TABLE 2.
The details of the most frequently recommended off-label drugs.
| Drug name | WHO EMLc | Types of off-label drug use | Indication | Types of references for recommendations |
|---|---|---|---|---|
| Acetaminophen | Yes | Unapproved age | Postoperative analgesia | Pediatric case-series |
| Pain control after tonsillectomy | Pediatric RCT; systematic review or meta-analysis | |||
| Pain management for acute severe colitis | Pediatric case-series | |||
| Postoperative pain management | Pediatric RCT; pediatric trial; pediatric cohort; pediatric case control study; pediatric case-series; systematic review or meta-analysis; review; pediatric guideline | |||
| Fentanyl | No | Unapproved population group | Postoperative pain management | Pediatric RCT; pediatric trial; pediatric case control study; pediatric case-series; systematic review or meta-analysis; review; pediatric guideline |
| Pain management for retinopathy of prematurity | Pediatric case-series | |||
| Ropivacaine | No | Unapproved population group | Postoperative pain management | Pediatric RCT; pediatric trial; pediatric cohort; pediatric case report; systematic review or meta-analysis; review; pediatric guideline |
| Regional anesthesia | Pediatric RCT; pediatric trial; adult trail; pediatric cohort; adult trail; pediatric case control study; adult case control study; pediatric case-series; pediatric case report; adult case report; systematic review or meta-analysis; review; consensus | |||
| Cyclosporine | No | Unapproved indication | Ulcerative colitis | Pediatric trial; pediatric cohort; pediatric case-series; adult case series; systematic review or meta-analysis |
| Warm autoimmune haemolytic anaemia | Adult RCT; pediatric case-series; review | |||
| Juvenile dermatomyositis | Pediatric case-series; pediatric guideline | |||
| Steroid-resistant nephrotic syndrome | pediatric RCT; pediatric case-series; systematic review or meta-analysis; review | |||
| Psoriasis | pediatric case-series; systematic review or meta-analysis | |||
| Psoriasis | pediatric case-series; pediatric case report; guideline or consensus; expert opinion | |||
| Glomerular diseases | adult RCT | |||
| Bupivacaine | Yes | Unapproved indication | Postoperative pain | Pediatric RCT; pediatric trial; pediatric cohort; pediatric case-series; systematic review or meta-analysis; review; pediatric guideline |
| Unapproved age unapproved route of administration | Regional anesthesia | Pediatric RCT; adult trail; pediatric cohort; pediatric case control study; adult case control study; pediatric case-series; pediatric case report; adult case report; adult case series; systematic review or meta-analysis; review | ||
| Tramadol | No | Unapproved population group | Postoperative pain | Pediatric RCT; pediatric trial; pediatric case-series; cross-sectional study; systematic review or meta-analysis; review; pediatric guideline |
| Tacrolimus | Yes | Unapproved indication | Ulcerative colitis | Adult RCT; pediatric cohort; pediatric case-series; adult case series; systematic review or meta-analysis |
| Ulcerative colitis | Adult RCT; adult trail; adult cohort; pediatric case-series; pediatric case report; systematic review or meta-analysis | |||
| Juvenile dermatomyositis | Systematic review or meta-analysis; guideline or consensus | |||
| Steroid-resistant nephrotic syndrome | Pediatric RCT; systematic review or meta-analysis | |||
| Psoriasis (topical) | Pediatric trial; pediatric case-series; adult case report; systematic review or meta-analysis | |||
| Glomerular diseases during the COVID-19 pandemic | Adult RCT | |||
| Infliximab | Yes | Unapproved age | Ulcerative colitis | Pediatric RCT; adult RCT; pediatric cohort; adult cohort; pediatric case control study; pediatric case-series; cross-sectional study; systematic review or meta-analysis; review |
| Ulcerative colitis | Pediatric RCT; adult RCT; adult trial; adult cohort; adult case series; cross-sectional study; systematic review or meta-analysis; guideline or consensus | |||
| Unapproved population group | Juvenile idiopathic arthritis | Pediatric RCT | ||
| Unapproved indication | Psoriasis | — | ||
| Psoriasis | Expert opinion | |||
| Mycophenolate mofetil | No | Unapproved indication | Autoimmune pancreatitis with a concomitant diagnosis of inflammatory bowel disease | — |
| Warm autoimmune haemolytic anaemia | Pediatric case-series; cross-sectional study; systematic review or meta-analysis; review | |||
| Juvenile idiopathic arthritis–associated Uveitis | Adult case control study | |||
| Immunoglobulin A vasculitis | Pediatric RCT; pediatric cohort; adult case control study; pediatric case-series | |||
| Juvenile dermatomyositis | Pediatric case-series | |||
| Steroid-resistant nephrotic syndrome | Pediatric RCT; pediatric cohort | |||
| Glomerular diseases during the COVID-19 pandemic | Pediatric case-series; guideline or consensus | |||
| Morphine | Yes | Unapproved population group | Postoperative pain | Pediatric RCT; pediatric trial; pediatric case control study; pediatric case-series; systematic review or meta-analysis; review; pediatric guideline |
| Regional anesthesia | Review | |||
| Pain for retinopathy of prematurity | Pediatric case-series | |||
| Needle procedures | Pediatric RCT; cross-sectional study |
WHO EMLc: World Health Organization Model List of Essential Medicines for Children (2021).