Clinical benefit, development, innovativeness, trials, epidemiology, and price for cancer drugs and indications with multiple special FDA designations
Menée à partir de données de la "US Food and Drug Administration", de la "Global Burden of Disease Study" et des bases "Medicare" et "Medicaid", cette étude examine les développements, les bénéfices cliniques, les innovations, les données épidémiologiques et les prix concernant les anticancéreux aux désignations spéciales et multiples (orphelin, procédure accélérée, approbation accélérée, examen prioritaire, thérapie révolutionnaire)
Résumé en anglais
Background : This study analyzes the development, US Food and Drug Administration (FDA) approval, benefits, innovativeness, trials, epidemiology, and price of cancer drugs with multiple special designations: orphan, fast track, accelerated approval, priority review, breakthrough therapy.
Methods : 355 FDA-approved cancer drug indications with 841 special designations were identified (2012-2022). Trial, epidemiology, and price data were collected from FDA labels, Global Burden of Disease Study, and Medicare and Medicaid. The association between efficacy outcomes and indications’ number of special designations were compared in meta-analyses.
Results : Median development times were 7.3, 7.8, and 5.4 months (p = 0.027) for drugs with 0-1, 2-3, and 4-5 special designations, respectively. Multiple special designations were associated with a higher biotechnological and clinical innovativeness. Median patient enrolment in trials was 615, 471, 398, 168, 104, and 120 (p < 0.001) for indications with 0-5 special designations. Drugs for rare diseases supported by open-label phase 1/2 trials of single-arm design were granted more special designations. Hazard ratios for overall survival (0.80 vs 0.73 vs 0.73 vs 0.69 vs 0.56 vs 0.52, p = 0.003) and progression-free survival (0.70 vs 0.61 vs 0.59 vs 0.44 vs 0.37 vs 0.67, p < 0.001) substantially declined, whilst tumor response increased with more special designations. Mean monthly prices increased for drugs with 0-4, yet not 5, special designations ($21596 vs $14753 vs $32410 vs $41240 vs $38703 vs $19184).
Conclusions : Multiple special designations are associated with faster clinical development and greater benefits for patients with unmet needs; yet, also with non-robust trial evidence and a tendency for higher drug prices.
