“They called him ‘The Boy.’ A tiny child with a devastating genetic disease who wasn’t supposed to blow out the candles on his first birthday cake. Who wasn’t supposed to sit up. Or talk. Not only did this baby survive to do all these things, but he became a poster child for gene therapy with the regulators at the U.S. Food and Drug Administration (FDA). Born with spinal muscular atrophy (SMA), the little boy was saved by the drug Zolgensma, a one-time intravenous infusion that restored the child’s defective SMN1 gene.
Gene-altering and other advanced medicines are radically rewriting the future for people with some of the rarest, and most devastating, diseases on earth. However, these complex treatments present challenges to developers over and above the well-documented hurdles of rare-disease research.
A new white paper, published by Xtalks in collaboration with experts at Medpace, Inc., aims to set up innovators for success with an up-to-the-minute review of current challenges (including COVID-19), best practices and hands-on wisdom from industry leaders.
The Xtalks White Paper, The Intersection of Rare Disease and Advanced Therapies: What it Means for Clinical Development, is available for free download. By weaving industry hands-on knowledge and the academic literature, the authoritative 20-page publication presents hard-won lessons learned and best practices surrounding the unique realities of advanced-therapy development.
Medpace leaders and a patient advocate share insiders’ gems with readers, backed up by the latest data, case studies and published commentary from regulators, including the U.S. FDA.
A key theme of the White Paper is collaboration. All clinical trials rely on a well-oiled machine of motivated professionals, but nowhere is this more true than in advanced-therapy studies in rare diseases where knowledge is sparse. From patient’s families, the de facto experts in the disease, through regulators adapting to the new realities, trial sites and ethics boards, to erudite international experts, collaboration is key, say the White Paper authors.
“In many conditions the natural history of the disease is relatively unknown,” said Terence Eagleton, MD, Vice-President of Medical Department, Medpace. “Because we need to learn we need to co-operate.”
The White Paper provides hands-on advice for mobilizing and sustaining these relationships. One chapter discusses how to form early partnerships with regulators. Another encourages readers to make use of the unique ground-zero experience of families and patients in all aspects of trial design – insight that is often under-valued by drug innovators. “Patient input is critical because something could be scientifically interesting but is not feasible for patients to do.” In a call to action, Ann Woolfrey, MD, Senior Medical Director at Medpace, commented that patient input “could be leveraged in more cases.”
The White Paper is also designed as a reference source for developers of advanced therapies. In addition to industry data, a definitive checklist on the operational, regulatory, scientific and human challenges facing developers will help strategic planners. There is also a one-stop guide to helpful regulatory mechanisms for developers in the U.S. and E.U.
The White Paper contents are:
- The current industry landscape
- The added challenges of advanced therapies in rare-disease clinical trials
- Setting up for success through
- Relevant and simple endpoints
- Integrating patients into all trial functions
- Partnering with regulators early
- Planning for site variability and long follow up
- Adjusting for COVID-19
Meantime, ‘The Boy’ continues to focus attention on success; on the rewards of getting it right when it comes to development of gene therapies and other advanced medicines. “The whole indication was turned upside down,” said Michelle Petersen, MS, Senior Director of Clinical Trial Management at Medpace. “We’ve seen patients who were never supposed to sit up preparing for preschool instead of dead at age one.”
Download the white paper here.
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