Small molecule orphan drugs: Balancing financial incentives and complex challenges


For companies pursuing an orphan drug, successfully traversing the path to regulatory submission requires striking the right balance of speed-to-clinic and cost. While there is no one-size-fits-all approach, shared characteristics of successful development projects include:

  • Early and frequent engagement with regulatory authorities
  • Alignment of CMC and clinical activities
  • Transparent communication among internal and external stakeholders
  • An integrated vendor strategy

In this whitepaper, our experts help guide you through the complex challenges of getting your small molecule orphan drug to market faster.