PRO Research Q and A with David Cella, PhD

Patient-centered outcomes research (PRO research) was the topic of a webinar hosted by the ECOG-ACRIN Cancer Research Group on December 5, 2017. The speaker was David Cella, PhD (Northwestern University), who co-chairs the Group’s Cancer Control and Survivorship Committee. The intended target audience was research personnel.

Below are the responses Dr. Cella gave to questions submitted by webinar attendees. The content was current as of the event date and will likely become outdated over time.

Q1. What are the differences, if any, in implementing PRO research in pragmatic trials vs. explanatory trials? Are there any considerations we should think about when designing such trials? Note to readers: Pragmatic trials test the effectiveness of an intervention in a broad routine clinical practice. Explanatory trials evaluate the effectiveness of an intervention in a well-defined and controlled setting.

A1. Special considerations include blinding (which might not be the case in many of these studies); whether or not you are obtaining informed consent; and the placebo/nocebo effects that many studies are vulnerable to (especially when blinding is not in place).


Q2. What opportunities are there for cancer care delivery research (CCDR) studies that look at the facilitators of the use of PROs in routine care delivery at community cancer centers?

A2. This is an exciting new area and the National Cancer Institute (NCI) is very interested in this. The NCI has published some funding announcements and more are likely to come, so this is a good time to get involved. A good source of information on available opportunities is the NCI Healthcare Delivery Research Program webpage.


Q3. What PROMIS® (Patient-Reported Outcomes Measurement Information System) evaluation seems best to determine pain and disability relief following spinal manipulation?

A3. Two good ones include PROMIS Pain Interference and PROMIS Physical Function. There may be other PROMIS measures relevant to additional characteristics on which the researcher is focusing.   More information, including this guide to measure selection, can be found on the HealthMeasures website.

Q4. How can other established web-based systems that capture PROs access research funds available at the Patient-Centered Outcomes Research Institute (PCORI)?

A4. Joe Selby, Executive Director of PCORI, recently circulated a letter affirming that PCORI is active and has money to fund future project proposals. For information, visit the PCORI Funding Opportunities webpage.


Q5. I am a pain physician at Stanford Pain Center and am interested in learning about how you use your registry for clinical care and research.

A5. We do not maintain a registry like the one you describe, but Stanford does have the Collaborative Health Outcomes Information Registry (CHOIR), which is headed up by Sean Mackey. For more information, visit the CHOIR webpage.


Q6. How might patient-reported outcome measures apply to home care?

A6. PROMIS banks cover the low end of functioning very well, so someone working in home care could go into the PROMIS banks and look at something like physical function to identify questions that are very basic (e.g., bed to chair transfers).


Q7. Our organization is interested in using PRO tools to evaluate the impact of population health-focused work, including elimination of health disparities. Some areas of focus include infant mortality, diabetes morbidity/mortality, substance use disorder, and complex care. Are there particular instruments that are recommended for any or all of these areas? 

A7. The PROMIS Global Health measure is recommended. The Centers for Disease Control and Prevention (CDC) is now deploying it in Healthy People 2020 and also plans to use it in its 2030 goals for the nation. The ECHO Program (Environmental Influences on Child Health Outcomes) supported by the NIH might be a good avenue for infant mortality. ECHO has just undergone a process of identifying a standard measure set and common data elements in this area. As for diabetes, the PROMIS Global Health measure is recommended in this area as well, in addition to looking at other measures. There is a whole set of item banks within PROMIS related to substance use. The US Center for Medicare and Medicaid Innovation (CMS) is recommending the PROMIS Global-10 or PROMIS-29 for complex care. By definition, complex care can’t be disease-specific, so you would really benefit from these more universal approaches.


Q8. Are PROs being developed for real-time (digital display) and, if so, has a standard platform been agreed upon and who is building it?

 A8. Yes, such PROs are being developed, though there is not currently a standard platform in use. There are multiple vendors who are building systems like PROMIS into their electronic health records. Unfortunately, there is no standard quite yet, but having multiple vendors will hopefully encourage future innovation. Additionally, there is an API available (more information available on the website).


Q9. What progress to date has been made in getting PROMIS into regular (i.e., integrated) use in large US HMOs.

A9. Some large HMOs (such as Kaiser in California) have adopted PROMIS widely. Others plan to do so as Epic brings computer adaptive tests (CATs) online (which happened with PROMIS). Progress is still stirring, but there are large systems like Cleveland Clinic and Partners that are all-in with PROMIS.


Q10. What current studies are using the PRO-CTCAETM?

A10. The ECOG-ACRIN Cancer Research Group is currently using PRO-CTCAE measures in a few therapeutic trials focused on renal cell carcinoma, melanoma, and advanced breast cancer. Looking more broadly, this NCI introductory slide presentation indicates that more than 100 early adopters in academic settings, along with several industry-sponsored trials, are using PRO-CTCAE in treatment trials and observational studies. For a complete roster of studies using these measures, contact vog.hin.liamnull@seiriuqnIEACTCORPICN.


Q11. Performance status is regarded as an inclusion/exclusion criterion in most treatment trials. Can quality of life (QoL) scores replace this (because performance status captures an investigator’s assessment of QoL)?

A11. We are not aware of any current efforts to replace or supplement performance status with QoL scores as standard criteria for measuring how a disease impacts a patient’s daily living abilities. It is an interesting idea for consideration.


Q12. Are there any costs involved for using the forms referenced in this webinar?

A12.  PDFs for PROMIS, FACIT and PRO-CTCAE measures are freely available in English and (in many cases) Spanish and other languages. Registration may be required. Please reference the respective terms of use for PROMIS, FACIT, and PRO-CTCAE for additional information.


Q13. Would you speak to the potential use of PRO-CTCAE “alone” or at a different frequency than CTCAE assessment to capture PROs on disease and treatment-related symptoms during a trial?

A13. Research on the use of PRO-CTCAE is ongoing, and your question represents an interesting future direction.

ECOG-ACRIN Cancer Research Group