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There is a recognised a gap in the availability of structured tools to help sponsors operationalise patient-centricity and identify and mitigate risks in rare disease clinical development.

Developed by ÃÛÌÒ´«Ã½, in collaboration with the Rare Disease Advisory Committee, this toolkit includes four digital resources designed to mitigate risks that frequently occur in rare disease clinical trials and support new patient-centric practices that promote trial participation helping sponsors save cost related to inefficiency, high trial dropout rates, protocol amendments, and inability to complete a trial on time.

Access these key resources to help all stakeholders overcome critical barriers in rare disease clinical trials:

• Patient-centric protocol risk assessment tool: Interactive, spreadsheet-based tool that enables sponsors to rapidly identify potential risks, track evolution of risk assessment through stages of the development process and identify potential mitigation strategies.
• Rapid participation burden survey tool: An easy-to-use questionnaire development guide that helps sponsors and patient advocates develop a rapid survey for patients and caregivers tailored to their clinical trial¡¯s specific context. The aim of this tool is to help sponsors quantify risk to the clinical programme through direct patient engagement.
• Patient involvement value dossier: This tool outlines the evidence of ROI/benefit involvement in the trial development process from extant literature and provides illustrative case studies from ÃÛÌÒ´«Ã½¡¯s Center for Rare Diseases.
• ¡°What to ask when you¡¯re interested in a clinical trial: A guide for rare disease patients and caregivers¡±: Helps prospective participants identify the barriers to participation they may encounter and request the support they need to enroll and stay in the trial.