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.
Toronto, ON (PRWEB) February 01, 2023
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:
1. The profoundly personal side of rare disease: Humanizing the therapeutic journey of patients
2. Ways to successfully collaborate with patients, families and advocacy groups
3. Supporting patient diagnosis and connectivity to recruitment for clinical trials
4. Ensuring clinical trial data can support regulatory and commercial decisions
Register to learn about breaking down barriers in support of patients with rare diseases.
Join Bill Hanlon, PhD, President CDCS and Chief Scientific Officer, Labcorp Drug Development; 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; Yukari Blumenthal, MD, Medical Lead, Astellas Pharmaceuticals; Philip Yeske, PhD, Science & Alliance Officer, United Mitochondrial Disease Foundation; Borbála Éva Szepes, MD, Clinical Team Lead, Early Phase Clinical Project Team, Global Clinical Operations, Global Medical Division, Gedeon Richter Plc.; Gina Calarco Smith, MPH, RN, CCRC, Scientific Director, Critical Path Institute; and Margaret Dean, MBA, Senior Director, Portfolio Oversight, Rare Disease and Pediatric Team, Labcorp Drug Development, for the live webinar on Wednesday, February 22, 2023, at 11am EST (4pm GMT/UK).
For more information, or to register for this event, visit Breaking Down Barriers in Support of Patients with Rare Diseases.
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