Rare Disease Studies: How to Run Successful Programs

Clinical Trials, Life Sciences, Pharmaceutical, Pharmaceutical Regulation,
  • Wednesday, March 11, 2015

This webinar will focus rare and ultra rare disease programs. The presenters will share sponsor and CRO recommendations for supporting successful rare and ultra-rare disease programs, in addition to practical considerations for varying stages of the development process.

Key topics:

  • Thoughtful Program Design including well understood natural history (comparability of baseline characteristics, reduce sources of bias in natural history) and validated endpoints
  • Thoughtful Protocol Design specifically reducing amendments
  • Challenging Recruitment & Retention in terms of number of sites and global considerations; Patient travel/transport support; Global network of sites to accept international patients; and anticipating questions (pediatric populations, development, growth)
  • Inspection Readiness


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.

Message Presenter

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.

Message Presenter

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).

Message Presenter

Who Should Attend?

VPs, Directors, Managers, Department Heads, Scientists and Researchers working within:

  • Clinical Affairs
  • Clinical R&D
  • Clinical Research
  • Clinical Pharmacology
  • Clinical Operations
  • Project Management
  • Regulatory Affairs
  • Medical Affairs

for pharmaceutical, biotechnology and medical device companies

Xtalks Partner

Premier Research

Premier Research is a leading global contract research organization serving biotech, Pharmaceutical and medical technology companies. The company has a wealth of experience in rare disease and pediatric research having managed about 100 projects in each area in the last five years alone. Its services include clinical research and regulatory outsourcing in the areas of analgesia; neurology; infectious, cardiovascular, and respiratory disease; dermatology; oncology; and medical devices.

Premier Research and operates in 50 countries. It employs 1,000+ clinical professionals dedicated first and foremost to fulfilling each customer’s requirements in a timely, accurate, and cost-effective manner. This includes a strong international network of monitors and project management professionals combined with regulatory, data management, statistical, scientific, and medical experts, and staff at its well-established network of dedicated clinical sites.

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