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Participant-focused tools designed to improve study success in rare disease clinical trials

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 a team of experts from our Center for Rare Diseases, 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 ICON’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.

Complimentary Patient-centric Trial Development Toolkit

Including Rapid Participation Burden Survey findings and Patient-centric Protocol Risk Assessment Tool