International Seminar on Orphan Drugs & Rare Disease
Live Seminar in New Jersey

Venue :
NJHA Conference & Event Center
760 Alexander Road, PO Box 1
Princeton, New Jersey
view location map
(Limited Seats!!!)
William C Maier MPH, PhD
Chief Scientific Officer/VP
Epidemiology & Risk Management,
Dr William C. Maier, is Chief Scientific Officer and Vice President Epidemiology and Risk Management at Registrat-Mapi ( the industry’s largest clinical research organization (CRO) dedicated solely to “real-world” clinical research.
Ronald A. Christensen, M.D
Chief Medical Officer,
Dr. Christensen has broad-based experience in academia, clinical research, and the biopharmaceutical industry. Following his fellowship training in Pediatric Endocrinology, he became Director of Endocrinology at Phoenix Children’s Hospital. As a Director in the Marketing Department at Genentech, Inc. he developed expertise in product commercialization, including development of registry studies.
International Seminar on
Orphan Drugs & Rare Disease

Date : Thursday, 12th September, 2013
Time : 09:00 AM - 02:00 PM EDT
Followed by Breakfast & Lunch

Drug development for rare diseases and orphan drug areas have been increasing over the past 5 years due to an increase in health care spending on specialty care in the USA and other regions. In addition, the drug approval process is often shorter and there is a different process for approval of medications to treat these conditions.

However, because of the difficultly of recruiting large populations in to clinical trials, the approval of these medications are usually accompanied by a requirement for more real-world post-marketing observational studies to provide more information about efficacy and safety of these products.

Real world studies are different from randomized controlled trials and are based on observational study designs. In addition, because these studies are intended to represent ‘real-world’, data collection could be directly from patients and include physicians who have no prior experience with clinical research. In addition, many health care payers are now adopting ‘pay for performance’ and conditional reimbursement programs that need to be supported by real world studies of how a new product is helping the payers’ actual population of patients given the drug.

This workshop will provide you with the knowledge to understand the process of drug approval for rare disease and orphan drugs in the USA and Europe, the tools to design and execute comparative effectiveness evaluations and other real world studies of product impact and examples of approved products and associated real world studies.

This workshop will teach you about:
  • Requirements in the USA and Europe for the approval of products for orphan drugs and rare diseases
  • Real World Studies: Design, approval, conduct and analysis.
  • Case studies of Real World Studies that helped to gain product approval and reimbursement.

Program Agenda
9:00 AM – 10:00 AM Spot Registration + Breakfast + Introduction
10:00 AM - 10:45 AM Review of drug approval process for rare disease/orphan drug – USA and Europe
10:45 AM - 11:00 AM Tea/Coffee Break
11:00 AM - 12:30 PM Observational study designs for rare disease and orphan drugs
12:30 PM - 01:15 PM Lunch
01:15 PM - 02:00 PM Examples of real world studies - drug approval and payer requirements.
02:00 PM Adjourn
REGISTRATION FEES : USD $599 (Early bird price till 30th July - Actual Price USD $699)
*Discounts on Group Registration Available
For discount on Group Bookings, please contact
or Call Us at 1-646-216-8860

PS : Companies who attended our earlier event includes:
Johnson & Johnson, PAR Pharmaceuticals, GlaxoSmithKline, Nektar, Novartis, Ferring, JSS Medical Research Inc, Lannett Company Inc, Foresight Group, Smith & Nephew, Inc, Roche, Bayer Healthcare, INC Research, Rising Pharmaceuticals, United BioSource (UBC) PLC Icon, Pfizer, Abbott and many more..