Pictured: The U.S. Capitol building/iStock, Mikhail Makarov

图为：美国国会大厦/伊斯托克，米哈伊尔·马卡罗夫

Sen. Bill Cassidy (R-La.), ranking member of the Senate Health, Education, Labor, and Pensions Committee, is asking stakeholders for feedback regarding gene therapies, with an eye toward crafting legislation that could make these expensive treatments more accessible for patients.

参议院健康、教育、劳工和养老金委员会资深成员参议员比尔·卡西迪（R-La.）正在征求利益相关者对基因疗法的反馈意见，以期制定立法，使这些昂贵的疗法更容易为患者所接受。

“Policymakers have a responsibility to evaluate the market structure and identify where targeted changes can be made to ensure market access for patients with ultra-rare diseases,” Cassidy wrote in Tuesday’s request for information. “Any proposal put forward by Congress should be informed by the current work of states and the private market.”.

卡西迪在周二的信息请求中写道：“决策者有责任评估市场结构，并确定可以在哪些方面进行有针对性的改变，以确保超罕见疾病患者的市场准入。”。“国会提出的任何提案都应了解各州和私人市场目前的工作。”。

Cassidy is looking for guidance on defining ultra-rare diseases and identifying patients who should be eligible for new coverage schemes. The Republican senator is also looking into the current payment and access practices for patients with ultra-rare diseases: how they pay for their therapy, how physicians provide access to these treatments and what programs—state-sponsored or otherwise—are there for them to leverage..

卡西迪（Cassidy）正在寻找有关定义超罕见疾病和确定应符合新覆盖计划条件的患者的指导。这位共和党参议员还研究了目前针对极罕见疾病患者的支付和获取方式：他们如何支付治疗费用，医生如何提供这些治疗的机会，以及国家资助或其他方面有哪些项目可供他们利用。。

In addition, Cassidy is asking stakeholders—including health insurers and manufacturers—how they decide on a list price for gene therapies and how they manage the financial risk of covering these expensive treatments.

此外，卡西迪正在询问包括健康保险公司和制造商在内的利益相关者，他们如何确定基因疗法的标价，以及如何管理覆盖这些昂贵疗法的财务风险。

Responses to Cassidy’s request for information are due by Jan. 22, 2024, and will help lawmakers determine the appropriate role of the federal government in maximizing access to gene therapies without inadvertently hurting innovation, according to the announcement.

根据公告，卡西迪的信息请求将于2024年1月22日前得到回应，这将有助于立法者确定联邦政府在最大限度地获得基因疗法而不无意中损害创新方面的适当作用。

“Given the current limited number of approved gene therapies, manufacturers and commercial health plans alike are currently able to absorb costs in order to provide patient access,” but this current market environment is unsustainable, according to Cassidy, particularly as approvals in this space “are expected to grow dramatically over the next decade.”.

卡西迪表示：“鉴于目前批准的基因疗法数量有限，制造商和商业健康计划目前都能够吸收成本，以便为患者提供治疗，”但目前的市场环境是不可持续的，特别是因为该领域的批准“预计在未来十年将大幅增长”。

Cassidy warned that “artificially low prices could promote patient access, but eliminate the incentive to bring these treatments to market.” At the same time, without pricing safeguards, “the small patient population runs the risk of bearing the full weight of the high price tag,” he noted.

卡西迪（Cassidy）警告说，“人为压低价格可能会促进患者获得治疗，但会消除将这些治疗推向市场的动机。”同时，如果没有价格保障措施，“患者人数少，就有可能承受高昂价格的全部负担，”他指出。

The initiative in the U.S. Senate to draft legislation tackling the access and affordability of gene therapies comes as the industry and the FDA anticipate a looming boom not only in cell and gene therapy applications, but also in approvals.

美国参议院提议起草立法，解决基因疗法的可获得性和可负担性问题。与此同时，该行业和FDA预计，不仅在细胞和基因疗法应用方面，而且在批准方面，都将迎来一个即将到来的热潮。

On Friday, the FDA is set to release its verdict on yet another gene therapy, Vertex Pharmaceuticals and CRISPR Therapeutics’ exagamglogene autotemcel (exa-cel), for the treatment of sickle cell disease. Neither company has indicated how much they plan to market the therapy, if approved, but it is likely to cost well above $1 million for a single dose..

周五，FDA将公布另一种基因疗法Vertex Pharmaceuticals和CRISPR Therapeutics的exagamglogene autotemcel（exa cel）的裁决，用于治疗镰状细胞病。两家公司都没有表示，如果获得批准，他们计划将该疗法推向市场，但单次剂量的成本可能远高于100万美元。。

In November 2022, the FDA approved CSL’s Hemgenix (etranacogene dezaparvovec-drlb) as the first gene therapy to treat adults with hemophilia B. The treatment costs $3.5 million per dose, a price tag that has won it the title of being the most expensive drug in the world.

2022年11月，FDA批准CSL的Hemgenix（etranacogene dezaparvovec drlb）作为第一种治疗成人血友病B的基因疗法。该疗法每剂售价350万美元，这一价格标签使其成为世界上最昂贵的药物。

Bluebird bio, also focused on cell and gene therapies, owns Zynteglo (betibeglogene autotemcel), an ex-vivo lentiviral treatment indicated for beta-thalassemia, and Skysona (elivaldogene autotemcel), indicated for active cerebral adrenoleukodystrophy. The therapies cost $2.8 million and $3 million, respectively..

蓝鸟生物也专注于细胞和基因治疗，拥有Zynteglo（betibeglogene autotemcel），一种用于β地中海贫血的离体慢病毒治疗，以及Skysona（elivaldogene autotemcel），用于活动性脑肾上腺白质营养不良。治疗费用分别为280万美元和300万美元。。

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

特里斯坦·马纳拉克（TristanManalac）是一位独立的科学作家，总部位于菲律宾马尼拉。联系方式tristan@tristanmanalac.com或tristan.manalac@biospace.com.