By Bhanvi Satija<\/p>\n
July 13 (Reuters) – An influential U.S.drug pricing watchdog raised the price estimate of two experimental gene therapies from Vertex Pharmaceuticals\/CRISPR Therapeutics and bluebird bio to as much as $2.05 mln, saying the new price can be cost effective to treat sickle cell disease.<\/p>\n
A price of up to $2.05 million a year, higher than the prior estimate in April of up to $1.9 million would meet some commonly used thresholds for cost effectiveness, the Institute for Clinical and Economic Review (ICER) said.<\/p>\n
Sickle cell disease leads to a shortage of healthy blood cells and Buy Paypal Account<\/a> restricts blood circulation, causing periodic episodes of pain in patients.<\/p>\n The higher estimated pricing reflects the ICER’s assumption of more pain attacks per patient, said David Rind, chief medical officer of ICER.<\/p>\n “The treatment is preventing more attacks and it’s worth more,” Rind said.<\/p>\n The ICER’s draft<\/a> report in April had assumed four attacks per patient in a year, compared with 5.1 attacks in its final report.<\/p>\n It estimated a price range between $1.35 million and $2.05 million a year for the gene therapies, which suggests the highest price a manufacturer in the United States should charge for a treatment.<\/p>\n Both the bluebird and Vertex\/CRISPR gene therapies are yet to be approved in the United States.<\/p>\n Recently approved gene therapies in the U.S.carry a high list price due to their potential to be one-time cures for rare conditions.<\/p>\n CSL’s Hemgenix for hemophilia B has a list price of $3.5 million in the U.S., while BioMarin Pharmaceutical’s therapy, Roctavian, for severe hemophilia<\/a> A is priced at $2.9 million.<\/p>\n Rind said the impact of high prices on healthcare budgets could potentially be an issue, but it was too early to assess.<\/p>\n “There’s a fair amount of nervousness about the new gene therapies,” he said.(Reporting by Bhanvi Satija in Bengaluru)<\/p>\n<\/div>\n <\/b><\/p>\n