The Toolkit for Patient-Focused Therapy Development website provides reliable information about the stages of therapy development and ways that rare disease patient group leaders can get involved. It was developed to provide engagement strategies for a broad range of patient advocacy groups, including smaller groups. The Toolkit was created by the National Center for Advancing Translational Sciences (NCATS) and members of patient advocacy groups.

The Toolkit includes:

• Easy-to-understand descriptions of the discovery phase, the early stage of identifying potential drugs that could be tested in future clinical trials
• Tips for preparing for and conducting clinical trials
• The Food and Drug Administration (FDA) review and approval process, and what occurs following FDA approval
• Definitions of common words used in research
• Links to helpful resources like online education and tips for working with the Centers for Medicare and Medicaid Services
• Tips for success

Stage(s) of Research Process: 

• Study Planning – The Toolkit provides recommendations for building relationships with key partners. In this section of the Toolkit, patient advocacy groups are given some strategies for identifying medical researchers and clinicians to serve on their scientific/medical advisory board.
• Study Conduct – Patient input on the clinical trial design is critical to ensuring that trial design is patient-friendly and inclusive. The Toolkit provides background information to assist patient advocacy groups in understanding key sections of a study protocol.
• Post-Study – After a research study is over, there are many ways to make sure the study’s results reach the right people. Patient advocacy groups can work with clinicians to develop screening programs that help identify people who can benefit from newly created treatments.

Who Might Benefit:

• Patients, Caregivers, and Patient advocacy groups can use the Toolkit to engage meaningfully with the therapy development process. The materials in the Toolkit were designed to be helpful for newly formed patient advocacy groups with limited resources and experience.
• Communities can benefit from the Toolkit if they serve on Institutional Review Boards or Data and Safety Monitoring Boards, participate in FDA Advisory Committee meetings, or want to play an active role in clinical research in other disease areas.
• Researchers can learn about how to partner with patient advocacy groups more effectively and how to accelerate translation of research observations into widely disseminated treatments by accessing information in the Toolkit.

Key Takeaways and Examples in Practice:

• Even newly formed patient advocacy groups can have an impactful voice in the therapy development process.
• The Toolkit utilizes five criteria when deciding to incorporate resources: generalizability (can be used by multiple patient groups), customizability (adaptable to different situations), actionability (has clear, concrete steps), uniqueness (fills a need not already met by other resources and content), and credibility (created by patient groups, created for patient groups, or already used by patient groups)
• Involving representatives of patient advocacy groups in the design and ongoing management of the Toolkit has enhanced its usability and informed the material contained in the Toolkit.
• Infrastructure provided by NCATS (website hosting, web-based process for toolkit feedback, management of the toolkit review group) has ensured long-term sustainability of the Toolkit.

This work was done with the direct support by each of the following partners who advance this work:

• FDA
• Global Genes
• National Organization for Rare Disorders
• Rare Diseases Clinical Research Network

Keywords: Methods of Engagement; Collaborative Decision-Making; Equitable Engagement; Sustainable Engagement & Capacity Building; Return of Research Value; Researchers; Communities; Patients, Caregivers, and Patient Advocacy Groups

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Links to Relevant Resources:

• The Toolkit for Patient-Focused Therapy Development website
• About NCATS Toolkit
• Contact the NCATS Toolkit

Definitions for each engagement objective can be found in the glossary.