What constitutes meaningful benefit of cancer drugs in the context of LMICs? A mixed-methods study of oncologists’ perceptions on endpoints, benefit, price, and value of cancer drugs

IF 7.1 2区医学 Q1 ONCOLOGY

ESMO Open Pub Date : 2024-11-01 DOI:10.1016/j.esmoop.2024.103976

S.S. Datta , V. Sharma , A. Mukherjee , S. Agrawal , B. Sirohi , B. Gyawali

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Abstract

Background

The importance of surrogate endpoints, magnitude of clinical benefit of cancer drugs, and their prices have often been debated in the oncology world. No study, however, has systemically explored oncologists’ perception regarding these issues.

Methods

We conducted a mixed-methods study including in-depth qualitative interviews of medical oncologists prescribing cancer drug therapy in India. Quantitative data were collected using a predetermined proforma. Qualitative in-depth interviews were audio-recorded, transcribed verbatim, anonymized, subsequently coded, and analyzed by generating basic and global themes.

Results

We interviewed 25 medical oncologists. Twenty-eight percent of oncologists rarely used cancer drugs that improved response rate (RR) but not overall survival (OS), and an equal percentage mostly/often used such drugs. For cancer drugs that improved progression-free survival (PFS) but not OS, 20% never/rarely used them while 48% mostly/often used them. Oncologists in India considered a 4.5-month (range, 1.5-12 months) advantage in median PFS as meaningful, and considered price of ∼120 United States Dollars (USD) per month (range, 48-720 USD per month) for those PFS gains as justified. For OS, median gains of 4.5 months (range, 2-24 months) and at a monthly price of ∼360 USD (range, 48-900 USD) was considered justified. Oncologists in India were aware and concerned that RR only meant tumour shrinkage not survival benefit, but many assumed that tumour shrinkage meant better quality of life. Many oncologists acknowledged the limitations of PFS but would use a drug with PFS benefit if it was cheaper than the drug with OS benefit.

Conclusions

Oncologists in India showed awareness of the limited surrogacy between RR/PFS and OS but assumed that RR/PFS correlated with improved quality of life and acknowledged price as a factor in deciding treatment choices. This is the first study providing a benchmark for minimum clinical benefit (4.5 months in PFS or OS) and maximum monthly price (120 USD for PFS, 360 USD for OS) deemed justifiable by oncologists practicing in low-and-middle-income countries.

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在低收入和中等收入国家，什么才是抗癌药物有意义的益处？肿瘤学家对抗癌药物终点、益处、价格和价值看法的混合方法研究

背景肿瘤学界经常就替代终点的重要性、抗癌药物临床疗效的大小以及抗癌药物的价格进行争论。方法我们开展了一项混合方法研究，包括对印度开具抗癌药物处方的肿瘤内科医生进行深入的定性访谈。定量数据使用预先确定的表格收集。深入定性访谈进行了录音、逐字转录、匿名、编码，并通过生成基本主题和总体主题进行分析。28%的肿瘤学家很少使用能提高反应率（RR）但不能提高总生存率（OS）的抗癌药物，同样比例的肿瘤学家大多/经常使用此类药物。对于能提高无进展生存期（PFS）但不能提高总生存期（OS）的抗癌药物，20%的人从不/很少使用，48%的人大部分/经常使用。印度的肿瘤学家认为，无进展生存期中位数提高 4.5 个月（范围为 1.5-12 个月）是有意义的，并认为无进展生存期提高的价格为每月 120 美元（范围为每月 48-720 美元）是合理的。至于 OS，中位收益为 4.5 个月（范围为 2-24 个月），每月价格为 360 美元（范围为 48-900 美元），被认为是合理的。印度的肿瘤学家意识到 RR 仅意味着肿瘤缩小而非生存获益，并对此表示担忧，但许多人认为肿瘤缩小意味着生活质量的提高。许多肿瘤学家承认 PFS 的局限性，但如果具有 PFS 益处的药物比具有 OS 益处的药物便宜，他们也会使用。这是第一项为中低收入国家的肿瘤学家提供最低临床获益（4.5 个月的 PFS 或 OS）和最高月价格（PFS 为 120 美元，OS 为 360 美元）基准的研究。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

ESMO Open Medicine-Oncology

CiteScore

11.70

自引率

2.70%

发文量

255

审稿时长

10 weeks

期刊介绍： ESMO Open is the online-only, open access journal of the European Society for Medical Oncology (ESMO). It is a peer-reviewed publication dedicated to sharing high-quality medical research and educational materials from various fields of oncology. The journal specifically focuses on showcasing innovative clinical and translational cancer research. ESMO Open aims to publish a wide range of research articles covering all aspects of oncology, including experimental studies, translational research, diagnostic advancements, and therapeutic approaches. The content of the journal includes original research articles, insightful reviews, thought-provoking editorials, and correspondence. Moreover, the journal warmly welcomes the submission of phase I trials and meta-analyses. It also showcases reviews from significant ESMO conferences and meetings, as well as publishes important position statements on behalf of ESMO. Overall, ESMO Open offers a platform for scientists, clinicians, and researchers in the field of oncology to share their valuable insights and contribute to advancing the understanding and treatment of cancer. The journal serves as a source of up-to-date information and fosters collaboration within the oncology community.