Formulating for rare and pediatric diseases presents unique obstacles: small patient populations, strict excipient limits, and the need for accurate, palatable dosing, every decision carries weight.
Liquid medications, while familiar, often susceptible to microbial growth, inconsistent dosing, and temperature-sensitive storage. Converting these liquids and suspensions into mini tablets or ODTs offers a safer, more stable, and patient-friendly alternative.
The Orphan Drug Challenge
These therapies demand smarter formulation choices that can make development viable, stable, and safe at any scale.The need is significant. Rare diseases affect an estimated 300 million people worldwide (1 in 20), yet fewer than 10% of known conditions have an approved treatment. Despite growing demand, orphan drug programs face limited patient reach and high development costs that make formulation efficiency essential.
Every excipient, every stability parameter, and every patient experience matters, especially for pediatric therapies where compliance and safety define success. Behind every rare or pediatric formulation is a patient population too small to attract large-scale investment, yet too critical to overlook.
$610B
12.2%
$6.1B
Why Orphan Drug Development Demands More Precision
With small-scale populations and narrow safety margins, success depends on stable, patient-friendly formulations that can reach those who need them most.
⚠️ Over 300 children died from contaminated cough syrups in 2022-2025 — Solid dosage forms reduce this risk.
The Liquid Formulation Crisis
- Contamination Risk: DEG/EG toxins causing kidney failure and death
- Microbial Instability: Short shelf life, cold chain requirements
- Dosing Errors: Inconsistent measurements in liquid suspensions
- Compliance Issues: Poor taste, difficult administration
- Supply Chain Vulnerability: Complex manufacturing, quality control failures
The Solid Dosage Advantage
- Zero Contamination: No liquid excipients = no DEG/EG risk
- Superior Stability: Extended shelf life, room temperature storage
- Accurate Dosing: Precise unit doses every time
- Patient-Friendly: Taste masking, fast dissolving ODTs
- Simplified Manufacturing: Robust processes, easier quality control
UltraBurst™ Platform Technology
Built for formulators ready to move beyond liquid limits. Suitable for pediatric syrups, antihistamine or antibiotic suspensions, pain management, nutritional supplements, and orphan drug formulations targeting rare metabolic, genetic, and oncology conditions. Designed for 503A and 503B facilities, specialty compounders, and pediatric-focused pharmacies.
Performance Highlights:
- High-Dose APIs: Handles challenging active ingredients with excellent compactibility
- Rapid Dissolution: Tablets dissolve in seconds for immediate relief
- Pediatric-Optimized: Easy to consume, patient-friendly formulations
- Content Uniformity: Precise dosing for small patient populations
Case Study: Diphenhydramine Mini ODTs
API: Taste-Masked APAP (Acetaminophen)
Challenge: High dose API with compactibility and content uniformity issues
Solution: Liquid suspension successfully converted to mini ODT using UltraBurst™ technology
