February 28, 2017

DrugDev Hosts New Educational Webinar on Overcoming the Challenges of Rare Disease Clinical Trials

Pharma industry innovators from AMO Pharma and Alnylam offer advice and best practices to improve trials and help patients suffering from rare disease

Over 8,000 recognized rare diseases afflict an estimated 350 million people worldwide – 50% of them children. In a new educational webinar on March 7 DrugDev will gather international experts to share challenges and provide insight and best practices to pharma professionals working in this especially difficult area.

Webinar: Overcoming the Challenges of Rare Disease Clinical Trials

Date: March 7, 2017

Time: 10:00-11:00 am EST


The webinar is complimentary

The Statistics are Startling

During the first 25 years of the Orphan Drug Act passed in 1983 only 326 new drugs were approved by the FDA and brought to market, leaving about 95% of rare diseases without a single FDA-approved treatment. Approximately 50% of rare diseases lack the support of a disease-specific foundation to drive drug development. Clinical trials are the only potential treatment option for so many of these patients. These statistics point out the importance of developing treatments for these rare disease patients.  

Experts Share Unique Insights and Advice

Ibraheem Mahmood, President and CEO, DrugDev and Chairman, AMO Pharma Ltd. will host a discussion and Q&A with industry experts Dr. Michael Snape, CEO and CSO, AMO Pharma Ltd; and Donna Mackey MS, Senior Director, Clinical Operations, Alnylam Pharmaceuticals. The presenters will review the particular challenges of rare disease trials and offer solutions to improve the conduct and ultimately, the potential for success of these unique trials. Our presenters will offer both the physician and sponsor perspective on the following:

  • Disease-specific challenges
  • Trial design
  • Recruitment
  • Site engagement
  • Disease awareness
  • Logistical challenges

About the Speakers

Ibraheem “Ibs” Mahmood, President and CEO, DrugDev and Chairman, AMO Pharma Ltd. – Mr. Mahmood defines the DrugDev vision: to provide technologies and services to clinical trial doctors that enable pharma to conduct 10 trials for the price of one. He has a global reputation for leading a transformational change in the clinical trial industry as pharma continues to collaborate and standardize on modern technologies.

Michael Snape PhD, CEO and CSO, AMO Pharma Ltd – Dr. Snape is a neurobiologist who has spent 27 years in pharma working for large and emerging biopharmaceutical companies. He has experience founding and funding companies from inception through IPO on the public markets. Dr. Snape’s principal experience is in demonstrating clinical proof-of-concept with novel targeted therapeutics in rare diseases. He is named an inventor in numerous patents and is a scientific advisor to several patient foundations.

Donna Mackey MS, Senior Director, Clinical Operations, Alnylam Pharmaceuticals, Inc. – Ms. Mackey has over 20 years of clinical research experience and has spent the last 15+ years focused on developing treatments for patients with rare medical conditions.  At Alnylam she is responsible for clinical oversight of several rare disease programs utilizing a new class of therapies based on RNA interference. Previously she was with Synageva BioPharma and Genzyme Corporation as the Therapeutic Clinical Leader for lysosomal storage disease programs well as a Global Team Leader. 

Join us to learn more about this compelling topic. A video recording will be provided to everyone who registers.

About DrugDev

DrugDev helps the world do more trials through industry-wide collaboration, standardization and a beautiful technology experience.  DrugDev Spark™, the unified clinical operations suite, is comprised of proven solutions used by 85 sponsors and CROs on over 1,800 clinical trials to transform the quality and efficiency of clinical trials from startup through closeout. DrugDev Spark is powered by the DrugDev Golden Number, the award-winning universal identifier for global site facilities and investigators used by TransCelerate and the Investigator Databank. Learn why pharma and biotech companies and CROs of all sizes worldwide trust DrugDev by scheduling a demo of DrugDev Spark at