How to Navigate the FDA Landscape for an Orphan Drug
Rare diseases are a public health priority. FDA has launched several programs and a final guidance to address the unmet need for orphan drugs.
Tag: Rare/Orphan Disease
Artificial Intelligence: What is it & how can it accelerate rare disease drug development?
Recently, artificial intelligence (AI) has been front and center of newspapers, trade journals, and social media. It gleams in the limelight the same way as a host of other past innovations. Like many scientists, I look at innovations as an advancement. However, little did I know what the business outlook on innovation was until I … Continued
A Bird’s Eye View on Rare Diseases: Key Drug Development Considerations for Thyroid Eye Disease
New therapeutics discovery and development for ocular diseases have been traditionally associated with a low probability of technical and regulatory success. The significant unmet medical needs for ocular diseases include but aren’t limited to glaucoma, age-related macular degeneration, and retinopathy. Recently, there has also been considerable focus on thyroid eye disease (TED). This research gained … Continued
World Orphan Drug Congress
The Rare Disease Drug Dilemma: Great Value, High Price, Limited Access?
This webinar will explain how pricing has evolved for the rare disease drug market and best practices for supporting value-based pricing.
Certara’s Best of the Blog 2019
A selection of short essays from our blog, written to empower our clients with modeling and simulation (M&S), regulatory science, and real-world value assessment solutions to help them solve the toughest drug development problems. In the Best of the Blog, Certara’s scientists and regulatory experts share their learnings, technological advances, and thought leadership.
Innovative Solutions for Fast-Tracking Drugs for Rare Diseases
This webinar discussed which pharmaceutical payer strategies work and which don’t in our rapidly changing regulatory and reimbursement world.
Found in Translation: Building an Early Development Strategy for Complex Biologics
This webinar described some important strategic considerations for successful development of complex biologics including appropriate data collection and timings, analyses requiring cross-functional data integration, and key pharmacology-related critical questions.
Immunogenicity Prediction and Dose Optimization using Clinically-Validated In Silico Modeling and Simulation
Biologic drug development is a rapidly evolving sector in the biopharmaceutical industry. Immunogenicity is an inherent challenge with this complex class of drugs. A quantitative systems pharmacology approach can be used to predict and better manage immunogenicity, and as a tool to guide clinical and regulatory decision-making in biologics drug development.
Making Sense of 6MWT Variability: Developing a Disease Progression Model for DMD
Duchenne Muscular Dystrophy (DMD) is a life-threatening, sex-linked, pediatric rare disease, primarily affecting boys. It is characterized by progressive muscle degeneration, weakness, and eventually functional loss. DMD is caused by a mutation in the dystrophin gene, a protein needed to maintain muscle integrity, and for improving signaling and growth in differentiation of the muscle tissue. … Continued