Rare Disease Studies: How to Run Successful Programs

Angi Robinson, Executive Director, Pediatrics and Rare Diseases, Premier Research

Angi has been with Premier Research for over 12 years and has provided executive oversight and full management support for numerous studies, including global studies with a focus in paediatrics and rare diseases. Angi has supported FDA Pre-IND meetings, IND submissions and BLA & NDA directorship. Her experience includes multiple study designs including PK/PD, adaptive design, FDA Fast Track designations and she has directed several studies requiring the oversight of Data Safety Monitoring Boards and Data Monitoring Committees. She has supported NDA submissions, including a pivotal studies resulting in product approval. Angi is the Global Project Director for Good Clinical Practice Journal’s (GCPj) 2008 Clinical Research Team of the Year for a pivotal clinical trial in a rare disease.

Patricia Nowowieski, Head, Global Clinical Operations, Cambridge, Alexion Pharmaceuticals

As a leader within the field of Global Clinical Operations, Pat has tremendous experience in global drug development trials, providing functional, operational, and strategic leadership to multi-functional teams. As Head of Alexion Pharmaceuticals’ Global Clinical Operations in Cambridge MA, she oversees operations from First in Man (FIM) to Proof of Concept (POC) and post-POC clinical programs. Prior to this, she held various leadership positions at Genzyme Corporation, the last as Therapeutic Area Lead for Genzyme’s Transplant & Oncology Division overseeing programs in rare hematological disorders (e.g., Chronic Lymphocytic Leukemia, Acute Myelogenous Leukemia and Myelodysplastic Syndromes).

Pat’s interest is in conducting global trials in specialized populations with complex logistical and regulatory challenges. She successfully led implementation of a patient registry for the 1st FDA-approved Biologics License Application for cell therapy approved under FDA cell therapy guidance. Many of these programs have undergone successful regulatory inspections following MAA/NDA filings.

Pat has a passion for mentoring new talent through organizations such as Healthcare Business Women’s Association (HBA) and volunteering for charities that support vulnerable populations.

Mallory Bissett, Associate Director, Global Clinical Operations, Alexion Pharmaceuticals

Mallory has worked in Global Clinical Operations, focusing solely on rare diseases for over 7 years. As global clinical operations lead for one of Alexion’s late-stage, ultra-rare disease products, she is responsible for the execution of the studies under the program. Prior to this, Mallory worked at Enobia Pharma Inc. after starting her career in rare diseases at Genzyme Corporation as a CRA in the Lysosomal Storage Disorders business unit working on Myozyme, Lumizyme (both Enzyme Replacement Therapies (ERT), Pompe Disease), and Cerdelga (eliglustat, Gaucher Disease).