How Electronic Data Capture Supports Patient Reported Outcomes in Clinical Trials
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In recent years, we have seen a move towards putting patients at the heart of healthcare and clinical research. This is not just about improving the patient experience, although that is an essential component. Increasingly, it is becoming important to measure how interventions improve outcomes that are meaningful for patients. But collecting this data easily, accurately and in real-time has been a challenge. This is where electronic data capture of patient-reported outcomes (PROs) can play a crucial role.
ePROs are an important component of electronic clinical outcomes assessment (eCOA) measures used to electronically capture patient and physician recorded outcomes in clinical trials. This blog focuses on the patient side of electronically capturing outcomes data.
What are PROs?
The FDA defines PROs as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” However, there are times when PRO data might be interpreted by a healthcare professional. Indeed, national surveys of patient experience are now a feature of NHS regulation and patient-reported outcome measures (PROMs) are now starting to be used globally as study endpoints in the development and evaluation of new treatments.
In the US, the move towards greater adoption of PROMs is being facilitated by the Patient-Centered Outcomes Research Institute (PCORI), a nonprofit institution that funds research that will ultimately help patients and caregivers make more informed health decisions. Key to this is the continuous collection of accurate, valid, accessible and reusable PRO data in real time. To this end, PCORI has also hosted workshops on building infrastructure for increasing the use of PROs in clinical and research settings.
What Types of Data are Captured as PROs?
PROs are considered anything the patient might report. Although PROs are commonly related to quality of life, they can also be information about the patient’s symptoms, or their functional ability (e.g. joint mobility) or their satisfaction with the intervention (e.g. the burden of taking a pill).
PROMs are the tools and/or instruments used to report PROs. Every PRO instrument contains a concept of measurement (often called a domain, which may be symptoms, sensation, functioning and quality of life) and way of measuring this (a rating scale of some kind).
Quality of Life (QoL) and health-related quality of life (HRQoL) are increasingly distinct domains of PRO. An example of a widely used QoL instruments is the EQ-5D-5L that measures generic health status.
Investigators on a clinical trial tend to be most interested in HRQoL. PCORI recommends that the outcomes measured should include ones that patients notice and care about – such as survival, function and symptoms.
How ePROs are Transforming Trials
For a long time, PROs were recorded by patients using paper diaries. However, there are many disadvantages with this. Data security is a problem, as it is potentially easy to mislay a paper diary and never be able to replace it. But one of the major issues is the compliance of patients filling it in, because there is no way to be sure that they entered data when they were meant to.
Moreover, in the past, electronic data capture (EDC) systems where data were captured into an electronic case report form (eCRF) were only accessible to investigator or study nurses. This meant site staff were entering data that patients wrote down on paper. Now, EDC software allows patients to log in to the system and enter data into the diary or answer questionnaires directly.
Today, PROs are collected using a wide array of electronic devices and interfaces available at home or in the clinic – and are known as ePROs. This has advanced from telephone-based interactive voice response systems to providing handheld touchscreen-based devices, to having patients input their data on their own device (so-called Bring Your Own Device, BYOD).
EDC systems nowadays have now the capability to receive data from external devices. This means that measurements can be taken with devices that a patient can use at home, such as blood pressure, glucose level, and these data automatically integrated into the actual study data. These tools have become the mainstay for off-site, unsupervised PRO data collection in clinical trials. This allows all study data to be stored a single place.
How is Clinical Trial PRO Data Used?
Primary and Secondary Endpoints
PROs might be used as either primary or secondary outcome measures. The FDA states that if a PRO is being used as a primary or secondary endpoint, its role and the instrument being used to measure it must be clearly defined in the research protocol, with evidence of the PROM instrument’s usefulness from the target population of patients.
Other guidelines go further. The CONSORT PRO extension highlights five checklist items recommended for trials in which PROs are primary or important secondary endpoints. An update to the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT) statement, SPIRIT-PRO Extension provides international, consensus-based, PRO-specific protocol guidance.
Adverse Reaction Reporting
PRO instruments are also used to measure the adverse effects of treatments. This is well established in the field of oncology, where PROs have been used for adverse event reporting with a high degree of patient engagement and compliance.
The Patient-Reported Outcomes Safety Event Reporting (PROSPER) Consortium developed guidance for the use of PROs in safety reporting, which outlines definitions and suitable taxonomies, the range of datasets that could be used, data collection mechanisms, and analytic methods.
Because some of the effects of illness are known only to those experiencing them, the FDA recommends that PRO instruments be used to assess efficacy outcomes in clinical trials. The European Medicines Agency is also working to increase the involvement of patients in evaluating benefits and risks on trials.
Again in oncology, online patient self-reporting of toxicity symptoms during chemotherapy has been shown to be feasible, even among patients with advanced cancer and high symptom burdens.
What are the Pros and Cons of PROs?
PROs provide clear advantages in terms of helping both patient and clinician to monitor results of an intervention. But they also have other, practical advantages, such as improving patient-clinician communication and patient experience. This is especially the case with ePRO tools which provide an easy route to foster patient engagement. A patient who has missed inputting their data can be reminded of the importance of taking part in the study and can be followed up immediately. It also provides a means for patients to flag concerning symptoms or side effects in real time.
PROs tools today are much better than those used in the past which were chiefly developed by clinicians. These tended to gather data that is important from the clinician’s perspective rather than patients and were often in a language and format that was a barrier to engagement and participation. Today – patients are involved in the early development of PROMs, and they are used to trigger further patient education and interventions, demonstrating to patients that the data they input is meaningful to their care.
Using mobile technology to collect PROs in clinical studies has many benefits, from collecting data such as blood pressure and blood glucose via Wi-Fi linked devices to the ability to directly transfer data via Bluetooth. This removes the need to manually transfer data (and reduces errors) and ensures an audit trail showing that the right person entered the right data at the right time and date.
Patients perceive these technologies as a “safer” means of providing private or highly personal answers and they also liberate clinicians and staff from coding and scoring PRO instruments.
Despite this, there are many ethical and practical issues to consider, such as how will the information be used? What happens when a patient reports a severe problem needing attention? How will privacy be maintained? The simplicity of technology can also create a tendency to collect much more information than is needed.
The benefits of allowing patients the familiarity of their own device can also be hindered by issues outside of the site team’s control – such as internet downtime, cross-device functionality and security if the device gets into others’ hands. However, the use of apps that capture data when offline, and security measures such as encryption, PINs, and two-factor authentication can solve many of these issues.
There are also risks of collecting data in an unsupervised setting that can cause data collection to be interrupted or delayed, but this issue is not unique to ePRO. It can apply to paper or any other data collection mode.
- PROs are increasingly being used to support regulatory submissions and to place what is important to patients at the heart of new treatment evaluation
- Anything that a patient might report can be considered a PRO, and this can be captured by paper, telephone or via web, tablet and mobile technologies
- PROs can be useful as primary and secondary endpoints, and for monitoring symptoms and adverse events
- Using electronic data capture to collect ePROs is transforming clinical trials, by facilitating straightforward data capture with an instant audit trail.
- Find out how Marvin’s WebPRO function can help PRO data capture on your trial