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Improving Drug Development for Rare Patients Post-COVID
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Journal Article
Improving Drug Development for Rare Patients Post-COVID
2021
Overview
Engaging the FDA from an early time point helped to paint a confident development roadmap as did the trailblazing work from an earlier program led by Dr. Timothy Yu at Boston Children's Hospital.2 Through this collaboration, a new way of advancing life-saving medicine is born-a furthering of the template first forged by Dr. Yu in an effort to define a path for families, academics and clinicians to treat even a single patient impacted by a genetic disease. Concepts from as the potential for 2D and 3D tissue modeling in lieu of an animal model to test efficacy to common delivery platforms in the case of AAV reducing the need for repetitive vector toxicology. A recent report by McKinsey cited that 45% of respondents (cell and gene therapy companies) expected delay's of 3-6 months on average for development programs.4 Diving into the details, delay's regarding site activation for clinical trials, patient recruitment for trials and follow up appointments of enrolled patients were top areas of delays with 55% of respondents citing these three areas as delayed. Lessons such as allowing virtual trials visits informed by diagnostic tests being done locally rather than having the patient go to a clinical trial site thereby limiting visits, trial burden and potential exposure (even in a post-COVID world).
Publisher
MultiMedia Healthcare Inc
Subject
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