Using Biosimulation to Support Approvals for Orphan Drugs

Rare diseases affect fewer than 1 in 2000 people. Each one affects only a small number of patients. Yet, there are over 7000 rare diseases. And, there are no treatments for 95 percent of them. Thus, many patients suffer from these diseases. The treatments for rare diseases are often referred to as "orphan drugs." Orphan drug developers face distinct challenges with rare diseases, including:

  • Heterogeneity in disease progress and treatment outcomes
  • Few patients to run new studies
  • Uncertain appropriate durations of treatment
  • Sparse existing data available from limited populations

Biosimulation methods include both top-down (empirical) and bottom-up (mechanistic) models. These methods use sparse data from small populations to inform dosing and trial designs. For example, population PK/PD models can test the influence of factors such as age, weight, and disease status on drug exposure and response. Likewise, combining drug and disease models can help distinguish between treatment effects on symptoms vs changes in disease processes. Model based approaches can support accelerated approval pathways that get treatments to patients faster.

Certara has supported the approval of scores of orphan drugs using its pharmacometric portfolio of solutions, including Physiologically-Based Pharmacokinetics (PBPK) and Population PK. Example cases include: