ISSN 0377-9556 (PRINT)
ISSN 2383-9457 (ONLINE)
ISSN 2383-9457 (ONLINE)
| NDA-NMEs and BLAs approved by FDA (N=288) | NDA-NMEs and BLAs without PRO information in labels (N=225) | NDA-NMEs and BLAs with PRO information in labels (N=63) | p value | |
|---|---|---|---|---|
| Application, n (%) | 0.569 | |||
| NDA-NME | 209 (72.6) | 161 (71.6) | 48 (76.2) | |
| BLA | 79 (27.4) | 64 (28.4) | 15 (23.8) | |
| Orphan status, n (%) | 0.002 | |||
| Non-Orphan | 136 (47.2) | 95 (42.2) | 41 (65.1) | |
| Orphan | 152 (52.8) | 130 (57.8) | 22 (34.9) | |
| Expedited approval pathways, n (%) | ||||
| Priority | 182 (63.2) | 152 (67.6) | 30 (47.6) | 0.006 |
| Fast track | 108 (37.5) | 91 (40.4) | 17 (27.0) | 0.071 |
| Breakthrough | 81 (28.1) | 69 (30.7) | 12 (19.0) | 0.098 |
| AA | 51 (17.7) | 51 (22.7) | 0 (0.0) | <0.001 |
| Not applicable | 90 (31.3) | 61 (27.1) | 29 (46.0) | |
| ICD-10, n (%) | <0.001 | |||
| Diseases of the Nervous System | 37 (12.8) | 15 (6.7) | 22 (34.9) | |
| Endocrine, Nutritional, and Metabolic Diseases | 40 (13.9) | 31 (13.8) | 9 (14.3) | |
| Diseases of the Skin and Subcutaneous Tissue | 17 (5.9) | 9 (4.0) | 8 (12.7) | |
| Diseases of the Blood and Blood-Forming Organs, Certain Immune Disorders | 17 (5.9) | 13 (5.8) | 4 (6.4) | |
| Diseases of the Respiratory System | 5 (1.7) | 1 (0.4) | 4 (6.4) | |
| Diseases of the Digestive System | 6 (2.1) | 2 (0.9) | 4 (6.4) | |
| Diseases of the Genitourinary System | 9 (3.1) | 6 (2.7) | 3 (4.8) | |
| Diseases of the Musculoskeletal System and Connective Tissue | 4 (1.4) | 2 (0.9) | 2 (3.2) | |
| Mental, Behavioral, and Neurodevelopmental Disorders | 7 (2.4) | 5 (2.2) | 2 (3.2) | |
| Certain Infectious and Parasitic Diseases | 33 (11.5) | 32 (14.2) | 1 (1.6) | |
| Diseases of the Circulatory System | 1 (0.3) | 0 (0.0) | 1 (1.6) | |
| Injury, Poisoning and Certain Other Consequences of External Causes | 3 (1.0) | 2 (0.9) | 1 (1.6) | |
| Neoplasms | 87 (30.2) | 86 (38.2) | 1 (1.6) | |
| Symptoms, Signs and Abnormal Clinical and Laboratory Findings, Not Elsewhere Classified | 6 (2.1) | 6 (2.7) | 1 (1.6) | |
| Pregnancy, Childbirth, and the Puerperium | 3 (1.0) | 3 (1.3) | 0 (0) | |
| Factors Influencing Health Status and Contact with Health Services | 3 (1.0) | 2 (0.9) | 0 (0) | |
| External Causes of Morbidity and Mortality | 2 (0.7) | 2 (0.9) | 0 (0) | |
| Diseases of the Eye and Adnexa | 7 (2.4) | 7 (3.1) | 0 (0) | |
| Codes for Special Purposes | 1 (0.3) | 1 (0.4) | 0 (0) | |
BLA, Biologic License Application; FDA, Food and Drug Administration; NDA, New Drug Application; NME, New Molecular Entity; AA, Accelerated Approval; ICD-10, International Classification of Diseases 10th revision
“Main endpoints” is when PRO is used as either a primary or secondary endpoint in studies.