Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2020 Feb;23(1):41-51.
doi: 10.1111/hex.12997. Epub 2019 Nov 13.

Including the patient voice in the development and implementation of patient-reported outcomes in cancer clinical trials

Bonnie Addario  1 , Jan Geissler  2 , Marcia K Horn  3 , Linda U Krebs  4 , Deborah Maskens  5 , Kathy Oliver  6 , Ananda Plate  7 , Erin Schwartz  8 , Nicole Willmarth  9
Affiliations
Review

Including the patient voice in the development and implementation of patient-reported outcomes in cancer clinical trials

Bonnie Addario et al. Health Expect. 2020 Feb.

Abstract

Context: Patient-reported outcomes (PROs) are used in parallel with clinical evidence to inform decisions made by industry, clinicians, regulators, health technology assessment bodies and other health-care decision-makers. In addition, PRO data can also guide shared decision making and individual patient choice. Yet, the quality of many PROs in cancer clinical trials is suboptimal and requires improvement to add value to health care and policy decision making.

Objective: To show how the integration of the patient and/or patient advocate at all stages of PRO development can help to realize the full potential of PROs.

Methods: We examined the literature to show that the patient voice is often absent from the planning and implementation of PROs in cancer clinical trials. Good practice examples from the literature were combined with guideline recommendations, training or educational resources, and our own experience to create detailed practical steps for the inclusion of patients and/or patient advocates throughout PRO development.

Results: Patient or patient advocates can play an active role in shaping PROs that are meaningful to the patient. They can contribute to content, choice of medium and implementation in a way that may support PRO completion and minimize missing data. Patients and their advocates can work to ensure PRO findings are disseminated appropriately in a way that is accessible to patients.

Conclusion: This practical guidance aims to optimize PRO development and implementation in clinical trials, resulting in robust, relevant data that reflect the patient experience and that support decisions made by all stakeholders involved in research and health care.

Keywords: cancer; clinical decision making; patient advocacy; patient-reported outcomes.

PubMed Disclaimer

Conflict of interest statement

Bonnie Addario, Marcia K Horn, and Linda U Krebs; I have no financial or other relationships, conditions, or circumstances that present a potential or actual conflict of interest that influenced what is written in the submitted work. Jan Geissler; Advisory roles and consultancy on meaningful patient engagement to Amgen, Biomarin, Bristol‐Myers Squibb, Janssen, Novartis, Pfizer, Roche, Servier, UCB. Contributions to IMI‐funded consortium projects HARMONY and EUPATI by EFPIA member companies. Deborah Maskens; I have no financial conflicts of interest except for some reimbursement of travel/accommodation costs to some essential meetings locally within Canada by associations that have obtained funding from the pharmaceutical industry. Any honoraria are donated in full to Kidney Cancer Canada (registered charity) for patient support, services and research. Kathy Oliver; I believe that I have no actual, perceived or potential conflict of interest with regard to this paper. The International Brain Tumour Alliance (IBTA) of which I am Chair and Co‐director accepts educational and non‐directed grants for its work from a number of pharmaceutical and medical device companies. IBTA also accepts a small number of donations from the general public and on occasion we have been supported by private trusts/bequests. In my role for Kathy Oliver Consulting, I have accepted honoraria for speaking engagements and, from time to time, providing advisory/consultancy services for a number of pharmaceutical and medical device companies. Ananda Plate; All funding and honoraria received are paid directly to Myeloma Patients Europe AISBL (non‐profit organization registered in Belgium) for member services and research to the benefit of the myeloma patient community. Erin Schwartz; The Max Foundation, for which I am VP of Global Engagement, is a non‐profit 501c3 entity which is independent and is a non‐governmental organization. The organization does receive funding from the pharmaceutical industry, including Novartis and other companies. In the last several years, I have participated in advisory boards with Pfizer and BMS which required consulting contracts. I am also on a high‐level committee with Novartis related to our patient access programme work. Nicole Willmarth; No conflict of interest to declare.

Figures

Figure 1
Figure 1
Levels of patient and patient advocate engagement (adapted from Wilson et al25)
Figure 2
Figure 2
Checklist for involving patient partners when initiating a PRO development project
Figure 3
Figure 3
Stages of PRO development and suggested input from the PRO development steering committee and patients/patient advocates at each stage
See this image and copyright information in PMC

References

    1. Fitzpatrick R, Davey C, Buxton MJ, Jones DR. Evaluating patient‐based outcome measures for use in clinical trials. Health Technol Assess. 1998;2(14):1‐74. - PubMed
    1. Dawson J, Doll H, Fitzpatrick R, Jenkinson C, Carr AJ. The routine use of patient reported outcome measures in healthcare settings. BMJ. 2010;340:c186. - PubMed
    1. European Medicines Agency . Appendix 2 to the guideline on the evaluation of anticancer medicinal products in man. The use of patient‐reported outcome (PRO) measures in oncology studies. London, UK: EMA: 2016.
    1. Mercieca‐Bebber R, King MJ, Calvert MC, Stockler MR, Friedlander M. The importance of patient‐reported outcomes in clinical trials and strategies for future optimization. Patient Relat Outcome Meas. 2018;9:353‐367. - PMC - PubMed
    1. Au HJ, Ringash J, Brundage M, et al. Added value of health‐related quality of life measurement in cancer clinical trials: the experience of the NCIC CTG. Expert Rev Pharmacoecon Outcomes Res. 2010;10(2):119‐128. - PubMed

Publication types