ESMO-MCBS Evaluation and French Derogatory Access Decision: Is There a Correlation?
Author(s)
Killian Carnet Le Provost, PharmD1, Arnaud Bayle, MD, PhD1, Christophe Massard, MD, PhD2, Isabelle BORGET, PharmD, PhD1.
1Office of Biostatistics and Epidemiology / INSERM CESP U1018 Oncostat, Gustave Roussy, Villejuif, France, 2Drug Development Department (DITEP) / INSERM U1015 & CIC1428, Gustave Roussy, Villejuif, France.
1Office of Biostatistics and Epidemiology / INSERM CESP U1018 Oncostat, Gustave Roussy, Villejuif, France, 2Drug Development Department (DITEP) / INSERM U1015 & CIC1428, Gustave Roussy, Villejuif, France.
OBJECTIVES: The ESMO-Magnitude of Clinical Benefit Scale (ESMO-MCBS) is a validated tool used to assess the clinical value of cancer drugs, based on the concept of substantial benefit (SB). In July 2021, France reformed its Early Access (EA) program to accelerate access to promising therapies. This study evaluates whether SB, as defined by ESMO-MCBS, correlates with French EA decisions.
METHODS: All EA applications for solid tumors submitted to French health authorities between July 2021 and April 2025, with an available ESMO-MCBS score, were included. SB is defined by ESMO-MCBS as grade A or B in curative settings or as a score of 4/5 in non-curative settings. The correlation between SCB and EA outcomes (approval or rejection), as well as decision timelines, was analyzed.
RESULTS: Among 77 applications (covering 40 drugs), most involved targeted therapies (60%) or immunotherapies (39%), used mainly in monotherapy (58%) and non-curative settings (81%). Supporting evidence was based on phase III (78%) or single-arm trials (21%), with endpoints including overall survival (31%) and progression-free survival (33%). Of these, 33 applications with SB were approved and 12 without SB were rejected, yielding a 58% concordance. However, 10 applications were refused despite a SB according to ESMO-MCBS —due to negative risk-benefit profiles, existing alternatives (often linked to treatment deferral), or lack of innovation—and conversely 22 without SB were approved. Finally, in 71% of cases, ESMO-MCBS preceded the EA decision, with a median delay of 134 days (interquartile range [IQR]: -343 to 14).
CONCLUSIONS: ESMO-MCBS is only moderately aligned with EA decisions. While SB may help inform access decisions, it currently appears to be a complementary, rather than stand-alone, tool for guiding early access assessments. Other clinical and regulatory factors might play an important role and require future investigations.
METHODS: All EA applications for solid tumors submitted to French health authorities between July 2021 and April 2025, with an available ESMO-MCBS score, were included. SB is defined by ESMO-MCBS as grade A or B in curative settings or as a score of 4/5 in non-curative settings. The correlation between SCB and EA outcomes (approval or rejection), as well as decision timelines, was analyzed.
RESULTS: Among 77 applications (covering 40 drugs), most involved targeted therapies (60%) or immunotherapies (39%), used mainly in monotherapy (58%) and non-curative settings (81%). Supporting evidence was based on phase III (78%) or single-arm trials (21%), with endpoints including overall survival (31%) and progression-free survival (33%). Of these, 33 applications with SB were approved and 12 without SB were rejected, yielding a 58% concordance. However, 10 applications were refused despite a SB according to ESMO-MCBS —due to negative risk-benefit profiles, existing alternatives (often linked to treatment deferral), or lack of innovation—and conversely 22 without SB were approved. Finally, in 71% of cases, ESMO-MCBS preceded the EA decision, with a median delay of 134 days (interquartile range [IQR]: -343 to 14).
CONCLUSIONS: ESMO-MCBS is only moderately aligned with EA decisions. While SB may help inform access decisions, it currently appears to be a complementary, rather than stand-alone, tool for guiding early access assessments. Other clinical and regulatory factors might play an important role and require future investigations.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HPR70
Topic
Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Reimbursement & Access Policy
Disease
Oncology