Previous Speakers

3rd Expanded Access Programmes Global Congress 2022

Alan J. Balch, PhD

CEO, National Patient Advocate Foundation

Dr. Balch has over fifteen years of executive leadership in the non-profit sector with an emphasis on consensus-building and collaboration. He has led numerous federal advocacy efforts on a range of issues both at the legislative and regulatory level. He became the CEO of both NPAF and PAF in 2013. From 2006 – 2013, he served as the Vice President of the Preventive Health Partnership — a national health promotion collaboration between the American Cancer Society, American Diabetes Association, and American Heart Association. Prior to 2006 Dr. Balch was the Executive Director of Friends of Cancer Research.

Dr. Balch has served on the Executive Board of the Patient Advocate Foundation and National Patient Advocate Foundation since 2007. He also serves on numerous advisory boards and committees.

He earned his PhD in environmental studies with a concentration in political economy in 2003 from the University of California, Santa Cruz; master’s degree in environmental sciences in 1997 from the University of Texas in San Antonio; his bachelor’s degree (cum laude) in biology in 1994 from Trinity University in San Antonio.

Donna Cowan

Associate Director, Expanded Access Programs Stealth BioTherapeutics

Donna Cowan is a Certified Clinical Research Coordinator/Project Manager with more than 20 years of biopharmaceutical experience. For the past several years Donna has concentrated on the management of Investigator Initiated Studies and Expanded Access Programs (Compassionate Use, Managed Access, Named Patient), leveraging her passion for patient care into providing early access to investigational therapies to those with serious or life-threatening conditions who may have no other treatment options. She has experience in various therapeutic areas including oncology, rare and orphan disease, endocrinology, neuroendocrinology, and infectious disease, participating in several successfully executed NDA applications in these areas. Ms. Cowan is the Associate Director of the Expanded Access Programs and Investigator Initiated Studies at Stealth BioTherapeutics (SBT), a Boston area-based biopharmaceutical company developing therapies to treat the mitochondrial dysfunction associated with genetic mitochondrial diseases and many common age-related diseases.

Robert Donnell

Executive Vice President, Medicines Access, Smartway Pharma

Robert has designed and led some of the most innovative and largest EAPs in recent years including in rare diseases and gene therapy. He has experience across Europe, USA and globally and has advised major blue-chip Pharma as well as smaller biotech companies. He started his career at P&G and AZ before moving to a number of international service providers working with pharma and biotech companies on supply chain solutions. He currently leads all global development and implementation of EAPs at Smartway where he specializes in working with manufacturers to develop the best Medicines Access plan for them.

Misty Gravelin, MPH, RAC

Project Lead, Expanded Access Support Unit, University of Michigan

Misty Gravelin, MPH, RAC, is the lead for expanded access support at the University of Michigan, as part of the Michigan Institute for Clinical and Health Research (MICHR). She has provided regulatory support or institutional coordination for hundreds of single-patient requests and dozens of multi-patient programs. In addition to this role, she is the project lead for Transforming Expanded Access to Maximize Support and Study (TEAMSS), an NIH-funded Collaborative Innovation Project to develop and disseminate resources for expanded access at academic medical centers. She has authored multiple papers on expanded access, including articles in the Journal of Clinical and Translational Science, the American Journal of Bioethics, and the Hastings Center Report.

Rachel Harrison

Associate Director, Early Access Program, Apellis Pharmaceuticals

Skilled at cross-functional collaboration between teams and driven to communicate successfully to obtain optimal results for the Expanded Access Programs I manage.  Successfully opened and closed an Early Access Program and currently manage a robust on-going Global Compassionate Use Program.   Knowledgeable in navigating the intricacies of Global Early Access Programs including Clinical Supply, Regulatory Requirements, Patient Access Portals, development of Physician facing materials, and submissions to the Institutional Review Board (IRB).  Proven success as an Alliance Manager with a demonstrated history of working in international settings. Thus far, was a team member for the launch of 2 FDA approved Orphan Drugs: COPIKTRA (duvelisib) and EMPAVELI (pegcetacoplan). Skilled in Communications, Global Alliance Management, Cross-functional Coordination, Corporate Development, Strategic Alliances, and Database Administration.  

Richard Klein

Director, Expanded Access Programs & Policy, GE2P2 Global Foundation

Richard Klein, an internationally recognized expert in expanded access to unapproved therapeutic agents, worked at the FDA for more than 40 years, before leaving the agency in 2017. While there he helped develop the revised expanded access regulations and guidelines, led the creation of the FDA expanded access website, and helped develop the streamlined application for individual patient access and the Reagan Udall Foundation Expanded Access Navigator. He also spearheaded the effort to waive full-board IRB review for individual patient access to unapproved drugs and biologics. As director of the FDA’s Patient Liaison Program, he interacted extensively with outside communities and the agency’s scientific and policy offices to advocate for patient interests and facilitate patient engagement in regulatory activities. He worked closely with patient communities in a variety of areas, including treatment access to unapproved drugs, product safety, and clinical trial design. Before taking on that role, he created the FDA’s HIV/AIDS program, working with AIDS activists and advocates to coordinate their input and participation in regulatory policy and decision-making related to HIV/AIDS. Prior to working in patient engagement, he helped develop policies and regulations for the protection of human research subjects, both at FDA and HHS, including the original Common Rule, and provided guidance for institutional review boards (IRBs).

Eugean Jiwanmall

Senior Research Analyst for Medical Policy & Technology Evaluation, Independence Blue Cross

Eugean Jiwanmall’s past professional experiences include basic science and clinical research. In his current role as a Senior Research Analyst for Medical Policy & Technology Evaluation at Independence Blue Cross’ Medical & Claim Payment Policy Department within Facilitated Health Networks and Medical Affairs, he has served as the subject-matter-expert, writer, reviewer, communicator, presenter, consultant, adviser/facilitator, and decision-maker for hundreds of medical policy topics and technology assessments within dozens of clinical disciplines and multiple business areas. Eugean has participated in numerous roles & capacities in a number of conferences, summits, and other national & international events etc. upon invitations. He has been invited by renowned organizations, evaluator bodies, & thinktanks etc. to be key informant on a variety of complex and challenging topics, and recognized in communications & publications for these works.

Eugean has taken and completed graduate coursework in bioethics at the University of Pennsylvania School of Medicine. He holds a bachelor’s degree in biological sciences with honors from Drexel University (minor was in business administration), a master’s degree of public health in evaluative clinical sciences from Dartmouth Medical School, and an MBA in healthcare management and economics with honors from Drexel University.

John Lagus

Executive Vice President, Business Development, Tanner Pharma Group

John Lagus is the Executive Vice President of Business Development for Tanner Pharma Group. John spent more than a decade at Orphan Medical where he held various roles in development, commercial, corporate development, and oversight of international. Prior to joining, John worked for 12 years at Idis (and subsequently Clinigen) with roles in commercial, operations, and general management. John has a BA in mathematics from St Olaf College and a MS in statistics from Iowa State University.
 

Nina Liu

Director, Clinical Product Management, Pulse Infoframe

Nina is a dynamic and engaging leader passionate about improving care quality and delivering products to market that will have a direct impact for patients. She is experienced product and business operations leader for a variety of fast-paced healthcare technology companies. Before joining Pulse Infoframe, Nina was the Director of Product Management at Wolters Kluwer Health. During her tenure she managed a suite of clinical decision support and formulary analytics products as well as innovative initiatives such as Pharmacovigilance, leveraging deep learning to identify signals of adverse effects and events for emergent and in market drugs. Additionally, Nina had direct responsibilities for driving top-line results; growing and developing products and service lines including uncovering market problems; driving product requirements through rapid prototyping and customer feedback

Senior Representative 16

Available Shortly.

Jennifer E. Miller

Associate Professor, Yale School of Medicine

Yale School of Medicine

Neena Nizar

President & Founder, The Jansens Foundation

Dr. Nizar serves as Founder and Executive Director of the Jansen’s Foundation. She is a rare disease advocate and Change Leader in the rare disease community, specifically in the field of ultra-rare skeletal dysplasias. Dr. Nizar has a Doctoral degree in Educational Leadership from Creighton University, Nebraska, is a TEDx speaker, a blogger and passionate voice for the special needs community. She is a Trailblazer for the Rare Advocacy Movement (RAM) and Nebraska’s Mother of The Year, 2018. Dr. Nizar is also a rare disease patient and a mother to two boys with Jansen’s Metaphyseal Chondrodysplasia – a disease that affects less than 30 people worldwide.

Monica L Weldon

President/CEO/Founder Bridge the Gap - SYNGAP Education and Research Foundation

In November of 2012, Monica’s twin son, Beckett, was the first to be diagnosed at Texas Children’s Genetics Clinic with the gene mutation SYNGAP1 (6p21.3). When Beckett was 4 months old, she noticed he was not meeting the same milestones as his twin sister. Monica then began a journey to find answers to help her son. She started to blog about his progress and this led to building a community of parents and caregivers that are now a strong support group. She is the Founder and President/CEO of Bridge the Gap – SYNGAP Education and Research Foundation. It is her passion to help support these families by raising awareness and creating a strong foundation that will accelerate a path to better therapies.

She retired in 2016 after 23 years in education teaching secondary science. Her new focus is on building the programs and mission of Bridge the Gap – SYNGAP Education and Research Foundation. She is the Primary Investigator on the SYNGAP1 (MRD5) Registry and Natural History Study. She is a life member of the Worldwide Association of Female Professionals and a member of the first class of 2017 Illumina Ambassadors established in the United States. In addition to leading the foundation, she is an author, public speaker, consultant on rare disease business strategies and advocates for rare disease legislation at both the federal and state levels. Several of her authored scientific publications include Nature Neuroscience, The Journal of Neurodevelopmental Disorders, and The Journal of Pediatrics. She has authored a book about her son Beckett’s diagnostic journey called “Slow Moving Stream – My Special Boy”. She is a graduate from East Texas Baptist University with a Bachelor’s of Science in Biology/Psychology (1991) and Secondary Certification in Education (1995). She will be attending Northwestern University Pritzker School of Law in the Fall of 2020 to earn her Masters in Science Law.  She has five beautiful children, Haleigh (28), Taylor, USMC (25), Sawyer (23), and the twins Beckett & Pyper (12). She was a Global Genes 2015 & 2016 RARE Champion in Advocacy Award nominee for her work in the rare disease community and WeGo Health Awards Nominee for years 2015, 2017, 2018, 2019, & 2020. She was named 2020 Global Shakers Rare Disease Champion.

Magali Taiel, M.D.,

Chief Medical Officer , GenSight-Biologics

Dr. Taiel completed her doctorate in Medicine with board certified in Ophthalmology from Lariboisiere Saint Louis University, Paris, France, in 1993, and her Associate Professor degree in 1998. Dr Taiel completed her internship at academic Paris hospitals, was an Associate Professor of Ophthalmology, served as an Ophthalmology Department Head, and ran Surgical and Medical Ophthalmology private practice.  After 13 years of Ophthalmology public and private practice, Dr Taiel has been engaged in the Pharma Industry for 20 years; she brings extensive experience and expertise in drug clinical development, gene therapy, and medical affairs. She started her career at Servier company headquarter, and then worked in Ophthalmology area at Pfizer for several years; she then held international and management positions in various therapeutic areas, including both technical and supervision duties, at Eli Lilly Company for many years. Then, as VP Clinical Development, she led Clinical Development and Operations, to develop antisense oligonucleotides in Inherited Retinal diseases at ProQR Therapeutics. She then moved to GenSight-Biologics in 2018, to supervise the Medical Department and lead Gene Therapy programs in Inherited Retinal and Neuro-Ophthalmology diseases, as the CMO of the company.

Dr. Taiel has authored numerous protocols and articles published in peer reviewed journals, and made critical contributions to successful clinical development and launch of many products. She brings extensive years of experience from both academic medicine and pharma industry.

Jeffrey Thibeault

Co-founder & Chief Technology Officer, MedaSystems

Jeffrey Thibeault is an entrepreneur and technology executive with over 20 years of experience building companies focused on innovative solutions and efficient processes. Jeffrey has provided leadership for technology teams of all sizes across multiple industries, most recently at Fintel Technologies, Inc., overseeing software development to help financial institutions navigate specific regulatory processes.  Prior to Fintel, Jeffrey led the technology group at i>clicker as the company embraced a digital transformation, introducing highly scalable, cutting-edge, mobile, and web technology solutions to augment its industry-leading hardware devices used each year by over 2 million higher education users. Jeffrey has several issued US patents for systems and algorithms in the artificial intelligence and data processing fields. He holds a B.S.E. in Computer Science and Engineering from the University of Connecticut, with Artificial Intelligence and Sociology concentrations.
 

Christine Von Raesfeld

Founder & CEO, People with Empathy

As Founder/CEO of People with Empathy, Christine Von Raesfeld is a leader in bringing a critically needed patient perspective to cutting-edge medical innovations. Committed to providing patients with chronic and rare diseases with the support they need, Christine works with patient advocacy organizations, industry representatives, and individual patients and their loved ones. Living with many rare and chronic diseases, she believes that the only way to ensure health equity is to leverage one’s own data. As a patient involved in Stanford’s Humanwide program, she speaks on the benefits and advantages of precision medicine, with a special interest in pharmacogenomics. 

Christine’s drive to make lives better for patients has been nationally recognized. In 2019, she spoke on stage at the Startup Health Festival as an invited guest of Sanguine Biosciences. In 2018, Christine served on the Team of Patient Advisors for PatientsLikeMe. She has been nationally recognized for her work in advocacy and when several awards including Silicon Valley Business Journal’s100 Women of Influence, 2019 and a 2020 Community Hero by Assemblyman Kansen Chu. In addition, Christine serves as a Virtual Advisor Team member for the All of Us research program, a Technical Expert Panelists for CMS and a patient advisor/consultant or multiple pharmaceuticals off branded campaigns and startup ventures.

Dawn Sullivan,

Founder/CEO, Sullivan Associates, LLC

Sullivan Associates, LLC

Anne B. Cropp

Chief Scientific Officer, Early Access Care

Anne Cropp Pharm.D. is Chief Scientific Officer for Early Access Care, a company exclusively providing global Expanded Access/Compassionate Use services.  Anne has led the development of Expanded Access programs for small, mid, and large pharma companies based in the US, Europe, and Asia. Prior to founding Early Access Care, Anne was Vice President in the Global Product Development Group of Pfizer Inc responsible for Phase 2 and 3 Clinical Development Programs and Head of the Compassionate Access Group for Pfizer.  She is a globally recognized expert in expanded access and compassionate use and has over 25 years experience in early access programs. 

Marjorie Zettler

Director of Clinical Science, Regor Pharmaceuticals, Inc.

Marjorie Zettler, PhD, MPH is Director of Clinical Science at Regor Pharmaceuticals, Inc., a pioneering drug discovery and clinical-stage pharmaceutical research company. An industry veteran with more than 18 years’ experience at companies in the pharma and healthcare sector including Lilly and Cardinal Health, her work has focused on clinical research, drug development, and regulatory strategy. She has published over 100 abstracts, manuscripts, and patents. Dr. Zettler received a doctorate in physiology and a bachelor’s degree (honors) in genetics from the University of Manitoba, Canada, as well as a master’s degree in public health from the University of Manchester, United Kingdom.  

Jean Godin

Head, Global Access Programme, Sanofi

Jean Godin holds a Doctorate in Medicine (MD) from the University of Montreal, a post Graduate diploma in Family Medicine from the University of Montreal and a MBA in Business Administration from McGill University. He is a licensed physician of the Quebec College of Physician & Medical Council of Canada (LMCC). He practiced medicine full time in the Montreal area in both university and community hospitals until he joined the pharmaceutical industry in December of 1999.

Jean headed scientific affairs in Canada at Teva Pharmaceuticals (innovative division) from 1999 to 2007. In June 2007, he became General Manager for the branded activities of Teva in Canada; he was then responsible for the development and commercialization of the innovative portfolio. In July 2012, Jean joined Novartis Canada in the position of Chief Scientific Officer and Vice-President, Clinical and Regulatory Affairs, reporting to the CEO. Jean joined Sanofi in May 2016.  He is currently the Head of Global Access Programs.

Jean currently lives in Cambridge, Massachusetts and maintains a part-time medical practice in Montreal.

Oliver White

Senior Program Manager, Tanner Pharma Group

Oliver White serves as Senior Program Manager for the Managed Access Programs (TannerMAP) division of Tanner Pharma Group. During his tenure with the company, Oliver has led the design and set-up of several Managed Access Programs for rare disease and oncology products. Oliver holds a master’s degree in chemistry from Durham University, England, and has undertaken pharmaceutical research placements at Vertex Pharmaceuticals and Université Grenoble Alpes, France.