Accessing miracles

With unanimous backing of an FDA advisory committee, overwhelming efficacy and a robust REMS, all signs suggest Novartis AG’s tisagenlecleucel-T is headed to the market for children and young adults with a rare leukemia. The question is how many patients will be able to access it, and not necessarily -- or at least not only -- because of the expected price for the CAR T therapy.

Novartis has not disclosed pricing plans; however, at least one analysis suggests that it would be cost effective at $500,000-$750,000. The pharma has said it is considering outcomes-based arrangements similar to those it used to secure reimbursement for its heart failure drug Entresto sacubitril/valsartan...