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BioWorld - Saturday, April 4, 2026
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Home » Regulatory, financial hurdles the gap between science and access
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Regulatory, financial hurdles the gap between science and access

June 5, 2025
By Mari Serebrov
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Children with solid tumors who relapse are being treated with the same chemotherapy they would have been given 40 years ago, as “there have been no major approvals for pediatric solid tumors,” Catherine Bollard, senior vice president and chief research officer at Children’s National Hospital, said at a June 5 FDA roundtable on cell and gene therapies (CGTs). The problem isn’t the science. Bollard said many groups are working on curative CGTs “for these children who have lost all other hope for survival.” The real gap is that “big pharma doesn’t see the business model because it’s a rare disease,” she added.
BioWorld Regulatory Cancer Rare disease Cell therapy Gene therapy U.S. FDA

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