Help Industry With Clinical Trial Design

Tips for Success

Watch Global Genes ’ video presentation Patient’s Role in Designing Clinical Trials (2016). The video covers patient engagement in the research and development process. The presenters speak about the development of a set of specific recommendations for patient engagement: Effective Engagement with Patient Groups Around Clinical Trials (2015). The presenters also provide case studies for turning these recommendations into real practice.
Read Guiding Principles for Interaction with Patient Advocacy Organizations , a list of principles that can be used by industry when interacting with patient advocacy organizations developed by Biotechnology Innovation Organization (BIO).
Review common issues encountered by Industry in rare disease therapy development in the FDA Draft, Rare Diseases: Common Issues in Drug Development Guidance for Industry (2019). Knowing these issues can help you determine information and data you may have or can develop to help Sponsor efficiently and adequately address them early on.

Resources

Clinical Outcome Assessments

Clinical Outcome Assessment (COA) Qualification Program U. S. Food and Drug Administration (FDA) (link)

Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims U. S. Food and Drug Administration (FDA) (link)

Clinical Outcome Assessment Compendium U. S. Food and Drug Administration (FDA) (link)

The Science of Clinical Outcome Assessment (COA) in Medical Product Development - An Intensive Online Educational Series University of Maryland School of Pharmacy (link)

Amyotrophic Lateral Sclerosis: Developing Drugs for Treatment Guidance for Industry U. S. Food and Drug Administration (FDA) (link)

Tips for Success

Patient's Role in Designing Clinical Trials Global Genes (link)

Effective Engagement with Patient Groups Around Clinical Trials Clinical Trials Transformation Initiative (CTTI) (link)

Guiding Principles for Interaction with Patient Advocacy Organizations Biotechnology Innovation Organization (BIO) (link)

Rare Diseases: Common Issues in Drug Development Guidance for Industry U. S. Food and Drug Administration (FDA) (link)