Recruitment strategies and geographic representativeness for patient survey studies in rare diseases: Experience from the living with myeloproliferative neoplasms patient survey

Abstract

Background Recruitment of individuals with rare diseases for studies of real-world patient-reported outcomes is limited by small base populations. Myeloproliferative neoplasms (MPNs) are a group of rare, chronic, hematologic malignancies. In this study, recruitment strategies and geographic representativeness from the Living with MPNs survey are reported. Methods The Living with MPNs online cross-sectional survey was conducted between April and November 2016. Individuals 18 to 70 years of age living in the United States and diagnosed with an MPN were eligible to participate. Recruitment approaches included direct contact via emails and postcards; posts on MPN-focused social media and patient advocacy websites; postcard mailings to doctors’ offices; and advertisements on medical websites, Google, and Facebook. Geographic representativeness was assessed based on the number of survey respondents living in each state or the District of Columbia and by the number of survey respondents per 10 million residents. Results A total of 904 respondents with MPNs completed the survey. The recruitment method yielding the greatest number of respondents was advertisements on MPN-focused social media (47.6% of respondents), followed by emails (35.1%) and postcards (13.9%) sent through MPN advocacy groups. Home state information was provided by 775 respondents from 46 states (range of respondents per state, 1–89). The number of respondents per 10 million residents in the 46 states with respondents ranged from 12.1 to 52.7. Conclusions Recruitment using social media and communications through patient groups and advocacy organizations are effective in obtaining geographically representative samples of individuals with MPNs in the United States. These approaches may also be effective in other rare diseases.

Patient‐reported outcomes among patients with systemic mastocytosis in routine clinical practice: Results of the TouchStone SM Patient Survey

“… 21 The small sample and relatively low participation (16%) may be partially explained by recruitment from a disease registry as opposed to broader community approaches. 21 , 22 , 34 , 35 However, our response rate is consistent with other published surveys. 36 The TouchStone Survey was also subject to standard biases inherent to survey‐based studies (i.e., question order effects, etc.).…”

“…Our sample size is smaller than some previous studies on the burden of mast cell disorders, which reported hundreds of patients, 22,34 although it is similar to another survey of 50 patients with myeloproliferative neoplasms 21 . The small sample and relatively low participation (16%) may be partially explained by recruitment from a disease registry as opposed to broader community approaches 21,22,34,35 . However, our response rate is consistent with other published surveys 36 .…”

General Practice and Digital Methods to Recruit Stroke Survivors to a Clinical Mobility Study: Comparative Analysis

“…However, although some studies have reported issues related to representativeness and selection bias [ 34 , 35 ], our data support previous findings from other studies that reported the successful use of SM to recruit comparable and representative samples, as described in the systematic review by Whitaker et al [ 36 ]. Additionally, Yu et al [ 37 ], for example, were able to recruit geographically representative samples of individuals with myeloproliferative neoplasms in the United States for a survey study using multiple recruitment strategies, including Google and Facebook.…”

Comparison of Web‐Based Advertising and a Social Media Platform as Recruitment Tools for Underserved and Hard‐to‐Reach Populations in Rheumatology Clinical Research

“…Up to 50% of studies are terminated early because of inadequate participant accrual (1), with many study locations failing to enroll even a single patient (2). Noninterventional studies can be affected even more severely than studies with therapeutic intent (3), and those involving uncommon diseases may be even more susceptible because of a limited potential population of participants (4). Typical recruitment methods involve coordination with local physicians, printed flyers posted in public spaces, events such as health fairs, and advertising in printed media.…”