SPIRIT-PRO Extension Adds 16 Items to Existing SPIRIT 2013

Inclusion of 16 items specific to patient-reported outcomes (PROs) can improve the quality of PRO data. The SPIRIT-PRO Extension has added 16 items to the existing SPIRIT 2013 items, including the statement of specific hypotheses and mentioning whether more than 1 language was used during the trial, according to an international project published in JAMA.¹

Clinical trial protocols do not contain the same information regarding PROs, like health-related quality of life or symptoms, which has been recognized as a necessity to inform clinical decision making and pharmaceutical labeling claims.

The goal of SPIRIT-PRO Extension was to develop an evidence-based extension of the SPIRIT 2013 statement by identifying additional PRO items for the inclusion of clinical trial protocols and to elaborate on the existing SPIRIT 2013 statement. A review of PRO-specific protocol was also conducted.

The SPIRIT-PRO Extension attendees followed the Enhancing Quality and Transparency of Health Research (EQUATOR) Network’s model to guideline development, including an international stakeholder survey of clinical trial research personnel, health economists, health advocates, and other types of researchers.

A total of 30 individuals were invited to the consensus meeting held on May 2017. The consensus meeting participants were informed of a previous Delphi exercise and its results. Overall, 137 national experts in PROs participated in the Delphi exercise to create a list of 56 candidate items that were rated in a survey.

During the SPIRIT-PRO Consensus meeting, 16 items were addressed. 5 elaborations were done to existing SPIRIT 2013 items to address PROs while 11 new extensions were added to existing guidance. Some of the added items include:

• SPIRIT-5a-PRO Elaboration: Specify the individual(s) responsible for the PRO content of the trial protocol.
• SPIRIT-6a-PRO Extension: Describe the PRO-specific research question and rationale for PRO assessment and summarize PRO findings in relevant studies.
• SPIRIT-10-PRO Extension: Specify any PRO-specific eligibility criteria (eg, language/reading requirements or prerandomization22 completion of PRO). If PROs will not be collected from the entire study sample, provide a rationale and describe the method for obtaining the PRO subsample.
• SPIRIT-18a(iv)-PRO Extension: When the trial context requires someone other than a trial participant to answer on his or behalf (a proxy-reported outcome), state and justify the use of a proxy respondent. Provide or cite evidence of the validity of proxy assessment if available.

Researchers believe that these recommendations among otherse will ensure the patient’s voice is heard throughout the study and in clinical decision making.

“The SPIRIT-PRO guidelines provide recommendations for items that should be addressed and included in clinical trial protocols in which PROs are a primary or key secondary outcome,” the authors concluded. “Improved design of trials including PROs could help ensure high-quality data that may inform patient-centered care.”

In an accompanying editorial,² authors explain that determining appropriate PROs has changed with new technology, but while the industry agrees PROs are important in the transition to a value-based healthcare system, "the potential of this vision has not been fully realized." The guidance offered by SPIRIT-PRO will enable a more uniform approach toward collecting and reporting on PRO data, which can help overcome barriers that have slowed the adoption of patient-centered tools at the point of care and reporting PROs.

"For now, PROs are essential as important outcome measures that matter to patients and will need to be incorporated in a systematic and standardized way in clinical trial protocols," the authors wrote in the editorial.

References

1. Calvert M, Kyte D, Mercieca-Bebber R, et al. Guidelines for inclusion of patient-reported outcomes in clinical trial protocols: the SPIRIT-PRO Extension. JAMA. 2018;319(5):483-494. doi:10.1001/jama.2017.21903.

2. Mehran R, Baber U, Dangas G. Guidelines for patient-reported outcomes in clinical trial protocols. JAMA. 2018;319(5):450-451. doi:10.1001/jama.2017.21541.