Researchers have known about Angelman syndrome for 50 years. Despite the research that has been done, there is still no treatment option available to patients. Ovid Therapeutics now faces several challenges in bringing a potential treatment through a critical Phase 3 trial.
By Simon Ellison,Thermo Fisher Scientific
Conducting the advanced therapy supply chain orchestra is more than waving a stick at people — it is an “end to end,” complicated, inter-related system that requires controlled, consistent management. Following are some things you need to think about to develop the capability to provide a harmonious supply chain management system.
By Justin Gambill,United BioSource LLC (UBC)
In the rare disease space, a product NDC may change for any of several reasons. It’s imperative to think through the potential impact that a product NDC change may have on your established patient population. Here are a few things to consider that could reduce potential therapy gaps for patients when an NDC change is imminent.
For biopharmaceutical sponsors, clinical trial data is both the greatest organizational asset and the greatest challenge. This paper discusses the principles of data governance and how they are used to build a business intelligence framework that advances data quality, acquisition, and integration to deliver actionable information for use across the drug development enterprise.
In an increasingly complex drug development and healthcare environment, it is extremely important for companies to be able to demonstrate value, even if their intention is to be acquired or to out-license their asset following proof-of-concept. Learn more about partnering solutions available to small and emerging biotech companies.
Advancements in science and technology have helped researchers develop new treatments for some of the most common diseases known to man. For rare diseases, however, patients have limited treatment options. Advocacy groups and regulatory authorities continue to encourage rare disease research, and more and more scientists are stepping up to the task.
As the director of CDER's Office of Generic Drugs' Clinical Safety Surveillance Staff, Dr. Howard Chazin leads a multidisciplinary team of pharmacists, medical officers, and data analysts to identify and assess emerging complex safety issues related to generic drugs. “Generic drugs provide considerable cost savings.