Orphan Drug Development

Orphan Drugs: Unique Challenges Require Unique Development Approaches

Developing drugs for rare diseases poses a range of clinical, regulatory and commercial challenges. The small number of patient populations are difficult to identify and recruit for clinical trials. Many orphan diseases are genetic. Often these patients have complex phenotypes that react very differently to proposed treatment protocols. The diseases may be poorly understood, making setting clinical endpoints, biomarkers or outcome measures difficult. Patients may include sensitive subpopulations, ranging from neonates and pediatrics to people with co-morbidities, where conducting clinical trials is ethically problematic.

Modeling and Simulation Streamline Orphan Drug Development

Modeling and simulation (M&S) approaches are not only ideal for the development of orphan drugs, but are encouraged by regulators. M&S allow quantifying the drug-disease-trial and exposure-response models from small populations, gaining insight into biomarkers and endpoints. It facilitates dose selection, identifying drug-drug and drug-food interactions, recommending the pediatric treatment when only adult data is available, and modeling the impact of the drug on other disease states. We have supported the approval of scores of orphan drugs, including nine in 2015 alone.

Supporting Regulatory Interactions

As seasoned drug developers, Certara’s team of scientists, clinical pharmacologists, regulatory strategists and regulatory writers work alongside orphan drug development teams on global drug approvals. Our Synchrogenix regulatory writing team has more than 100 writers with proficiency in orphan, pediatric, oncology and global filings, as well as a deep understanding of how to articulate the results derived from M&S.

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


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