Patient involvement in health research: A contribution to a systematic review on the effectiveness of treatments for degenerative ataxias

Serrano-Aguilar, P.; Trujillo-Martín, M.M.; Ramos-Goñi, Juan Manuel; Mahtani-Chugani, Vinita; Perestelo-Pérez, Lilisbeth; Paz, Manuel Posada de la

doi:10.1016/j.socscimed.2009.07.005

Cited by 66 publications

(83 citation statements)

References 31 publications

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“…All studies were observational; none employed experimental or quasi-experimental designs. Eleven studies used qualitative or survey methods to obtain input from patients, 4,7,[18][19][20][21][22][23][24][25][26] and 17 studies were narrative reports describing one group's experiences with engagement. [27][28][29][30][31][32][33][34][35][36][37][38][39][40][41][42][43] The remaining seven studies were descriptions of specific initiatives reported on websites [44][45][46][47][48][49][50] ( Table 1 and Table 2).…”

Section: Resultsmentioning

confidence: 99%

“…cians. 20,38,39,47,49 Seven studies were classified as sufficiently descriptive 7,18,19,[23][24][25][26] The purposes of engagement included identifying patient-centered research topics or agendas, 19,23,29,36,39 identifying outcomes important to patients or developing measures relevant to patients' needs, 7,19,20,23,25,26 increasing recruitment or enrollment through development of patient-centered study designs, 4,18,21,24,27,30,31,41 and incorporating the patient perspective into study design 19,25,35,39,40 (Table 2, Table 3). Patients and other stakeholders were most commonly engaged in the preparatory stage (n= 19 for agenda setting) 4, 7, 19, 20, 23, 25, 28, 29, 32, 35, 36, 39- (Table 4 [online]).…”

Section: Resultsmentioning

confidence: 99%

“…Five studies used either qualitative or survey methods to obtain information directly from patients about the outcomes that were important to them. 7,19,23,25,26 These reports permitted rankings of symptoms by importance 7,25 and also clarified how patients describe symptoms. 7,19,23 For example, due to patient input, these studies included measures of pain, stiffness, sleep, motor function, quality of life, and the economic impact of disease.…”

Section: Approaches For Patient and Other Stakeholder Inputmentioning

confidence: 97%

“…18,31 Third, several authors expressed concerns about possible bias among patients and patient representatives chosen for engagement due to higher motivation, high educational levels, methods of recruitment (e.g., via internet or annual meetings) or possible conflicts of interest (e.g., relationships between patient organizations and industry groups). 23,25,27,30,33,43 Other challenges identified include lack of researcher and patient experience in or training for engagement, 25,32 an absence of policies facilitating engagement, 25 the potential for both researchers and patient partners to undervalue engagement, 25 ethical concerns (e.g., patient rights 43 ), and the possibility of uncoordinated efforts by competing patient-led groups. 43 …”

Section: Approaches For Patient and Other Stakeholder Inputmentioning

confidence: 99%

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A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

et al. 2014

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BACKGROUND: Patients with rare diseases have limited access to useful information to guide treatment decisions. Engagement of patients and other stakeholders in clinical research may help to ensure that research efforts in rare diseases address relevant clinical questions and patient-centered health outcomes. Rare disease organizations may provide an effective means to facilitate patient engagement in research. However, the effectiveness of patient-engagement approaches, particularly for the study of rare diseases, has not been well studied. OBJECTIVES: To synthesize evidence about engagement of patients and other stakeholders in research on rare diseases, including the role of rare disease organizations in facilitating patient-centered research. METHODS/RESEARCH DESIGN: A systematic review and gray literature search were guided by a technical expert panel composed of patient representatives, clinicians, and researchers. English-language studies that engaged patients or other stakeholders in research on rare diseases or evaluated engagement were included. Studies were assessed on how well key research questions were answered, based on the level of detail describing engagement activities and whether outcomes from engagement were assessed. RESULTS: Thirty-five studies were included, although many reported minimal information on engagement. Patients and other stakeholders were most commonly engaged to identify patient-centered research agendas, to select which study outcomes were important to patients, to provide input on study design, and to identify strategies for increasing enrollment in trials. Rare disease organizations mainly helped provide access to patients and communicated research opportunities and findings. They also helped promote collaborative networks and provided financial support for research infrastructures. Although authors reported benefits of engagement and identified changes to their research processes, no empirical assessments of engagement practices and their effectiveness were found. CONCLUSIONS: Researchers studying rare diseases can obtain patient input regarding which research questions and health outcomes to study; however, the most effective approaches to engagement have not been well defined.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Approaches For Patient and Other Stakeholder Inputmentioning

confidence: 97%

Section: Approaches For Patient and Other Stakeholder Inputmentioning

confidence: 99%

See 2 more Smart Citations

A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

et al. 2014

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“…Future trials should be designed to address these needs. Since some of the patients´ self-perceived degenerative ataxia-related health problems have never been investigated so far [17], they should be considered to improve future research projects and adapt them to the patients' needs.…”

Section: Discussionmentioning

confidence: 99%

Appropriateness of Treatments for Patients With Degenerative Ataxias: Recommendations by a Panel of Experts

Trujillo-Martin

2012

J Neurol Res

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Is The Cochrane Collaboration Prepared for the Era of Patient-Centred Outcomes Research?

Gartlehner

Flamm

2013

Cochrane Database of Systematic Reviews

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Patient involvement in health research: A contribution to a systematic review on the effectiveness of treatments for degenerative ataxias

Cited by 66 publications

References 31 publications

A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

A Systematic Review of Approaches for Engaging Patients for Research on Rare Diseases

Appropriateness of Treatments for Patients With Degenerative Ataxias: Recommendations by a Panel of Experts

Is The Cochrane Collaboration Prepared for the Era of Patient-Centred Outcomes Research?

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