PRA ‘toolkit’ pinpoints rare-disease clinical trial risks

By Jenni Spinner

- Last updated on GMT

(Traitov/iStock via Getty Images Plus)
(Traitov/iStock via Getty Images Plus)

Related tags Pra health sciences Rare disease orphan disease Clinical trials software

The free Patient-Centric Trial Development Toolkit is aimed toward helping sites and sponsors identify and minimize risks in rare-disease clinical studies.

PRA Health Sciences has launched its Patient-Centric Trial Development Toolkit, a resource designed to aid clinical trial teams in identifying risks that could stymie the effectiveness and efficiency of their research, and deal with those problems. Outsourcing-Pharma recently spoke with Scott Schleibner, senior vice president of the Center for Rare Diseases at PRA Health Sciences, about the toolkit, the unique challenges rare disease studies face, the importance of patient-centric trials, and more.

OSP: Could you please talk about some of the reasons why it is important that clinical research professionals focus on rare disease work?

SS: Rare diseases represent a large medical challenge, most of which have no cure. However, the appropriate treatment plan can greatly improve the quality of life and extend life expectancy for patients.

Currently, there are 7,000 rare diseases in the world, with 360m people living with a rare disease globally. With viable drug treatments available for only 5% of these indications, now is the time to solve this crisis.

Additionally, the economic impact of diagnosing and managing rare diseases is significant – rare disease patients often need more diagnostic tests than patients with more common diseases, lack of standards and guidelines of care make it difficult for rare disease patients to make health coverage decisions, many patients have to travel long distances to seek treatment, and the list goes on. These costs can quickly add up for patients coping with a rare disease.

OSP: What are some of the challenges unique to studies centered on rare and orphan diseases?

SS: For sites, the ability to identify eligible patients is a challenge. Coordinating home health nursing visits, telehealth, and on-site visits for each patient enrolled can be challenging to sites with minimal staff and little resources.

Sponsors can incur significant costs related to inefficiencies, experience high trial dropout rates, protocol amendments, and sometimes are not able to complete a trial on time. It can also be challenging for sponsors to find patients and making studies realistic for patients.

Then, last but not least, patients probably incur the most obstacles when it comes to participating in a clinical study -- cost, travel, misdiagnoses, multiple doctors’ visits, just to name a few. The burden of clinical trial participation is still extensive, and participation in a rare disease clinical trial often involves a child, a parent, or a caregiver.

In short, the entire family “enrolls” and these factors need to be considered when designing a clinical trial that will be feasible for rare disease patients.

OSP: Could you please talk a bit about the benefits of the individual kit components, and why together they serve as a good resource for trial teams?

Scott Schleibner, SVP, Center for Rare Diseases, PRA Health Sciences

SS: The components of the Patient-Centric Trial Development Toolkit were developed to support sponsors, patient communities, and other stakeholders to operationalize true patient-centricity in clinical development. We believe in empowering every stakeholder to make rare disease clinical research as efficient, effective, and successful as possible; these tools work together to support self-assessment of risks to a rare disease clinical program that may exist due to factors that affect participants’ ability to enroll and/or remain in a trial. This toolkit was developed in response to the gap in availability of structured tools.

  • Patient-Centric Protocol Risk Assessment Tool – Interactive, spreadsheet-based tool that enables sponsors to rapidly identify potential risks, track the 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 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 Dossier – This tool outlines the evidence of ROI/benefit involvement in the trial development process from the 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” – This tool helps prospective participants identify the barriers to participation they may encounter and request the support they need to enroll and stay in the trial.

OSP: Is there anything you’d like to add we didn’t touch upon above?

SS: We chose to develop this toolkit as a free, open-access set of tools for sponsors, patients, and patient organizations to use. Our aim is to ensure that all clinical trials – regardless of the trial phase, indication, geography, or sponsor – include the patient perspective within the study development stage, so that burdens to participation can be minimized to ensure patients can, and will, enroll. 

The PRA Patient-Centric Trial Development Toolkit can be downloaded for free at

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