. Author manuscript; available in PMC: 2015 Jun 13.

Published in final edited form as: J Am Stat Assoc. 2014 Jun 13;109(506):525–536. doi: 10.1080/01621459.2014.881740

Table 2.

Simulation study comparing the five phase I–II designs in Case 2. The probability pairs given in parentheses for each dose level are (π_E(d_r)^true, π_T(d_r)^true).

Method		Dose Level						N_E/N_T	Duration (Days)

		1	2	3	4	5	None
	Scenario 1	(0.05, 0.03)	(0.10, 0.05)	(0.20, 0.07)	(0.25, 0.08)	(0.35, 0.10)
	δ^true	0.52	0.53	0.58	0.61	0.67
LO-EffTox	% selected	0.0	0.4	1.1	2.2	90.9	5.4	12.7/3.9	237
	# patients	3.58	4.25	5.99	4.91	27.75
One Down	% selected	0.0	4.8	94.2	1.0	0.0	0.0	2.9/1.6	246
	# patients	38.74	9.23	0.03	0.00	0.00
Look Ahead	% selected	0.0	0.0	1.6	1.9	91.0	5.5	13.3/4.0	399
	# patients	3.25	3.49	4.54	6.11	29.26	(49.2)
Complete Case	% selected	0.0	2.7	7.3	6.3	80.7	3.0	6.2/2.4	244
	# patients	28.96	3.64	3.91	3.35	7.97
TiTE-CRM	% selected	0.0	0.0	0.0	0.0	99.9	0.1	14.2/4.3	240
	# patients	3.22	3.23	3.18	3.40	34.92
	Scenario 2	(0.02, 0.10)	(0.10, 0.15)	(0.40, 0.20)	(0.45, 0.30)	(0.50, 0.60)
	δ^true	0.43	0.43	0.59	0.52	0.32
LO-EffTox	% selected	0.0	9.4	62.5	21.4	5.4	1.3	16.4/12.6	244
	# patients	3.35	8.30	18.77	10.09	7.03
One Down	% selected	0.0	1.8	96.3	1.4	0.0	0.5	1.6/5.2	246
	# patients	38.72	9.17	0.04	0.00	0.00
Look Ahead	% selected	0.1	8.9	56.9	27.9	5.7	0.5	17.3/13.2	436
	# patients	3.27	6.46	17.54	13.38	7.24	(58.8)
Complete Case	% selected	0.0	5.3	29.2	32.0	33.2	0.3	7.9/9.1	246
	# patients	29.11	3.65	5.10	5.09	5.04
TiTE-CRM	% selected	0.0	0.7	14.0	70.8	13.3	1.2	18.3/15.7	238
	# patients	3.99	4.39	8.27	18.97	11.89
	Scenario 3	(0.30, 0.10)	(0.35, 0.20)	(0.45, 0.40)	(0.50, 0.60)	(0.55, 0.65)
	δ^true	0.63	0.55	0.43	0.32	0.31
LO-EffTox	% selected	68.1	27.9	3.4	0.4	0.0	0.2	16.4/9.2	247
	# patients	26.19	13.53	5.82	1.99	0.46
One Down	% selected	44.9	8.0	47.0	0.1	0.0	0.0	14.8/5.6	246
	# patients	38.98	8.99	0.02	0.00	0.00
Look Ahead	% selected	58.3	32.2	8.7	0.7	0.0	0.1	16.7/9.7	420
	# patients	22.21	16.28	7.54	1.69	0.23	(54.0)
Complete Case	% selected	85.1	11.3	2.0	1.5	0.1	0.0	14.9/6.2	247
	# patients	39.77	5.24	2.39	0.57	0.03
TiTE-CRM	% selected	1.1	30.1	62.3	5.1	0.0	1.4	20.1/17.4	238
	# patients	5.32	12.13	18.08	8.98	2.90
	Scenario 4	(0.18, 0.20)	(0.28, 0.24)	(0.55, 0.28)	(0.74, 0.31)	(0.79, 0.33)
	δ^true	0.44	0.46	0.62	0.76	0.78
LO-EffTox	% selected	3.5	3.8	10.4	20.9	61.0	0.4	27.0/13.5	246
	# patients	9.02	5.84	7.97	11.51	13.52
One Down	% selected	17.0	7.5	73.1	1.2	0.0	1.2	9.6/9.9	245
	# patients	38.64	9.09	0.05	0.00	0.00
Look Ahead	% selected	6.4	6.1	14.8	23.1	48.5	1.1	27.1/13.4	445
	# patients	7.58	6.17	9.38	10.55	13.93	(62.0)
Complete Case	% selected	11.4	6.1	7.8	20.1	54.4	0.2	15.3/11.0	246
	# patients	31.64	4.12	3.79	3.82	4.60
TiTE-CRM	% selected	1.1	5.6	19.1	28.0	40.2	6.0	25.9/12.8	231
	# patients	6.37	6.89	9.16	10.50	12.62
	Scenario 6	(0.20, 0.10)	(0.50, 0.19)	(0.52, 0.34)	(0.54, 0.44)	(0.56, 0.54)
	δ^true	0.55	0.69	0.53	0.45	0.38
LO-EffTox	% selected	20.3	63.4	13.9	1.8	0.4	0.2	20.8/10.9	246
	# patients	11.86	20.78	10.87	3.45	0.96
One Down	% selected	8.0	12.4	79.2	0.4	0.0	0.0	12.4/5.6	247
	# patients	38.73	9.24	0.03	0.00	0.00
Look Ahead	% selected	16.7	52.2	26.3	3.8	0.9	0.1	21.6/11.5	402
	# patients	9.10	20.03	13.71	4.26	0.86	(48.6)
Complete Case	% selected	27.8	40.2	22.8	7.1	2.0	0.1	14.4/7.6	246
	# patients	32.84	7.63	5.38	1.82	0.33
TiTE-CRM	% selected	0.0	11.8	51.6	30.2	4.6	1.8	23.5/16.7	237
	# patients	4.47	7.42	14.68	13.57	7.10
	Scenario 8	(0.02, 0.10)	(0.05, 0.25)	(0.35, 0.55)	(0.40, 0.60)	(0.50, 0.70)
	δ^true	0.43	0.34	0.29	0.28	0.27
LO-EffTox	% selected	0.0	1.9	8.1	0.4	0.1	89.5	7.9/13.6	117
	# patients	3.36	7.21	13.99	4.18	1.79
One Down	% selected	0.0	4.2	90.1	1.4	0.0	4.3	1.3/6.1	247
	# patients	38.62	9.31	0.05	0.00	0.00
Look Ahead	% selected	0.0	1.6	18.8	1.1	0.1	78.4	8.2/13.4	367
	# patients	3.22	8.10	16.11	3.79	1.17	(53.5)
Complete Case	% selected	0.0	8.2	33.5	8.7	3.9	45.7	5.0/10.6	204
	# patients	28.81	4.85	7.18	2.84	1.15
TiTE-CRM	% selected	5.8	58.7	31.7	0.9	0.0	2.9	9.1/17.5	236
	# patients	8.00	17.23	14.29	5.65	1.69