11606_2014_2895_MOESM1_ESM.docx - static …10.1007... · Web view2. patient centered care/ or...

Table 4. Ways in which patients and other stakeholders were engaged

Study Engaged stakeholder(s) Identification and selection of patients and other stakeholder(s)

Roles of patients, patient organizations, and other stakeholder(s)

Engagement training for patients and/or researchers

Bedgood, 2007(27)

Patients, disease-specific patient organization (Achalasia Support Group (ASG))

Disease-specific patient organization (ASG)

Patient organization provided access to the online support group members.

NR

Boon, 2010(28) Disease-specific patient organization (Dutch neuromuscular disease association (VSN)

NA Patient organization established networks of researchers and stakeholders in order to influence research agendas, conveyed patient wishes to these groups, and assisted with and imitated clinical trials.

NR

Bruinsma, 2011(29)

Disease-specific patient organization (Tall Girls Inc)

NR Patient organization petitioned the government for a follow-up study after receiving controversial treatment, helped to recruit participants to retrospective cohort study of girls with tall stature, provided input on study measures.

NR

Carroll, 2012(18) Patients Clinics Patients provided feedback through interviews. NR

Cystic Fibrosis Foundation, Therapeutics Development Network(47)

Patients, families, clinicians

NR Patient organization drives the provision of access to information about ongoing clinical trials for patients and families to enroll in. Additionally, patients and clinicians serve on a protocol review committee to evaluate a sponsor/ industry protocol for research.

NR

de Blieck, 2013(30)

Disease-specific patient organization (The Batten Disease Support and Research Association (BDSRA))

NR Patient organization provided funding to launch a disease registry, supported enrollment in the registry (e.g., through recruitment at the group’s annual meeting).

NR

DeWard, 2013(31) Patient organizations NR Patient organizations helped to identify and recruit patients.

NR

Edwards, 2011(19) Disease-specific organization, parents

Disease-specific patient organization

Parents provided feedback through semi-structured interviews; patient organization recruited parents for the interviews and reviewed the supporting materials that explained scientific concepts for the interviewees.

NR

Eleftheriadou, 2012(20)

Patients, clinicians NR Patients and clinicians provided feedback through surveys.

NR

1




EMA (European Medicines Committees), Fifth report on the interaction with patients' and consumers' organizations (2011)(33)

Patients, patient organizations

NR Patient representatives participated as experts on scientific committees, specifically the Committee for Orphan Medicinal Products, with the following roles: assess applications for rare disease drug designations, identify external experts to review applications, collaborate on preparation of public summaries of opinion for rare disease designations.

NR

EMA (European Medicines Committee), The role of patients as members of the EMA Human Scientific Committees(32)

Patients, parents/ family/friends, patient organizations

NR EMA supports the presence of patient representatives on the Committee for Orphan Medicinal Products in order to bring the patient perspective to the discussions of the committee.

NR

EUPATI (European Patients’ Academy on Therapeutic Innovation)(44)

NA NA *EUPATI attempts to (among other things) increase the capacity of patients and the lay public to be effective advocates and advisors in clinical trials, with regulatory authorities, and in ethics committees, and to provide tools and knowledge to empower meaningful involvement of patients in pharmaceutical innovation.

NR

EURORDIS, Activity Report 2012 & Work plan 2013(34)

NA NA *Patients and their representatives serve on EMA scientific committees to provide input regarding the most relevant outcome measures and endpoints for clinical trials.

EURORDIS promotes engagement of patients in research and on ethical committees, provides input to international policy-making groups to ensure the involvement of patients in decision-making processes, provides training for patients regarding clinical research, and contributes to the promotion and maintenance of rare diseases as a priority in European research policy and funding schemes. It informs, educates, and raises awareness about rare diseases.

NA

2




Fern, 2013(21) Patients NR Patients developed the study invitation, helped develop interview guide, conducted interviews, and reviewed researcher findings.

Trained in research methods (provided by the study) through formal training, patient and professional cancer research conferences, and in-house sessions.

Landy, 2012(4) Non-specific patient organizations

NR Patient organizations reported involvement in recruiting research subjects, collecting data, supporting a registry or biobank, preparing research reports, analyzing data, disseminating findings, writing funded proposals to initiate their own projects, and providing financial support to researchers.

NR

Langston, 2005(35)

Disease-specific organization, patients, caregivers

Disease-specific patient organization

Patient organization representatives reviewed trial protocol, advised on how to present the study to lay audiences, marketed the study to clinicians at professional meetings and to potential patient participants (via newsletter, patient days, and information sessions); patients provided feedback on patient information leaflets and trial conduct (e.g., language used with participants).

NR

The Lupus Research Institute, A patient’s voice in lupus research(48)

Patients A patient applied to review proposals.

Patient representative nominated by the disease-specific patient organization to serve as a reviewer for research proposals

Three to five full days of intense pre-proposal discussion meeting preparation.

Mai, 2012(36) Parents/Family NR Family members of children with Li-Fraumeni syndrome participated in a workshop.

NR

Marshall, 2004(37)

Disease-specific patient organization (PXE International)

NR Patient organization representative helped with DNA extraction, lab tests, and manuscript writing. This representative was involved at a level significant enough to merit inclusion as an inventor on the patent for a genetic test. The organization helps to mobilize support for scientific studies on an international scale.

NR

3




Mavris, 2012(22) Non-disease-specific patient organization (EURORDIS, European Organization for Rare Diseases), other patient organizations

NA *Patient organizations provide financial support to research, provide links between patients and researchers, identify patients for clinical trials, and provide input on trial design.

NR

McCormack, 2013(38)

Patients, parents, patient organizations, clinicians, legal academics, sociologists, and ethicists.

NR Patients and other stakeholders participated on an ethics committee to provide advice on a variety of issues related to the study of neuromuscular disorders (e.g., ethical and legal governance of patient registry data).

NR

Mease, 2007(7) Patients NR Patients provided feedback through focus groups and a Delphi exercise.

NR

Molster, 2012(39) Patients, parents, patient support groups, clinicians, funders, industry, policy makers, regulators

NR Patients and other stakeholders participated in multiple workshop discussions.

NR

Nierse, 2010(23) Patients, parents Disease-specific patient organization (Dutch Patient Association for neuromuscular disorders)

Patients participated in interviews, focus groups, expert meetings, a questionnaire, and a dialogue meeting to identify and prioritize research topics.

NR

Ntshanga, 2010(40)

Community members, community leaders, religious leaders, traditional healers, school principals, community health workers

NR The Community Advisory Board (CAB) is tasked with ensuring that research is sensitive to cultural and traditional practices, preparing the community for participation in clinical trials, alerting researchers to community concerns, and informing the community about research findings.

Community representatives attended a 2-day workshop to provide basic knowledge about TB and research on TB.

Orphanet, Researchers' wish-list for rare disease research in Europe(45)

Patients NR Patient representatives participated in a multi-stakeholder workshop (including biopharmaceutical industry representatives, other government agencies, etc.) to provide specific recommendations for optimizing rare disease research in Europe (size and type of projects, infrastructure needs and scientific priorities).

NR

4




The Parkinson Pipeline Project(49)

Patients, clinicians treating Parkinson’s Disease patients

NR Patients and clinicians serve as consultants in the pre-approval, clinical trial phase of Parkinson’s drug development to advise the FDA and drug sponsors on topics such as clinical trial design, endpoint determination, protocol development, and clinical trial patient-recruitment strategies.

Rare-disease organizations drive and promote policies that allow for provision of the patient perspectives to industry sponsors and investors, clinical scientists, and government regulation agencies; aim to include patients’ voices in decision making at all levels of the clinical trial process.

NR

Rare disease matters, Patient-initiated research foundations(46)

Patient organizations NR Patient organizations drive the provision of fundraising tools for patients to raise money for research.

NR

Rothwell, 2013(24)

Parents Professional agency in two states recruited parents from the general public

Parents provided input through focus groups about a pilot newborn screening study for spinal muscular atrophy.

Patients received an informational video.

Schwartz, 2013(41)

Disease-specific patient organization (PC Project)

NR Patient organization created connections between researchers, physicians, and patients, provides access for patients to ongoing research projects, disseminates research findings, and developed collaborations with industry groups to initiate clinical trials.

NR

5




Seminara, 2010(42)

Non-disease-specific patient organization (Coalition of Patient Advocacy Groups),Disease-specific group (National Urea Cycle Disorders Foundation)

NR Representatives of the patient organization joined a steering committee of the Rare Diseases Clinical Research Network, which develops policies, procedures, and standards for data collection. A representative from the patient organization also served on the executive committee of the Urea Cycle Disorders Consortium to help with study enrollment, protocol development, content evaluations, progress reporting, and website content. The patient organization also designed and fielded a survey about study participation and shared information about consortia research projects with its membership via the web and newsletters.

NR

Serrano-Aguilar, 2009(25)

Patients Patient organizations Patients provided input through a Delphi exercise. NR

von Hippel-Lindau Alliance (VHLA)(50)

Patients, families NR A patient-driven registry will be launched in November 2013: Cancer in Our Genes International Patient Database (CGIP) for VHL (plus HLRCC and BHD).

Patients and families are members of the VHLA strategic planning committee that is developing a blueprint to expand current programmatic efforts and provides direction for the organization.

NR

Workman, Engaging patients in information sharing and data collection: The role of patient-powered registries and research networks prepared for AHRQ(43)

Patients, families, and patient organizations

NA Patients, family members, and representatives from patient organizations drive, manage, and/or control the research agenda, data collection, and/or translation and dissemination findings for patient-powered registries or research networks.

Rare-disease organizations initiate and run registries or research networks

NR

Wu, 2012(26) Patients, Parents NR Patients and their parents provided feedback through a focus group.

NR

6

NR= Not reported, such that information was relevant to the included study but was not described in the publication/website; NA= not applicable, such that the information was not relevant for the specific type of study included. * Study described engagement or roles of patient organizations but did not report on engagement conducted directly by authors.

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Table 5. Impact of engagement in research on rare diseases on the design, conduct, dissemination, and relevance of the research

Study name Patient engagement leading to changes in the research

Engagement increasing the number of patients invited to participate

Engagement reducing study attrition

Engagement leading to patient-reported measure or patient-reported outcome measure adopted in research

Bedgood, 2007(27) NR NR NR NRBoon, 2010(28) Patient organization

cofounded collaborative research networks.

NA NA NA

Bruinsma, 2011(29) Authors reported that engagement resulted in improved study design and acceptance of study findings.

NR NR NR

Carroll, 2012(18) NR NR NR NR

Cystic Fibrosis Foundation, Therapeutics Development Network(47)

NR NR NR NR

de Blieck, 2013(30) Engagement supported recruitment.

Authors suggest that the patient organization was critical for recruitment.

NR NR

DeWard, 2013(31) NR NR NR NR

Edwards, 2011(19) The trial design and outcomes measured were identified based on input from parents of patients.

Authors reported high rates of recruitment relative to similar studies.

Retention in the trial was excellent with outcome data on 94% and only nine children withdrawing⁄ lost to follow-up.

Parents suggested a range of factors relevant to their child’s quality of life.

Eleftheriadou, 2012(20)

Based on patients’ feedback, the authors recommended some outcomes to be included in future research about vitiligo (e.g., cosmetically acceptable repigmentation)

NR NR NA- one purpose of engagement was to inform future studies by other investigators.

8





EMA (European Medicines Committees), Fifth report on the interaction with patients' and consumers' organizations (2011)(33)

NR NR NR NR

EMA (European Medicines Committee), The role of patients as members of the EMA Human Scientific Committees(32)

NR NR NR NR

EUPATI, EUPATI (European Patients’ Academy on Therapeutic Innovation)(44)

NA NA NA NA

EURORDIS, Activity Report 2012 & Work plan 2013(34)

NA NA NA NA

Fern, 2013(21) Engagement helped to develop interview guide questions. Authors reported receiving more in-depth interview responses from young adults via patient interviewers compared to researcher interviewers.

NR NR NA

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Landy,2012(4) NR Patient organizations responding to a survey about engagement efforts felt that engagement increased study participation rates.

NR NR

Langston, 2005(35) Changes were made in language used to communicate with participants, new study sites were recruited through the patient organization, patient requests to participate as a subject were received.

Yes High response rates (98%) to follow-up quality-of-life assessments.

NR

The Lupus Research Institute, A patient’s voice in lupus research(48)

Engagement helped research directed toward clinically relevant issues

NR NR NR

Mai, 2012(36) NR NR NR NR

Marshall, 2004(37) NR NA NA NA

Mavris, 2012(22) NR NR NR NR

McCormack, 2013(38)

NA NA NA NA

Mease, 2007(7) NR NA NA NA

Molster, 2012(39) Patients identified an outcome important to study that was not identified by clinician-investigators.

NA NA NA- purpose of engagement was to inform future studies by other investigators

Nierse, 2010(23) NR NA NA NA

Ntshanga, 2010(40) A research agenda shared by patients, clinicians, and researchers was identified.

NR NR NR

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Orphanet, Researchers' wish-list for rare disease research in Europe(45)

NR NR NR NR

The Parkinson Pipeline Project(49)

NR NR NR NR

Rare disease matters, Patient-initiated research foundations(46)

NR NR NR NR

Rothwell, 2013(24) NR NR NR NR

Schwartz, 2013(41) NR (parents engaged in study approved the approach proposed by the research team)

NR NR NR

Seminara, 2010(42) New projects were initiated. NR NR NRSerrano-Aguilar, 2009(25)

Authors report that patient organization representatives provided greater access to patients and made significant contributions to protocol development, recruitment, website content, and other aspects of their work.

NR NR NR

von Hippel-Lindau Alliance (VHLA)(50)

Authors report that the patient engagement enhanced the design and conduct of the systematic literature review.

NA NA NA

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Workman, Engaging patients in information sharing and data collection: The role of patient-powered registries and research networks prepared for AHRQ(43)

NR NR NR NR

Wu, 2012(26) NA NA NA NA

Bedgood, 2007(27) A new module was developed for a quality-of-life tool to capture additional domains (the socioeconomic consequences of hemophilia).

NA NA A new module was developed for a quality-of-life tool to be used in future studies of hemophilia.

NR= Not reported, such that information was relevant to the included study but was not described in the publication/website; NA= not applicable, such that the information was not relevant for the specific type of study included.

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Search Strategy

The specific terms used for the search are listed by database below. As recommended by the Cochrane Handbook for Systematic Reviews of Interventions (section 6.6.2.2), the searches are reported exactly as run with line numbers for each search set.

MEDLINE/EMBASE

1. patient participation/ or consumer participation/ or patient advocacy/ or consumer advocacy/

2. patient centered care/ or ((patient* or consumer* or stakeholder* or user* or lay* or client* or

citizen* or communit* or public or advoca* or carer* or caregiver* or surrogate* or parent* or relative)

adj2 (important or perspective or centered or centred or participa* or collaborat* or partner* or voice*

or unvoiced)).mp. [mp=title, abstract, original title, name of substance word, subject heading word,

keyword heading word, protocol supplementary concept, rare disease supplementary concept, unique

identifier]

3. ((patient* or consumer* or stakeholder* or user* or lay* or client* or citizen* or communit* or public

or advoca* or carer* or caregiver* or surrogate* or parent* or relative) adj2 (involv* or represent* or

consult* or contribut* or engage* or activat* or opinion* or dialog* or partner* or input*)).mp.

[mp=title, abstract, original title, name of substance word, subject heading word, keyword heading

word, protocol supplementary concept, rare disease supplementary concept, unique identifier]

4. or/1-3

5. exp evaluation studies as topic/ or exp methods/

6. 4 and 5

7. 6 and (outcome*.mp. or quality of life/ or patient preferences/ or risk assessment/ or patient

satisfaction/) [mp=title, abstract, original title, name of substance word, subject heading word, keyword

heading word, protocol supplementary concept, rare disease supplementary concept, unique identifier]

8. 6 and ((utilities or values or empiric* or feedback* or communication*).mp. or health priorities/ or

research priorities/)

9. 6 and exp clinical trials as topic/

10. 6 and (physician-patient relations/ or patient acceptance of health care/)

11. health services research/ or needs assessment/

13

12. 6 and 11

13. 6 and agenda*.mp. [mp=title, abstract, original title, name of substance word, subject heading word,

keyword heading word, protocol supplementary concept, rare disease supplementary concept, unique

identifier]

14. 4 and *research design/ and (choice behavior/ or cooperative behavior/)

15. 6 and (choice behavior/ or cooperative behavior/)

16. 7 or 8 or 10 or 12 or 13 or 15

17. 16 and ((panel* or jury or juries or forum).mp. or qualitative research/ or interview*.mp.)

18. 16 and (recruit* or participat* or "focus group*" or instrument* or scale* or questionnaire* or

consultant* or questionnaire* or survey* or interview* or "nominal group" or delphi*).mp. [mp=title,

abstract, original title, name of substance word, subject heading word, keyword heading word, protocol

supplementary concept, rare disease supplementary concept, unique identifier]

19. 17 or 18

20. 16 and observation*.mp. [mp=title, abstract, original title, name of substance word, subject heading

word, keyword heading word, protocol supplementary concept, rare disease supplementary concept,

unique identifier]

21. (19 or 20) and exp clinical trial as topic/

22. 19 or 20

23. limit 22 to (consensus development conference or consensus development conference, nih or

multicenter study or "research support, american recovery and reinvestment act" or research support,

nih, extramural or research support, nih, intramural or research support, non us gov't or research

support, us gov't, non phs or research support, us gov't, phs)

24. 22 and (*patient satisfaction/ or *consumer satisfaction/ or *patient-center care/ or *patient

preferences/)

25. 22 and (technology assessment, biomedical/ or community-based participatory research/ or px.fs.)

26. 21 or 24 or 25

27. 23 and 2

14

28. 26 or 27

29. limit 28 to (comment or editorial or interview or introductory journal article or legislation or letter or

news or newspaper article or patient education handout or retracted publication or "retraction of

publication")

30. 28 not 29

31. limit 30 to humans

32. 31 not animals/

33. ((patient* or consumer* or stakeholder* or user* or lay* or client* or citizen* or communit* or

public or advoca* or carer* or caregiver* or surrogate* or parent* or relative) adj2 (important or

perspective or centered or centred or participa* or collaborat* or partner* or voice* or unvoiced)).ti,ab.

34. ((patient* or consumer* or stakeholder* or user* or lay* or client* or citizen* or communit* or

public or advoca* or carer* or caregiver* or surrogate* or parent* or relative) adj2 (involv* or

represent* or consult* or contribut* or engage* or activat* or opinion* or dialog* or partner* or

input*)).ti,ab.

35. (24 or 33 or 34) and 32

EBM Reviews - Cochrane Methodology Register

1 (participat* adj2 research).mp. [mp=title, abstract, subject heading word] 141 Advanced

2 "CMR: Evaluation methodology - patient involvement".kw. 521 Advanced

3 "CMR: Other methodology - patient based outcome measures".kw. 252 Advanced

4 (design* or planning or priorit* or agenda* or participat*or decision*).mp. and (2 or 3) [mp=title, abstract, subject heading word] 244 Advanced

5 (2 or 3) and (perspective* or preference*).mp. [mp=title, abstract, subject heading word] 128 Advanced

6 5 not 4 78

PsycINFO

1 client participation/ 1087 Advanced

15

2 experimentation/ or exp consumer research/ or exp interdisciplinary research/ or exp qualitative research/ or exp experimental design/ or exp methodology/ 147651 Advanced

3 1 and 2 128 Advanced

4 1 and (priorit* or participatory or engage* or planning or design* or perspective* or preference*).mp. [mp=title, abstract, heading word, table of contents, key concepts, original title, tests & measures] 594 Advanced

5 3 or 4 655 Advanced

6 limit 5 to (all journals and human) 545 Advanced

7 1 and (agenda* or involve*).mp. [mp=title, abstract, heading word, table of contents, key concepts, original title, tests & measures] 413 Advanced

8 limit 7 to (all journals and human) 336 Advanced


10 9 and outcome*.mp. [mp=title, abstract, heading word, table of contents, key concepts, original title, tests & measures] 155 Advanced

11 9 and (2 or methodol*.mp.) [mp=title, abstract, heading word, table of contents, key concepts, original title, tests & measures] 124 Advanced


13 *client participation/ and 12 202

Same limits

S11 S8 and S9 Limiters - Exclude MEDLINE records

Search modes - Boolean/Phrase

View Results (224) .View Details .Edit .Interface - EBSCOhost

Search Screen - Advanced Search

Database - CINAHL .

S10 S8 and S9 Search modes - Boolean/Phrase



Database - CINAHL .

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S9 method* Search modes - Boolean/Phrase



Database - CINAHL .

S8 S2 AND (S6 OR S7) Search modes - Boolean/Phrase



Database - CINAHL .

S7 (MH "Study Design+") Search modes - Boolean/Phrase



Database - CINAHL .

S6 (MH "Research+") OR (MH "Behavioral Research") OR (MH "Medical Practice, Research-Based") Search modes - Boolean/Phrase



Database - CINAHL .

S5 S2 and S4 Search modes - Boolean/Phrase



Database - CINAHL .

S4 (MH "Action Research") Search modes - Boolean/Phrase



Database - CINAHL .

S3 (MH "Patient Centered Care") Search modes - Boolean/Phrase

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Database - CINAHL .

S2 (MM "consumer participation" OR "consumer advocacy") Search modes - Boolean/Phrase



Database - CINAHL .

S1 input* OR perspective* OR involve* OR planning OR design* OR outcome* Search modes - Boolean/Phrase

View Results (529181)

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