World Orphan Drug Congress USA 2018
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World Orphan Drug Congress 2018 Pharmerit Whitepaper


Patient-Reported Outcomes in Orphan Drug Clinical Trials

Innovative Approaches to Improving Study Data

This paper focuses on the critical need for development and refinement of effective tools to identify patient-reported outcomes (PRO) in pre-approval assessments of orphan drugs. Background is provided on the dearth of PROs in the labels of pharmaceuticals for rare diseases and challenges inherent to orphan drug analyses, such as limited and heterogeneous patient populations. However, the author identifies and discusses innovative approaches now being used to develop and implement new tools for PROs, including improvements in qualitative and psychometric/statistical approaches, which can address the unique requirements of orphan drug analyses.

This white paper may have utility for research professionals involved in the development and oversight of patient-reported outcomes and clinical outcome assessments of drugs under development for rare diseases.

Readers can expect to:

• Understand the background and challenges inherent to orphan drug assessment, for both patient and clinical outcome analyses
• Describe the progress being made to improve and use PRO measurement tools for orphan drug studies
• Identify examples of innovative tools and approaches to improve PROs in orphan drug analyses


To learn more about Pharmerit, visit their website.


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