The pandemic caused our industry to change the way we collect data and engage with patients. The rapid deployment of virtual trials proved that we can safely and effectively gather data remotely from patients using electronic clinical outcome assessments (eCOAs). These tools give clinicians, patients and caregivers an easy way to record insights about the patient’s disease, the trial experience and the treatment’s impact on quality of life without setting foot in a clinic. There are many elements to consider when deciding on the best COAs to implement. We’ll dive into those and cover best practices when developing your eCOA strategy.

COAs and Trial Design

While clinician reported outcomes (ClinROs) and performance outcomes (PerfOs) must be completed by medical professionals, patient reported outcomes (PROs) and observer reported outcomes (ObsROs) are more nebulous. Patients and caregivers complete these assessments, so sponsors must trust them to follow the rules of the assessment and to complete them accurately and on-time, which requires training and support structures that may not always be part of the trial design.

When designing a trial, sponsors need to consider whether the eCOA will be appropriate through the life of the trial, and what happens when and if it is no longer effective. For example, in pediatric trials that last for several years, a patient may not be mature enough to complete an ePRO in the first few years but may be fully able to record their own experiences later on. Their first-person insights could be a valuable addition to the trial data, but only if planners consider when and how they will shift from ObsRO to PRO, and how they will combine and evaluate data from both formats.

In trials of patients with debilitating or terminal illnesses, like ALS, the patient may be able to complete PROs early on but lose that physical or mental ability over time or during certain phases of the disease. If sponsors aren’t clear about how these assessments will be used, and what to do when patients no longer can (or should) be completing them on their own, it is left to patients and their caregivers to make these decisions.

That can create problems with the data and confusion for participants. In some cases, the caregiver may complete PROs on the patient’s behalf assuming it must be completed for them to remain in the trial. This could be acceptable if the patient is dictating their answers, but not appropriate if the caregiver is speaking on behalf of the patient. In these instances, the data could become unusable, compromising the trial integrity. Conversely, patients may choose to stop completing the assessment all together, creating gaps in the database once they can no longer comply.

Both scenarios can be avoided if sponsors think through the use of eCOAs as part of the end-to-end trial planning. With a little forethought and user training, these tools will bring valuable patient perspective to the trial data.

eCOA best practices

To make the best use of eCOAs, sponsors need to consider where and when they will be used throughout the trial, who should complete the eCOA and how to align these tools to the study’s broader goals.

Ensure the right data is captured while improving the patient experience by following these best practices for successfully implementing an eCOA:

1. Involve patients and caregivers in decision-making. The value of ePROs and ObsROs is to capture the patient’s experience with their disease and the treatment in terms that matter most to them. That only works if the eCOAs ask the right questions in the right frequency to engage patients without over-burdening them with too many activities. Involving patients in the eCOA planning process ensures the assessment will be fit for purpose, and helps trial planners identify problems with their use that they might not otherwise have considered – like what should a patient do if they lose the mobility to complete a survey on their phone, or if their caregiver speaks another language? Once an eCOA is developed, include training for patients and caregivers on how to use it, why it is important to the trial, and what they should do if they can no longer complete it on the expected schedule. Include information on who to contact (site staff, concierge) if they need help.
2. Plan for every scenario. When choosing an ePRO or ObsRO, think through how the patient or caregiver will use the assessment at every stage of the trial, and whether there will be scenarios when ownership of the assessment should change hands.
   If there is a chance that a transition from an ePRO to an ObsRO (or vice versa) will be necessary, determine how it should be handled, including who makes that decision, how the decision is made, and how the transition will be recorded in trial data.
   Questions to consider:
   • What should a caregiver do if a patient is temporarily or permanently unable to complete the PRO?
   • At what point is a child fit to complete their own PRO, and how will this be decided?
   • How will site staff determine that a patient’s PRO responses are no longer reliable due to cognitive issues, and how should they treat that data?
   These questions will guide trial planners toward creating a robust eCOA strategy that plans for every scenario.
3. Consider complimentary eCOAs. Asking both caregivers and patients to complete assessments can provide deeper perspective on the patient experience and create benchmarks for comparison for validating patient responses (to vet cognitive function), and to seamlessly fill gaps if the patient can no longer compete the PRO. However, the decision to add a complimentary ObsRO must be balanced against the added time and burden on caregivers to complete these assignments.
4. Make BYOD an option. Allowing patients and caregivers to complete eCOAs has become common practice in clinical trials. Regulators in the US and EU encouraged BYOD policies during the pandemic, and patients have indicated a preference for using their own phones vs. carrying a separate provisioned device.

BYOD policies make eCOAs cheaper and easier to deploy and allows patients and caregivers to manage all trial information, alerts and activities in one place, therefore reducing the patient burden.

Make the most of eCOAs

eCOAs will be a permanent part of the trial landscape going forward. They give sponsors an easy and effective way to capture the patient perspective remotely, and to use that data to inform research decisions.

When sponsors work with partners with deep experience in this space, it will give them the confidence that they are choosing the right tools for the trials, and ensuring patients and caregivers have the training and resources they need to use these tools effectively throughout the trial. Email us at ecoa@iqvia.com with any questions you might have about our experience and how to make an eCOA work for your next trial.