Rare diseases often face unique obstacles such as small patient populations, sensitive clinical profiles, and complex regulatory pathways. Certara integrates model-informed drug development (MIDD) strategies to address these complexities. By leveraging quantitative models, regulatory expertise, and real-world data, we empower our partners to fast-track innovative therapies while meeting stringent regulatory standards.
Rare and Neglected Diseases
Unique challenges require unique approaches
Partner with Certara to accelerate rare disease drug development
Advancing breakthroughs
in rare disease drug development
Accelerated approvals
Certara’s modeling and simulation strategies have led to expedited approvals, including rare and neglected diseases achieving accelerated pathways.
Expert regulatory guidance
With over 200 regulatory experts, Certara ensures seamless rare and neglected disease filings and compliance with global standards.
Quantifying small populations
Our clinical pharmacology expertise enables insights into biomarkers, endpoints, and exposure-response models from limited patient data.
Market access solutions
We employ robust frameworks to justify pricing, reimbursement, and access strategies for rare disease therapies.
Certara’s proven leadership in Rare Disease innovation
Certara combines cutting-edge biosimulation technology with decades of expertise to deliver impactful solutions for rare disease drug development.
Explore Certara’s Rare Disease solutions
Early development solutions
Start with the end in mind.
Pharmacometrics services
Quantitative modeling for informed decisions.
Regulatory affairs consulting
Comprehensive regulatory strategy and support.
Clinical pharmacology & translational medicine
Expertise in exposure-response models.
Market access and pricing
Robust frameworks for reimbursement success.
CMC services
Early development manufacturing strategies.
DMPK solutions
Advance your compounds from candidate selection to clinical development.
Nonclinical development services
Preclinical insights for rare diseases.
Early HTA engagement
Proactive health technology assessments.
Drug development stewardship
End-to-end program management.
PK/PD data programming support
Data-driven pharmacokinetics insights.
Real-world evidence
Leverage patient data for actionable insights.
Certara’s Rare Disease specialists
Innovative solutions for rare diseases
View allCollaborate with Certara’s experts
Transform your rare disease drug development with Certara’s integrated solutions. From preclinical insights to market access strategies, we provide unparalleled expertise to guide you through every stage of the drug development process.
Comprehensive services: End-to-end support for rare disease programs.
Global leadership: Trusted by leading pharma and biotech companies.
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Certara’s comprehensive drug development solutions
Frequently asked questions about rare disease drug development
What makes rare disease drug development challenging?
Small patient populations, sensitive profiles, and complex regulations create unique hurdles. Certara addresses these with advanced biosimulation and regulatory expertise.
How does Certara ensure regulatory success?
With over 200 experts, we provide proactive support from pre-IND to postmarketing stages, ensuring compliance and success.
Why is modeling and simulation critical in rare disease research?
M&S enables precise insights into small patient populations, biomarkers, and endpoints, improving trial outcomes and accelerating approvals.
