TPP: For aerosolized surfactant therapy in neonates with respiratory distress syndrome in low and middle income countries

Target product profile for aerosolized surfactant therapy in neonates with respiratory distress syndrome in low- and middle-income countries

Indication

Target product profile (TPP) for the minimal and optimal characteristics of an aerosolised surfactant that can be used for neonates (aged 0-28 days) with respiratry distress syndrome (RDS) in low and middle income countries (LMICs).

Intended use

Minimal: Treatment of RDS not responding to use of noninvasive respiratory support devices (NRS) (e.g. CPAP)

Optimal: Treatment of RDS regardless of use of noninvasive respiratory support devices (NRS) (e.g. CPAP)

Target population

Minimal: Neonates > 1000g birth weight or > 28 weeks gestational age with RDS

Optimal: Any neonate with RDS

Use setting

Target facilities in LMIC

Minimal: Facilities providing level 2 special care and able to refer to level 3 facilities 

Optimal: Facilities providing level 2 special care regardless of referral network

 

Initiation time

Minimal: If treatment of infant with RDS is started up to 12 hours after birth it will still have maximum effect

Optimal: If treatment of infant with RDS is started up to 48-72 hours after birth it will still have maximum effect

 

Frequency

Minimal: Needs no more than 3 doses for maximum effect equivalent to current preparations

Optimal: Needs only one dose for maximum effect

 

Dose calculation

Minimal: Dosing is ml/kg body weight

Optimal: Dose is independent of body weight or calculated in ‘bands’ of body weight (e.g. <0.5kg, 0.6-1.0kg, 1.1-1.5kg etc.)

 
Performance

Time of onset of effect

Minimal: Must have measurable clinical improvement (such as improved oxygenation, reduced

Optimal: Must have measurable clinical improvement within 15 min of treatment completion

Efficacy
Minimal: > 20% reduction in mortality or “failed CPAP” in infants who receive CPAP plus surfactant compared to infants who receive CPAP alone18 Optimal: > 30% reduction in mortality or “failed CPAP” in infants who receive CPAP plus surfactant compared to infants who receive CPAP alone18 The same or better efficacy than currently available surfactant given intratracheally. 
Safety
Minimal: Comparable to adverse events from current surfactant therapy Optimal: Lower than adverse events from current surfactant therapy
Product type
Medicine