Patty Spears, Research Patient Advocate and program manager of Cancer Information & Support Network, wrote this post about the patient voice in outcome assessments for Breast Cancer Clinical Trials Matter to You.
I have been interested in how clinical trials are developed and conducted since my diagnosis and treatment of breast cancer in 1999. My diagnosis led me to participate in two clinical trials, which was an easy decision for me. I have been involved in basic research for the past 30 years studying microbial pathogenesis, so participating in a clinical research study was a natural choice. However, this is not the case with most cancer patients.
Having been a patient advocate for more than 10 years, I have had multiple opportunities to be involved in the development and conduct of clinical trials at both the local and national level. During this time there have been many changes in the way trials are conducted, and changes in the expectation of patients who volunteer to participate in the trials. Patients today are more involved in clinical research and in their own healthcare than in the past. Patients today want to have a voice. And they need information from other patients about their experiences on clinical trials to make informed decisions about their own current treatments. To make informed decisions, patients need to have information about benefits and harms that are important to them. Assessing potential benefits and harms is very subjective and needs to be done consistently and reliably to accurately predict what is important to patients.
One way to measure benefits and harms is by using Patient Reported Outcome (PRO) assessments through the administration of questionnaires answered directly by the patient. PROs are defined 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.” The incorporation of PROs in clinical trials measures not only what is the matter with the patient, but also measures what matters to the patient1.
There not only needs to be a shift in what is being measured and reported by patients, there also needs to be an effort to make the questionnaires more acceptable to patients to complete during clinical trials. I have participated in a clinical trial that used questionnaires (PROs) and I have also asked many other patients about their experiences and feelings about questionnaires regarding barriers and incentives to completing them including their acceptability of how they are administered.
Patients want to understand how the information they provide will be used to inform future patients. Asking the same question multiple times (redundant questions), which is likely to occur when multiple questionnaires are packaged into one trial, is irritating to patients. This was the case in the questionnaires I completed. I would actually go back to find what I answered on the previous page and I wondered whether they were actually testing me to see if my answers were consistent. This does not build trust with patients.
There were also some barriers and acceptability issues to completing questionnaires including the time it takes to fill them out, the number of questions on each questionnaire and how often questionnaires are given to the patient. There is not one answer, there is not a perfect number of questions to ask, and it will depend on the trial and the predetermined PRO endpoint. For example, a questionnaire with fewer questions may be able to be given more often and may capture trends in how patients are feeling and functioning immediately after therapy and before their next appointment, which may provide very useful information for future patients. The longer questionnaires are best given less frequently but can still add value to capturing the entire patient experience. With the move toward electronic PRO assessments there are more opportunities to incorporate PROs into all clinical trials in ways that are acceptable to patients and provide information that is important to future patients.
It is time to transform PROs to be more meaningful by measuring what is important to patients and to ensure the information is reported back to patients and clinicians to guide informed decision-making.
What is needed to ensure PROs are in all clinical trials and that they truly measure what is important to patients? Patients need to be involved in informing which questions should be asked based upon specific disease condition and therapies being tested. Every trial is unique and will require different PRO assessment strategies so that the questions asked are relevant to that specific situation. Patient relevant endpoints need to be identified early in trial development so the appropriate questions can be asked in the clinical trail. The purpose or endpoint based upon the PRO assessment needs to measure what is important to patients and should guide what questions are asked and how often they are asked. The clinical trial needs to be conducted and the PRO measurements collected with the same rigor as other measurements (e.g., imaging) within the clinical trial.
How do you develop targeted, precision PRO assessments for patients?
Ask patients to report what matters to them
- Make PRO measurements acceptable to patients to complete
- Make PRO measurements an important part of the trial with well-defined purpose and use
- Make the PRO endpoints meaningful to patients
- Make the PRO information available to patients
In the end, it is about developing Precision PRO Assessments: asking the right question, at the right time, in the right way, so that what is being measured is important to patients.
1Accelerating Anticancer Agent Development and Validation, 2015. Patient and Stakeholder Engagement in Risk/Benefit Decision Making in Cancer Drug Development, Sandra Finestone
2Paul G. Kluetz, Ashley Slagle, Elektra J. Papadopoulos, Laura Lee Johnson, Martha Donoghue, Virginia E. Kwitkowski,Wen-Hung Chen, Rajeshwari Sridhara, Ann T. Farrell, Patricia Keegan, Geoffrey Kim, and Richard Pazdur Focusing on Core Patient-Reported Outcomes in Cancer Clinical Trials: Symptomatic Adverse Events, Physical Function, and Disease-Related Symptoms, Clin Cancer Res. 2016 Apr 1;22(7):1553-8. doi: 10.1158/1078-0432.CCR-15-2035