Table 2 Variables collected in step 1 for each cancer drug-indication pair

 Brand name

(Character string)

As accepted by the US Food and Drug Administration (FDA) and used in the US.

 Generic name

(Character string)

According to US Adopted Names.

 Type of active compound

“NME”; “NBE”

NME (New Molecular Entity, that is, a small molecule) or NBE (New Biologic Entity; that is, a biologic product).

 Date of marketing authorization

(Date)

Format: YYYY-MM-DD.

 Innovation class

“First-in-class”; “Advance-in-class”; “Addition-to-class”

Drug innovation class, following the definitions and categories described by Lanthier et al. [17]. New molecular or new biological entities are categorized as “First-in-class” if they define a new drug class, as “Advance-in-class” if they offer significant therapeutic advance (that is, they were granted priority review by the FDA) over existing drugs in the same class, or “Addition-to-class” in any other case.

 FDA-approved indication

(Character string)

Medical condition for which the drug of interest has been approved, according to the first-ever available FDA drug label.

 Line of treatment

“1st”; “2nd”; “3rd”; “4th”

The clinical order the treatment is given

 NDA/BLA number

(Integer)

FDA’s Original New Drug Application (NDA) or Biologics License Application (BLA) number. A unique identifier assigned to each application for approval submitted to the FDA.

 Site of disease

“Breast”; “Digestive”; “Gastrointestinal”; “Endocrine and Neuroendocrine”; “Genitourinary”; “Gynecologic”; “Leukemia”; “Lymphoma”; “Musculoskeletal”; “Neurologic”; “Other - Multicentric Castleman’s Disease”; “Other - Other”; “T-cell malignancies”; “Respiratory/Thoracic”; “Skin”

Cancers by body location/system (following the classification by the National Cancer Institute (www.cancer.gov/types/by-body-location).

 Priority review

“Standard”; “Priority”

Priority review is an expedited FDA review program for drugs that provide a significant improvement over existing therapies.

 Accelerated approval

“Yes”; “No”

Expedited FDA approval pathway for drugs that (a) treat serious conditions, (b) provide a meaningful advantage over available therapies, and (c) demonstrate effects on a surrogate endpoint that is reasonably likely to predict clinical endpoints. Accelerated approved drugs do not meet regulatory standards for traditional or full approval and are therefore required to provide evidence of clinical benefit in subsequent pivotal trials.

 Breakthrough therapy designation

“Yes”; “No”

An expedited program at FDA introduced in 2012 for drugs that are (a) intended to treat serious conditions and (b) provide preliminary clinical evidence of substantial improvement over existing therapies.

 Orphan designation

“Yes”; “No”

A status assigned by the FDA to rare disease indications if less than 200,000 people in the US are affected.