Table 1 Inclusion and exclusion criteria

P

1. Studies in which at least 50% of participants were 18 years old or older and the study was not indicated as pediatric.

2. Patients with a single type of solid tumor or hematological malignancy at any stage

1. Pediatric studies.

2. Patients with benign tumors or other diseases only, without cancer.

I

1. Studies that test agents based on the molecular profiling of an individual patient’s tumor, defined as a method of testing genetic characteristics as well as any unique biomarkers of a cancerous tumor. The results are used to identify and create targeted therapies that work most effectively for specific cancer tumor profiles [4].

1. Studies that did not test agents based on the tumor molecular profiling.

2. Multiple molecularly targeted therapies (monoclonal antibodies or small molecule or antibody-drug conjugates).

3. Combination of both: molecularly targeted therapies and chemotherapy.

4. Targeted therapy combined with surgery.

2. Chemotherapy only in the experimental arm—cytotoxic drug schedules, monotherapy, or polytherapy.

3. Studies in which hormone therapy, immunotherapy, surgery, or radiotherapy were the only treatment.

4. Radiotherapy or immunotherapy used together with targeted therapy.

5. Supportive care without anticancer agents and other types of drugs and treatments, i.e., antiviral agents or non-specific immunotherapy (e.g., interferon, interleukins, cytokines, immunostimulator, GM-CSF granulocyte-macrophage colony-stimulating factor), cancer vaccine, and oncolytic virus therapy.

5. Drugs administered systemically.

6. No systemic administration (e.g., topical only).

C

1. Standard of care/placebo.

2. Experimental arm only in case of a non-match arm.

–

O

1. Measures of benefit: objective response rate or progression-free survival.

2. Measures of risk: grade 3, 4, or 5 drug-related events.

3. Additional outcomes: disease control rate, overall survival, time to progression, and duration of response.

1. No data on measures of benefit and/or risk.

S

1. Umbrella trials defined as studies that have many different treatment arms within one trial (participants are assigned to a particular treatment arm of the trial based on their type of cancer and the specific molecular makeup of their cancer) [4].

2. Sub-studies testing targeted therapy or a combination of targeted therapy and chemotherapy that were part of the umbrella master protocol.

3. Platform umbrella trials as classified by the study authors and/or umbrella trials utilizing Bayesian response-adaptive randomization and/or umbrella trials in which sub-trials are added or suspended continuously [7, 13].

4. Interventional studies of all phases (i.e., I, II, III).

5. Publications of sub-studies that were part of one umbrella study.

1. Studies without umbrella design (e.g., studies testing only one targeted therapy based on the patient’s molecular makeup).

2. Studies that tested targeted therapies on multiple tumor types.

3. Observational studies, review articles, and articles describing only umbrella study design without results.

6. Full articles and abstracts.

4. No full text available.