Advancing Rare Disease Clinical Trials: A High-Touch, Patient-Centered Approach For Biotechs

By Alex Kavros, Executive Vice President, Global Scientific & Regulatory Affairs; John Mann, Executive Vice President, North American Operations; and Kevin Leach, Senior Vice President, Global Scientific and Regulatory Affairs

Rare diseases, which affect millions of patients worldwide, present a unique challenge for drug sponsors looking to design effective clinical trials to deliver impactful therapies to patients. This is due to patient scarcity, complex disease progression, limited natural history data, and intricate regulatory pathways. To ensure that a trial gathers sufficient data to bring a drug to market, drug sponsors must prioritize an innovative, patient centric approach throughout trial design and execution.

By developing a high-touch model across every stage of trial development, drug sponsors can execute a trial that centers the patient experience from diagnosis to treatment. Sponsors should also consider how to leverage advanced statistical approaches like Bayesian modeling and procure guidance on regulatory compliance and accelerated regulatory pathways for their rare disease trial. Download the full article to delve into strategies for improving patient recruitment and retention, fostering relationships with patient advocacy groups, and building a trial that is not only scientifically sound but deeply empathetic.