TABLE 1.

Characteristics of Included Studies

Study	Study Population	No. of Patients	Intervention	Comparator	Primary Study End Point(s)	Biomarker Testing Method(s)
Drilon et al17 (phase I/II study)	Locally advanced or metastatic NTRK fusion-positive tumors (age 4 months to 76 years) previously treated with therapy other than kinase inhibitors (where available); ECOG PS 0-2 (ECOG PS 0–3 were eligible)	55 (8 adults and 12 pediatric patients from the phase I trial; 35 adults and adolescents from the phase II trial); 1 patient with pancreatic cancer	Larotrectinib (orally, 100 mg twice daily for adults or children with a BSA ≥ 1 m2)	No comparator	Overall (complete and partial) response rate	TRK fusions were identified using NGS (50 patients) or FISH (5 patients) as routinely obtained by each participating site
Doebele et al18 ALKA- 372–001 (phase I), STARTRK-1 (phase I), STARTRK-2 (phase II global basket study)	Locally advanced or metastatic NTRK fusion-positive solid tumors; ECOG PS 0–2; minimum life expectancy of 3 months (ALKA or STARTRK-1) or 4 weeks (STARTRK-2) and adequate organ function	54 patients with advanced or metastatic NTRK fusion- positive solid tumors (6%; 3 patients with pancreatic cancer); 51 patients from STARTRK-2; 2 patients from STARTRK-1; 1 patient from ALKA-372–001	Entrectinib (orally, at least 600 mg once per day in capsule form)	No comparator	Objective response rate and median duration of response	Patients enrolled on the basis of local testing, including FISH, quantitative PCR, or DNA- based or RNA-based NGS; STARTRK-2 phase II study patients required to provide tumor tissue for NGS
Golan et al11 (phase III POLO trial, 119 sites, 12 countries)	Patients with platinum-sensitive metastatic pancreatic cancer and germline BRCA1 or BRCA2 mutations who received at least 16 weeks of platinum-based chemotherapy (therapy continued as long as there was no evidence of disease progression)	154 patients	Maintenance therapy with olaparib tablets (300 mg twice daily)	Placebo	Progression-free survival	“[D]etection of a germline BRCA mutation by central testing with the use of the BRACAnalysis CDx text (Myriad Genetic Laboratories) or by local testing with subsequent confirmation with the use of BRACAnalysis CDx test after randomization”

Abbreviations: BSA, body surface area; ECOG PS, Eastern Cooperative Oncology Group performance status; FISH, fluorescence in situ hybridization; NGS, next-generation sequencing; PCR, polymerase chain reaction.