Advancements in artificial intelligence, machine learning, and computing technologies have fueled progress in the use of sophisticated computational modeling and simulation as a tool for facilitating preclinical and clinical research in rare diseases. In this white paper, we explore the...
Expertise: Rare Disease 84 results
In a world of ever-increasing clinical trial costs, sponsors can feel overwhelming pressure to stretch their budgets without sacrificing the data they need for regulatory approval. In rare diseases, this pressure is intensified with the many challenges that often accompany...
Rare diseases are a distinctive and complex field within clinical development, characterized by significant hurdles from start to finish. Addressing these complexities requires innovative approaches to achieve a future of adaptability, quality, and efficiency. Here are 5 key steps to...
Designing patient-centric programs can have a resoundingly positive impact on clinical research and the health and life of patients. Consider these six ways to “reverse engineer” development strategy to accommodate the true rare disease patient journey.
It is an unfortunate reality that many diseases and conditions affect such small numbers of patients that, when a sponsor develops a drug or biological product to treat them, relatively little return on investment is generated to offset the high development costs....
Rare Disease
Daniel Zamfir, MBA
Rare Disease
Operationalizing Gene Therapy Trials
To achieve operational excellence in gene therapy trials, sponsors must understand – and overcome – obstacles ranging from start-up regulations and site selection to patient recruitment and retention. Gene therapy holds promise for treating a wide range of diseases, from...
Rare Disease