Health Services Researchers

• 1998: First self-governed Consumer Advisory Council (CAC)The mission of the Consumer Advisory Council (CAC) is to advocate for consumers and provide for strong public and consumer input in healthcare reform policies in Connecticut. The purpose of the Consumer Advisory Council is to ensure significant consumer participation in the planning and implementation process. portal.ct.gov/OHS/Pages/Consumer-Advisory-Council was established as part of Canadian Arthritis Network/CAN The Canadian Arthritis Network (CAN) is a not-for-profit organization that supports integrated, trans-disciplinary research and development. can.arthritisalliance.ca, the first federally funded arthritis research institute in Canada. After morphing into the Alliance for the Canadian Arthritis Program (ACAP) in 2012, then Alliance for Arthritis in in Canada (ACC), the group was disbanded in 2019. 
• 2000: Arthritis Research Canada (ARC) ARC was created in 2000 in recognition of the tremendous potential that research can bring to arthritis treatment in Canada, and indeed, the world. Arthritis Research Canada has earned international attention as a leading arthritis research environment. We have built a strong multi-disciplinary research team of outstanding medical doctors and research scientists, and we willingly collaborate with medical professionals around the world. www.arthritisresearch.ca and established the Arthritis Patient Advisory Board (APAB).The Arthritis Patient Advisory Board (APAB) is comprised of volunteer advocates with arthritis who bring personal experience and arthritis knowledge to research decision making at Arthritis Research Canada. Their goal is to ensure the patient perspective is represented on research matters related to prevention, treatment and self-management of arthritis. Individually and collectively, they communicate research information and findings to arthritis patients, professional organizations and the general public. www.arthritisresearch.ca 
• Additionally, Patient Organization, Arthritis Consumer Experts/JointHealthⓇArthritis Consumer Experts/JointHealthⓇ, a national organization that provides free, science-based information and education programs in both official languages to people with arthritis. https://jointhealth.org/ , was established.

CoPI: Advisory panels

Context: Research & Development

• 2002: The Childhood Arthritis and Rheumatology Research Alliance (CARRA) Founded by a small group of pediatric rheumatologists who aimed to create an investigator-led collaborative research network by establishing a registry. The information is provided by the medical team and by patients and their families. The registry was created to monitor the long-term safety of the medications used to treat pediatric rheumatic diseases. https://carragroup.org pediatric registry is established.  The founding members quickly realized that one of the best ways to achieve its primary focus was to begin a registry project. The CARRA Registry was later founded as a means to collect information about how childhood-onset rheumatic diseases are treated and how they affect patients by collecting information from the medical team, patients, and their families. United States
• 2006: The National Institute for Health Research (NIHR) (NIHR) was established in 2006 under the government’s health research strategy Best Research for Best Health to “create a health research system in which the NHS supports outstanding individuals, working in world-class facilities, conducting leading-edge research focused on the needs of patients and the public”. Since that time, we have transformed research in and for the NHS and helped to shape the health research landscape more broadly. Our mission is unchanged: to improve the health and wealth of the nation through research. We deliver against this mission through five core workstreams. www.nihr.ac.uk establishes the ‘Patient Research Ambassadors’ network and supporting GenerationRGenerationR is a National Network (GenerationR Alliance) of Young People’s Advisory Group’s (YPAGs) based across the United Kingdom to support the design and delivery of paediatric research. GenerationR Alliance YPAGs are a resource available to any health researcher (both academic and life science industries) who seek the input of children, young people, and families in their research projects www.invo.org.uk , a series of young people’s advisory groups across the country enabling children and young people under 25 to be involved in research. United Kingdom

CoPI: Advisory panels

Context: Research & Development, dissemination (knowledge translation), children, registries, young adults

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.
  eular.org Europe
• 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.
  www1.uwe.ac.uk/
• 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

CoPI: Patient Research Partners (PRPs), Public Involvement in Research (PIR)/Patient and Public Involvement in Research (PPI)

Tools: Recommendations – patient-researcher engagement; Reports – patient researcher engagement; Guidance documents – patient researcher engagement and briefing notes

Context: Guideline development, Patient-reported outcomes (PROs) 

• 2013: PCORnet PCORnet is a national resource that offers the kind of research ecosystem that has long been pursued: a fully integrated network where vast, highly representative health data, research expertise, and patient insights are built-in and accessible from the very start. The infrastructure of PCORnet is well established, meaning that the community knows how to maximize the value of these connections to deliver fast, trustworthy answers that advance public health. pcornet.org –  The era of big data, loosely defined as the development and analysis of large or complex data sets, the Patient-Centered Outcomes Research Institute (PCORI) The Patient-Centered Outcomes Research Institute (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.
  www.pcori.org establishes PCORnet, bringing new opportunities to empower patients and their families to generate, collect, and use their health information for both clinical and research purposes. Through PCORnet, a “network of networks” was established– Patient-Powered Research Networks (PPRNs), built and governed by stakeholder groups focused on specific conditions and community interests. Learn more about PPRN’s .

Two rheumatology specific PPRN’s were developed, including:

• 2015: ArthritisPowerVisit the ArthritisPower.org website for products and/or info about how to get involved. led by CreakyJointsCreakyJoints is a digital community for millions of arthritis patients and caregivers worldwide who seek education, support, advocacy, and patient-centered research. CreakyJoints is a part of the non-profit Global Healthy Living Foundation, which receives funding from grants and sponsorships from pharmaceutical manufacturers, private foundations, and government-sponsored research grants. They frequently collaborate on research with major research institutions and universities.
  creakyjoints.org. ArthritisPower is a non-profit, patient-inspired, and patient-managed research initiative that helps patients make a difference in the fight against arthritis and related conditions. View ArthritisPower overview, includes links to publications .
• 2015: Patients, Advocates and Rheumatology Teams Network for Research and Service (PARTNERS) PARTNERS is a PPRN linking patients, family members, caregivers, researchers, healthcare providers, and other people interested in pediatric rheumatic diseases. Through PARTNERS, patients, family members, and other caregivers are able to share research ideas, work on proposals, and join study teams. www.arthritis.org/science ) – led by healthcare providers, Childhood Arthritis and Rheumatology Research Alliance (CARRA)Childhood Arthritis and Rheumatology Research Alliance (CARRA) was founded by a small group of pediatric rheumatologists who aimed to create an investigator-led collaborative research network by establishing a registry. The information is provided by the medical team and by patients and their families. The registry was created to monitor the long-term safety of the medications used to treat pediatric rheumatic diseases.
  carragroup.org/, the US Arthritis FoundationThe 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. https://www.arthritis.org/, Friends of CARRA, the Lupus Foundation of AmericaLupus Foundation of America’s mission is to improve the quality of life for all people affected by lupus through programs of research, education, support, and advocacy.
  www.lupus.org/, and a quality-improvement learning network (Pediatric Rheumatology Care and Outcomes Improvement Network, or PR-COIN)Pediatric Rheumatology Care and Outcomes Improvement Network (PR-COIN) works to improve outcomes and provide safe, effective, efficient, timely, patient-centered, and equitable care for all children with rheumatic conditions. Our goals are to increase remission and clinically inactive disease rates among children with JIA, improve quality of life including optimal physical function and pain, and support families in managing their child’s condition.
  https://pr-coin.org/. View summary overview of PARTNERS .
• 2015 PCORnet Assessment:  Report evaluating the strengths and weaknesses of the initial phase dedicated to building its governance, technical, and research infrastructure. View report .

CoPI: Patient-Centered Outcomes Research (PCOR), co-production of stakeholder groups, patient-powered networks

Tools: Reports – patient research engagement

Context: Registries, patient-powered registries, children

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.
  www.fda.gov.
•  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.

CoPI: Patient-Focused Drug Development (PFDD), regulatory reviewers

Tools: Guidance Documents – patient-focused drug development (PFDD); Reports – patient-focused drug development (PFDD)

Context: Regulatory process, clinical trials, Research & Development, guideline development

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.

CoPI: Regulatory reviewers, working groups, advisory panels, Patient Preference Information (PPI)

Tools: Frameworks – regulatory; Guidance Documents – patient engagement

Context: Regulatory process, clinical trials, Research & Development, health technology assessment (HTA), decision aids, young adults

Led by patients who have helped shaped patient engagement in this space, the ACTion CouncilLearn more about the ACTion Council at www.rheumactioncouncil.org unites a global representation of key stakeholders in the rheumatology research community to record the history of patient engagement and track its continued expansion so efforts are not duplicated, value measurements can be improved, and new initiatives can evolve from existing successes.

CoPI: Co-production of Stakeholder Groups

• 2017: While Precision Medicine (PM) Precision Medicine is a medical approach that proposes to prevent and treat disease based upon a person’s unique genetic makeup and their lifestyle habits. Precision medicine, the ability to accurately measure specific predictors of patient outcomes is implemented in personalized clinical care. research has progressed in oncology for years, only recently has there been a focus in the rheumatology spaces. As treat-to-target (T2T)T2T is a medical strategy that sets remission – defined as the absence of signs and symptoms of significant inflammatory disease activity – or low disease activity as a goal. Specific disease management targets are set. Then disease activity may be measured as frequently as monthly through lab tests and clinical examinations. If the targets are not reached, medications and/or doses are adjusted according to a predefined protocol. The process continues until the goal is achieved. www.aiarthritis.org/precisionmedicine treatment protocols prove successful, and the complexity of rheumatic diseases is realized, efforts to advance science towards precision treatments have continued to evolve.
• 2019: Preparing Patients for Precision Medicine Project – (Underway) – Developing a patient-led shared decision-making tool to help patients talk to their doctors about participating in future precision medicine trials, which will include patients who will may differ from those currently enrolled (general patient population versus atypical presentation). Learn more. – International Foundation for Autoimmune & Autoinflammatory Arthritis (AiArthritis) AiArthritis helps others, like us, living with autoimmune or autoinflammatory diseases that include inflammatory arthritis as a major clinical component, have a voice – alongside other stakeholders as equals – so, together, we can solve problems that impact education, advocacy, and research.www.aiarthritis.org
  , professional advisor OMERACT.

CoPI: Research advocacy, Mentor Assisted Research (MAR)

Context: Precision medicine, clinical trials, decision aids

With the onset of the global pandemic of COVID-19COVID-19 is the disease caused by SARS-CoV-2, the coronavirus that emerged in December 2019. www.hopkinsmedicine.org/health/conditions-and-diseases/coronavirus , rheumatology registries – come existing and some forming solely for this reason – began focusing strongly on tracking cases in rheumatology patients. Some of these registries included the patient voice in various phases of data collection.

• 2020: COVID-19 Global Rheumatology Alliance (GRA)The mission of the GRA is to collect, analyze and disseminate information about COVID-19 and rheumatology to patients, physicians, and other relevant groups to improve the care of patients with rheumatic disease.
  rheum-covid.org/ was developed to collect, analyze, and disseminate information about COVID-19 and rheumatology to patients, physicians, and other relevant groups to improve the care of patients with rheumatic disease. In addition to establishing a Patient Board, patients were invited to submit and review questions as part of the Patient Experience Survey and help develop lay summaries. International
• 2020: The Arthritis & Rheumatic Disease COVID-19 Project From conception to development and launch, the Arthritis and Rheumatic Disease COVID-19 project included patients during all phases of the project, which began by learning about the impact of COVID-19 on people who are living with underlying chronic disease through multiple conversations. Patients also collaborated with researchers and clinicians to help develop and refine the survey, prioritize survey questions, provide input on the study timeline, and strategize on dissemination efforts.
  rheumcovid19project.org/ – A patient-powered study of the Autoimmune Research Collaborative, from conception to development and launch, the Arthritis and Rheumatic Disease COVID-19 project included patients during all phases of the project. Respondents are asked to participate at multiple time points to better understand concerns, behaviors, and the pandemic’s overall impact on them over time. The study will also inform the preparedness of patients and clinicians for future pandemics and provide a better understanding of concerns with infection in general among patients with autoimmune disease. Led by Global Healthy Living Foundation/CreakyJoints. United States, Canada

CoPI: Advisory panel, working group, co-production of stakeholder groups

Context: Registries, lay summaries, dissemination (knowledge translation)