Design and Oversight
This project comprised both a survey development stage, in which the questions were created, and a subsequent two-part deployment stage, in which surveys were used in clinical studies (Fig. 1). The project was organized and overseen by a cross-functional taskforce within UCB Pharma using a disease-agnostic approach and conducted in accordance with ethical standards for market research and data privacy regulations. Alignment with UCB Pharma best practice for protocol simplification meant that the PES (which were not associated with study endpoints) were not included as a required activity within the clinical study schedule of events. Thus, potential responders had to provide separate consent before participation. When the online surveys were made available to patients, the first page (landing page) explained the survey aim, UCB Pharma sponsorship, and assurances around confidentiality and informed consent. All participants who viewed the landing page at the end of their study participation were offered the opportunity to opt-in to undergo an interview and receive plain language summaries, even if they chose not to complete the survey. No monetary incentives were provided for survey participation.
In 2016, UCB Pharma started development of PES, which were modeled on a self-administered close-out survey designed by an independent market research organization . That model survey was informed by a literature review and patient interviews, and included a 50-item set of closed-ended questions (multiple choice or 5-point Likert scale) on the themes (identified as preferred from patient interviews) of communication, site experience, convenience, relationship building and support, compensation, and helping self and others .
Recognizing the importance of capturing the patient experience at key moments during their clinical study journey, UCB Pharma planned to develop 3 separate surveys: (1) “Introductory survey,” administered after informed consent, (2) “Interim survey,” administered during the clinical study at a time determined by the study team, and (3) “Close-out survey,” administered at the end of study participation (i.e., at study completion or patient withdrawal). For each survey, UCB Pharma adapted the model survey by adding and reformulating questions to fulfill UCB Pharma’s strategy to partner with patients. An iterative process was used involving an internal, cross-functional team with expertise in psychometrics, global clinical operations and development, human-centered design, survey research, data analytics, market research, legal, ethics and compliance, data privacy, and commercialization management.
The new survey questions underwent health literacy testing via a patient engagement co-creation process conducted by the independent market research organization on UCB Pharma’s behalf. An online survey/telephone interview was administered to a sample of 6 past clinical study participants (4 women, 2 men, all Caucasian, aged 36–64 years) residing in the United States (US) and Canada who were recruited through a proprietary database. The new questions were then critically evaluated based on comprehensive review of patient feedback.
Between March and August of 2017, UCB Pharma deployed the PES in 7 global phase 2/2a studies at sites in the US, Canada, Poland, Australia, and Japan (Fig. 1). The planned use of the PES was included in a submission to independent ethics committees (IECs) after the study had started. The PES were submitted to a central institutional review board (IRB) in the US first. To establish precedent and enable a request for no changes to the surveys, the US IRB approval was included in subsequent IRB/IEC submissions. Ethics committee approvals were granted before the PES were initiated. Training materials on the PES, including their purpose and deployment, were distributed to sites via email and/or video. Study participants were invited to participate in the online survey via a printed postcard (in the participant’s own language), which guided the participant to the online PES landing page. Site staff were asked to distribute postcards to all study participants at the appropriate study visit. Because the PES were not included in the study protocol, site staff were also sent reminders (e.g., direct-to-site letters, study portals, newsletters) about survey distribution. The UCB Pharma Site Engagement Team was asked to obtain feedback from site staff on survey deployment.
Following the initial deployment in Part 1, the PES were deployed at the start of 5 phase 2/2a and 3 global clinical studies between January and August 2018 (Fig. 1). All 5 studies in Part 2, as well as 2 of 7 studies in Part 1, are ongoing as of September 2019. The process of survey deployment in Part 2 was modified slightly based on experience obtained during Part 1. Site staff training materials were similar to those in Part 1; however, unlike in Part 1, the PES were included in the initial (i.e., before study start) IRB/IEC submissions, discussed during site initiation visits and prestudy investigator meetings, and deployed at the start of each study.
Most studies in Part 2 used postcard invitations, as in Part 1. However, in one study, a “myUCB 4me” digital app was developed by UCB Pharma to support clinical outcome assessments. The app was optimized to issue the PES invitations at the appropriate study visit and direct participants to the landing page so that they could access the survey on the same device. Site staff and UCB Pharma staff were interviewed to gain feedback on PES deployment.
The surveys were translated by qualified translation services into 6 languages or local dialects (e.g., Canadian French, Australian English) for Part 1 and 18 languages/dialects for Part 2. The meaning of the questions was checked when translated into other languages, including via back-translation.
Data Management and Statistics
A data management plan was developed before the study that addressed the confidentiality, acquisition, management, analysis, storage, reporting, and sharing of survey data. An invitation-only system was used to prevent unauthorized PES access; study participants could only participate if they received a postcard invitation from site staff or an invitation from the myUCB 4me app.
To reassure participants regarding confidentiality, no identifying information was collected. Therefore, individual participant responses could not be linked from one survey to the next survey. Site-specific feedback was only available once at least 5 surveys had been completed and was provided in aggregate form to protect patients from being identified. This threshold also applied at the study level and country level.