Confirmed Speakers

Orphan Drugs & Rare Diseases Global Congress 2021 Americas

Karmen Trzupek

Director, Clinical Trial Services, InformedDNA

Scott Dorfman

President and CEO, Odylia Therapeutics

Wendy Erler

Vice President, Patient Experience, STAR and Advocacy, Alexion Pharmaceuticals

Philip John Brooks

Program Director, Office of Rare Diseases Research, NCATS, NIH

Khrystal Davis

Founder & President, Texas Rare Alliance, Advisory Member, Texas Newborn Screening

Alan J. Balch, PhD

CEO, National Patient Advocate Foundation

Samuel Seward, Jr., MD, FACP,

Professor of Medicine and Site Chair, Department of Medicine, MSMW/Mount Sinai Health System, New York City

Debra Miller

CEO and Founder, CureDuchenne

Lisa Kearns, MS, MA,

Senior Researcher, Division of Medical Ethics, NYU Grossman School of Medicine

Simu Thomas PhD,

Vice President, Global Head HEOR, Alexion Pharmaceuticals

Mike Page

Vice President, Global Regulatory Affairs Development Strategy, Alexion Pharmaceuticals.

Magali Taiel, M.D.,

Chief Medical Officer , GenSight-Biologics

Tara J. Britt

Founder and President, Rare Disease Innovations Institute, Inc.

Robert Donnell

Executive Vice President – Medicines Access, Smartway Pharma, a leading provider of Early / Expanded Access Programs (EAP).

Kristin Smedley

President, Curing Retinal Blindness Foundation

Robert Keel

Executive Vice President, Managed Access Programs, Tanner Pharma Group

John Lagus

Executive Vice President, Business Development, Tanner Pharma Group

Oliver White

Senior Program Manager, Tanner Pharma Group

Patricia Weltin

CEO and Founder of the Beyond the Diagnosis.

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Barry Ticho, M.D., PhD., 1

Chief Medical Officer, Stoke Therapeutics, Inc.

As Chief Medical Officer Dr. Ticho is responsible for Stoke’s efforts to develop first-in-class RNA based disease-modifying medicines to treat severe neurologic genetic diseases. Prior to joining Stoke Barry was Head of R&D for Cardiovascular and Metabolic Diseases at Moderna Therapeutics.  He was previously Head of External R&D Innovation for Cardiovascular and Metabolic Diseases at Pfizer.  Prior to that he was Vice President of Clinical Development at Biogen where he led clinical development for the Tysabri program for MS and led the aducanumab program for Alzheimer’s Disease. Barry obtained his M.D. and Ph.D. degrees from the University of Chicago and completed Pediatrics training at Northwestern University and a Cardiology fellowship at Children’s Hospital in Boston. He was on clinical staff at Harvard Medical School and Massachusetts General Hospital. 

Rahul Ballal, PhD

President & CEO, Imara Inc.

Rahul has served as Imara’s President and Chief Executive Officer and as a member of the board of directors since June 2018. He has more than 15 years of life sciences experience in business development, strategic partnerships, venture capital, and company financing. Rahul joined Imara from Northern Biologics and Versant Ventures, where he served as Chief Business Officer and an Entrepreneur-in-Residence, respectively. At Northern Biologics, he built a first-in-class immuno-oncology portfolio, led a broad strategic partnership with Celgene, and managed several key functional roles at the executive level. At Versant, he negotiated foundational assets for existing portfolio and new companies, sourced several new deals, and participated in investment team activities.

Rahul was previously Vice President, Business Development at Flexion Therapeutics, where he led several business development transactions, supported multiple functions during Flexion’s initial public offering, and completed a key $175 million strategic partnership with Patheon UK. Prior to Flexion, he held business development and leadership positions at Novartis Venture Fund, Broad Institute of MIT and Harvard, and Redmind Inc. Rahul earned a B.S. in biology from Brown University, a M.S. in bioinformatics from Johns Hopkins University, and a Ph.D. in biochemistry from Georgetown University. He is also a Kauffman Fellow.

Andrea C. Furia-Helms, M.P.H.

Director, Patient Affairs Staff, Office of Clinical Policy and Programs, Office of the Commissioner, U.S. Food and Drug Administration

Andrea Furia-Helms is the Director of the Patient Affairs Staff in the Office of Clinical Policy and Programs, Office of the Commissioner. In her role, she collaborates with patient communities, the FDA medical product Centers, and other offices to incorporate patient and caregiver perspectives in cross-cutting regulatory meetings.  Ms. Furia-Helms spent over ten years in the FDA’s Office of Health and Constituent Affairs where she directed the FDA Patient Representative Program and coordinated patient engagement activities for the agency.

Prior to FDA, Ms. Furia-Helms was Director of the Back to Sleep (now Safe to Sleep) campaign, a public-private partnership to educate communities on Sudden Infant Death Syndrome (SIDS), at the National Institutes of Health.  She developed SIDS outreach initiatives for African American, American Indian, and Latino communities.

Ms. Furia-Helms has a B.A. in psychology from Framingham State University, a B.S. degree in community health education from the University of Maryland, and a Master of Public Health degree from The George Washington University.

 
 
 

Christopher Dandrea

Vice President, Global Program Management, uniQure

Chris Dandrea is a 25 year veteran in the Biopharmaceutical Research, Development and Commercialization space.  His current role as uniQure’s Vice President of Global Program Management is focused on scale-up, acceleration and productivity of uniQure’s growing Research & Development Pipeline of CNS/Neuro and Rare Gene Therapy programs.  Prior to uniQure, Chris was Sanofi’s Head of Oncology Project Management tasked to build a next-generation project management organization to support rapid oncology portfolio growth.   Additional experience includes Rare Disease Program Leadership at Genzyme, Life Science Management Consulting, Medical Device Engineering and service as a US Naval Officer.  Education includes Engineering degrees from Duke University and an MBA from Carnegie Mellon University.  

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.

Haya Taitel

Senior Vice President, Chief Commercial Officer, Kadmon

Ms. Taitel is Senior Vice President, Chief Commercial Officer at Kadmon, where she is responsible for the strategy, tactical execution and forecasting of in-line promoted products, portfolio contracting strategies and the launch readiness of pipeline assets.

Haya leads commercial operation disciplines including marketing, sales, national accounts and contracting strategies across several therapeutic areas. She also oversees the commercial operation infrastructure, including the business analytics, market segmentation and customer service units.

Prior to joining Kadmon, Haya led the U.S. commercial franchises of both the Pharma and Device sectors of Johnson & Johnson. In her 25-year tenure with J&J, she launched new chemical entities in several therapeutic areas including neurology, analgesia, peripheral diabetic neuropathy, women’s health, and urology, with focus on interstitial cystitis.

Cherry T. Thomas, M.D.,

SVP Clinical Development, Caribou Biosciences, Inc.

Coming Soon

Deborah Collyar

President, Patient Advocates In Research (PAIR)

Coming Soon 

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.

Dr. Neena Nizar

Founder and Executive Director, Jansen's 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.

Melinda Bachini

Director of Advocacy, Cholangiocarcinoma Foundation

Melinda Bachini, is an 11-year survivor of cholangiocarcinoma. She was treated at the National Cancer Institute starting in 2012 with a clinical trial using Adoptive Cell Therapy. Due to her exceptional and breakthrough response to treatment at NIH, her story was published in the New York Times and other media. 

Melinda is married and the mother of six children, grandmother to one. She is passionate about patient advocacy. Her personal experience with previous and current treatments allows for great insight to patients participating in clinical trials.  Her position as Director of Advocacy for the Cholangiocarcinoma Foundation, allows Melinda to interact, support and educate others touched by this cancer.  She shares her story with as many cancer patients as possible in hopes of giving courage to those in need.

Melinda had previously volunteered for seven years with the Cholangiocarcinoma Foundation and in August of 2017 came on as staff. She also worked for over 15 years in the Emergency Medical Field as an EMT Paramedic.

Melinda serves as patient advocate for the NCI Hepatobiliary Task Force, the NCI Patient Advocate Steering Committee (PASC), ECOG-ACRIN GI Committee, ECOG-ACRIN Cancer Research Advocacy Committee, and the NCRA.  Melinda attended the 2016, 2017, and 2018 NCCS, CPAT Symposium and advocated on Capitol Hill. She participated as a panelist in the Cancer Moonshot – Community Oncology Event at the White House, told her personal story and was introduced by her state Senator for the Innovation and Access in Quality Cancer Care at the US Capitol. In June of 2017 she spoke with nine US Senators at NIH to share her personal story of how research has prolonged her life.  Melinda has served as a Consumer Reviewer for the Department of Defense for several years.

Luke Rosen

VP, Accelerated Development & Community Engagement, Ovid Therapeutics

Luke Rosen is Co-Founder of KIF1A.ORG, a non-profit organization working to discover treatment for children living with KIF1A Associated Neurological Disorder, a rare genetic disorder affecting his daughter, Susannah. Started in 2016, KIF1A.ORG works to forge efficient collaborations within the scientific and patient communities to accelerate development of therapeutics for KIF1A. 

Luke also serves as VP, Patient Engagement and Government Affairs at Ovid Therapeutics, a biotech working to develop treatments for rare neurological disorders. In his role at Ovid, Luke works closely with members of the rare disease community to encourage efficient and impactful collaboration among all stakeholders. Working closely with every function at Ovid, Luke ensures the patient and caregiver voice is central in decision making, clinical trial readiness and public policy.

Co-Executive Director, Bensfriends.org

Co-Executive Director, Bensfriends.org

Clasina is a retired Canadian teacher and school counselor who lives near Ottawa, ON. She puts her teaching and counseling skills to good use in managing the Member Experience side of Ben’s Friends. She guides community moderators, deals with members who are having difficulties on the sites, and takes responsibility for the overall experience of Ben’s Friends members. Having a rare disease herself, when she’s not working on Ben’s Friends, Clasina enjoys being home, but she also loves going away and indulging in international travel.

Angela Ramirez Holmes

Founder/President, Cal Rare

Angela Ramirez Holmes started her advocacy in rare diseases in 2013 when her then 8 year old son Andrew was diagnosed with a rare brain condition, an arteriovenous malformation or AVM. Angela is the founder and president of Cal Rare, a non-profit coalition of rare disease stakeholders dedicated to improving the lives of rare disease patients in California. Cal Rare started the California Rare Disease Legislative Caucus and was able to secure $2 million in the 2018 budget for a Whole Genome Sequencing Pilot Project for undiagnosed pediatric patients. In 2018, Angela was the recipient of the Rare Voice Award for State Advocacy from the EveryLife Foundation. She is the Principal of Alliance Campaign Strategies, a political consulting and public affairs firm working on local campaigns and issues. Angela has nearly twenty years of political and legislative experience on the national, state, and local levels. She is also an elected official, currently serving her second term on the Board of the Zone 7 Water Agency. Angela graduated from Saint Mary’s College of California with a B.A. in Politics and U.S. History.

Tom James Lambdin

Co-Executive Director, Bensfriends.org

Tom James, after a wide and varied career connected to research, medicine and business, looks after the technical and business side of Ben’s Friends. With his background in many aspects of medicine, and his long list of friends and contacts, he is also the Ben’s Friends medical fact checker.  Tom, a rare disease patient himself, lives in the western USA and if he ever had any spare time, he’d be fly fishing.

Femida Gwadry Sridhar

CEO and Founder, Pulse Inforframe Inc.

Femida is a pharmacist, clinical epidemiologist, and methodologist with over 25 years of experience in clinical trials, patient registries, knowledge translation, health analytics, and clinical disease outcomes. Femida has received more than $10 million in research funding as a principal investigator or co-investigator. She was awarded a career scientist award in 2006 from the Canadian Institute of Health Research and through her work founded the first knowledge translation health informatics lab in North America, creating a collaborative ecosystem for interdisciplinary research. Recently recognized as a lifetime fellow of CIHR for her contributions to global research with impact. Femida has published over 200 manuscripts and abstracts in top-tier journals and actively continues her professional interests in enabling technologies for better provider workflow and patient engagement. 

Femida is a founding member of the International Society for Pharmacoeconomics and Outcomes Research medication adherence group, which has provided an early glimpse into how to conduct research in adherence. She is an expert reviewer in North America and Europe on large-scale research network grants and a frequent international speaker on patient engagement, patient registries, innovation, health informatics, and data visualization. The recipient of numerous nominations, awards, and positive press, Femida has joined Springboard Enterprises as a female entrepreneur in digital health.

David A Pearce, PhD

President of Innovation, Research, & World Clinic, Sanford Health

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 heads the leading lab in Juvenile Batten disease research.  He has been researching Juvenile 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 vice-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.

Karmen Trzupek

Director, Clinical Trial Services, InformedDNA

Karmen Trzupek currently directs clinical trial services at InformedDNA.  As a clinical genetic counselor, Karmen developed the first nationwide telemedicine program for ocular genetic counseling & genetic test coordination services.  She has managed many rare disease outreach programs in collaboration with biopharma companies & patient foundations. Today, she develops strategies & programs that drive patient recruitment and enrollment in rare disease clinical trials.  As a national telemedicine practice, InformedDNA uses telehealth services to screen patients for clinical trials, remove barriers to genetic services, and engage patients and families in the clinical research process.

Founded in 2007, InformedDNA is the country’s largest applied genomics solutions company, helping more people harness the full power of the genomics revolution. With the largest, lab-independent staff of board-certified genetics specialists in the U.S., InformedDNA helps to ensure that pharmaceutical and biotech companies, health plans, clinicians, and patients all have access to the highest quality genetic services.

Key offerings for pharmaceutical and biotech companies include design support for genetics-based clinical trials and studies, patient engagement and recruitment, physician education and engagement, clinical genetic counseling, and test ordering and interpretation services.

For more information, visit www.InformedDNA.com.

 

Scott Dorfman

President and CEO, Odylia Therapeutics

Scott Dorfman is president and CEO of Odylia Therapeutics, a non-profit biotech company whose mission is to facilitate the translation of rare disease with proof-of-concept from the lab into clinical trials. Prior to Odylia, Mr. Dorfman founded and was Chairman, & CEO of Innotrac Corporation (NASDAQ: INOC), provider of eCommerce services. Founded in 1984, Innotrac employed over 2,500 employees with $250 million in revenues. The company merged with eBay Enterprises (division of eBay) and sold to Belgium Post (Bpost).

Mr. Dorfman served on the Board of Visitors for Emory University and is a past member of the Board of Directors of the Partnership against Domestic Violence (PADV). In addition to serving on the Radial board, he has also sat on the Board of Directors for Chatham Capital, Return.com and Market Velocity, Inc.

Mr. Dorfman currently serves as an Operations Partner for Fulcrum Equity Partners and sits on the Board of Nacuity Pharmaceuticals Inc., Dropoff Inc., Complemar Corporation, Fulcrum Equity, Odylia Therapeutics and Usher 2020 Foundation. Mr. Dorfman received a BBA from The University of Georgia in 1979.

Wendy Erler,

Vice President, Patient Experience, STAR and Advocacy, Alexion Pharmaceuticals

Wendy has built her patient advocacy career in many rare disease communities and oncology. Wendy Erler is the Vice President of Patient Experience, STAR and Advocacy at Alexion Pharmaceuticals where she is responsible for leading the enterprise innovation model to gather and incorporate patient insights throughout the drug development and commercialization processes. She leads the Global Patient Advocacy function and has responsibility for the advocacy team. Previously, Ms. Erler was on the executive leadership team at Wave Life Sciences where she led Patient Advocacy and Commercial.

Wendy graduated from Miami University and earned her MBA from St. Joseph’s University.

Philip John Brooks

Program Director, Office of Rare Diseases Research, NCATS, NIH

Philip John (P.J.) Brooks is a Program Director in the Office of Rare Diseases Research in the National Center for Advancing Translational Sciences (NCATS). Prior to taking on this role, he was in the NCATS Division of Clinical Innovation, where he was the lead program director for the Clinical and Translational Science Awards (CTSA) Program Collaborative Innovation Awards, designed to fund projects that will result in novel and creative approaches to overcoming roadblocks in translational science. In addition to his work in NCATS, Brooks is the Working Group Coordinator for the NIH Common Fund Somatic Cell Genome Editing Program https://commonfund.nih.gov/editing.

Dr. Brooks earned bachelor’s and master’s degrees in psychology and received his Ph.D. in neurobiology from the University of North Carolina at Chapel Hill. After completing a postdoctoral fellowship at the Rockefeller University, Brooks became an investigator in the intramural program of the National Institute on Alcohol Abuse and Alcoholism. He developed an internationally recognized research program focused on two distinct areas: the molecular basis of alcohol-related cancer, and rare neurologic diseases resulting from defective DNA repair.

Khrystal Davis

Founder & President, Texas Rare Alliance, Advisory Member, Texas Newborn Screening

Khrystal joined the rare disease community in 2011 when her newborn son, Hunter, was diagnosed with Spinal Muscular Atrophy (SMA) Type 1, the leading genetic cause of mortality in children under the age of two. SMA Type 1, often described as ALS in babies, robs the ability to move, swallow, and ultimately breathe. Khrystal is the author of Hunt for a Cure: An Unexpected Adventure to Save a Life. Khrystal founded Zebra Leaf Publishing to provide the rare disease community with a platform to promote rare disease awareness.

In May of 2016, Khrystal advocated alongside the FAST Movement (Families for the Acceleration of Spinal Muscular Atrophy Treatments) in a meeting with top FDA representatives for access to Spinraza, an SMA treatment in clinical trials at the time. Together with other FAST members, she asked the FDA to stop placebo trials, provide a means of access for the weakest SMA patients, accelerate the approval, and approve the treatment for all SMA patients regardless of age or type of SMA. In an interim look completed on August 1, 2016, the FDA found the treatment met trial objectives. An Expanded Access Program for SMA Type 1 patients commenced August 12, 2016, and the FDA approved Spinraza for children and adults with SMA on December 23, 2016. Khrystal is committed to improving health outcomes in those with rare diseases through improved access to rare disease treatments. She advocates for the expansion of newborn screening programs and insurance policies that conform to FDA labels for orphan drugs. She is a proponent of patient-driven access to rare disease treatments.

Khrystal holds a Juris Doctorate from Stetson University College of Law and is certified in Clinical Trial Design and Interpretation by Johns Hopkins through the Coursera program. She is a proud wife and mother of five who enjoys travelling the world with her family.

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.

Samuel Seward, Jr., MD, FACP

Professor of Medicine and Site Chair, Department of Medicine, MSMW/Mount Sinai Health System, New York City

Dr. Seward received his M.D. from the University of Texas Southwestern School of Medicine and completed his training in Internal Medicine and Pediatrics.  He is internationally known for his work with the Hermansky-Pudlak Syndrome (HPS) patient community, also serving on the Board of Directors of the HPS Network and its Scientific Advisory.  He is the co-author of the definitive treatment protocol for HPS patients and has been in the forefront driving a patient-centered approach to clinical research programs involving this community.  In addition, Dr. Seward maintains an active clinical practice which focuses on prevention including in adult survivors of rare congenital disorders.
 

Debra Miller

CEO and Founder, CureDuchenne

Debra Miller is CEO and Founder of CureDuchenne. She and her husband started the organization in 2002 after their only son, Hawken, was diagnosed with Duchenne. Debra and her team have brought many firsts to the Duchenne community, including a Duchenne-focused venture philanthropy model, an annual nationwide community education event tour (virtual for now), a Duchenne Physical Therapy Certification program and an open access Biobank. Debra proudly serves as the Patient Representative on TREAT-NMD’s Executive Committee and reviews Duchenne applications for the Department of Defense’s Congressionally Directed Medical Research Program. A native Californian, Debra graduated from Mariner’s High School in Huntington Beach and earned a Bachelor of Arts in Communication Studies from the University of California Los Angeles. Prior to CureDuchenne, Debra had a career in publishing with positions at IDG Communications, Cahners Publishing, Ziff-Davis Publishing, and Scholastic Publishing. She also worked in management at PC Magazine and was an independent stock trader.

Lisa Kearns, MS, MA,

Senior Researcher, Division of Medical Ethics, NYU Grossman School of Medicine

Lisa Kearns, MS, MA, is a senior researcher in the Division of Medical Ethics at NYU Grossman School of Medicine. She is a member of the Division’s working groups on Compassionate Use and Preapproval Access (CUPA) and Pediatric Gene Therapy and Medical Ethics (PGTME). For the past six years she has studied the ethical issues surrounding pre approval/expanded access to investigational drugs, including state and federal “right to try” legislation. More recently she has been studying the ethics of “bespoke” N-of-1 gene therapies and of pre approval access to gene therapies. Since March she has been researching the ethical allocation of drugs, vaccines, and medical equipment during the novel coronavirus pandemic and access to investigational and unproven therapies for Covid-19. She is the deputy chair of the Compassionate Use Advisory Committees (CompAC), which advise Janssen Pharmaceuticals on the ethical allocation of its investigational drugs.

Simu Thomas PhD,

Vice President, Global Head HEOR, Alexion Pharmaceuticals

Dr. Simu Thomas is the Vice President and Global Head of Health Economics & Outcomes Research at Alexion Pharmaceuticals. Simu brings 20 years of leadership experience in the area of Value, HEOR, RWE and Access, building and leading teams across the world.  

Prior to Alexion, Simu was the Global Head of Value & Access for Rare Diseases at Novartis and previously, Global Head for the Cell & Gene Therapy Unit, where he developed the value access strategy for the first CAR-T therapy approved in the world. He was also the founding architect of the IMI HARMONY data initiative with public and EFPIA private partners in Europe, as the leading industry partner in the consortium.  Simu ran the Economic Modeling organization at Novartis, was the Global Head of HEOR for Neuroscience and Ophthalmics franchises and US Oncology.

He has authored more than 35 manuscripts and 75 congress presentations and co-authored book chapters in the field of Health Economics. Simu holds a PhD in Pharmaceutical Economics from the University of Maryland and MS in Pharmacy Administration from the University of Toledo and Pharmacy degrees. Simu also serves as Adjunct Assistant Professor at University of Maryland and Rutgers University of New Jersey.

Mike Page

Vice President, Global Regulatory Affairs Development Strategy, Alexion Pharmaceuticals.

Mike leads a global team of strategists who define and deliver innovative regulatory approaches across Alexion’s rare disease portfolio. With over twenty years regulatory experience in Europe and the United States and has worked across all stages of development. Prior to joining Alexion, Mike led the US Regulatory team for oncology at Eisai and managed the regulatory aspects of Eisai’s monoclonal antibody products. Prior to that, Mike was a regulatory affairs consultant at United BioSource Corporation, before which he was a Director of Regulatory Strategy at Pfizer, both in the UK and the US.  With a particular interest in late stage development and product registration, Mike has global experience in therapeutic areas including oncology, hematology, psychiatry, neurology, sexual health, and addiction disorders.

Mike is a graduate of the University of Hull, UK.

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.

Tara J. Britt

Founder and President, Rare Disease Innovations Institute, Inc.

Tara is the President and Founder of Rare Disease Innovations Institute, RDII. RDII is a global non-profit focused on educating, engaging and equipping the rare disease community to achieve a higher quality of life, accelerating diagnosis and enabling access and treatment through policy. Through this non-profit and her rare disease network, RDII has developed exclusive toolkits for other states to build upon the learning and success of North Carolina enabling creation of successful councils and unique rare disease networks. The toolkits are state tailored to the unique rare disease population.   RDII also partners with rare disease advocacy groups, academia, foundations, healthcare systems, state and federal government and industry to build disease specific models to support targeted disease populations and the challenges to accessing care and managing their disease.  These models are currently developed in North Carolina and can be modeled globally by the RDII team.

Tara also currently serves as Associate Chair of the North Carolina Rare Disease Advisory Council and Network.  The council was created as a result of co-authoring legislation with a North Carolina patient advocate to create a Rare Disease Advisory Council signed into law, August 2015.   Tara has created a unique rare disease ecosystem within the state that brings together patients, caregivers, academics, clinicians, foundations, industry and any entity in rare disease.  
Information about RDII can be found here:
https://rdii.org/
tjbritt@rdii.org

More information about the council and the network can be found on the following url:
https://ncrarediseasenetwork.org/ 

Robert Donnell

Executive Vice President – Medicines Access, Smartway Pharma, a leading provider of Early / Expanded Access Programs (EAP).

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.

 

Kristin Smedley

President, Curing Retinal Blindness Foundation

Kristin Smedley’s mantra is “Life is funny… sort of.” Her fun-loving spirit and energetic personality guided her life in the direction of a career in teaching. However, when her two sons were born blind, Kristin walked away from the classroom to teach her sons how to navigate a world they could not see.

With the right foundation and a multitude of resources, her children have achieved more than most sighted children their age including recording artist, international Braille competition finalists, top blind athlete in the United States, high school Commencement Speaker, and most recently, Michael was honored with the President’s Award at Penn State University for being in the top 1% of the 75,000 undergrad students and Mitchell scored in the top 3% of all high school students that took the SAT.

In 2011, Kristin launched a nonprofit to fund research and resources for children living with the rare eye disease her sons have, CRB1 LCA/RP. In just ten years, the Curing Retinal Blindness Foundation has raised over 1.4 million dollars and achieved a National Rare Eye Disease Awareness Day.  That legislation, H.R. #625, was the first in US history to be submitted in Braille and it advocates for better resources for blind and visually impaired Americans.
 
In 2019, Kristin published her first book called Thriving Blind: Stories of Real People Succeeding Without Sight.  It achieved #1 New Release and Best Seller on Amazon for paperback and kindle.  Kristin partnered with TMobile and the National Braille Press to make the book available in Braille. She used that book to catapult an engaged and supportive online community called Thriving Blind Academy which connects families living with blindness to each other and to mentors and resources for succeeding without sight.

Kristin lives in suburban Philadelphia with her three children and two rescue dogs.

Robert Keel

Executive Vice President, Managed Access Programs, Tanner Pharma Group

Rob Keel is the Executive Vice President of Managed Access Programs (TannerMAP) and Managing Director in Switzerland for Tanner Pharma Group. He has lived and worked extensively on an international basis and feels it is a privilege to be able to help others. He has held global general management, supply chain and finance roles within the airline, agriculture, and pharmaceutical industries across the EMEA and Asia-Pacific regions. Prior to joining Tanner, Rob was part of the EMEA senior management team at AstraZeneca and held various positions at Alliance Boots (now Walgreens) and Genentech. Rob holds an Industrial Engineering and Management Sciences degree from Northwestern University and an MBA from Harvard Business School.

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.

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.
 

Patricia Weltin

CEO and Founder of the Beyond the Diagnosis.

Beyond the Diagnosis began working on a project to increase research and raise awareness of rare diseases within the medical community through art. Professional artists paint portraits of children living with a rare disease, the portraits then become part of a traveling exhibit for medical schools, research institutes and hospitals. This exhibit has also touched the hearts and minds of the general public. Since the debut, Beyond the Diagnosis has visited the NIH, Broad Institute, Hofstra Medical, Harvard Medical, the FDA and many more.

Patricia, the mother of two children with Ehlers-Danlos Syndrome, began working in this space by creating a new business model of working by state. Her award winning work in state-level advocacy grew to national advocacy efforts. Today, Patricia proudly works to
successfully use art as a powerful tool to create awareness and increase innovation into orphan and neglected diseases.

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