Table 2. Spending by CMS for Drugs Granted FDA Accelerated Approval Before and After Conversion to Standard Approval, 2012 to 2017.
| Drug characteristic | All drugs | Not converteda | Converted to standard approvalb | ||
|---|---|---|---|---|---|
| Total | Before | After | |||
| Overall CMS drug spending (%), $ in millions | |||||
| All drugs | 67 873.1 | 5795.1 | 62 078.0 | 11 093.2 | 50 984.8 |
| Type | |||||
| Small molecule | 40 883.9 (60) | 2894.2 (50) | 37 989.6 (61) | 4988.3 (45) | 33 001.4 (65) |
| Biologic | 26 989.2 (40) | 2900.9 (50) | 24 088.4 (39) | 6104.9 (55) | 17 983.4 (35) |
| Therapeutic area | |||||
| Cancer | 64 265.8 (95) | 3.837.1 (66) | 60 428.8 (97) | 10 220.7 (92) | 50 208.1 (98) |
| Non-cancer | 3607.3 (5) | 1958.0 (34) | 1649.3 (5) | 872.5 (8) | 776.8 (2) |
| Orphan drug designation | |||||
| Yes | 54 990.8 (81) | 2991.4 (52) | 51 999.4 (84) | 9100.1 (82) | 42 899.3 (84) |
| No | 12 882.3 (19) | 2803.7 (48) | 10 078.6 (16) | 1993.1 (18) | 8085.5 (16) |
| Confirmatory trial primary outcome | |||||
| Clinical outcome | 17 946.3 (26) | 4861.4 (84) | 13 084.9 (21) | 1007.4 (9) | 12 077.6 (24) |
| Surrogate end point | 40 286.3 (59) | 892.6 (15) | 39 393.7 (63) | 4123.5 (37) | 35 270.2 (69) |
| No data | 9640.5 (14) | 41.1 (1) | 9599.4 (15) | 5962.3 (54) | 3.637.1 (7) |
| Supplemental indication | |||||
| Yes | 62 664.9 (92) | 3316.9 (57) | 59 348.0 (96) | 10 085.0 (91) | 49 263.0 (97) |
| No | 5208.2 (8) | 2478.1 (43) | 2730.0 (4) | 1008.2 (9) | 1721.8 (3) |
| Annualized CMS drug spending, median (IQR), $ in millions | |||||
| All drugs | 71.8 (14.6-277.7) | 23.3 (1.2-143.0) | 132.4 (27.9-578.4) | 35.0 (9.2-150.2) | 199.0 (41.3-718.4) |
| Type | |||||
| Small molecule | 62.6 (8.6-192.2) | 7.9 (0.8-86.3) | 132.4 (27.6-332.4) | 48.9 (21.8-180.4) | 199.0 (42.9-475.9) |
| Biologic | 328.0 (26.7-605.7) | 328.0 (175.7-341.6) | 319.8 (28.5-1351.3) | 9.8 (5.7-25.7) | 403.7 (42.8-1746.7) |
| Therapeutic area | |||||
| Cancer | 85.3 (21.6-390.5) | 23.3 (6.4-162.1) | 175.9 (32.6-691.0) | 39.4 (13.1-145.7) | 276.1 (52.5-859.9) |
| Noncancer | 14.6 (0.3-129.6) | 36.4 (0.4-115.1) | 14.6 (7.3-146.2) | 6.2 (3.1-143.7) | 18.0 (9.0-150.3) |
| Orphan drug designation | |||||
| Yes | 51.3 (10.6-224.8) | 9.3 (0.9-71.8) | 89.0 (26.7-496.4) | 30.6 (8.6-136.5) | 122.0 (38.6-617.8) |
| No | 355.2 (341.6-1041.5) | 341.6 (334.8-348.4) | 1727.8 (1727.8-1727.8) | 956.7 (956.7-956.7) | 2156.1 (2156.1-2156.1) |
| Confirmatory trial primary outcome | |||||
| Clinical outcome | 71.8 (23.3-277.7) | 129.6 (47.6-242.3) | 30.3 (17.6-216.8) | 8.6 (5.1-25.7) | 47.0 (23.1-225.9) |
| Surrogate end point | 67.5 (11.0-253.3) | 0.9 (0.3-29.2) | 175.9 (42.4-551.0) | 45.7 (19.5-145.7) | 276.1 (58.7-684.9) |
| No data | 803.1 (404.8-1201.5) | 6.4 (6.4-6.4) | 1599.9 (1599.9-1599.9) | 1431.0 (1431.0-1431.0) | 2078.3 (2078.3-2078.3) |
| Supplemental indication | |||||
| Yes | 249.2 (48.6-648.3) | 328.0 (245.1-341.6) | 241.1 (33.9-776.3) | 39.4 (15.1-154.8) | 315.5 (55.4-967.9) |
| No | 9.3 (0.9-71.8) | 7.9 (0.8-54.8) | 14.6 (3.1-175.9) | 6.2 (0.7-52.2) | 18.0 (3.6-276.1) |
Abbreviations: CMS, Centers for Medicare & Medicaid Services; FDA, US Food and Drug Administration.
a
Excludes 1 drug (deferiprone) for which CMS payments could not be differentiated between multiple products with the same name.
b
Prorated based on the month of conversion to standard approval.