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Hematology

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The chimeric antigen receptor T-cell therapy tisagenlecleucel has been approved for a second type of blood cancer; the National Institutes of Health has started recruiting individuals for a database that will include data on more than 1 million people; Kansas’ request to impose a 3-year lifetime limit on Medicaid benefits is testing just how open the Trump administration is to allowing states flexibility.

Sancilio Pharmaceuticals announced that it has received the European Medicines Agency’s Orphan Designation for its SC411 (which it plans to market as Altemia), a proposed treatment for sickle cell disease in pediatric patients, in the European Union. The FDA granted the proposed drug a similar designation—the Rare Pediatric Disease designation—in 2017.

This week, the top managed care stories included Cigna's deal to buy Express Scripts; Seema Verma, Alex Azar, and Scott Gottlieb, MD, made the rounds at health conferences; an analysis finds FDA's accelerated approval pathway has been a success with hematology and oncology drugs.