摘要：2024年11月14日至17日，国际细胞与免疫治疗（CTI）大会在中国杭州盛大举行，汇聚了全球细胞治疗和免疫治疗领域的顶尖专家、学者和行业领袖。此次大会不仅是一个学术交流的国际平台，更是一个展示最新科研成果、推动领域发展的重要窗口。会议期间，《肿瘤瞭望-血液时

2024年11月14日至17日，国际细胞与免疫治疗（CTI）大会在中国杭州盛大举行，汇聚了全球细胞治疗和免疫治疗领域的顶尖专家、学者和行业领袖。此次大会不仅是一个学术交流的国际平台，更是一个展示最新科研成果、推动领域发展的重要窗口。会议期间，《肿瘤瞭望-血液时讯》特别邀请到杜克-新加坡国立大学医学院(Duke-NUS)、新加坡国立癌症中心首席执行官William Hwang ying khee教授，就CAR-T细胞疗法的成本效益进行了深入的探讨，为我们揭示了CAR-T细胞疗法在肿瘤治疗领域的独特优势，以及其在成本效益方面所展现出的巨大潜力。现整理成文，以飨读者。

Q1

您如何看待CAR-T细胞疗法在提高癌症患者生存率和生活质量方面的成本效益？在您看来，哪些因素影响了CAR-T细胞疗法的成本效益？

William Hwang ying khee教授：当我们深入探讨CAR-T细胞疗法的成本效益时，必须着眼于不同疗法所展现的长期有效性。相比之下，其他一些疗法往往要求患者长时间内每月接受给药，且其中不乏复发率较高的疗法。因此，在计算成本时，我们不得不将这些疗法持续给药所需的费用、患者复发后追加的治疗成本，以及因长期治疗而导致患者生活质量下降所带来的间接成本纳入考量。然而，CAR-T细胞疗法作为一种治疗手段，其独特之处在于患者接受治疗后，能够在相当长的一段时间内免于进一步的治疗干预。更为乐观的是，部分患者甚至可能因此实现疾病的完全缓解，达到临床治愈的状态。从长期效益的视角出发，CAR-T细胞疗法无疑展现出了更高的成本效益。

当然，在评估成本效益时，我们还需兼顾治疗产品的特异性和疾病的特异性。不同产品之间，其成本效益可能存在显著差异。同时，针对特定的适应症，也可能存在更为经济高效的治疗选择。此外，我们还必须认识到，不同国家之间的增量成本效益存在差异，这主要归因于各国生活成本和人均国内生产总值的显著差异。同时，CAR-T细胞疗法在不同国家的定价也是影响成本效益评估的重要因素。

因此，在综合考虑上述所有因素的基础上，每个国家都需要针对特定适应症的具体CAR-T细胞疗法，开展独立的成本效益评估工作。

Oncology Frontier-Hematology Frontier：How do you view the cost-effectiveness of CAR-T cell therapy in improving the survival rate and quality of life for cancer patients?In your opinion, what factors influence the cost-effectiveness of CAR-T cell therapies?

Dr.William Hwang ying khee: When we consider the cost-effectiveness of CAR-T cell therapies, we need to consider the lifetime horizon of the effectiveness of these therapies. Some other therapies may require monthly administration for a long time. And then some of these other therapies may have a higher incidence of relapse. We have to take into consideration the cost of continued administration of these therapies. The cost of relapse after the patient gets a relapse and also the quality of life these patients suffer because of being on continued therapies . With CAR-T cell therapies these are one of therapies and the patient is completely treatment-free. After that for at least a long period of time. And some of these patients may be cured. When we consider the lifetime horizon for the benefit of these therapies, CAR-T cell therapies can be more cost-effective.

However, we have to consider product specificity and disease specificity. Certain products may be more cost-effective than other products. There may be also more cost-effective options, only for particular indications. We also have to consider the incremental cost-effectiveness in different countries, because the cost of living, the GDP per capita varies significantly between countries. And also we have to consider the price of CAR-T cell therapies in these different countries. We take that all into consideration, each country will have to do its own assessment of cost-effectiveness for specific CAR-T cell therapies for specific indications.

Q2

针对不同类型的血液癌症，CAR-T细胞疗法的成本效益是否存在差异？如果有，这些差异主要体现在哪些方面？

William Hwang ying khee教授：不同适应症CAR-T细胞疗法在成本效益方面所呈现出的主要差异，主要源自针对特定适应症与特定产品所开展的大型临床试验的结果。具体而言，在二线CAR-T细胞疗法的范畴内，有一款特定产品已在长期生存率及无事件生存率方面展现出显著的优越性。基于这一卓越表现，当我们针对该CAR-T细胞疗法在诸如弥漫性大B细胞淋巴瘤二线适应症中的成本效益进行精确计算时，发现其展现出了更高的成本效益比。然而，对于旨在治疗如多发性骨髓瘤等其他疾病的某些CAR-T细胞疗法产品而言，其有效性比率并未达到如此显著的程度，因此，这些不同疗法的成本效益目前仍处于评估与计算之中。

Oncology Frontier-Hematology Frontier：Are there differences in the cost-effectiveness of CAR-T cell therapies for different types of hematological cancers? If so, what are the main areas of these differences?

Dr.William Hwang ying khee: The main areas of differences in the cost-effectiveness of CAR-T cell therapies for different indications are based on the results of the large clinical trials. They have been performed for these particular indications for that particular product. For example, in second-line CAR-T cell therapies, one of the specific products has been shown to be significantly superior in overall survival and event-free survival in the long-term horizon. So when we calculated the cost-effectiveness for this CAR-T cell therapy for the second-line indications, for diffuse large B-cell lymphoma, it was found to be more cost-effective. For some of the products for other diseases like multiple myeloma. The effectiveness ratio was not so significant. The cost-effectiveness for these CAR-T cell therapies is still currently being worked out.

Q3

您认为应通过哪些方法来提高CAR-T疗法的成本效益？

William Hwang ying khee教授：成本效益的增强可通过两大核心策略达成：首要策略是降低成本，其次则是提升疗效。随着技术的日新月异，我们已能够采取更为高效的方式制造这些细胞，从而大幅度降低成本。在此方面，中国在降低CAR-T细胞疗法成本方面已处于领先地位。另一方面，提升疗效同样占据举足轻重的地位。若能使CAR-T细胞疗法的疗效实现质的飞跃，远超当前的标准疗法，达到更持久的疗效且伴随更少的副作用，那么其成本效益亦将随之显著提升。

此外，CAR-NK细胞疗法的运用则为我们开启了一个全新的视角，即一名供体有可能为众多患者提供治疗的可能性。若这能够降低每位患者的制造费用与给药成本，那么其成本效益亦将得到相应的提升。我对这些研究成果充满了浓厚的兴趣与期待。举例来说，MD安德森癌症中心（MDACC）已经展示了高度有效的CAR-NK细胞能够成功消除肿瘤，并且这种来源于脐带的CAR-NK细胞在移植后至少能够存活一年。我迫切地期望看到更多关于此方面的研究成果。或许，这将成为未来一个极具创新性且颠覆性的发展领域。

Oncology Frontier-Hematology Frontier：What methods do you think should be employed to improve the cost-effectiveness of CAR-T therapy?

Dr.William Hwang ying khee: Cost-effectiveness can be improved through two things. One is by reducing costs, and the other is by improving effectiveness. Reducing costs can be done as we improve technology to be able to manufacture these cells more efficiently. Then we can significantly reduce costs. China is one of the leaders in trying to reduce the costs of CAR-T cell therapies. The other thing is to improve their effectiveness. If you can make CAR-T cell therapies more effective, so that they are significantly better than the standard of care, lasting longer and having less side effects, then in that case we can improve the cost-effectiveness of CAR-T cell therapies.

The use of CAR-NK cell therapies allows us to use this in an energetic setting whereby one donor can potentially donate to several patients. This could improve the cost-effectiveness if it reduces the cost of manufacture and the cost of administration for each individual patient. I'm interested and excited with the results.For example, MD Anderson just showed a highly effective CAR-NK cells being able to eliminate tumors, as well as to be able to last for at least a year after transplant from this cord-derived CAR-NK cell, I'm interested to look at more results for this. So that perhaps this could be a novel, game-changing area of development in the future.

总 结

CAR-T细胞疗法在肿瘤治疗领域展现出了显著的成本效益优势。其长期有效性、治疗产品的特异性和疾病的特异性，以及不同国家间的成本效益差异等因素，共同构成了评估其成本效益的复杂框架。随着技术的不断进步和成本的逐步降低，CAR-T细胞疗法的成本效益将得到进一步提升。同时，CAR-NK细胞疗法的出现为我们提供了新的治疗视角，其在成本效益方面的潜力同样值得期待。未来，随着更多研究成果的涌现，CAR-T及CAR-NK细胞疗法有望在肿瘤治疗领域发挥更加重要的作用，为患者带来更为有效且经济的治疗方案。

来源：肿瘤瞭望