Why does gene therapy frequently sell at "skyrocketing prices" with a price of 2.9 million US dollars?

Tech 2023-07-04 18:20:37 Source: Network

(Tan Qixin, health client reporter of People's Daily, Zhang Shuang) On June 29, the Food and Drug Administration announced that it had approved the listing of Biomarin's gene therapy Roctavian for the treatment of severe type A Haemophilia patients, priced at 2.9 million US dollars

(Tan Qixin, health client reporter of People's Daily, Zhang Shuang) On June 29, the Food and Drug Administration announced that it had approved the listing of Biomarin's gene therapy Roctavian for the treatment of severe type A Haemophilia patients, priced at 2.9 million US dollars. People's Daily Health Client reporter noticed that in the past year, at least 5 gene therapies have been approved and launched globally, with prices exceeding 2.5 million US dollars.

Why is gene therapy so expensive? Ding Sheng, founding president of the School of Pharmacy of Tsinghua University and director of the Global Health Drug development Center (GHDDI), analyzed to the health client reporter of People's Daily that there are multiple reasons for the high price of gene therapy, and Pricing needs to comprehensively consider various factors such as R&D investment, production cost, potential efficacy and market demand.

A type of gene therapy refers to a biological therapy method that introduces exogenous normal genes into target cells through gene transfer technology to correct or compensate for diseases caused by gene defects and abnormalities, ultimately achieving the therapeutic goal. Compared with ordinary drugs, this treatment method has a high threshold for early research and development, and the design, optimization, and even carrier selection of therapeutic genes are not easy. The chain of gene therapy is very long, and the choice of carrier is not easy The therapeutic genes of different fragments and the technology used in the gene therapy process need to be designed and inspected according to the patient's situation in each step to ensure safety, which makes it difficult for some types of drugs to be mass-produced and the production cost remains high Ding Sheng explained.

Ding Sheng told reporters that from the perspective of clinical development, the larger the indications of emerging treatment methods are, the better. Emerging technologies are often easier to break through in the field of diseases that seriously affect people's quality of life or life. At present, most gene therapy companies will choose Rare disease for experiments. On the other hand, due to the fact that gene therapy often targets only one indication for a single gene fragment and cannot achieve multiple adaptations, it is only possible to adopt a high price strategy. Without high prices, it is difficult for companies to move forward, whether in foreign or domestic markets

In this context, how can ordinary people use the currently expensive treatment techniques? In order to further improve the accessibility of gene therapy in the future, it is necessary to first encourage and support enterprises to continuously develop new drugs and treatment technologies, forming full competition. More importantly, as treatment plans continue to increase and companies' participation increases, competition will become more intense, which will correspondingly reduce treatment costs and increase accessibility. In addition, medical insurance, commercial insurance, charitable drug giving, and Innovative payment methods such as installment payments can improve the accessibility of emerging treatment options Hao Xiangwen, founder and CEO of Beijing Jiyun Huikang Technology Co., Ltd., told People's Daily Health Client reporter.

In order to further benefit patients with expensive drugs, gene therapy research and development companies have been actively exploring emerging payment methods. For example, when Zynteglo, priced at 2.8 million US dollars, went public in the United States in August 2022, its research and development company Blue Bird Biology introduced a new payment method, which is to refund 80% of the treatment fee after a one-time prepayment if the patient does not achieve the treatment effect of blood transfusion independence (no blood transfusion required within 12 months).

Gene therapy is expected to achieve a complete cure for single gene genetic diseases, and high pricing is reasonable. Increasing production and reducing production costs are the pain points and difficulties that gene therapy urgently needs to overcome. Currently, the scientific community has begun to develop a standardized gene carrier system. With a standardized gene carrier system, gene therapy can achieve standardized/large-scale production in the near future, benefiting more patients Ding Sheng said.

Disclaimer: The content of this article is sourced from the internet. The copyright of the text, images, and other materials belongs to the original author. The platform reprints the materials for the purpose of conveying more information. The content of the article is for reference and learning only, and should not be used for commercial purposes. If it infringes on your legitimate rights and interests, please contact us promptly and we will handle it as soon as possible! We respect copyright and are committed to protecting it. Thank you for sharing.(Email:[email protected])

Mobile advertising space rental

Tag: Why does gene therapy frequently sell at skyrocketing prices