The Challenges and Opportunities of Rare Disease Drug Development

New therapeutics development in rare diseases presents both opportunities and complexities. Because of the small patient pool available in these indications, there are challenges in designing and conducting clinical trials and the data interpretation that follows, and the ultimate path to registration.

The top 3 critical downfalls in rare diseases development include

Poor understanding of the disease process and natural history
Incomplete understanding of clinically meaningful endpoints
Inability to assess clinical benefit and achieve full approval

This whitepaper by drug development experts, Drs. Julie Bullock and Rajesh Krishna, reflect on some of these challenges and opportunities in rare diseases drug development.

What you’ll learn

Biology and genetics of rare diseases

How to leverage regulatory frameworks to reduce regulatory uncertainty

The challenges of the accelerated approval mechanism

How to streamline rare disease drug development using model-informed drug development

Download the white paper

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About Certara

Certara is dedicated to transforming drug discovery and development for good. We harness the power of biosimulation, advanced analytics, and regulatory expertise to create a future where treatments reach patients faster and more efficiently.

From discovery to market access and commercial, we tailor solutions to meet our clients’ most pressing challenges. Through strategic leadership and advanced predictive technologies, Certara provides comprehensive solutions to optimize drug development processes, reduce risks, and improve outcomes. Our clients include more than 2,400 biopharmaceutical companies, academic institutions, and regulatory agencies across 70 countries.