A hub and spoke model for treating and referral sites allows efficient recruitment planning and wide patient study access, however, challenges the study execution with long-distance or cross-border patient management.
TORONTO (PRWEB)
February 13, 2023
Clinical development is becoming increasingly complex with continuing innovations in cell and gene therapies, especially when factoring in the hurdles of rare disease research. To achieve cell and gene therapy approvals in rare diseases, it is essential to overcome challenges specific to these trials.
Common clinical challenges to consider when planning trials include the complexities related to gene therapy and underlying conditions, as well as needing to monitor patient visits closely and efficiently after administration to ensure study endpoint protection. Operational considerations are often associated with patient recruitment, retention, compliance and challenges behind long-term patient follow-up. A hub and spoke model for treating and referral sites allows efficient recruitment planning and wide patient study access, however, challenges the study execution with long-distance or cross-border patient management.
Additionally, the regulatory landscape continues to evolve to meet challenges with the ongoing development of innovative types of gene therapy, including gene editing, as well as the need for regulatory guidance and flexibility to ensure that a rare disease therapy’s development strategy will meet regulator expectations. Rare disease studies must consider the constraint of very low patient numbers while also needing to achieve appropriate statistical significance to demonstrate efficacy and meet regulatory expectations; having early and regular interactions with regulators to ensure agreement of proposed study designs is essential. Receiving Orphan Drug Designation for a product in development is an important milestone and provides numerous incentives, including regulatory assistance, which helps to facilitate interactions with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) and other authorities from around the world.
Register now for the webinar on Rare Disease Day 2023 to join the discussion with Medpace medical, regulatory and operational experts on successful approaches for achieving cell and gene therapy approvals in rare diseases.
Join experts from Medpace, Terence Eagleton, MB BS, BSc, Senior Medical Director; Marco Tangelder, MD, PhD, Senior Medical Director; Laura Omoboni, MS, Executive Director, Clinical Trial Management; and Trevor Walker, DPhil, Senior Director, Regulatory Affairs, for the live webinar on Tuesday, February 28, 2023, at 1pm EST (10am PST).
For more information, or to register for this event, visit Successful Approaches from Rare Disease and Cellular and Gene Therapy Product Approvals.
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