Rare disease trials of new drugs often encounter patient recruitment challenges, small sample sizes, and ethical complexities. This Drug Discovery & Development article explains how biosimulation and virtual trial technology can help pharma companies overcome these barriers, streamline development, and de-risk decision-making across the product lifecycle.
The Inner Circle Newsletter
February 2026
The leading monthly newsletter to help transform drug discovery and development for good, together.
The Inner Circle Newsletter
August 2025
The leading monthly newsletter to help transform drug development for good, together.
Transforming Rare Disease Drug Development Through Innovation
February 28th marks Rare Disease Day, a global recognition of the millions of people living with rare conditions and the urgent need for faster, smarter drug development. While each rare disease may affect a small population, collectively they represent a tremendous unmet medical need for everyone. In 2025, more than 50% of FDA novel approvals were for rare disease indications and all but one were Certara clients. Rare disease research uniquely requires innovative approaches that Certara Drug Development strategy and modeling and simulation approaches fill in.
This month, we’re spotlighting how innovative approaches, from virtual twin models and biosimulation to novel regulatory frameworks that are reshaping rare disease drug research, accelerating clinical insights and helping get these critical new treatments to the patients that urgently need them.
Article
How Biosimulation and Virtual Trials Can Bust Through Clinical Trial Roadblocks
Blog
Certara’s Virtual Twin® QSP Models: Transforming Rare Disease Research into Clinical Insights
Rare disease programs often face limited patient populations and sparse clinical data. This blog explores how Quantitative Systems Pharmacology (QSP) and virtual twin models are helping researchers simulate disease progression, evaluate treatment response, and make more confident decisions earlier in development.
Podcast
On Virtual Twin® Models in Rare Disease Research with Piet van der Graaf
In this episode of the Pharmaforum Podcast, web editor Nicole Raleigh speaks with Piet van der Graaf, Senior Vice President at Certara. They discuss how virtual twin models are being applied to rare disease therapy research, enabling data-driven insights when traditional clinical trial approaches are limited.
Article
To Accelerate Rare Disease Progress, Take a Sandbox Approach
This thought-provoking Biospace article highlights the need for regulatory and development “sandboxes” to test innovative approaches in rare disease drug development. By fostering collaboration and flexibility, stakeholders can reduce barriers and accelerate therapies to patients who urgently need them.
White Paper
Rare Oncology Product Launch Playbook: Universal Strategies for Success in the U.S. and Europe
Launching a rare oncology therapy presents unique commercial and access challenges. This white paper outlines strategic considerations for successful product launch in the U.S. and Europe, including market access, stakeholder engagement, and cross-functional planning.
Resources
Rare Disease Drug Development Services
Explore how specialized expertise, integrated modeling and simulation, and regulatory strategy support can help advance rare disease drug development programs from early discovery through regulatory approval. Certara’s services are designed specifically to meet the challenges of rare disease development.
Take the Tufts Center for the Study of Drug Development Survey on Modeling & Simulation
The Tufts Center for the Study of Drug Development at Tufts University School of Medicine is conducting a global study to better understand how modeling and simulation are being used in drug development.
We greatly value your perceptions and experience. Please complete this 10-minute survey. To thank you for your participation, the Tufts CSDD will send you a summary report of the study findings. All responses are strictly confidential and reported only in aggregate.
Greater Certainty in Rare Disease Drug Development
Meet the Certainty US 2026 Keynote Speaker
David Fajgenbaum, MD, MBA, MSc.
Co-Founder and President of Every Cure
As the Co-Founder and President of Every Cure and a physician-scientist at the University of Pennsylvania, Dr. Fajgenbaum has advanced numerous life saving treatments for cancer and rare disease with support from ARPA-H and TED’s Audacious Project. Bestselling author of "Chasing My Cure", he also discovered and repurposed treatment for his own battle with a deadly disease.
Join us at Certainty US on April 14-15 in Boston, MA to hear from one of our industry's most forward-thinking and patient-first leaders.
Accelerate Your 2026 Goals with Expert Insights
Check out our rotating lineup of webinars led by scientific experts — don't miss out on the latest insights in drug discovery and development.
Model-Based Meta-Analysis (MBMA), Quantitative Systems Pharmacology (QSP), and Advanced Modeling in Early Reimbursement Strategy
Tuesday, March 3, 2026
11am - 12pm ET
Leveraging the full potential of PBPK models to accelerate drug development
Wednesday, March 11, 2026
11am - 12pm ET
Explore where to connect with Certara next
ACDM 2026
March 15-17 | Berlin, Germany | Booth #13
SOT Annual Meeting and ToxExpo 2026
March 22-25 | San Diego, CA | Booth #2024
Phuse US Connect 2026
March 23-25 | Austin, TX
DIA Europe 2026
March 24-26 | Rotterdam, Netherlands | Booth #E5
