Case Study

TrialMax® Makes Infusion Data Capture Easier in Rare Disease Studies

Case Study

Rare diseases can be di�icult to manage and very stressful for patients. Their quality of life can be impacted, causing them to become very sick, admitted to the hospital for treatments, and absent from personal and professional activities. Some patients will have limited life expectancy. It's incredibly taxing, but enrolling in a clinical study can improve the life of future generations and can be encouraging for those living with a rare disease. Some common challenges in rare disease trials include:

Introduction

Recently, CRF Health's TrialMax® electronic Clinical Outcome Assessment (eCOA) platform was selected to collect data from subjects in a rare immunological disease study. Trial participation would entail subjects answering a wide-variety of questions during weekly infusions, contributing to the existing burden of managing this disease. To reduce stress on the subject, a digital solution for accurately tracking infusions and their dosage was requested by the study team.

Study Overview

The Challenge

It can be di�icult to find voluntary participants when so few people have the rare disorder. Sponsors and study teams may need to work with specialist sites and consultants (principle investigators/key opinion leaders) when actively searching for study participants.

Keeping a small number of patients engaged is important to ensure that there is enough data available for evaluation. Every single patient's participation is critical in assessing the drug's e�ects, so retaining patients for the duration of the study is imperative. The patient experience should be as smooth and supportive as possible.

Rare disease studies o�en leverage geographically dispersed patient groups in order to obtain as many subjects as possible. This creates a burden on the sponsor when it's time to train sites and then for site sta� to train patients. Providing them with the resources needed for easy data capture and study success, despite their geographic location, is imperative for data accuracy.

Ensuring enough data is collected to make actionable decisions is more challenging in smaller studies and significant e�ort should be made to make every data point count.

In the event of physical, mental, or emotional limitations, some accommodations may be needed to provide equal opportunity for subject participation, especially in smaller study groups.

Patient Recruitment Patient Engagement and Retention

Logistics and ScaleMeaningful, Comparative Data

Special Accommodations

In past experiences with this client and indication, subjects used paper to record their infusions which produced multiple issues. Following each infusion, the subject would need to record all of these data points immediately following their treatment:

This then required sites to manually calculate the rate of infusion, which meant doing the math on every single entry. Subjects also had to remember where they put their pen and paper, the exact time they began the infusion, the location of the infusion, as well as all of the dosing information. If they forgot to record even one infusion, the data's accuracy was compromised. This retrospective data entry then required sponsors to spend countless hours to manually correct the data with data clarification forms, or DCFs, adding on extra time that delays the study and possibly the drug's approval. Other challenges with paper on this study included:

Infusion site location

Total number of infusions

Total volume infused

Total pumped infusion rate

Infusion rate per site

Infusion interruption details

Infusion site reactions

Lost or missing data

Confusion about infusion volumes

Security and privacy

Illegible handwritten answers

Inaccurate responses/partially completed responses

Noncompliance with the study protocol

Case Study

Study Design

CRF Health was tasked with alleviating this burden on subjects. In addition, because the questionnaire was not previously validated, the assessment needed to be designed in an electronic format by CRF Health. EXAMPLE

Pictured le�: A sample paper version of the infusion dosing diary showcases how overwhelming it can be to see all of the questions at once.

Protocols(two-arm study)

24 Weeks(6 months)

52 Weeks(12 months)

60subjects

30sites

2countries

2languages

15TrialMax Touch®

handheld devices deployed

infusion pumps*

2

80subjects

40sites

7countries

6languages

25TrialMax Touch®

handheld devices deployed

infusion pumps*

2

*diary was built for 1 pump but can accommodate 2

Case Study

SolutionCRF Health provided TrialMax Touch® handheld devices to alleviate the burden of subject data entry on this study. The electronic handheld device easily integrated into the lives of subjects who needed to perform multiple infusions on a weekly basis to manage their condition.

The ability to fill in their infusion data remotely with a device that seamlessly integrates into their everyday lifestyle is hugely beneficial for subjects. However, because subjects with this rare disease are sometimes very ill, they may experience issues entering data within the expected timeframes. Sites are able to eliminate this burden by extending data entry windows to give them extra time. Unlike paper where subjects may fill in their diary with inaccurate dates or entry times to make up for the fact that they couldn't complete their questionnaire at the time required, the eCOA solution prevented blank or incomplete entries and accurately captured the infusion details. The entire questionnaire was built to be extremely patient- centric so that the experience of entering data was easy to do remotely, eliminating any additional stressors for subjects already managing a di�icult rare disease.

The questionnaire was designed to only ask one question at a time, enabling subjects to focus on the task or question at hand and eliminating confusion. Alarms and reminders encouraged subject compliance and prevented them from forgetting to enter their data (or entering it retrospectively). This ensured data was entered at the right time, increasing data accuracy and maintaining consistency for better outcome assessments. Sites were also able to enter data via a separate login if subjects were too sick to complete their questionnaire themselves. All of these elements enabled easy data entry that, most importantly, improved data accuracy.

Pictured above: Screenshots showcasing how easy data entry was for subjects. Simply select the pump, infusion location, and the number of infusions per location. Separating each question onto one screen at a time allowed the subject to focus on the question at hand.

Reduce Patient Burden with Remote Data Capture Functionality

Simplify Data Entry with Patient-Centric Design

Case Study

Study stakeholders worked closely with CRF Health to design the assessment, which included branch logic that automated the process for subjects. This enabled the proper questions/requests to appear based on logic connected to subject responses. In addition to branch logic, edit checks were also included in the solution to flag missing or abnormal data, such as low or high dosing amounts. Both the logic and edit checks prevented incomplete and inconsistent data entries and standardized response options to ensure consistency.

Finally, a drastic increase in e�iciency for sites came from the eCOA solution automatically calculating the total infusion rates. If infusion rates were recorded for two (2) pumps, the numbers would be calculated to reflect the total rate. A confirmation screen would appear so that the subject could confirm the total. This also helped prevent patients from entering data for two (2) pumps if they intended to only enter data for one (1). This feature reduced countless hours of math and streamlined data collection for sites.

Also protecting subjects from entering incorrect information was functionality that required infusion pump selection. Subjects were able to choose from one of two pumps or both, and then enter the appropriate data for each following their selection.

Making the switch from paper to eCOA for this infusion diary was invaluable for subjects. Simple, patient-friendly diary design reduced burden and provided real-time access to data. Showing subjects one question or one command at a time eliminated confusion and allowed them to focus on the task at hand. Increased subject engagement and compliance was prevalent due to reminders, alerts, edit checks, and branching logic that enabled more accurate, reliable data collection.

The client was so pleased with the success of the initial two studies that they awarded CRF Health an additional study.

Streamline E�iciencies with Built-In Logic and Real-Time Edit Checks

Study Results CRF Health's Rare Disease Trial Experience

30,842,500 data pointscollected

30+ Trials

Pediatric, Adolescent and Adult studies

1-240+ Sites

16-1500+ Subjects

Pictured above: A sample edit check to ensure the number of mL/hr was entered as intended by the subject.

Pictured le�: The option to enter data for one pump at a time reduces errors.

About CRF HealthCRF Health is the leading provider of patient-centered eSource technology and service solutions for the life sciences industry. With experience in more than 800 trials, over 100 languages and across 74 countries, CRF Health’s TrialMax® platform consistently demonstrates the industry’s highest data accuracy, patient and site compliance, and patient retention.

CRF Health’s eSource solutions improve trial engagement by fitting into the lives of patients and seamlessly integrating into sites to maximize protocol compliance. The integrated TrialMax platform includes eCOA solutions for collecting PROs (Patient Reported Outcomes), ObsROs (Observer Reported Outcomes), ClinROs (Clinician or Rater Reported Outcomes), and PerfOs (Performance Outcomes), and features TrialConsent™, an electronic solution for collecting informed consent in clinical trials.

For more information, please visit www.crfhealth.comor email info@crfhealth.com

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