How to collect patient-reported outcomes in clinical trials
Patient-reported outcomes (PROs) are "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," according to the FDA. They are also a way to include the patient voice in clinical trials.
More than a quarter of clinical trials now use patient-reported outcomes, and with the FDA's increased focus on PROs, that number is expected to grow in the coming years. In 2009, the FDA released guidelines stating that if label claims are to be used in marketing, trials must collect PROs related to the patient experience.
In addition to the FDA's focus on PROs, including them in a trial design can lead to better therapy with meaningful benefits to patients. The timeline for including PROs can be long, however, so it's essential to plan ahead and start engaging patients, PRO vendors, and even the FDA early on in the process.
How to collect PROs in a clinical trial
Step 1: Research existing PRO instruments for each condition area.
It can be helpful to begin the process by researching the literature to understand what PRO instruments have been used and if they were implemented successfully in an FDA-approved label claim. Existing PRO measures may suit the trial's needs, or it may be necessary to modify an existing PRO or start from scratch.
Step 2: Conduct patient interviews or focus groups.
If modifying an existing PRO measure or creating a new one, the next step is to talk with patients. Options include conducting patient interviews, running focus groups, or contacting patient organizations. Patient input can help determine what it's like to live with your trial's condition and what quality of life improvements would be most meaningful for patients.
Step 3: Decide whether to use electronic patient-reported outcomes or paper forms.
As of 2014, around half of clinical trials that used PRO used electronic devices to capture data. While that number may seem low, use is expected to grow by 15% between now and 2025. While using electronic patient-reported outcomes (ePRO) involves up-front costs, advocates say that going digital helps reduce the time spent on data reconciliation. It also may provide more accurate data, as patients are more likely to record symptoms and answer questions in real time through an electronic device. If the trial uses ePRO, talk with potential vendors to ensure their devices are compatible with the existing clinical trial technology.
Step 4: Formulate questions and timing.
PROs are ideal for capturing data on how a patient's quality of life has improved since beginning the treatment. They can also be used to measure adverse events in a clinical trial. PROs can also capture information about symptoms not measured during clinical visits. It's particularly valuable to measure questions related to pain using PROs.
When creating patient-reported outcome questions, it's also crucial to understand how to ask each question. Nine out of ten American adults struggle with health literacy – many people struggle to understand medicine bottle labels, doctor instructions, and other health materials. Use clear, unambiguous language when formulating questions. It's also a best practice to capture information about symptoms and adverse events as they occur when possible. Experts also recommend keeping in mind the burden of questions on the patient, and limiting the length of the required questions to increase engagement rates.
Step 5: Conduct user acceptance testing.
Once the questions are formulated and the PRO system is ready, it's time to start user acceptance testing (UST). If working with an ePRO vendor, staying in close contact with them throughout this process is crucial in case tweaks need to be made to the e-diary or other tools based on user feedback.
Step 6: Submit your materials to your Institutional Review Board (IRB).
Throughout the PRO design process, it's essential to consider your IRB. One of the prominent roles of an IRB is to consider whether a trial impedes patient participation. In general, IRBs are more likely to look at the content of PRO design rather than whether the trial uses paper or an electronic device, but keep the patient population in mind when deciding to go digital. For example, there may be cases where an IRB determines that electronic devices aren't the right fit, such as Alzheimer's patients.
Step 7: Train your site staff and develop a user guide.
No matter how intuitive the PRO instrument may be, patients and site administrators will likely have questions once your trial launches. Based on your findings from user acceptance testing, create a training guide for site staff that includes instructions on using the PRO instrument and answers to anticipated questions. Depending on the device being used, you may need to prepare your staff to answer questions about the user experience on several devices.
Step 8: Start recruiting patients.
Once your materials have been approved and your sites have been selected, it's time to enroll patients in your trial. We created a clinical trial patient recruitment template to help. Download your free copy below, and let us know if Antidote can help you connect engaged patients with research.
