on healthcare compliance and FDA regulatory matters. In her current role as Vice President, Legal at Amicus Therapeutics, she provides legal advice to many departments, including Patient & Professional Advocacy, Medical, Commercial, Clinical and Regulatory. She previously served as in-house counsel at Novo Nordisk and Abbvie and spent a number of years in private practice at Wiley Rein in Washington DC. Heather received a BS in Biochemistry & Molecular Biology from The Pennsylvania State University and a JD from Emory University School of Law. She lives in the Princeton, NJ area with her husband, two teenaged sons, two fluffy dogs and one large cat.

Michele is Head of Global Advocacy Relations and Patient Engagement at Savara, Inc. Partnering with the patient community and healthcare/professional associations to support people with pulmonary disease-autoimmune pulmonary disease –autoimmune pulmonary alveolar proteinosis (aPAP) She has more than a decade of experience leading patient affairs and advocacy across pharmaceutical and nonprofit industries. Michele is embedded within the rare disease and oncology patient communities through both her extensive professional work and her personal experience with a cancer diagnosis at age 20 and rare disease diagnosis at age 21. Partnering with these communities, she has led patient interviews/advisory boards and represented the patient perspective, which has directly informed trial design, patient support services, and disease education programs, among other efforts. Michele previously served as Head of Patient Affairs at Enzyvant where she helped build the Patient Affairs function, developing and maintaining both global patient advisor and advocacy partnerships across disease areas. During her time there, the company was nominated for a Global Genes 2019 RARE Champions of Hope award, in part, due to its expanding efforts in Patient Advocacy. Prior to her work at Enzyvant, Michele served as Global and R&D Patient Advocacy Lead at Takeda Oncology, Head of Global Patient Affairs at Bluebird Bio, and Director of both Strategic Initiatives and Program Initiatives at the National Brain Tumor Society. In addition, Michele has served as Chair of the MassBio Patient Advocacy Roundtable since 2017, is a member on the Massachusetts Rare Disease Advisory Council, is a founding board member for Costs of Care, an organization dedicated to helping clinicians and health systems deliver better care at lower cost and was named a 2019 PharmaVOICE 100 honoree. Michele received her MPH from Yale School of Public Health and her MBA from Yale School of Management.

Jamie has over 20 years of patient advocacy experience in the life sciences and is the founder of Jamie Ring Advocacy Consulting, an independent advocacy consulting firm. She most recently served as the Head of Patient Advocacy at Spark Therapeutics, a leader in the field of gene therapy. In her role, Jamie was responsible for creating the patient advocacy function and leading activities related to Spark’s disease areas of interest. She initiated and sustained corporate engagement in the Inherited Retinal Disease space where she supported the launch of the first FDA approved gene therapy for a genetic disease, Luxturna. Prior to joining Spark, Jamie spent 10 years at Genzyme where she most recently had served as the Vice President of Patient Advocacy and Humanitarian Programs – Rare Diseases. In that role, Jamie provided strategic oversight of all advocacy programming and led the global advocacy function in addition to managing external partnerships related to Genzyme’s global humanitarian programs. Prior to Genzyme, Jamie worked at Biogen supporting patient programs for both the Multiple Sclerosis and Non-Hodgkin’s Lymphoma disease communities. Additionally, Jamie worked at the ALS Therapy Development Institute as the Associate Director of Programs, responsible for ALS disease awareness initiatives and fundraising campaigns. In 2013, Jamie was voted by PharmaVoice magazine as one of the 100 most inspiring leaders in the pharmaceutical industry. Jamie holds a B.A. in Sociology from Union College, Schenectady, NY and an M.P.H. in Social & Behavioral Sciences from Boston University. Jamie serves on the Board of Directors for the National Tay Sachs and Allied Diseases Association and is on the advisory board for the BU School of Public Health Pharmaceutical Certificate Program.

Michelle has more than 20 years of agency advocacy relations and healthcare communications experience, starting in oncology on behalf of Bristol-Myers Squibb and Genentech and most recently, working with biotech companies focused on treatments for rare diseases. In 2019, she launched Slattery Health Communications to meet client needs at the intersection of advocacy relations and clinical trial recruitment. Ask Michelle about her recent work producing patient videos, moderating virtual patient advisory boards, researching strategies for improving diversity in clinical trials, and helping clients win internal advocates for their patient advocacy vision and approach. You can also ask Michelle about her evolving Wordle strategy or life in Kansas City with three teenage daughters.

Chris has more than 25 years of experience in healthcare communications, including leadership positions with global pharmaceutical, biotechnology and public relations firms. As an expert in rare disease communication, he knows how to address the scientific, medical, clinical, social and economic aspects of orphan drug development. His work spans more than a dozen biopharma companies, and includes patient advocacy support, patient panels, patient meet-ups, media relations, investor events, patient days, talent acquisition, clinical trial recruitment, corporate branding and website development, pipeline communications, data announcements, and more. Chris has worked across diverse therapeutic categories and has represented many of the world’s leading healthcare companies, including Alexion, AstraZeneca, Johnson & Johnson, Pfizer and Sanofi. He is a founding member of the Rare Collective®, a group of trusted independent advisors in orphan drug development.

Rob is a writer and editorial consultant with a specialty in rare diseases. For more than 20 years, he has provided editorial support and guidance to medical institutions, voluntary health organizations and pharmaceutical and biotechnology companies. Additionally, he provides strategic consultation on patient advocacy topics to industry and the nonprofit sectors. Rob’s extensive experience and style of writing on rare diseases and disorders assures that both individuals and physicians are provided with a clear understanding of rare diseases. During his tenure as the Managing Editor at the National Organization for Rare Disorders (NORD), Rob oversaw the development and expansion of the NORD Rare Disease Database as well as interacted with rare disease experts to ensure that each report was medically peer reviewed. His understanding of the unique needs of the rare disease community serves as the impetus to foster better communication and understanding between these diverse groups and the industry representatives that work with them. Rob has worked for several years with JF Campbell Consultants to help facilitate the relationship between patient communities and pharmaceutical companies and provide guidance to these organizations on a wide variety of educational and business activities. He has helped to mentor fledging nonprofit patient advocacy organizations, oversee grants programs, and written news articles that have appeared in magazines or websites including Exceptional Parent magazine and WebMD. Rob has also worked as an editorial consultant at the Leukemia & Lymphoma Society and currently serves as Managing Editor to RareShare, a rare disease community run by Rare Genomics.

Dr. Jill Weimer has directed the science arm of Amicus Therapeutics since June 2019, first as the Senior Vice President of Discovery Research and Gene Therapy Science and more recently as the Chief Science Officer. Dr. Weimer is a developmental neuroscientist by training and started her academic research program at Sanford Research and the University of South Dakota in 2009 as Assistant Scientist/Assistant Professor, focused on the molecular mechanisms mediating development of the cerebral cortex and how disruption in these processes can lead to a whole host of neural pediatric disorders, including Batten’s disease and Neurofibromatosis Type 1. Work in Dr. Weimer’s lab led to the first ever gene therapy trial programs for CLN3 and CLN6 Batten disease. Today she plays a unique dual role, holding leadership positions with both Amicus and Sanford Research, serving most recently in the role of Senior Director of Therapeutic Development and Scientist at Sanford. Dr. Weimer grew up in north central Missouri and moved to upstate New York where she received her bachelor’s degree and Ph.D. in neuroscience from the University of Rochester. She completed her postdoctoral training in the Neuroscience Research Center at the University of North Carolina in Chapel Hill with a focus on developmental neuroscience. Dr. Weimer also serves as a scientific advisor to a number of rare disease foundation as well as serving as the President of the Alumni Council for her alma mater, the University of Rochester School of Medicine and Dentistry.

Barbara serves as Chairman and co-founder of the Professional Patient Advocates in the Life Sciences organization. She has over 20 years of experience working with patients, patient organizations, and clinicians in the rare disease community. In 2018 she established her own consulting business. She is currently the Vice-President of the Battens Disease Support and Research Association board.