The Scary Future of Rare Disease Management
In a world of rising healthcare costs, the greatest nightmare for payers is reimbursing drug…
CertaraSeptember 23, 2021
Using a PBPK-PD Receptor Occupancy Model to Determine the Optimal Dose of Radiprodil for Neonates
Radiprodil is a selective allosteric modulator of the NR2B N-methyl-D-aspartate receptor GluN2B-NMDA that was being…
CertaraAugust 26, 2021
Simcyp’s Age-Defining Feature Facilitates Everolimus Dosing in Neonates
Everolimus (Afinitor®) was recently approved as adjunctive therapy for tuberous sclerosis complex (TSC)-associated partial seizures…
CertaraAugust 26, 2021
Eliglustat for Gaucher Disease: Quantifying the Impact of Pharmacogenetic Status on DDIs
In 2014, Eliglustat (Cerdelga®) was approved by the FDA as the first long-term treatment for…
CertaraAugust 26, 2021
DDI Regulatory Poster Child using the Simcyp Simulator: Ibrutinib (Imbruvica®)
Pharmacylics and J&J sought to bring ibrutinib, its new tyrosine kinase inhibitor therapy targeting rare…
CertaraJuly 29, 2021
Voxelotor for Treating Sickle Cell Disease: Leveraging Dose Prediction Model for DDI Prediction Without Any Clinical Studies
In November 2019, the US FDA granted accelerated approval for Oxbryta™ (voxelotor) tablets for the…
CertaraJuly 14, 2021
The Promises and Pitfalls of Gene Therapy for Rare Diseases
The state of gene therapy faces much uncertainty as recent regulatory and clinical setbacks have…
CertaraApril 29, 2021
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…
CertaraFebruary 19, 2020
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…
CertaraAugust 10, 2019
