Medical Writing Patient-reported outcomes Publication planning and patient-reported outcomes: Demonstrating value in a multi-stakeholder era

Volume 27, Issue 4 - Patient-reported outcomes

Publication planning and patient-reported outcomes: Demonstrating value in a multi-stakeholder era

Abstract

Patient-reported outcomes (PROs) are an essential element to demonstrate the value of a health intervention. In many ways, PROs represent the ultimate “real-world” data, yet the drive towards “Big Data” has focused on
routinely collected data from healthcare databases, which often do not include assessments of PROs or the patient voice. Effective planning of PRO publications requires an in-depth understanding of the planned
studies, the opportunities these provide for publications, and how clinicians, patients, and caregivers may contribute as authors to provide validation of results. Mainstream clinical journals and conferences should be targeted wherever possible, considering the availability and objectives of “enhanced publication” options and open access to increase reach, comprehension, and impact. PRO publications must be written in a clear and engaging way, explaining the instrument in simple terms, and addressing the “so what?” question – ideally with an accompanying plain language summary. And PRO publications must always thank the patients.

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References

  1. United States Government. Clinical trials registration and results information submission. Final rule [Internet]. 2016 [cited 2018 Sep 6]. Available from: Available from: https://www.federalregister.gov/document s/2016/09/21/2016–22129/clinicaltrials- registration-and-results-informationsubmission.
  2. European Commission. Commission Guideline – Guidance on posting and publication of result-related information on clinical trials in relation to the implementation of Article 57(2) of Regulation (EC) No 726/2004 and Article 41(2) of Regulation (EC) No 1901/2006. 2012 [cited 2018 Sep 6]. Available from: Available from: https://ec.europa.eu/ health//sites/health/files/files/eudralex/ vol-10/2012_302–03/2012_302–03_en. pdf.
  3. White R, Willett J, Field P. HEOR: why it is changing the world of medical publications. The MAP newsletter [Internet]. 2017 [cited 2018 Sep 6]. Available from: Available from: https://ismpp-newsletter.com/2017/03/ 14/heor-why-it-is-changing-the-world-ofmedical- publications/.
  4. Vakil NB, Halling K, Becher A, Ryden A. Systematic review of patient-reported outcome instruments for gastroesophageal reflux disease symptoms. Eur J Gastroenterol Hepatol. 2013;25(1):2–14. 10.1097/MEG.0b013e328358bf74. Available from: http://
  5. Jones R, Junghard O, Dent J, Vakil N, Halling K, Wernersson B, et al. Development of the GerdQ, a tool for the diagnosis and management of gastrooesophageal reflux disease in primary care. Aliment Pharmacol Ther. 2009;30(10):1030–8. 10.1111/j.1365– 2036.2009.04142.x. Available from: http://
  6. Jones R, Coyne K, Wiklund I. The gastrooesophageal reflux disease impact scale: a patient management tool for primary care. Aliment Pharmacol Ther. 2007;25(12):1451–9. 10.1111/j.1365– 2036.2007.03343.x. Available from: http://
  7. Mitchell P, Bressler N, Tolley K, Gallagher M, Petrillo J, Ferreira A, et al. Patientreported visual function outcomes improve after ranibizumab treatment in patients with vision impairment due to diabetic macular edema: randomized clinical trial. JAMA Ophthalmol. 2013;131(10):1339– 47. 10.1001/jamaophthalm01.2013.4592. Available from: http://
  8. Kay S, Ferreira A. Mapping the 25-item National Eye Institute Visual Functioning Questionnaire (NEI VFQ-25) to EQ-5D utility scores. Ophthalmic Epidemiol. 2014;21(2):66–78. 10.3109/09286586.2014.888456. Available from: http://
  9. Hongo M, Kinoshita Y, Shimozuma K, Kumagai Y, Sawada M, Nii M. Psychometric validation of the Japanese translation of the Quality of Life in Reflux and Dyspepsia questionnaire in patients with heartburn. J Gastroenterol. 2007;42(10):807–15. 10.1007/ s00535–007–2098–9. Available from: http://
  10. Talley NJ, Wiklund I. Patient reported outcomes in gastroesophageal reflux disease: an overview of available measures. Qual Life Res. 2005;14(1):21–33. Available from: http://
  11. Chassany O, Shaheen NJ, Karlsson M, Hughes N, Ryden A. Systematic review: symptom assessment using patientreported outcomes in gastroesophageal reflux disease and dyspepsia. Scand J Gastroenterol. 2012;47(12):1412–21. 10.3109/00365521.2012.712999. Available from: http://
  12. Nagy P, Hage A, Coghill DR, Caballero B, Adeyi B, Anderson CS, et al. Functional outcomes from a head-to-head, randomised, double-blind trial of lisdexamfetamine dimesylate and atomoxetine in children and adolescents with attention-deficit/ hyperactivity disorder and an inadequate response to methylphenidate. Eur Child Adolesc Psychiatry. 2016;25(2):141–9. 10.1007/s00787–015–0718–0. Available from: http://
  13. Calkwood J, Cree B, Crayton H, Kantor D, Steingo B, Barbato L, et al. Impact of a switch to fingolimod versus staying on glatiramer acetate or beta interferons on patient- and physician-reported outcomes in relapsing multiple sclerosis: post hoc analyses of the EPOC trial. BMC Neurol. 2014;14:220. 10.1186/s12883–014–0220–1. Available from: http://
  14. Hunter SF, Agius M, Miller DM, Cutter G, Barbato L, McCague K, et al. Impact of a switch to fingolimod on depressive symptoms in patients with relapsing multiple sclerosis: An analysis from the EPOC (Evaluate Patient OutComes) trial. J Neurol Sci. 2016;365:190–8. 10.1016/j.jns.2016.03.024. Available from: http://
  15. Bakhai A, Sandberg A, Mittendorf T, Greiner W, Oberdiek AM, Berto P, et al. Patient perspective on the management of atrial fibrillation in five European countries. BMC Cardiovasc Disord. 2013;13:108. 10.1186/1471–2261–13–108. Available from: http://
  16. Fridman M, Banaschewski T, Sikirica V, Quintero J, Erder MH, Chen KS. Caregiver perspective on pediatric attention-deficit/ hyperactivity disorder: medication satisfaction and symptom control. Neuropsychiatr Dis Treat. 2017;13:443– 55. 10.2147/NDT.S121639. Available from: http://
  17. Muller-Lissner S, Tack J, Feng Y, Schenck F, Specht Gryp R. Levels of satisfaction with current chronic constipation treatment options in Europe – an internet survey. Aliment Pharmacol Ther. 2013;37(1):137– 45. 10.1111/apt.12124. Available from: http://
  18. Price D, Fletcher M, van der Molen T. Asthma control and management in 8,000 European patients: the REcognise Asthma and LInk to Symptoms and Experience (REALISE) survey. NPJ Prim Care Respir Med. 2014;24:14009. 10.1038/npjpcrm.2014.9. Available from: http://
  19. Ryden A, Martin M, Halling K, Niklasson A. Night-time symptoms and their impact on sleep in patients with gastroesophageal reflux disease who have a partial response to proton pump inhibitors: a qualitative patient interview study. Patient. 2013;6(4):307–14. 10.1007/s40271–013–0024–1. Available from: http://
  20. Price DB, Roman-Rodriguez M, McQueen RB, Bosnic-Anticevich S, Carter V, Gruffydd-Jones K, et al. Inhaler errors in the CRITIKAL study: type, frequency, and association with asthma outcomes. J Allergy Clin Immunol Pract. 2017;5(4):1071–81 e9. 10.1016/ j.jaip.2017.01.004. Available from: http://
  21. Calvert M, Kyte D, Mercieca-Bebber R, Slade A, Chan AW, King MT, et al. Guidelines for inclusion of patient-reported outcomes in clinical trial protocols: the SPIRIT-PRO extension. JAMA. 2018;319 (5):483–94. 10.1001/ jama.2017.21903. Available from: http://
  22. International Committee of Medical Journal Editors. Recommendations for the conduct, reporting, editing, and publication of scholarly work in medical journals. [cited 2018 Sep 6]. Available from: Available from: http://www.icmje.org/ icmje-recommendations.pdf.
  23. Kantor D, Bright JR, Burtchell J. Perspectives from the patient and the healthcare professional in multiple sclerosis: social media and participatory medicine. Neurol Ther. 2018;7(1):37–49. 10.1007/s40120–017–0088–2. Available from: http://
  24. Kantor D, Bright JR, Burtchell J. Perspectives from the patient and the healthcare professional in multiple sclerosis: social media and patient education. Neurol Ther. 2018;7(1):23–36. 10.1007/s40120–017–0087–3. Available from: http://
  25. Calvert M, Blazeby J, Altman DG, Revicki DA, Moher D, Brundage MD, et al. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA. 2013;309(8):814–22. 10.1001/jama.2013.879. Available from: http://
  26. Brundage M, Blazeby J, Revicki D, Bass B, de Vet H, Duffy H, et al. Patient-reported outcomes in randomized clinical trials: development of ISOQOL reporting standards. Qual Life Res. 2013;22(6):1161–75. 10.1007/ s11136–012–0252–1. Available from: http://
  27. International Society For Pharma co - economics and Outcomes Research. Good practices for outcomes research. 2018 [cited 2018 Sep 6]. Available from: Available from: https://www.ispor.org/heor-resources/ good-practices-for-outcomes-research.
  28. O’Brien BC, Harris IB, Beckman TJ, Reed DA, Cook DA. Standards for reporting qualitative research: a synthesis of recommendations. Acad Med. 2014;89(9):1245–51. 10.1097/ACM.0000000000000388. Available from: http://
  29. Tong A, Sainsbury P, Craig J. Consolidated criteria for reporting qualitative research (COREQ): a 32-item checklist for interviews and focus groups. Int J Qual Health Care. 2007;19(6): 349–57. 10.1093/intqhc/mzm042. Available from: http://
  30. European Medicines Agency. Summaries of clinical trial results for laypersons. 2018 [cited 2018 Sep 6]. Available from: Available from: https://ec.europa.eu/health/sites/health/ files/files/eudralex/vol-10/2017_01_26_ summaries_of_ct_results_for_ laypersons.pdf.

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Articles

Patient reported outcomes
President's Message
EMWA News
Measuring health outcomes: The foundation of contemporary healthcare decision-making
Measuring quality of life – theoretical background
Quality of life measures – an overview
Publication planning and patient-reported outcomes: Demonstrating value in a multi-stakeholder era
Patient-reported outcome measure translation: An overview
Systematic hospital collection of patient-reported outcome data via patient apps
The PROMIS of electronic patient-reported outcomes
Interview with Professor Matthias Rose on developing patient-reported outcomes and the PROMIS initiative
The UK pharmaceutical industry braces for Brexit, be it mild, severe, or doomsday
Awareness of medical writing as a profession and its career prospects
Estimands – closing the gap between study design and analysis
Protecting the rights of clinical trial patients through disclosure: The significance of plain language
News from the EMA
Regulatory Matters & Regulatory Public Disclosure
Medical Communications
Medical Devices
Journal Watch
In the Bookstores
Good Writing Practice
Getting Your Foot In The Door
My First Medical Writing
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