Guidance Documents Pathway
- 2005: Cochrane Musculoskeletal (CM)Cochrane Musculoskeletal (CM) is made up of health care professionals, researchers and consumer representatives that belong to Cochrane, an international, non-profit organization that aims to help people make well-informed decisions about health care by preparing, maintaining and promoting reviews on the effects of health care treatments. They produce reliable, up-to-date reviews of interventions for the prevention, treatment or rehabilitation of musculoskeletal disorders in the form of systematic reviews. CM is also dedicated to making our systematic reviews available to those interested in treatments for musculoskeletal diseases (dissemination). In 1993, they formed a Consumer Group (CMSG), enlisting patient systematic reviewers to join health care professionals and researchers belonging to Cochrane. Canada musculoskeletal.cochrane.org develops guidance for systemic review authors and for building partnerships in research.Consumer‐driven health care: Building partnerships in research, 2005. Shea B, Santesso N, Qualman A, Heiberg T, Leong A, Judd M, et al. Consumer-driven health care: building partnerships in research. Health Expectations 2005;8(4):352-9.
- 2006: Cochrane Musculoskeletal Consumer Group (CMSG)The CMSG formed in 1993, enlisting patient systematic reviewers to join health care professionals and researchers belonging to Cochrane. “The feedback provides a layperson’s perspective to complement the feedback provided by our clinical experts.”musculoskeletal.cochrane.org develops initial recommendations to aid knowledge translation and exchange between clinicians and consumersSantesso N, Maxwell L, Tugwell PS, Wells GA, O’connor AM, Judd M, Buchbinder R. Knowledge transfer to clinicians and consumers by the Cochrane Musculoskeletal Group. J Rheumatol. 2006 Nov;33(11):2312-8. PMID: 17086612.. These were updated in 2014.
- 2008: Assessment of SpondyloArthritis International SocietyAssessment of SpondyloArthritis International Society is an international group of experts in the field of spondyloarthritis.
asas-group.org/European Alliance of Associations for Rheumatology European League Against Rheumatism (EULAR) aims to reduce the burden of rheumatic diseases on the individual and society and to improve the treatment, prevention, and rehabilitation of musculoskeletal diseases.
eular.org (ASAS/EULAR) collaborated with patients to translate recommendations for the management of ankylosing spondylitis for the first time into a language easily understood by patients.
As a few years have passed since patient engagement in rheumatology began, guidance and recommendations are developed based on lessons learned.
- 2010: Patient Research Partner (PRP): As a result of patient-researcher collaboration with Outcome Measures in Rheumatology (OMERACT) OMERACT is a global community for the development of Core Outcome Sets in the field of rheumatology. Patients participate as research partners in all phases of the research and are equal members in any OMERACT working group.
omeract.org; omeractprpnetwork.orgin 2002, Eueropean Alliance of Associations for Rheumatology European Alliance of Associations for Rheumatology (EULAR) European Alliance of Associations for Rheumatology (EULAR) is the organization which represents the people with arthritis/rheumatism, health professionals (HPR), and scientific societies of rheumatology of all the European nations. The aims of EULAR are to reduce the burden of rheumatic diseases on the individual and society and to improve the treatment, prevention, and rehabilitation of musculoskeletal diseases. To this end, EULAR fosters excellence in education and research in the field of rheumatology. It promotes the translation of research advances into daily care and fights for the recognition of the needs of people with musculoskeletal diseases by the governing bodies in Europe.
eular.org publishes recommendations for patient-researcher collaboration and defines the role of Patient Research Partner (PRP)Patient Research Partner (PRP) is defined as a person with a relevant disease who participates or has participated as an active research team member on an equal basis with professional researchers, thus adding the value of experiential knowledge to a research project.
De Wit M, et al. European League Against Rheumatism recommendations for the inclusion of patient representatives in scientific projects. Annals of the rheumatic diseases 2011;70(5):722-6.
- 2010: The University of the West of England (UWE)The University of the West of England (UWE) rheumatology group is involved in projects of regional, national, and global significance.www.uwe.ac.uk rheumatology group publishes a dedicated paper Patient collaboration in the design of Patient-Reported Outcome Measures: Capturing the Experience of Fatigue in Rheumatoid Arthritis
Nicklin J, et al. Collaboration with patients in the design of patient-reported outcome measures: Capturing the experience of fatigue in rheumatoid arthritis. Arthritis Care & Research 2010;62(11):1552-8. on patient-researcher collaboration in the development of a new patient-reported outcome (PRO) for fatigue in rheumatoid arthritis (BRAF). United Kingdom. Measures were updated in 2017.Bristol rheumatoid arthritis fatigue scales (BRAFS) are free to use for clinical or academic reasons.
- 2011: European Alliance of Associations for Rheumatology (EULAR) EULAR is the organization that represents the people with arthritis/rheumatism, health professionals (HPR), and scientific societies of rheumatology of all the European nations. The aims of EULAR are to reduce the burden of rheumatic diseases on the individual and society and to improve the treatment, prevention, and rehabilitation of musculoskeletal diseases. To this end, EULAR fosters excellence in education and research in the field of rheumatology. It promotes the translation of research advances into daily care and fights for the recognition of the needs of people with musculoskeletal diseases by the governing bodies in Europe.
www.eular.org establishes the network of Patient Research Partners and develops patient engagement Reference Cards and Background document . Europe
- 2012: INVOLVEINVOLVE, Establishment 1996 and funded by the National Institute for Health Research (NIHR) to support active public involvement in National Health Services (NHS) public health and social care research. As a national advisory group, the role of INVOLVE is to bring together expertise, insight, and experience in the field of public involvement in research (PIR), with the aim of advancing it as an essential part of the process by which research is identified, prioritized, designed, conducted and disseminated.
www.invo.org.uk publishes updated briefing notes for researchers . INVOLVE notes some of the content is drawn from earlier editions is substantially different in its content, reflecting the changing environment since the original briefing notes were written in 2004. United Kingdom
As the decade progresses, international efforts to expand the patient voice in rheumatology research continues. As a result, more programs that focus on patient-reported needs, guidance for involving patients in research, and reports that assess patient involvement emerge.
- 2013: Patient-Centered Outcomes Research Institute (PCORI)PCORI is an independent nonprofit, nongovernmental organization and was authorized by Congress in 2010. PCORI is the largest funder of comparative effectiveness research (CER) in the United States with the mandate to improve the quality and relevance of evidence available to help patients, caregivers, clinicians, employers, insurers, and policymakers make better-informed health decisions. In addition to requiring patient and stakeholder engagement in PCORI funded research, PCORI also engages the public through its own institutional activities, such as Merit Review, which includes patients and stakeholders as reviewers of research applications, ensuring dollars spent are aligned with research outcomes that are meaningful to the patient population. www.pcori.org began the novel funding opportunity, Eugene Washington Engagement Awards ProgramThe Eugene Washington PCORI Engagement Award program, named in honor of the first chair of PCORI’s Board of Governors, is intended to bring more patients, caregivers, clinicians, and other healthcare stakeholders into the research process. The goal is to support projects that will build a community better able to participate in patient-centered outcomes research (PCOR) and comparative clinical effectiveness research (CER) and serve as channels to disseminate study results. www.pcori.org/engagement , which supports projects that will build a community better able to participate in patient-centered outcomes research (PCOR) and comparative clinical effectiveness research (CER) and serve as channels to disseminate study results. Example of award: Matching Actions to Needs: What Matters Most to the Arthritis Community? – Arthritis Foundation, United States
- 2014: Canada’s Strategy for Patient-Oriented Research (SPOR)Endorses the active partnership of patients, researchers, health professionals and decision-makers in research so as to build a sustainable and accessible health care system that optimizes the health of Canadian citizens. cihr-irsc.gc.ca publishes A Resource Guide for Research Teams and Networks , a living guide to support researchers, decision-makers and other relevant stakeholders involved in a wide range of SPOR-related activitiesThis resource aims to:
1) Clarify key concepts and terms relevant to involving patients in health research such as what we mean by patients, the public and stakeholders, and the different levels and types of involvement, and the rationales and perceived benefits of patient involvement
2) Describe, at a very high level, the current state of the evidence about patient engagement with respect to the effectiveness of different methods in relation
to process and outcome measures
3) Assemble in one place, a selection of resource documents collected from leading patient engagement organizations around the world to provide basic guidance for researchers about principles and methods for involving patients
in health research.
- 2014: Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA)Group for Research and Assessment of Psoriasis and Psoriatic Arthritis (GRAPPA) is organized exclusively for non-profit, educational, and scientific purposes, specifically to facilitate the sharing of information related to psoriasis and psoriatic arthritis, networking among different medical disciplines that see psoriasis and psoriatic arthritis patients, and to enhance research, diagnosis, and treatment of psoriasis and psoriatic arthritis.
www.grappanetwork.org publishes Patient Participation in Psoriasis and Psoriatic Arthritis Outcome Research report , co-authored by Patient Research Partners (PRPs)Patient Research Partner (PRP) is defined as a person with a relevant disease who participates or has participated as an active research team member on an equal basis with professional researchers, thus adding the value of experiential knowledge to a research project. After this publication, GRAPPA publishes annual patient reports through 2018.
- 2014: Cochrane Musculoskeletal Consumer Group (CMSG)Formed in 1993, enlisting patient systematic reviewers to join health care professionals and researchers belonging to Cochrane. “The feedback provides a layperson’s perspective to complement the feedback provided by our clinical experts.” musculoskeletal.cochrane.org updates recommendations to aid knowledge translation and exchange between clinicians and consumers .
Patient-focused drug development (PFDD) is a systematic approach to help ensure that patients’ experiences, perspectives, needs, and priorities are captured and meaningfully incorporated into drug development and evaluation. The primary goal of patient-focused drug development is to better incorporate the patient’s voice in drug development and evaluation
- 2012: The Food and Drug Administration (FDA)Food and Drug Administration’s (FDA) evaluation of new medicines (United States). During clinical trials, researchers study whether new medicines are safe and effective for patients and whether the medicine’s benefits outweigh the risks.
www.fda.gov/ established the PFDD initiative under the Prescription Drug User Fee Act (PDUFA V)2012, the US President signed into law the Food and Drug Administration Safety and Innovation Act (FDASIA) to include the reauthorization of the Prescription Drug User Fee Act (PDUFA) that provides FDA with the necessary resources to maintain a predictable and efficient review process for human drug and biologic products.
- 2016: The National Psoriasis FoundationNational Psoriasis Foundation (NPF) drives efforts to cure psoriatic disease and to improve the lives of those affected.
www.psoriasis.org a public meeting to hear perspectives from psoriasis patients, caregivers, and other patient representatives on the most significant dermal or skin-related effects of the disease. Publishes Voice of the Patient report . While attendees were not asked to focus on the arthritic component, a large percentage of comments and concerns included those with Psoriatic Arthritis.
- 2017: The Arthritis Foundation
The Arthritis Foundation boldly pursues a cure for America’s #1 cause of disability while championing the fight against arthritis with life-changing resources, science, advocacy, and community connections. www.arthritis.org hosts the first externally ledExternally-led means it was not commissioned by the FDA so nonprofit organization(s) raise funds to host a meeting offsite. Patient-Focused Drug Development (PFDD) meeting for Osteoarthritis and, as a result, publishes OA Voice of the Patient ReportVisit www.arthritis.org to view videos and more information about this PFDD. You can also download the Report HERE .
- 2017: Lupus and Allied Diseases AssociationThe Lupus and Allied Diseases Association, Inc., is an all-volunteer national non-profit dedicated to enhancing quality of life for those impacted by lupus and allied diseases and other conditions of unmet needs by fostering collaboration among stakeholders, promoting unity in the community, and participating in innovative advocacy, awareness and biomedical research initiatives www.ladainc.org , the Lupus Research AllianceThe Lupus Research Alliance is the largest nongovernmental, nonprofit funder of lupus research worldwide. The organization aims to transform treatment while advancing toward a cure by funding the most innovative lupus research; fostering diverse scientific talent; stimulating collaborations; and driving discovery toward better diagnostics, improved treatments and, ultimately, a cure for lupus. www.lupusresearch.org , and the Lupus Foundation of America unite to host an externally-led PFDD meeting for lupus. Publishes Lupus: Patient Voices reportVisit www.lupuspfdd.org to learn more about this PFDD. The full report can be downloaded HERE .
- 2018 – 2019: FDA develops PFDD guidance materialsInformation and documents related to FDA’s development of the methodological PFDD guidance, including public workshops, draft guidance, and hypothetical scenarios, which are all intended to serve as a basis for dialogue.
View guidance materials at:
www.fda.gov to improve efforts to gain patient input and assessing outcomes in regulatory engagement.
- 2019: FasterCuresFasterCures, a center of the Milken Institute works to build a system that is effective, efficient, and driven by a clear vision: working with our partners to build a patient-centric system where science is accelerated, unnecessary barriers are overcome, and lifesaving and life-enhancing treatments get to those who need them as rapidly as possible.
https://milkeninstitute.org publishes a guide for patient advocacy organizations interested in hosting, or are currently planning, an externally led PFDD meeting. The guide includes links to other tools for planning PFDD meetings, including the PFDD Meeting Tracker, the PFDD Readiness Assessment, and the PFDD Community Toolbox.
Patient testimony in the regulatory process expands in the second half of this decade and the Food and Drug Administration (FDA)Food and Drug Administration’s (FDA) evaluation of new medicines (United States). During clinical trials, researchers study whether new medicines are safe and effective for patients and whether the medicine’s benefits outweigh the risks.
www.fda.gov and the European Medicines Agency (EMA) EMA is a decentralized agency of the European Union (EU) responsible for the scientific evaluation, supervision, and safety monitoring of medicines in the EU www.ema.europa.eu join forces!
In addition to the European Medicines Agency EMA recently revising their original framework for interaction between the agency and patients and consumers and their organizations (2014) :
- 2015: The FDA launches the Patient Engagement Advisory Committee (PEAC)Patient Engagement Advisory Committee (PEAC) – Food and Drug Administration (FDA) committee providing advice to the Commissioner or designee on complex issues relating to medical devices, the regulation of devices, and their use by patients.
www.fda.gov/patients to provide advice relating to medical devices . CoPI: Patient Preference Information (PPI)PPI: Qualitative or quantitative assessments of the relative desirability or acceptability to patients of specified alternatives or choices among outcomes or other attributes that differ among alternative health interventions. is established to describe this patient engagement. PPI framework and guidance released for the Center for Devices and Radiological Health (CDRH)(CDRH) recognizes that scientists, clinicians, device developers, and regulators play critical roles in evaluating and communicating the benefits and risks of medical devices. However, only patients live with their medical conditions and make daily choices regarding their health care. Their voice and perspective are critical to understanding the impact of medical devices.
www.fda.gov/about-fda/fda-organization medical device decision-making.
- 2016: FDA and the EMA create a new workgroup on patient engagement called the FDA/EMA Patient Engagement ClusterWork group that allows FDA and EMA to share best practices involving patients along drug and biologic regulatory lifecycles. Information that is discussed is covered by confidentiality agreements signed by the FDA and EMA.
www.fda.gov/patients to share best practices involving patients along with drug and biologic regulatory lifecycles.
- 2017: FDA launches Patient Engagement Collaborative The Patient Engagement Collaborative (PEC) is a group of patient organizations and individual representatives who discuss how to achieve more meaningful patient engagement in medical product development and other regulatory discussions at the FDA. The PEC was established by the FDA and the Clinical Trials Transformation Initiative (CTTI), a public-private partnership that brings together organizations and individuals representing academia, clinical investigators, government and regulatory agencies, industry, institutional review boards, patient advocacy groups and others to develop evidence-based solutions to clinical research challenges. www.fda.gov/patients with the Clinical Trials Transformation Initiative (CTTI)The FDA, together with the Clinical Trials Transformation Initiative, established a group of patient organization and individual representatives to discuss topics focusing on enhancing patient engagement in medical product development and regulatory discussions at FDA.
www.ctti-clinicaltrials.org/ to develop guidance materials for including patients in the R & D continuum. This collaborative was modeled after the European Medicines Agency (EMA)’s Patients’ and Consumers’ Working PartyEMA is a decentralized agency of the European Union (EU) responsible for the scientific evaluation, supervision, and safety monitoring of medicines in the EU. The Patients’ and Consumers’ Working Party (PCWP) provides a platform for exchange of information and discussion of issues of common interest between EMA and patients and consumers. www.ema.europa.eu/en/committees.
- 2017: EMA involves young people in their activities and establishes the principles for the involvement of young patients, consumers, and their carers, in the Agency’s scientific committees and working parties in a consistent and efficient manner .
- 2017: EMA begins holding public hearings to give European citizens a voice in the evaluation of the safety of medicines and empower them to express their views on issues related to the safety of certain medicines and the management of risks.
- 2017: EMA expands opportunities to acquire patients’ perspectives on benefit/risk considerations within the Committee for Medicinal Products for Human Use (CHMP)(CHMP) is the European Medicines Agency’s (EMA) committee responsible for human medicines. The CHMP replaced the former Committee for Proprietary Medicinal Products (CPMP) in May 2004.www.ema.europa.eu/en/committees meetings.
- 2018: INVOLVEINVOLVE was established in 1996 and funded by the National Institute for Health Research (NIHR) to support active public involvement in National Health Services (NHS) public health and social care research.As a national advisory group, the role of INVOLVE is to bring together expertise, insight, and experience in the field of public involvement in research (PIR), with the aim of advancing it as an essential part of the process by which research is identified, prioritized, designed, conducted and disseminated.www.invo.org.uk
produces a set of six draft Standards for Patient Engagement that are currently piloted and evaluated in different areas of health research. United Kingdom
- 2018: Canadian Institute of Health Research (CIHR)The Canadian Institutes of Health Research (CIHR) is Canada’s federal funding agency for health research. Composed of 13 Institutes. It collaborates with partners and researchers to support the discoveries and innovations that improve our health and strengthen our health care system.https://cihr-irsc.gc.ca publishes draft Ethics Guidance for Developing Research Partnerships with PatientsThe proposed ethics guidance builds on the SPOR Patient Engagement Framework and, once finalized, could be adapted in various formats by SPOR leads and other initiatives, organizations, and institutions to serve as an educational resource for all those involved in research partnerships with patients.
http://www.cihr-irsc.gc.ca, in support of Canada’s Strategy for Patient-Oriented Research (SPOR)Endorses the active partnership of patients, researchers, health professionals and decision-makers in research so as to build a sustainable and accessible health care system that optimizes the health of Canadian citizens.https:cihr-irsc.gc.ca. The project is a response to ethical issues that were initially identified by the SPOR SUPPORT Unit Patient Engagement Working GroupPatient engagement means fostering a climate in which researchers, health care providers, decision-makers and policy-makers understand the value of patient involvement and patients see the value of these interactions.Engaging patients is an integral component in the development and implementation of all elements of SPOR https://cihr-irsc.gc.ca and reiterated at various conferences and workshops.
- 2019: ESCEO (European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis and Musculoskeletal Diseases)ESCEO is a not-for-profit organization, dedicated to a close interaction between clinical scientists dealing with bone, joint, and muscle disorder, pharmaceutical industry developing new compounds in this field, regulators responsible for the registration of such drugs and health policymakers, to integrate the management of Osteoporosis and Osteoarthritis within the comprehensive perspective of health resources utilization.
The objective of ESCEO is to provide practitioners with the latest clinical and economic information, allowing them to organize their daily practice, in an evidence-based medicine perspective, with a cost-conscious perception.
www.esceo.org & World Health Organisation (WHO) publish “Best practice principles for engaging patients in health research, treatment guidelines, and regulatory processesM. de Wit e.a., Principles for engaging with patients in health research, treatment guidelines and regulatory processes: Results of an expert group meeting organized by the World Health Organization (WHO)WHO is the United Nations agency that connects nations, partners and people to promote health, keep the world safe and serve the vulnerable – so everyone, everywhere can attain the highest level of health.www.who.int and the European Society for Clinical and Economic Aspects of Osteoporosis, Osteoarthritis, and Musculoskeletal Diseases (ESCEO), Lancet, April 2019”.
Tools: Guidance documents – ethics
2019: International Consortium for Health Outcome Measurement (ICHOM)International Consortium for Health Outcomes Measurement (ICHOM) aims to unlock the potential of value-based healthcare by defining global Standard Sets of outcome measures that matter most to patients and driving adoption and reporting of these measures worldwide to create better value for all stakeholders.
https://ichom.org unites an international working group – including patients – to develop the Inflammatory Arthritis Standard Set to enable health care providers to implement the value-based health care framework and compare outcomes that are important to patients with inflammatory arthritis. (Covers patients with rheumatoid arthritis, axial spondyloarthritis, psoriatic arthritis, and juvenile idiopathic arthritis.)
CoPI: Working groups
Tools: Guidance Documents – value-based healthcare