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.”

"PRA is so sincerely dedicated to putting the patient's experience first,” said Terry Jo Bichell, Founder & Director of COMBINEDBrain, RDAC Member. It is easy to give lip service and small concessions to patients, but PRA is actually digging deep into what it means for patients and their families to be a part of clinical trials. Even when a patient wants a new treatment, it is still stressful, scary, and time-consuming to take part in a trial. PRA is trying to understand that and make it better.”

While the Patient-Centric Trial Development Toolkit does focus on lessening the burden on patients to participate in clinical research, there are also several key benefits for sponsors such as avoiding significant costs related to inefficiency, high trial dropout rates, protocol amendments, and not being able to complete a trial on time.

To learn more about the importance of patient-centricity in clinical development or to download the toolkit, visit https://prahs.com/centers/center-for-rare-disease/trial-development-toolkit.

For more information about the Center for Rare Disease, please visit https://prahs.com/centers/center-for-rare-disease.

About PRA Health Sciences

PRA Health Sciences is one of the world’s leading global contract research organizations by revenue, providing outsourced clinical development and data solution services to the biotechnology and pharmaceutical industries. PRA’s global clinical development platform includes more than 75 offices across North America, Europe, Asia, Latin America, Africa, Australia and the Middle East and approximately 19,000 employees worldwide. Since 2000, PRA has participated in approximately 4,000 clinical trials worldwide. In addition, PRA has participated in the pivotal or supportive trials that led to U.S. Food and Drug Administration or international regulatory approval of more than 95 drugs. To learn more about PRA, please visit www.prahs.com.

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