Help Industry With Clinical Trial Design
Patient Group Assessment by Industry
Knowing the criteria Industry uses to assess your group can help you maximize and articulate your expertise and assets.
- The vision, mission, and goals:
- Ensure that your group’s vision, mission, and goals are clearly stated in all materials and demonstrate that your vision, mission, and goals are at the center of your activities and are reasonable in respect to your budget.
- Your goals and objectives are summarized in your vision and mission statements.
- A vision statement concentrates on the future; what is your ultimate goal? The vision statement should be clearly written and engaging, but at the same time it should also express realistic desires and give a clear timeline. A vision statement guides later decision-making.
- A mission statement is about the present; it outlines your current goals and the processes you have in place to meet those objectives. What is the purpose of your organization, who does your organization serve, and what is its primary focus? This statement should avoid the use of generic endpoints (e.g., our goal is to lower therapy prices) and should instead be as specific as possible (e.g., our goal is to provide support for the Orphan Drug Act by broadening public knowledge of Wilson’s Disease).
- Group operations are evaluated in several places to ensure that the group is sophisticated enough to demonstrate a measurable impact.
- Specifically, it is important to have scientific and medical community members involved either directly or as advisers.
- Your outreach methods to partner with other groups, researchers, and Centers of Excellence will also be evaluated.
- Check your ratings with the Better Business Bureau and Charity Navigator and take the necessary steps to correct any inaccuracies.
- The amount of your income is less important than the diversity of sources and fundraising capacity.
- The ways in which you use your resources is also relevant; most of your budget should be going towards operations and activities instead of overhead.
- Groups are also evaluated on the effectiveness of their communication system, including their social media presence, publications, and outreach activities.
- The impact of informal publications (e.g., blogs, press releases) and formal publications (e.g., white papers) is also considered.
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)
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
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)