Relationships Between ICER Clinical Evidence Ratings and FDA Approval Decisions
Author(s)
Philip J. Membrino, BSc, Connor Davies, BA;
Costello Medical, Boston, MA, USA
Costello Medical, Boston, MA, USA
Presentation Documents
OBJECTIVES: ICER recently assessed MDMA-assisted therapy for PTSD, concluding the clinical evidence was insufficient for decision making. Thereafter, the FDA rejected the NDA. This analysis aimed to determine whether relationships exist between ICER clinical evidence ratings and FDA approval decisions.
METHODS: Completed ICER assessments from 2018-2024 were reviewed for inclusion in the analysis. Key information extracted from the ICER assessments included the therapy, comparators, disease area, clinical evidence rating, and year of report. For therapies with multiple comparators, the evidence rating compared to usual care was prioritized. FDA approval decisions were reviewed for all therapies assessed within the included ICER reports.
RESULTS: A total of 63 ICER assessments were reviewed. Fifty-four were included for analysis encompassing 98 therapies and nine were excluded. Reasons for exclusion were COVID-19 related conditions, outdated FDA decisions, or repetition. As assessed by ICER, of the 98 therapies, 18 received a high certainty rating with 15.8% (16/98) assessed to have “Superior”, 1% (1/98) “Incremental” and 1% (1/98) “Comparable” net health benefits, respectively. Sixty-four therapies had moderate certainty with 28.6% (28/98) assessed to have “Incremental or Better”, 10.2% (10/98) “Comparable or Better”, 8.2% (8/98) “Comparable or Incremental”, and 18.3% (18/98) “Promising but Inconclusive” benefit respectively. Sixteen therapies had low certainty with 15.8% (16/98) assessed to have “Insufficient” evidence. Excluding pending FDA decisions, 100% (16/16) of therapies with high certainty evidence, 88.9% (56/63) with moderate certainty, and 81.3% (13/16) with low certainty were approved, respectively.
CONCLUSIONS: A strong alignment between ICER’s high- and moderate-certainty clinical evidence ratings and FDA approval outcomes, with therapies rated “Superior”, “Incremental”, “Comparable”, “Incremental or Better”, and “Comparable or Incremental” achieving 100% approval. Lower-certainty ratings such as “Promising but Inconclusive” and “Insufficient” showed variable approval rates.
METHODS: Completed ICER assessments from 2018-2024 were reviewed for inclusion in the analysis. Key information extracted from the ICER assessments included the therapy, comparators, disease area, clinical evidence rating, and year of report. For therapies with multiple comparators, the evidence rating compared to usual care was prioritized. FDA approval decisions were reviewed for all therapies assessed within the included ICER reports.
RESULTS: A total of 63 ICER assessments were reviewed. Fifty-four were included for analysis encompassing 98 therapies and nine were excluded. Reasons for exclusion were COVID-19 related conditions, outdated FDA decisions, or repetition. As assessed by ICER, of the 98 therapies, 18 received a high certainty rating with 15.8% (16/98) assessed to have “Superior”, 1% (1/98) “Incremental” and 1% (1/98) “Comparable” net health benefits, respectively. Sixty-four therapies had moderate certainty with 28.6% (28/98) assessed to have “Incremental or Better”, 10.2% (10/98) “Comparable or Better”, 8.2% (8/98) “Comparable or Incremental”, and 18.3% (18/98) “Promising but Inconclusive” benefit respectively. Sixteen therapies had low certainty with 15.8% (16/98) assessed to have “Insufficient” evidence. Excluding pending FDA decisions, 100% (16/16) of therapies with high certainty evidence, 88.9% (56/63) with moderate certainty, and 81.3% (13/16) with low certainty were approved, respectively.
CONCLUSIONS: A strong alignment between ICER’s high- and moderate-certainty clinical evidence ratings and FDA approval outcomes, with therapies rated “Superior”, “Incremental”, “Comparable”, “Incremental or Better”, and “Comparable or Incremental” achieving 100% approval. Lower-certainty ratings such as “Promising but Inconclusive” and “Insufficient” showed variable approval rates.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR160
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling
Disease
No Additional Disease & Conditions/Specialized Treatment Areas