DISEASE BURDEN, INNOVATION, AND EVIDENCE GAPS IN FDA ONCOLOGY APPROVALS: A 10-YEAR CROSS-CANCER ANALYSIS
Author(s)
Minhea Lee, BS, MS, RPh1, Hankil Lee, BS, MS, RPh, PhD2.
1College of Pharmacy, Ewha Womans University, Seoul, Korea, Republic of, 2College of Pharmacy, Graduate Program in Regulatory Science Convergence, Ewha Womans University, Seoul, Korea, Republic of.
1College of Pharmacy, Ewha Womans University, Seoul, Korea, Republic of, 2College of Pharmacy, Graduate Program in Regulatory Science Convergence, Ewha Womans University, Seoul, Korea, Republic of.
OBJECTIVES: To assess whether cancer-specific disease burden is associated with innovation, evidence maturity, and regulatory pathway characteristics in FDA oncology approvals and to evaluate the relationship between innovation and evidence maturity.
METHODS: FDA Oncology Approval Notifications issued from 2016-2025 were reviewed retrospectively. Oncology indications were mapped to 21 American Cancer Society cancer groups and linked to corresponding 5-year survival estimates as an indicator of disease burden. Innovation was classified using a modified Olivier framework as novel mechanism of action (MoA), next-in-class, or subsequent indication. Regulatory pathway (regular vs. accelerated approval) and overall survival (OS) evidence availability were extracted. Pearson correlation analyses were used to evaluate associations among disease burden, innovation, evidence maturity, and regulatory pathway.
RESULTS: Among 379 oncology approvals, 58 (15.3%) were classified as novel MoA therapies. Approval activity was concentrated in leukemia (n=74) and lung cancer (n=71), whereas pancreatic (n=2) and esophageal cancers (n=3) received few approvals despite having among the lowest 5-year survival rates. No meaningful associations were observed between 5-year survival and approval volume (r=0.02), novel MoA proportion (r=-0.12), OS evidence availability (r=-0.09), or accelerated approval proportion (r=0.12). Interestingly, cancer groups with higher proportions of novel MoA therapies demonstrated lower rates of OS evidence availability (r=-0.50) and higher proportions of accelerated approvals (r=0.41).
CONCLUSIONS: These findings indicate that oncology drug development is driven more by innovation dynamics and regulatory flexibility than by unmet clinical need based on disease burden. Notably, approval volume itself was not reflective of therapeutic innovation, as cancer types with more approvals did not necessarily have higher proportions of novel MoA therapies. Greater policy attention to prioritizing drug development in cancer types with high disease burden and limited therapeutic innovation may be warranted to address persistent gaps in cancer treatment.
METHODS: FDA Oncology Approval Notifications issued from 2016-2025 were reviewed retrospectively. Oncology indications were mapped to 21 American Cancer Society cancer groups and linked to corresponding 5-year survival estimates as an indicator of disease burden. Innovation was classified using a modified Olivier framework as novel mechanism of action (MoA), next-in-class, or subsequent indication. Regulatory pathway (regular vs. accelerated approval) and overall survival (OS) evidence availability were extracted. Pearson correlation analyses were used to evaluate associations among disease burden, innovation, evidence maturity, and regulatory pathway.
RESULTS: Among 379 oncology approvals, 58 (15.3%) were classified as novel MoA therapies. Approval activity was concentrated in leukemia (n=74) and lung cancer (n=71), whereas pancreatic (n=2) and esophageal cancers (n=3) received few approvals despite having among the lowest 5-year survival rates. No meaningful associations were observed between 5-year survival and approval volume (r=0.02), novel MoA proportion (r=-0.12), OS evidence availability (r=-0.09), or accelerated approval proportion (r=0.12). Interestingly, cancer groups with higher proportions of novel MoA therapies demonstrated lower rates of OS evidence availability (r=-0.50) and higher proportions of accelerated approvals (r=0.41).
CONCLUSIONS: These findings indicate that oncology drug development is driven more by innovation dynamics and regulatory flexibility than by unmet clinical need based on disease burden. Notably, approval volume itself was not reflective of therapeutic innovation, as cancer types with more approvals did not necessarily have higher proportions of novel MoA therapies. Greater policy attention to prioritizing drug development in cancer types with high disease burden and limited therapeutic innovation may be warranted to address persistent gaps in cancer treatment.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HPR70
Topic
Clinical Outcomes, Health Policy & Regulatory, Methodological & Statistical Research
Topic Subcategory
Approval & Labeling
Disease
Oncology