PRA’s Center for Rare Diseases Launches Toolkit to Identify and Mitigate Risks to Rare Disease Clinical Programs

Four-part, patient-focused toolkit is available for download at no cost and allows sponsors to identify risks to the efficiency and success of their trials

RALEIGH, N.C., March 22, 2021 (GLOBE NEWSWIRE) -- PRA Health Sciences (NASDAQ: PRAH) announced today the launch of its Patient-Centric Trial Development Toolkit, available now to clinical development sponsors focusing on rare diseases. Developed by PRA’s Center for Rare Diseases in collaboration with PRA’s Rare Disease Advisory Committee (RDAC) and other patient stakeholders, the toolkit includes four digital resources designed to mitigate risks that frequently occur in rare disease clinical trials. The toolkit also introduces and affirms new patient-centric practices that promote trial participation.

The Patient-Centric Trial Development Toolkit is available at no cost and can be downloaded at https://prahs.com/insights/patient-centric-trial-development-toolkit.

“The main purpose of the toolkit is to guide sponsors in taking a more patient-centric approach in developing clinical trials,” said Scott Schliebner, MPH, Senior Vice President, Center for Rare Diseases at PRA Health Sciences. “As an example, the toolkit includes a risk assessment tool that clinical development teams can use to identify risk to the efficiency of a clinical program. The risks that are identified are usually real world burdens for participants, and the tool provides risk mitigation strategies and solutions for sponsors to consider.”

PRA’s rare disease experts and RDAC members recognized a gap in the availability of structured tools for operationalizing patient-centricity in rare disease clinical development. Patients, advocates, and industry leaders in rare diseases collaborated and consulted with PRA to develop four parts of the Patient-Centric Trial Development Toolkit:

• 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 program 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 PRA’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.

“Rare disease patients and trials face unique challenges compared to those in more common indications,” said Tracy Dixon-Salazar, PhD, Director of Research & Strategy at LSG Foundation, RDAC member. “As a rare disease patient advocate, I appreciate the genuine care that PRA gives to their patients and family caregivers. In sharing these resources with sponsors and the rare disease community, PRA is taking another step in making clinical research more accessible and ensuring the patient and their family is top of mind when developing a clinical trial program.”