Short Abstract

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.