The correlation between the costs and clinical benefits of PD-1/PD-L1 inhibitors in malignant tumors: An evaluation based on ASCO and ESMO frameworks
- Cancer drug innovation has significantly accelerated in the 21st century, with novel drug approvals and expenditures increasing notably.
- Assessment frameworks ASCO-VF and ESMO-MCBS were used to evaluate the clinical benefit of PD-1/PD-L1 inhibitors, finding that nearly half of the trials met "meaningful clinical benefit" thresholds.
- There is no significant correlation between the price of cancer drugs and their clinical benefit, consistent with prior studies.
- High drug prices are partly due to the high costs and risks of pharmaceutical research and development, and sometimes an overestimation of drug benefits during expedited FDA approvals.
- ASCO-VF and ESMO-MCBS frameworks show moderate agreement, but differences in their criteria and scoring methods exist.
- Growing expenditures on cancer drugs may impact the allocation of resources for other essential medicines, highlighting the need for balanced investment.
- Limitations of the study include reliance on available and English-only trials, exclusion of non-approved agents, and potential biases due to incomplete data.
- The study suggests that a comprehensive cost-benefit assessment should guide oncological drug approvals, and there should be coordinated efforts to control drug costs.
Survival benefits of patients with a malignant tumor have been improved significantly over the years, partially attributed to the employment of novel anti-cancer therapies. Recent success in immunotherapy propels cancer treatment to an exciting new era after traditional chemotherapy and targeted therapy. To date, approximately 4000 clinical trials focusing on programmed cell death protein-1/programmed cell death protein ligand-1 (PD-1/PD-L1) inhibitors have been carried out in at least 20 types of cancer, including both solid and hematological tumors; the total number of subjects worldwide is more than 20,000. For the moment, approximately six PD-1/PD-L1 inhibitors are commonly used in clinical practice: Nivolumab, Pembrolizumab, Atezolizumab, Avelumab, Durvalumab, and Cemiplimab. These PD-1/PD-L1 inhibitors are demonstrated to have the preeminent potential for long-term survival, but along with dramatic high drug costs. Although the rapid development of novel therapies has provided insights into the future direction of treatments for malignancy, the high cost of cancer treatment has become a major concern for patients and the society. The financial toxicity may lead to psychosocial distress, poor quality of life (QOL), and worse patient outcomes. Thus, the focus that if the survival benefit and living quality are in proportion to the economics expenditure has been in the spotlight...
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