U.S. 監視者 解除するs price 見積(る) for Bluebird, Vertex/CRISPR 遺伝子 therapies to $2.1 mln

By Bhanvi Satija

July 13 (Reuters) - An 影響力のある U.S. 麻薬 pricing 監視者 raised the price 見積(る) of two 実験の 遺伝子 therapies from Vertex 製薬のs/CRISPR 治療力のあるs and bluebird bio to as much as $2.05 mln, 説 the new price can be cost 効果的な to 扱う/治療する sickle 独房 病気.

A price of up to $2.05 million a year, higher than the 事前の 見積(る) in April of up to $1.9 million would 会合,会う some 一般的に used thresholds for cost 有効性, the 学校/設ける for 臨床の and 経済的な Review (ICER) said.

Sickle 独房 病気 leads to a 不足 of healthy 血 独房s and 制限するs 血 循環/発行部数, 原因(となる)ing periodic episodes of 苦痛 in 患者s.

The higher 概算の pricing 反映するs the ICER's 仮定/引き受けること of more 苦痛 attacks per 患者, said David Rind, 長,指導者 医療の officer of ICER.

"The 治療 is 妨げるing more attacks and it's 価値(がある) more," Rind said.

The ICER's 草案 報告(する)/憶測 in April had assumed four attacks per 患者 in a year, compared with 5.1 attacks in its final 報告(する)/憶測.

It 概算の a price 範囲 between $1.35 million and $2.05 million a year for the 遺伝子 therapies, which 示唆するs the highest price a 製造業者 in the 部隊d 明言する/公表するs should 告発(する),告訴(する)/料金 for a 治療.

Both the bluebird and Vertex/CRISPR 遺伝子 therapies are yet to be 認可するd in the 部隊d 明言する/公表するs.

Recently 認可するd 遺伝子 therapies in the U.S. carry a high 名簿(に載せる)/表(にあげる) price 予定 to their 可能性のある to be one-time cures for rare 条件s.

CSL's Hemgenix for hemophilia B has a 名簿(に載せる)/表(にあげる) price of $3.5 million in the U.S., while BioMarin 製薬の's therapy, Roctavian, for 厳しい hemophilia A is 定価つきの at $2.9 million.< /p>

Rind said the 衝撃 of high prices on healthcare 予算s could 潜在的に be an 問題/発行する, but it was too 早期に to 査定する/(税金などを)課す.

"There's a fair 量 of nervousness about the new 遺伝子 therapies," he said. (報告(する)/憶測ing by Bhanvi Satija in Bengaluru)

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