EARLY ENGAGEMENT OF THE ‘PATIENT VOICE’ TO INFORM SELECTION OF OUTCOME MEASURES IN RARE DISEASE: AN EXAMPLE FROM A SURVEY STUDY IN ACQUIRED THROMBOTIC THROMBOCYTOPENIC PURPURA (ATTP)
Author(s): ,
Stephen John Holmes
Affiliations:
Medical Affairs,Sanofi Genzyme,Reading,United Kingdom
,
Ewelina Rzepa
Affiliations:
OPEN Health Communications,Marlow,United Kingdom
Fleur Chandler
Affiliations:
Health Outcomes,Sanofi Genzyme,Reading,United Kingdom
EHA Library. John Holmes S. 06/12/20; 298389; PB2477
Mr. Stephen John Holmes
Mr. Stephen John Holmes
Contributions
Abstract

Abstract: PB2477

Type: Publication Only

Background
The collection of patient-reported outcomes (PROs) is central to understanding the true impact of illness from the patient perspective, e.g. according to quality of life (QoL). In rare diseases, selection of relevant PROs is particularly complex, due to the variability and heterogeneity of illnesses (1,2). Here, we describe the methods used to inform the selection of PROs for a non-interventional real-world study in aTTP.

Aims
Define appropriate outcome assessments in aTTP from the patient perspective

Methods
A 4-step process was followed: 1) a targeted review of published literature in aTTP; 2) elicitation of relevant concepts identified as important by patient support group members; 3) concept mapping to select relevant PROs and concepts measured; 4) further validation with patients of the selected instruments and  developed bespoke questions.

Results

The literature review revealed a lack of concept elicitation studies, with just one published registry study of patients with TTP which reported QoL data according to the generic 36-item Short Form Survey (SF-36) (3). Discussion with the patient group confirmed that some concepts from the SF-36 are relevant (e.g. shopping, cleaning, playing with children) to aTTP. Further concepts, such as “worries about travelling”, “inability to return to work”, “memory and concentration problems”, and “flashbacks” were also identified as important. Conceptual mapping showed that some concepts (e.g. “memory and concentration problems”) could be addressed by the PRO Measurement Information System (PROMIS) - Cognitive Function Abilities - Short Form 6a, and other concepts (employment status, absenteeism due to health issues and perceived productivity in the past week) measured according to the Work Productivity and Activity Index (4), and a set of bespoke questions.

Three individual patients were asked to ratify the selected PRO instruments and bespoke questions. The instruments selected were described as “easy to understand” and relevant to aTTP, although the total number of items was considered burdensome for individuals early in their diagnosis.

Conclusion

Early patient engagement allowed for incorporation of the patient perspective to identify and validate a disease-relevant PRO measurement strategy.  Hearing direct from patients about how to measure concepts important to them is a valuable step for ensuring patient-centric data collection for a real-world outcomes study.

 

1.            Slade A, Isa F, Kyte D, Pankhurst T, Kerecuk L, Ferguson J, et al. Patient reported outcome measures in rare diseases: a narrative review. Orphanet Journal of Rare Diseases 13, 61 (2018).

2.            Benjamin K, Vernon MK, Patrick DL, Perfetto E, Nestler-Parr S, Burke L. Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report. Value in Health. 2017 Jul;20(7):838–55.

3.            Lewis QF, Lanneau MS, Mathias SD, Terrell DR, Vesely SK, George JN. Long-term deficits in health-related quality of life after recovery from thrombotic thrombocytopenic purpura. Transfusion. 2009 Jan 1;49(1):118–24.

4.            Reilly MC, Zbrozek AS, Dukes EM. The validity and reproducibility of a work productivity and activity impairment instrument. PharmacoEconomics 1993; 4(5):353-65

Session topic: 35. Quality of life, palliative & supportive care, ethics and health economics

Keyword(s): Outcome, Thrombotic thrombocytopenic purpura (TTP)

Abstract: PB2477

Type: Publication Only

Background
The collection of patient-reported outcomes (PROs) is central to understanding the true impact of illness from the patient perspective, e.g. according to quality of life (QoL). In rare diseases, selection of relevant PROs is particularly complex, due to the variability and heterogeneity of illnesses (1,2). Here, we describe the methods used to inform the selection of PROs for a non-interventional real-world study in aTTP.

Aims
Define appropriate outcome assessments in aTTP from the patient perspective

Methods
A 4-step process was followed: 1) a targeted review of published literature in aTTP; 2) elicitation of relevant concepts identified as important by patient support group members; 3) concept mapping to select relevant PROs and concepts measured; 4) further validation with patients of the selected instruments and  developed bespoke questions.

Results

The literature review revealed a lack of concept elicitation studies, with just one published registry study of patients with TTP which reported QoL data according to the generic 36-item Short Form Survey (SF-36) (3). Discussion with the patient group confirmed that some concepts from the SF-36 are relevant (e.g. shopping, cleaning, playing with children) to aTTP. Further concepts, such as “worries about travelling”, “inability to return to work”, “memory and concentration problems”, and “flashbacks” were also identified as important. Conceptual mapping showed that some concepts (e.g. “memory and concentration problems”) could be addressed by the PRO Measurement Information System (PROMIS) - Cognitive Function Abilities - Short Form 6a, and other concepts (employment status, absenteeism due to health issues and perceived productivity in the past week) measured according to the Work Productivity and Activity Index (4), and a set of bespoke questions.

Three individual patients were asked to ratify the selected PRO instruments and bespoke questions. The instruments selected were described as “easy to understand” and relevant to aTTP, although the total number of items was considered burdensome for individuals early in their diagnosis.

Conclusion

Early patient engagement allowed for incorporation of the patient perspective to identify and validate a disease-relevant PRO measurement strategy.  Hearing direct from patients about how to measure concepts important to them is a valuable step for ensuring patient-centric data collection for a real-world outcomes study.

 

1.            Slade A, Isa F, Kyte D, Pankhurst T, Kerecuk L, Ferguson J, et al. Patient reported outcome measures in rare diseases: a narrative review. Orphanet Journal of Rare Diseases 13, 61 (2018).

2.            Benjamin K, Vernon MK, Patrick DL, Perfetto E, Nestler-Parr S, Burke L. Patient-Reported Outcome and Observer-Reported Outcome Assessment in Rare Disease Clinical Trials: An ISPOR COA Emerging Good Practices Task Force Report. Value in Health. 2017 Jul;20(7):838–55.

3.            Lewis QF, Lanneau MS, Mathias SD, Terrell DR, Vesely SK, George JN. Long-term deficits in health-related quality of life after recovery from thrombotic thrombocytopenic purpura. Transfusion. 2009 Jan 1;49(1):118–24.

4.            Reilly MC, Zbrozek AS, Dukes EM. The validity and reproducibility of a work productivity and activity impairment instrument. PharmacoEconomics 1993; 4(5):353-65

Session topic: 35. Quality of life, palliative & supportive care, ethics and health economics

Keyword(s): Outcome, Thrombotic thrombocytopenic purpura (TTP)

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