While not specific to rheumatology, including patients in the regulatory process paves the way for advances in therapeutic outcomes.

• 2000: 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 invites patients as committee members 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 expands the Patient Representative role to serve as consultants to scientific and regulatory reviewers.
• 2003: EMA expands to patient inclusion in working groups.
• 2005: EMA establishes their first collaboration framework for regulatory practices between EMA and patients and consumers and their organizations, which was revised in 2014. 
• 2006: The EMA establishes the Patients’ and Consumers’ Working Party (PCWP) EMA 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, which provides a platform for the exchange of information and discussion of issues of common interest between EMA and patients and consumers. The PCWP provides recommendations to EMA and its human scientific committees on all matters of interest in relation to medicines.

CoPI: Working groups, regulatory reviewers, consumer reviewers

Context: Regulatory process

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) 

• 2011: PCOR – Patient-Centered Research Outcomes (PCOR)Established by PCORI, this research approach is a new form of comparative effectiveness research (CER) that considers the needs and perspectives of patients and stakeholders by engaging them throughout the entire research process. www.pcori.org is established by 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 and enlists patients to participate in clinical research grant evaluationsPCORI also engage 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. United States View other PCORI projects that included rheumatology patients in research.
• 2012: National Institute for Arthritis and Musculoskeletal and Skin diseases (NIAMS)The mission of the National Institute of Arthritis and Musculoskeletal and Skin Diseases is to support research into the causes, treatment, and prevention of arthritis and musculoskeletal and skin diseases; the training of basic and clinical scientists to carry out this research; and the dissemination of information on research progress in these diseases.
  www.niams.nih.gov adds one patient to the grant application review team. 
• 2012: The US Department of Defense (DoD) The Department of Defense is America’s largest government agency. With our military tracing its roots back to pre-Revolutionary times, the department has grown and evolved with our nation. Our mission is to provide the military forces needed to deter war and ensure our nation’s security. www.defense.gov created the Peer Reviewed Medical Research Program (PRMRP)PRMRP is one of 35 research programs managed by the Congressionally Directed Medical Research Programs (CDMRP). Patients/survivors/individuals living with the diseases and conditions with research programs managed by the CDMRP were incorporated into all aspects of the program cycle with equal voice to include research focus, investment strategy, and programmatic review in 1993, and into peer review in 1995.
  cdmrp.army.mil adds patients/individuals living with rheumatoid arthritis and osteoarthritis as peer reviewers starting in 2012 and lupus was added in 2015. In 2017, lupus became its own research program within the Congressionally Directed Medical Research Programs (CDMRP). The CDMRP is a Department of Defense (DOD) program. that receives congressional appropriations explicitly for. biomedical research in specific, congressionally identified. health matters. cdmrp.army.mil However, patients/individuals living with lupus originally participated in peer review starting in 2005 and continue doing so in the Lupus Research Program.
• 2013: Establishment of Foundation for Research in Rheumatology (FOREUM)FOREUM Foundation for Research in Rheumatology is devoted to promote research in rheumatic and musculoskeletal diseases (RMDs) as an independent research funding body in rheumatology research. Basic and applied research of highest quality is supported to reduce the burden of disease for people with RMDs.
  www.foreum.org, supported by the European League Against Rheumatism (EULAR)EULAR is the organization that represents 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. As such, they have always had their own educational program at the annual EULAR Congress meeting, but in the earlier years, the symposia they organized was mostly independent of the other congress sessions. eular.org, involves patients in the government of the foundation as well as in the review of research grant applications.
• 2013: 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 policy makers make better-informed health decisions.
  www.pcori.org established the Advisory Panel for Patient EngagementBy inviting the patient and caregiver community to help generate research questions, review research proposals, assist in the conduct of research, help disseminate information and evaluate impact, patient engagement ensures authentic patient-centeredness in the research process. www.pcori.org which is an advisory body that provides input and recommendations to PCORI Board of Governors, Methodology Committee, and staff to help plan, develop, implement, improve, and refine efforts toward meaningful patient-centered research. Panel members assure the highest patient engagement standards and a culture of patient-centeredness in all aspects of its work.
• Most pharmaceutical companies realize the need for an internal patient engagement department. The use of Patient Advisors becomes common.

CoPI: Advisory panels , consumer reviewers, Patient-Centered Outcomes Research (PCOR)

Context: Assessing grant applications, Research & 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 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

• 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”.

CoPI: Strategy for Patient-Oriented Research (SPOR), Patient Involvement in Research (PIR)/Patient and Public Involvement (PPI), working groups

Tools:  Guidance documents – ethics

Context: Ethical review, regulatory process, clinical research, guideline development