. 2021 Oct 18;181(12):1–9. doi: 10.1001/jamainternmed.2021.5983

Table 3. Top 20 Oral Targeted Cancer Drugs by Spending in 2018^a.

Rank	Generic name	Spending, $ million^b	No. of exposed patients	Spending per patient, $	First approval time	Cancer type	RCT^c	OS benefit^d	OS primary end point^e	Orphan drug designation	FDA approval type^f
Rank	Generic name	Spending, $ million^b	No. of exposed patients	Spending per patient, $	First approval time	Cancer type	RCT^c	OS benefit^d	OS primary end point^e	Orphan drug designation	Fast track	Priority review	Breakthrough therapy	Accelerated approval
1	Palbociclib	209.0	2539	82 301	2015	Breast cancer	Yes	No	No	No	No	Yes	Yes	Yes
2	Abiraterone acetate	95.8	1497	63 962	2011	Prostate cancer	Yes	Yes	Yes	No	No	Yes	NA	No
3	Ruxolitinib phosphate	75.8	821	92 290	2011	Myelofibrosis/polycythemia vera	Yes	No	No	Yes	No	Yes	NA	No
4	Enzalutamide	74.3	1156	64 234	2012	Prostate cancer	Yes	Yes	Yes	No	Yes	Yes	No	No
5	Pomalidomide	69.3	734	94 452	2013	Myeloma	Yes	Yes	No	Yes	Yes	No	No	Yes
6	Osimertinib mesylate	61.9	650	95 176	2015	Lung cancer	Yes	No	No	Yes	Yes	Yes	Yes	Yes
7	Cabozantinib s-malate	38.1	485	78 455	2012	Thyroid cancer/renal cell carcinoma	Yes	Yes	No	Yes	Yes	Yes	No	No
8	Olaparib	28.2	440	63 983	2014	Ovarian/fallopian tube/peritoneal/breast cancer	Yes	Yes	No	Yes	No	Yes	No	Yes
9	Ixazomib citrate	24.5	404	60 644	2015	Myeloma	Yes	No	No	Yes	No	Yes	No	No
10	Alectinib hydrochloride	23.8	222	106 999	2015	Lung cancer	Yes	No	No	Yes	No	Yes	Yes	Yes
11	Neratinib maleate	20.2	351	57 443	2017	Breast cancer	Yes	No	No	No	No	No	No	No
12	Abemaciclib	19.7	377	52 126	2017	Breast cancer	Yes	No	No	No	Yes	Yes	Yes	No
13	Trametinib dimethyl sulfoxide	19.0	378	50 259	2013	Melanoma/lung cancer/thyroid cancer	Yes	Yes	No	Yes	Yes	No	No	No
14	Bosutinib monohydrate	16.4	170	96 592	2012	Leukemia	Yes	No	No	Yes	No	No	No	No
15	Dabrafenib mesylate	16.3	346	47 184	2013	Melanoma/lung cancer/thyroid cancer	Yes	Yes	No	Yes	Yes	No	No	No
16	Lenvatinib mesylate	16.1	215	74 758	2015	Thyroid cancer/renal cell carcinoma/hepatocellular carcinoma	Yes	No	No	Yes	No	Yes	No	No
17	Niraparib tosylate	14.4	254	56 743	2017	Ovarian/fallopian tube/peritoneal cancer	Yes	No	No	Yes	Yes	Yes	Yes	No
18	Crizotinib	13.0	143	91 250	2011	Lung cancer	Yes	No	No	Yes	Yes	Yes	NA	Yes
19	Ponatinib hydrochloride	13.0	87	149 362	2012	Leukemia	No	No	No	Yes	Yes	Yes	No	Yes
20	Venetoclax	12.9	318	40 588	2016	Leukemia	Yes	No	No	Yes	No	Yes	Yes	Yes

Abbreviations: FDA, US Food and Drug Administration; NA, not applicable; OS, overall survival; RCT, randomized clinical trial.

^{^a}

Oral targeted cancer drugs with only cancer indications.

^{^b}

Sum of total gross payments associated with dispensing events.

^{^c}

At least 1 RCT supporting approval for at least 1 indication by December 31, 2018.

^{^d}

Documented overall survival benefit by December 31, 2018.

^{^e}

The primary end point in label-reported pivotal RCTs by December 31, 2018.

^{^f}

Data NA when the drug was approved before breakthrough therapy designation option (2012).

Table 3. Top 20 Oral Targeted Cancer Drugs by Spending in 2018a.

Table 3. Top 20 Oral Targeted Cancer Drugs by Spending in 2018^a.