Planning patient-reported outcome publications
A publication plan for patient-reported outcomes covers the studies that develop and validate an instrument, the results of using it, the authors who interpret those results and the audiences who need to understand them.
Publication opportunities
Developing and using a patient-reported outcome instrument creates publication opportunities beyond a trial's endpoint results.
| Purpose | Study or publication opportunity |
|---|---|
| Identify available instruments | Systematic literature review |
| Develop a conceptual framework and draft instrument | Patient and physician focus groups and cognitive interviews |
| Confirm the framework and assess the instrument's properties | Validation in relevant patient samples |
| Collect, analyse and interpret patient-reported outcome data | Clinical trials incorporating these endpoints |
| Determine patient health state utilities | Mapping the instrument to a generic health-related quality-of-life or utility measure |
| Modify the measure for wider use | Cultural adaptations, translations and evaluations in related diseases |
Early systematic reviews of instrument use in an indication or population provide the due diligence supporting instrument selection and merit publication rather than remaining internal fact-finding exercises. In 2018, demand was increasing for standalone publications of patient-reported outcome results from randomised trials or real-world studies, even after the primary publication had disclosed the top-line data. Thematic interviews, preference studies and patient or caregiver surveys also offer publication opportunities and context on the impact of disease and treatment. In 2018, these studies were often overlooked as publication opportunities despite increasing demand from mainstream peer-reviewed journals.
Studies and messages in the plan
Planning should begin with close review of trial protocols to identify the patient-reported outcome data that will become available; ideally, the protocols describe these endpoints in line with SPIRIT-PRO. The publication plan should set the scene and provide context for those results. The next step is to review the plans for other patient-reported outcome studies led by internal functions, which may sit in patient-reported outcomes, patient centricity or health economics and outcomes research. A collaborative approach across functions coordinates effort and avoids inconsistent, siloed communications.
Audience needs should guide the choice of publications. For a new instrument, or its first use in an indication, the plan needs introductory articles explaining why it was developed and how it works. For an instrument already established in the disease area, reviews of its previous publications can frame the new studies.
Authorship
A multicentre trial publication typically involves the study writing committee and relevant sponsor representatives, such as the responsible medical director and study statistician. ICMJE authorship guidance also applies to systematic reviews of instrument use, qualitative patient research and validation studies, although their authorship may be less obvious. For these commonly outsourced studies, limiting authorship to the specialist vendor and sponsor's outcomes research representatives is common practice rather than best practice.
The recommended authorship approach is to:
- Include clinicians as authors to provide clinical perspective and validate the practical relevance of the data.
- Include patients or caregivers as authors where appropriate to validate whether the data reflect their real-life experience.
These contributions can be particularly effective for interview- or survey-based research. Their involvement should be planned early so that they can contribute fully to the study and publication and meet authorship criteria.
Journals and conferences
The appropriate venue gives the data maximum exposure to the most appropriate audience in the timeliest fashion. Technical patient-reported outcome journals and conferences should not necessarily be the default: they typically do not reach physicians, patients, payers and other decision makers.
The recommended venue strategy is to:
- Submit to mainstream clinical journals and conferences wherever possible, using technical venues for methodological aspects.
- Publish full papers open access so that interested readers can obtain them without paying.
Alongside impact factor, lead times and geography, journal selection should assess a journal's receptiveness to patient-reported outcome publications, its record of publishing the relevant study types and whether its editorial board has expertise in these outcomes to support meaningful peer review.
Publication enhancements
Materials beyond the article or conference presentation are essential to help non-specialists understand patient-reported outcome results. The format should be chosen according to its objective.
| Objective | Materials or channels |
|---|---|
| Improve comprehension | Educational formats, such as plain language summaries and explanatory videos |
| Increase reach | Media, email, healthcare professional communities and social sharing |
| Translate evidence into clinical impact | Tools such as apps and decision algorithms |
Comprehension is generally the primary concern because these outcomes are poorly understood and their clinical application is often unclear. Reach matters when the initial venue will miss intended readers, such as clinicians who do not read a technical journal. Clinical impact may be an objective when there is potential to increase adoption of an instrument in practice. The materials should be integrated with wider medical affairs communication planning, and compliance with the relevant regulatory and promotional guidelines for disseminating data is essential.
Options include an infographic, an author video, an animation, an interactive annotated publication and a glossary, which can also encourage social sharing. For journal articles, these should ideally be peer-reviewed supplementary materials associated with the article. For conference presentations, augmented reality is one way to connect physical materials with embedded digital content. A patient-reported outcome publication should have a tailored plain language summary explaining the instrument and the meaning of the results clearly and engagingly; ideally it is a peer-reviewed supplement.
An example of these enhancements accompanied qualitative research on patient and physician perspectives in multiple sclerosis, with summary slides and an author video.