There are over 7,000 rare diseases, and one in 10 individuals worldwide have a rare disease. Over 675 diseases have some research and development targeting supportive care, treatment or a cure for these patients, with approximately only 5% of indications having a treatment available. There is tremendous opportunity and an even larger patient need.
A limiting challenge remains the complexity of rare disease development and a rapidly evolving landscape. An increasing number of diseases are targeted by large research and development pipelines with increasing complexity to complete studies in a timely manner, as evidenced by the almost 31,000 studies started in the last 10 years across the 675 indications. In addition, there is a growth of indications for which research has never been completed.
Success in this space requires collaboration across many stakeholders including families and patients, sites, investigators, labs, advocacy groups, regulators and the various service providers that support clinical trial delivery. Rapid engagement and work with these stakeholders is important in the search for answers.
This webinar will provide important perspectives from these stakeholders on:
- The profoundly personal side of rare disease: Humanizing the therapeutic journey of patients
- Ways to successfully collaborate with patients, families and advocacy groups
- Supporting patient diagnosis and connectivity to recruitment for clinical trials
- Ensuring clinical trial data can support regulatory and commercial decisions
Register to learn about breaking down barriers in support of patients with rare diseases.
Bill Hanlon, PhD, President CDCS and Chief Scientific Officer, Labcorp Drug Development
Dr. Bill Hanlon is President CDCS, Clinical Therapeutic and Regulatory Sciences and Chief Scientific Officer of Labcorp Drug Development. He has responsibility for all the scientific, therapeutic, market access & regulatory expertise, patient recruitment, medical device & diagnostics development, cell & gene therapy growth and digital innovation for the clinical trials business.
Dr. Hanlon has over 30 years of scientific, drug development and business leadership experience as well as extensive background in international business. Starting his career at Merck, Inc. (MSD) in 1988, he quickly advanced in responsibility, broadening his expertise in discovery research, global regulatory affairs and driving strategic product development. Starting at Covance in 2012 as Head of Global Regulatory Affairs, he built a regulatory strategy and product development consulting group to support biotech companies in developing their products.
Dr. Hanlon is a member of the Board of Trustees for the Institute of Life Science Entrepreneurship (ILSE), a 501(c)(3) not-for-profit organization that is accelerating life sciences innovation in medicine, devices and other technologies to improve human health. He also represented the American Clinical Laboratory Association on the inaugural Governing Committee of the National Evaluation System for health Technology Coordinating Center (NESTcc) (2017-2021). Dr. Hanlon served as a Board observer for MC-10, Inc. (2017-2020), a medical digital device company and currently is a Board Observer at BioSpectal, a European medical software as a device company.
Frederick Derosier, D.O., Vice President and General Manager, Rare Diseases, Advanced Therapies and Pediatrics Team (RAPT) and Clinical Lead, Enterprise Cell and Gene Therapy, Labcorp Drug Development
Dr. Frederick Derosier joined Labcorp Drug Development in 2017 and has over 10 years of private practice experience (internal medicine) and more than 20 years of clinical development experience, primarily in neurosciences and rare diseases, in the pharmaceutical industry with large/mid-sized pharma and biotechs. Dr. Derosier’s experience broadly extends from translational/early phase development through product launch to late phase/post-marketing studies and lifecycle management. He provides medical/scientific leadership for clinical development programs involving rare diseases and/or advanced therapeutics at Labcorp Drug Development.
Yukari Blumenthal, MD, Medical Lead, Astellas Pharmaceuticals
Dr. Blumenthal is a surgical doctor and a researcher by profession, She graduated from St. George’s University in London Medical School, conducted her internship and Clinical Fellowship for Surgery (Urology) at the Cambridge University Hospital, UK.
Apart from her formal job, Dr. Blumenthal is also a founder of the Germany-based NPO Kindness United Neuroscience Org, (K.U.N.O.) aiming to support families and research for Alexander disease and other rare diseases. She writes a column based on the theme “Circle of Kindness” and serves as a consultant member to a government committee advising on guidelines for transitional medical care in Japan.
Dr. Blumenthal is also a mom. After her son, Kuno was diagnosed with Alexander disease, she shifted her research focus to rare diseases and collaborates with Kyoto University CiRA in Japan. As Associate Medical Director for Astellas Pharma International, Yukari promotes patient advocacy and research in rare conditions with high unmet medical needs.
Philip Yeske, PhD, Science & Alliance Officer, United Mitochondrial Disease Foundation
Dr. Philip Yeske is the United Mitochondrial Disease Foundation (UMDF)’s Science & Alliance Officer. He received a bachelor’s degree in chemistry from Allegheny College and a doctorate in organic chemistry from Emory University. Prior to joining UMDF, he served as President and CEO of Fluorous Technologies, a Pittsburgh-based, early-stage life sciences company. He also worked at Bayer Corporation in positions ranging from basic research to global account management and co-founded the drug discovery company, MS2 Array.
Dr. Yeske has been active in the mitochondrial disease community since 2004, first as a parent of an affected child, then as a trustee of the United Mitochondrial Disease Foundation from 2010 to 2012. In 2013, he accepted the position of UMDF Science & Alliance Officer, responsible for leading the foundation’s research mission and managing all scientific and business development efforts related to improved diagnoses, development of treatments and cures and optimized patient care.
Borbála Éva Szepes, MD, Clinical Team Lead, Early Phase Clinical Project Team, Global Clinical Operations, Global Medical Division, Gedeon Richter Plc.
Dr. Borbála Éva Szepes is a clinical operations professional passionate about early phase drug development. Dr. Szepes is well-versed in the management and execution of Phase I-II -studies, globally, in the CNS therapeutic area. She graduated as a medical doctor and then joined Gedeon Richter Plc., where she gained multi-angle insights into the broad spectrum of original compound development with innovative clinical trial programs. With a general solution-oriented, pragmatic attitude, holistic view and creative mindset she has been part of the team that drove the early scientific networking activities of Gedeon Richter Plc. in its first rare disease study.
Gina Calarco Smith, MPH, RN, CCRC, Scientific Director, Critical Path Institute
Gina Calarco Smith is a Scientific Director of Pediatrics at the Critical Path Institute, a non-profit organization working in a collaborative manner to assess the needs and develop solutions to expedite rare disease drug development. Gina is a pediatric nurse with a master’s in public health that she earned from the University of Kansas. Nearly her entire career has been focused within pediatrics beginning as a neonatal ICU nurse and moving into study coordination at a large pediatric academic center and most recently working within the CRO setting as a Senior Director of Strategy and Planning developing proposals and supporting delivery of complex and rare indication pediatric clinical trials.
Margaret Dean, MBA, Senior Director, Portfolio Oversight, Rare Disease and Pediatric Team, Labcorp Drug Development
Margaret Dean has 18 years of experience in the healthcare industry across pharmaceutical, healthcare IT and CRO industries within rare disease, cardiovascular and oncology fields. Her broad functional experience spans strategic planning, business development, deal negotiation, marketing, sales and program management.
Who Should Attend?
- Chief Medical Officer, Senior Medical Director, Medical Director
- Chief Executive Officer
- Clinical Development Director
- Research and Development Director
- Clinical Outcome Assessment Specialist
- Senior Operational Director, Operational Director
- Senior Clinical Director, Clinical Director
- Clinical Research Director
- Project Director, Project Manager
- Patient Recruitment Director
- Patient Engagement Director
- Global Head of Patient Engagement and Recruitment
What You Will Learn
Attendees will gain insights into:
- Labcorp’s commitment to collaborate with patient community, patient advocacy groups, academia and sponsors in support of patients with rare diseases in clinical trials
- Labcorp’s strategy to overcome critical barriers with innovative solutions to efficiently conduct rare disease studies
- Global regulatory strategy and operational execution in rare disease clinical development
- Scientific leadership on rare disease treatment endpoint selection, development and validation
- Early scientific networking in a rare disease study by partnering with patient advocacy groups
- Engagement and retention of patients in clinical trials, expanded access programs and patient registries
- How working with partners like the United Mitochondrial Disease Foundation (UMDF) are supporting rare disease clinical development through sponsored patient testing programs
Labcorp is a leading global life sciences company that provides vital information to help doctors, hospitals, pharmaceutical companies, researchers and patients make clear and confident decisions. Through our unparalleled diagnostics and drug development capabilities, we provide insights and accelerate innovations to improve health and improve lives. Learn more about Labcorp at www.Labcorp.com or follow us on LinkedIn and Twitter @Labcorp.