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Table 2 Characteristics of participants in pivotal trial aggregated at the indication level providing the basis of U.S. Food and Drug Administration approval of novel therapeutic agents between 2011 and 2013

From: Participation of the elderly, women, and minorities in pivotal trials supporting 2011–2013 U.S. Food and Drug Administration approvals

 

All indications

Indications that should involve underrepresented patient groupsa

U.S. population

Age

   

 Medianb (IQR)

53.1 (40.6–60.6)

56.4 (50.5–62.5)

37.2 (n/a)

 Aged ≥65 yearsc, proportion of patients (95 % CI)

28.9 % (23.5–34.4 %)

32.6 % (26.9–38.4 %)

13.0 %

Sex, proportion of patients (95 % CI)

 Male

50.3 % (45.3–55.2 %)

46.4 % (41.8–50.9 %)

49.2 %

 Female

49.7 % (44.7–54.7 %)

53.6 % (49.0–58.2 %)

50.8 %

Race, proportion of patients (95 % CI)

 White

79.2 % (75.9–82.6 %)

77.7 % (74.3–81.2 %)

72.5 %

 Black

7.4 % (5.5–9.3 %)

8.0 % (6.0–10.0 %)

13.2 %

 Asian

7.4 % (5.2–9.7 %)

7.9 % (5.4–10.3 %)

5.4 %

 Otherd

5.9 % (4.4–7.5 %)

6.4 % (4.7–8.0 %)

8.8 %

Ethnicity, proportion of patientse (95 % CI)

 Hispanic

13.3 % (10.3–16.3 %)

n/a

16.3 %

 Non-Hispanicd

86.7 % (83.7–89.7 %)

n/a

83.7 %

  1. n/a not applicable
  2. aIncludes only those indications for which the prevalence of the disease is evenly spread across patients of all ages, sexes, or races. Indications for which the prevalence in three key demographic subgroups (age ≥65 years, female sex, nonwhite race) is less than half that of the prevalence among other patients were excluded.
  3. bMean age was unavailable for 28 trials. In these cases, we assumed that the median age, which was universally reported for these trials, approximated the mean.
  4. cIncludes patients in subgroups defined as >65 as well as ≥65 years old. Age breakdown was not available for 31 (33.7 %) of 92 in overall analysis and 19 (27.1 %) of 70 in the sensitivity analysis.
  5. dIncludes patients whose race/ethnicity was not explicitly defined
  6. eEthnicity information was not available for 37 (40.2 %) of 92 indications in overall analysis. Sensitivity analysis by ethnicity was not performed.
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ISSN: 1745-6215