White Paper

Rising To The Rare Disease Challenge: Key Considerations In Large-Molecule Orphan Drug Development

Source: Thermo Fisher Scientific

By Christy Eatmon, Global SME, Thermo Fisher Scientific


Rare diseases present a unique set of challenges within drug development due to small-scale manufacturing, limited time/data, small sample sizes, and costs.

This whitepaper provides targeted guidance for overcoming these challenges and ensuring that enough high-quality data is generated from the start of your orphan drug program to guide evidence-based decision making, based on the following:

  • Process design decisions for clinical batches across the three stages of validation
  • Key formulation considerations for sterile dosage forms based on the physical, chemical, and biologic characteristics of the drug substances
  • Optimal API quantities for preclinical development and Phase 1 studies

Moving the needle in the fight against rare disease requires innovative and flexible drug development approach that spans API production phases to sterile product manufacture and takes into account the data limitations of orphan drug research and the inherent vulnerability of biologics at every stage of the development process.

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