Comparison of Long-term Survival Benefits in Trials of Immune Checkpoint Inhibitor vs Non-Immune Checkpoint Inhibitor Anticancer Agents Using ASCO Value Framework and ESMO Magnitude of Clinical Benefit Scale
- Importance: Anticancer agents, especially immune checkpoint inhibitors (ICIs), have shown potential for long-term durable survival in some patients. However, traditional clinical benefit measures may not accurately capture this, leading to proposed amendments in valuation frameworks.
- Objectives: The study aimed to determine the frequency of long-term durable survival trends in ICI vs non-ICI anticancer agents using ASCO-VF v2 and ESMO-MCBS v1.1 frameworks and to analyze the agreement between these frameworks.
- Design, Setting, and Participants: This cohort study analyzed anticancer agents from phase 2 or 3 randomized clinical trials (RCTs) cited for clinical efficacy in FDA drug approvals between January 2011 and March 2018. Data were extracted to determine the frequency of survival benefits awarded by ASCO-VF v2 and ESMO-MCBS v1.1.
- Main Outcomes and Measures: The study measured ASCO-VF v2 tail-of-the-curve bonuses and ESMO-MCBS v1.1 immunotherapy-triggered long-term plateau adjustments.
- Results:
- 247 RCTs were identified; 100 RCTs involving 57,164 patients were included.
- RCTs awarded ASCO-VF v2 bonuses more frequently than ESMO-MCBS v1.1 adjustments (ASCO-VF v2: 45.0% vs ESMO-MCBS v1.1: 2.6%).
- ICIs were not more likely to receive ASCO-VF v2 bonuses or ESMO-MCBS v1.1 adjustments than non-ICI RCTs.
- Poor agreement was found between ASCO and ESMO frameworks in identifying long-term survival benefits from RCTs (κ=0.01; 95% CI, -0.23 to 0.22; P=.50).
- Conclusions and Relevance: ASCO-VF v2 and ESMO-MCBS v1.1 frameworks may need refinement to accurately capture durable long-term survival benefits, or ICIs may not provide long-term survival as previously thought.
Conventional non–immune checkpoint inhibitor (ICI) anticancer agents typically improve patients’ overall survival (OS) and progression-free survival (PFS). However, long-term survival benefits are limited by acquired biological resistance. In contrast to non-ICI agents, ICI agents have the potential to show long-term survival, represented by a plateau at the tail of the survival curve in small patient populations for cancer types including melanoma. While ICI agents are often preferred as treatment options, they are often costlier than non-ICI agents.
As such, there are concerns regarding the relationship between price and clinical benefit. Saluja et al have demonstrated that while the costs of novel oncology drugs have risen during the last decade, the clinical benefits of these medications have not experienced a proportional increase. Efforts to evaluate the association of efficacy with the cost of novel therapies have led to the development of various value frameworks.
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