Earlier this year, the FDA released its fourth and final guidance on the incorporation of the patient’s voice in medical product development and regulatory decision-making. With a focus on the successful implementation of clinical outcome assessments (COAs), the US Food and Drug Administration’s (FDA) strategy sought to provide clinical trial organisers with the guidance required to successfully implement COAs in their research.
For trial providers, the guidance couldn’t come soon enough. The COA process has historically been rife with challenges. Often completed by healthcare providers or clinical staff, traditional paper-based COAs have not always accurately reflected the patient experience, and are often subject to human error and missing data.
This is where electronic Clinical Outcome Assessments (eCOAs) come into play. eCOAs have gained popularity in clinical trials as they offer a more precise and comprehensive understanding of a patient’s experience with a disease or condition. They provide immediate feedback to patients and include features to prevent missing data or inconsistent responses.
Most significantly, eCOAs provide a more patient-centric approach, giving them a voice in the research process, enabling researchers to gain a better understanding of their experience, and ultimately improving healthcare outcomes.
The decentralisation of clinical research
A key reason why eCOA has become a more attractive choice in today’s clinical trial landscape is the role they play in decentralised and virtual trials, which involves the utilisation of eCOAs to collect patient-reported outcomes (PROs) electronically.
With the industry still learning to adjust to a post-Covid world, virtual trials remain commonplace, and 2022 proved to be a leading year for decentralised trials (DCTs). According to GlobalData survey results, 41% of industry participants predict that DCT and virtual clinical trials will be even more frequently used in the next one to two years.
DCTs have long been lauded as the new frontier in patient-centricity and eCOAs are gathering momentum as the next natural step in the path to patient-first trials.
According to Dan Herron, VP of digital offerings at leading translation and localisation provider RWS Group: “With eCOAs, patients can directly input their health data and communicate with their healthcare providers, improving real-time visibility.
“[eCOAs are] providing patients with more control over their healthcare, enabling timely and accurate reporting of symptoms. Capturing patient-reported outcomes that are meaningful to patients increase patient satisfaction and engagement as well.”
The eCOA roadmap
To achieve safer and more effective treatments, as well as scientifically valid assessments and better regulatory decision-making, a patient-centric eCOA strategy is critical. Below are the five recommended steps to successfully implement a patient-centric eCOA strategy in accordance with FDA guidance.
- Electronic data capture
Using electronic systems to collect data directly from patients can help to ensure accuracy, and provide additional layers of security and privacy protection. Similarly, electronic devices that include alarm functions and daily reminders to capture electronic data can help reduce occurrences of missing data, and automatically record timestamp information.
According to Herron: “If using electronic devices, it is also recommended to have a backup plan in case of malfunctions. Furthermore, when using subject-provided devices, sponsors should use a single platform to minimise variation, and provide a detailed plan for FDA review.”
- Development and validation
According to the FDA, the same rigorous development and validation processes that apply to paper-based instruments also apply to eCOAs. For example, to minimise the risk of sub-quality data, sponsors should perform usability testing, including patient cognitive interviews, after user acceptance testing (UAT) to ensure device functionality, questionnaire comprehension, and ease of use in the patient population.
- Training and education
To ensure accurate data collection, the FDA’s guidance stresses the necessity of adequate training for patients, clinicians and study personnel on the correct use of eCOAs, including educating groups on their purpose and benefits, how the devices or platforms should be used, and best practice for troubleshooting.
“It is imperative that sponsors and investigators ensure that FDA regulatory requirements are met for record keeping, maintenance and access. Source-data control should be maintained by the clinical investigator(s) to ensure data integrity,” says Herron.
Data sourced via the eCOA must comply with all FDA regulatory guidelines, as well as the International Conference on Harmonization’s (ICH) Guideline for Good Clinical Practice.
- Linguistic validation
For true patient-centricity, all materials should be unambiguous and easy to understand; an outcome achieved through linguistic validation. This adapts instruments for use in different languages and cultures while maintaining their conceptual equivalence, and ensuring the translated versions have similar measurement properties to the originals.
According to the FDA’s third paper in the guidance series, Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments: “A robust process of translation and/or cultural adaptation increases confidence that all trial participants, regardless of their language and/or cultural backgrounds, understand the measure’s instructions, items or tasks, and response options similarly.”
It is therefore crucial for eCOAs to undergo a rigorous linguistic validation process to ensure the data collected is valid across all relevant languages and cultures.
RWS stands as the leading provider of linguistic validation services. Its approach, aligned with the FDA’s proposed strategy and the recommendations set forth by The International Society for Pharmacoeconomics and Outcomes Research, encompasses a comprehensive process. This includes a thorough analysis of the source instrument, a review of relevant literature, cultural adaptation, cognitive debriefing interviews, and meticulous documentation of the linguistic validation procedure.
According to Herron: “Leveraging RWS’s linguistic validation service empowers trial sponsors to ensure the accurate translation and cultural adaptation of their eCOA instruments, preserving their measurement properties across different languages and cultures. This helps mitigate the risk of data integrity issues, regulatory challenges, and enhances the likelihood of obtaining regulatory approval for global clinical trials.”
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