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Partnering With Patients to Rapidly Develop a Quality-of-Life Measure in Mycosis Fungoides/Sézary Syndrome Type Cutaneous T-cell Lymphoma

Paul Wicks, PhD; Marjan Sepassi, PharmD; Gaurav Sharma, PharmD; and Margot Carlson Delogne
The field of measurement in Mycosis Fungoides/Sézary syndrome type cutaneous T-cell lymphoma lacked a specific quality-of-life measure to describe patient experience or guide treatment decisions. Using an online platform with an engaged patient community, we developed and psychometrically validated a new measure in just under a year.
Developing the Instrument

While a full description of the study methods is beyond the scope of this article (for more, see Towers, et al33), we will summarize the approach taken. Following a literature review and interviews with clinical experts, 21 patients reporting a physician-confirmed diagnosis of MF/SS CTCL were invited to complete open-text concept elicitation items (eg, “How is your physical well-being affected by MF/SS CTCL?”). In addition, 10 patients out of the 21 were invited for further telephone interview to probe their answers. Qualitative analysis suggested that content saturation was reached after 15 patients and thematic content analysis identified 6 major themes that were subsequently used to generate a preliminary version of the 31-item questionnaire. This long-form questionnaire was administered to 42 patients for their item-level feedback, where multiple-choice and open text survey items allowed patients to identify issues with any items they found lacked clarity or relevance. After removing redundant items, an abbreviated 14-item scale was administered to a sample of 126 patients for psychometric validation, including comparator measures. Sixty-six patients completed test-retest performance, 5 days later, with known-group validity analysis.

Psychometric analysis showed good internal consistency, test-retest reliability, and that the items were appropriately ordered in terms of severity. We also found that the MF/SS CTCL QoL is capable of discriminating between individuals with low and high levels of interference in their QoL, and that the items adequately covered the varying levels of interference with QoL due to MF/SS CTCL. Further work detailing the validation process and final instrument is under preparation.
The use of the ORE platform enabled patient feedback to be easily incorporated throughout every step of the development process. Additionally, thanks in large part to a relationship with the Cutaneous Lymphoma Foundation, recruitment was faster and more cost effective than traditional methods of sourcing patients through clinical centers. Information collected from key opinion leaders and the literature helped to inform a scientifically grounded and relevant measure that accurately depicts the patient experience with MF/SS CTCL, particularly given the variability that patients often encounter, as they cope with the daily demands of the condition. Online feedback replaced more time and resource-expensive interviewer-led cognitive debriefing interviews. In all, the process took less than a year, and the total cost was a fraction of what traditional instrument development costs.

The greatest limitation of the study is the sample size, due in part to the rarity of the condition. A number of psychometric validation measures were, therefore, unable to be completed and could be addressed in future research. Additionally, while it is impossible to guarantee that those who registered on the website actually have MF/SS CTCL, a recent study in other conditions, on PatientsLikeMe, found that around 95% of patients could be identified via IMS Health medical and pharmacy claims (Eicher et al, In Press). Further, the sample of patients who participated in this study were largely in Stage 1 of mycosis fungoides, or Sézary syndrome, with few patients reporting more advanced stages of the syndrome (Stage III,  n = 2; Stage IVa, n = 4; Stage IVb, n = 1). Inclusion of more individuals at later stages of the syndrome would allow for empirical testing of differential item function by later stages of the syndrome.

The MF/SS-CTCL PRO tool project represented a unique collaboration between a pharmaceutical company and a patient network, using an innovative platform to work on a new measure for a rare disease that would otherwise have never been developed. We hope use of the MF/SS CTCL-QoL tool, which is freely available, will enable better communications about QoL between patients and their care teams, and improve and inform clinical management and treatment decision making related to MF/SS-CTCL. EBO

Author information
Paul Wicks, PhD, is vice president of innovation, PatientsLikeMe, Inc.
Marjan Sepassi, PharmD, is associate director, Medical Affairs, Actelion Pharmaceuticals, Inc. 
Gaurav Sharma, PharmD, is associate director, Medical Affairs, Actelion Pharmaceuticals, Inc.
Margot Carlson Delogne, is vice president of communications, PatientsLikeMe, Inc.
Address for correspondence
Paul Wicks, PhD
160 2nd Street,
Cambridge, MA
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