Newsletter | July 15, 2019

07.15.19 -- A Rare Disease Trial Requires An Objective Scale And Site Training

 
Featured Editorial
A Rare Disease Trial Requires An Objective Scale And Site Training
 
By Ed Miseta, chief editor, Clinical Leader

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.

Why All The Talk About Real-World Evidence?
By Ed Miseta, chief editor, Clinical Leader

Real-world evidence (RWE) has become a topic everyone wants to discuss. Several executives I have spoken to recently believe it will continue to grow in importance. But why the sudden interest in RWE?

Industry Insights
Conducting The Advanced Therapy Supply Chain Orchestra
Article | 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.

How A Product NDC Change Could Impact Your Rare Disease Patient Base
Article | 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.

Data Governance In The Clinical Trial Ecosystem
White Paper | By Jaime Cook, YPrime

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.

Partnering Solutions For Emerging Biopharma: Alternative Outsourcing Strategies
White Paper | IQVIA Biotech

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.

Gene Therapies In Rare Disease: From R&D To Regulatory Approval
White Paper | Medpace

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.

Challenges In Generic Drug Safety And Surveillance
Podcast | DIA

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.

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