Improving Patient-Reported Outcomes Guidelines for Clinical Trial Protocols

A group of researchers from the UK, Australia and Canada have proposed changes to international guidelines for clinical trial protocols to improve the use of patient-reported outcomes. The special communication – which was published in JAMA – calls for the addition of 11 extensions and five elaborations to the Standard Protocol Items: Recommendations for Interventional Trials (SPIRIT) 2013, currently a 33-item checklist designed to help ensure that clinical trial protocols are complete.

Clinical trial protocols include all aspects of how a study will be conducted, including the purpose and design, as well as the methods and outcomes measures to be used. Well-written clinical trial protocols can help ensure that the health and wellbeing of patients participating the clinical trial are protected, and that data collected is of good-quality.

Historically, patient input on how the trial, or the intervention being tested, has affected their quality of life, has not always been considered. However, as the clinical trials industry focuses on being more patient-centric, interest in the inclusion of patient-reported outcomes measures has increased.

“Patient-reported outcome data from clinical trials can provide valuable evidence to inform shared-decision making, pharmaceutical labelling claims, clinical guidelines, and health policy; however, clinical trial protocols often lack important information regarding the collection of quality of life and symptom data,” said Professor Melanie Calvert, Director of the Centre for Patient-Reported Outcomes Research (CPROR) at the University of Birmingham. “Working in collaboration with international stakeholders we have developed consensus-based, patient-reported outcomes-specific protocol guidance to help ensure high-quality data to inform patient-centred care.”

Calvert and her colleagues recommended a total of 16 changes to the current SPIRIT 2013 checklist, which have collectively been dubbed the SPIRIT-PRO Extension. These specific recommendations would apply to clinical trial protocols where patient-reported outcomes would be used as a primary or key secondary outcome.

“As more new cancer treatments are tested as part of clinical trials, it is vital that we know about the impact they have on patients’ lives,” said Professor Jane Maher, Joint Chief Medical Officer at Macmillan Cancer Support, a charitable organization that provided funding for this research. “This will enable people to decide whether or not to have a particular treatment in the future.

“New cancer treatments can add months or even years to life, but they can also have side effects which can really have an impact on quality of life, in some cases long after treatment finishes. Such outcomes have often not been reported,” said Maher. “These recommended changes to international guidelines will help ensure that people’s experiences of new treatments are properly recorded and published, and I would urge researchers to adopt them.”

The proposed changes focus on how patient-reported outcomes impact key trial features, including the rationale, objectives and inclusion criteria for the study. In addition, they give guidance on patient-reported outcomes instrument selection, translation of the protocol into other languages for the purposes of obtaining informed consent, and deciding whether patient-reported outcomes data will be collected and used during the course of the trial to shape how patients are being cared for.

“While this guidance has been developed for trials where patient-reported outcomes are a primary or key secondary outcome, we are actively encouraging protocol writers to consider use of this guidance in all trials or clinical research studies where patient-reported outcomes are collected,” said Calvert. “The guidance does not aim to be prescriptive, but instead to encourage and facilitate careful planning of patient-reported outcomes components of trials, and thereby improve patient-reported outcomes trial design, which we hope will help staff and patients understand the rationale for patient-reported outcomes assessment, improve patient-reported outcomes data completeness and quality, facilitate high quality analysis and reporting, and ultimately improve the quality of the global patient-reported outcomes evidence base.”