The Importance Of Patient-Reported Outcomes In Clinical Trials

Accurate, complete and compelling clinical trials data is an essential piece in securing US Food and Drug Administration (FDA) approval for a new treatment. Though this data is collected based on a variety of clinical trial-specific outcomes measures – such as tumor size reduction and progression-free survival – it might not always be collected from the patient’s perspective.

While clinical trials may do a good job of assessing a drug or treatment’s efficacy at treating a disease, they often fail to take into account the patients’ experience when participating in a clinical trial. Clinical trials most often rely upon observer-reported outcomes whereby a physician, clinician or clinical trials staff member records outcomes-related data based on their interpretation of the treatment’s efficacy.

Once a medication is approved for use by the FDA, patient-reported outcomes become an important tool to guide patients and clinicians to make the best treatment decisions possible. In a field like oncology where patients may have a number of treatment options, patient-reported outcomes can help match the patient to the treatment that will be most effective, while minimizing side effects. If two oncology treatments offer equivalent survival benefits, patient-reported outcomes could be used to differentiate between the treatment choices by identifying which treatment had fewer negative effects on patient health.

Relying on traditional observer-reported outcomes alone, physicians only know what results were seen in the trial participants as a group. Individual patient outcomes are consistent with the trend toward precision medicine, to ensure cancer patients are given more personalized treatment.

As well as taking the clinical trial participants’ experience into account, patient-reported outcomes have the benefit of supporting certain label claims, making them potentially very valuable to the drug developer. The FDA recommends that sponsors include patient input when designing a patient-reported outcomes measure, to ensure the instrument is developed to produce credible results.

How are Patient-Reported Outcomes Measured?

Patient-reported outcomes can be collected using a number of self-report tools, including patient logs, questionnaires or even patient interviews. If the latter approach is taken, the clinician recording the patient responses must not confound the data in any way by adding their own observations or opinions.

Patient-reported outcomes measures most often assess factors that matter most to the patient, such as symptoms, functioning and quality of life. Even if observer-reported outcomes suggest a treatment option is effective at reducing tumor size, patients may not report an equivalent benefit in terms of their own quality of life.

Measures of symptoms may address a broad range of disease-associated impairments, or they could focus on a specific symptom, such as pain. Whether a questionnaire is designed to measure a single patient characteristic (unidimensional) or addresses a number of characteristics (multi-dimensional), it’s important that the tool has been confirmed to measure what it has been designed to measure.

When assessing a patient’s self-reported ability to function, patient-reported outcomes measures should be designed to determine how well a clinical trials participant believes that he or she is able to perform personal care activities, as part of daily life. Quality of life measures go beyond assessing symptoms and patient functionality in isolation, and instead aim to measure a patient’s broader emotional response to their limitations.

As with any tool used to collect or measure during a clinical trial, patient-reported outcomes measures must be fully validated for the appropriate patient population before being applied in the clinic. While generic questionnaires are available which apply to wide patient groups, condition-targeted outcomes measures can provide clinicians with a more specialized tool designed assess patients with a given disease.

Barriers Preventing Inclusion Of Patient-Reported Outcomes In All Clinical Trials

There are a number of barriers to implementing patient-reported outcomes data collection on a clinical trials industry-wide scale. Clinicians’ main concerns are that the additional data collection will be burdensome, as it adds to their own workload as well as that of any data entry professionals.

Interestingly, some clinical trials professionals feel that patient-reported outcomes are not necessary provided that have already established a good relationship with their study participants. In this case, the clinicians believe that their own assessment of the treatment benefits is consistent with the patient’s own experience, which may not always be the case.

Building patient-reported outcomes measures into clinical trials isn’t the only hurdle facing the oncology community. A recent study published in the Journal of Clinical Oncology, found that most newly-approved oncology drugs don’t include patient-reported outcomes in their package inserts. The researchers found that only three out of 40 new cancer drugs approved in a four-year period – between January 2010 and December 2014 – included patient-reported outcomes information in the drug insert.

Compared to the 24 percent of non-oncology treatments that were found to include patient information over the same time period, it’s clear that something is preventing cancer drug developers from collecting and sharing this information. While not required by the FDA, the regulatory body does encourage pharmaceutical companies to include patient experiences alongside indications information and possible side effects to help inform treatment decisions.

Interestingly, these same researchers found that one third of oncology drugs included patient-reported outcomes measures in their clinical trial data collection, but decided against displaying such information on the product labelling. Despite their efforts to collect the patient-reported data, something must have prevented them from including the information in the product insert.

As oncology clinical trials tend to be smaller, it’s possible that patient-reported outcomes would be less valuable to the wider patient population. In addition, many new cancer drugs – including most of those reviewed in the current study – are granted priority review by the FDA, potentially decreasing the amount of time available for clinicians to collect meaningful, long-term patient experience information.

Now that patient-reported outcomes are becoming a standard way to gauge the patient experience during a clinical trial, the next step for clinicians will be to ensure that this patient data is being put to good use. By using these patient reports to guide treatment decisions in the clinic, oncologists will be able to provide more personalized medicine to oncology patients.