Here are some common terms and organizations families and patients might encounter during their journey.
Adverse Event: An undesired change in the health of a participant that happens during a clinical study or within a certain time period after the study has ended. This change may or may not be caused by the intervention being studied.
Arthritis: Inflammation of one or more joints. The two most common types of arthritis are osteoarthritis (wear and tear related) and rheumatoid arthritis (see rheumatoid arthritis).
Arthritis Foundation: Founded in 1948, the Arthritis Foundation is focused on finding a cure and championing the fight against arthritis with life-changing information, advocacy, science and community. They are boldly leading the fight against juvenile arthritis and childhood rheumatic diseases. The Arthritis Foundation is strongly committed to making sure you have easy access to the resources, community and care you need – and making each day another stride towards a cure. www.arthritis.org
Autoinflammatory Syndrome: A set of disorders characterized by recurrent episodes of systemic and organ-specific inflammation.
Blind/Double Blind Study: Blind means study participants do not know which treatment they are getting. Double blind means doctors and study participants do not know which treatment is being used. Treatment could also include a placebo (see placebo).
CARRA: The Childhood Arthritis and Rheumatology Research Alliance was created to ease research aimed at finding the cause and cure for childhood rheumatic diseases. Nearly every pediatric rheumatologist in North America is a member of CARRA. CARRA’s vision is to have every patient participate in research in some way. www.carragroup.org
CARRA Registry: A database of information collected from children and young adults with pediatric-onset rheumatic diseases such as juvenile idiopathic arthritis (JIA) and lupus. The CARRA Registry allows patients from all over North America to share information including clinical outcomes, patient reported outcomes, and blood sample collection. That research is then shared with researchers to better understand pediatric rheumatic diseases.
Clinical Research: A type of research designed to inform treatment decisions by providing evidence that can be compared on the effectiveness, benefits, and harms of two or more treatment options.
Clinical Study/Trial: A research study that uses human subjects to evaluate biological or health-related outcomes, or results. Examples include interventional trials and observational trials (see interventional and observational).
Comparative Effectiveness Research (CER): A type of clinical research designed to inform treatment decisions by providing evidence that can be compared on the effectiveness, benefits, and harms of two or more treatment options. Research that compares treatment choices. For example: which medication will work better for my disease, drug X or drug Y?
Consensus Treatment Plan (CTP): Commonly used treatment approaches for a condition. Developed by CARRA so treatments can be compared to each other.
Controlled Study/Trial: A type of clinical trial in which the results of the group that received the intervention being studied (see intervention) are compared to those of the control group which received either the standard of care or no treatment.
Cure JM Foundation®: Founded in 2003, Cure JM Foundation is the only organization solely dedicated to supporting Juvenile Myositis (JM) research and improving the lives of families affected by JM. Cure JM Foundation's mission is to increase awareness, provide support to the families battling this disease, and fund research into better treatments and an eventual cure for JM. www.curejm.org
Electronic Health/Medical Record (EHR/EMR): An electronic version of a patient’s medical history that is maintained by the provider over time, such as past medical history and medications.
Health Insurance Portability and Accountability Act (HIPPA): Passed in 2003, HIPAA is a law that protects privacy and patient medical records. HIPAA allows patients to control how their health facts are used and shared.
Inclusion/Exclusion Criteria: Factors that allow someone to participate in a study.
Informed Consent Form (ICF): A document that is signed by a research participant before joining a research study that documents willingness to participate and understanding of study and activities and its risks.
Institutional Review Board (IRB): A group that reviews and approves research on people. The purpose of the IRB is to make sure that all human research is conducted in accordance with all federal, institutional, and ethical rules to protect patients.
Intervention: Something taken or done to try to improve health outcomes. Interventions may be drugs, medical devices, procedures, vaccines, and other products that are either in a research/development phase or already approved by the Food and Drug Administration and available for use. Interventions can also include nonmedical approaches, such as educational programs.
Juvenile Idiopathic Arthritis (JIA): The most common form of arthritis in children and adolescents. (“Juvenile” in this context refers to an onset before age 16, “idiopathic” refers to a condition with no defined cause, and “arthritis” is the inflammation of the joint lining.)
Learning Healthcare System (LHS): Using technology to collect and share healthcare data from patients, clinicians, and the general population to drive better care with faster results.
Localized Scleroderma: A form of scleroderma (see scleroderma) that involves isolated patches of hardened skin on the face, hands, and feet, or anywhere else on the body, with no internal organ involvement.
Lupus: An autoimmune disease in which the body's immune system mistakenly attacks healthy tissue in many parts of the body. Common symptoms include painful and swollen joints, fever, chest pain, hair loss, mouth ulcers, swollen lymph nodes, feeling tired, and a red rash which is most commonly on the face. Often there are periods of illness, called flares, and periods of remission during which there are few symptoms.
Lupus Foundation of America: Founded in 1977, the Lupus Foundation of America, Inc. is devoted to solving the mystery of lupus, one of the world’s cruelest, most unpredictable, and devastating diseases, while giving caring support to those who suffer from its brutal impact. They strive to improve the quality of life for all people affected by lupus through programs of research, education, support, and advocacy. www.lupus.org
Observational Study: A type of clinical study in which the researcher studies a defined group but does not apply an intervention to or influence any aspect of the group’s care or activities (in contrast to an interventional study; see interventional). The researcher collects information/data and makes observations or draws inferences, often about cause and effect. Researchers and/or participants decide their treatment as opposed to being assigned a treatment.
Outcome Measures: Study results that are evaluated according to a predefined measure. An outcome measure provides a tool or method for objective assessment of the effect of an intervention (see intervention). The outcome of interest is identified and the ways in which it will be measured are determined before a study begins. A way to check if an intervention has helped improve a patient’s health.
PARTNERS: Patients, Advocates, and Rheumatology Teams Network for Research and Service. Building the framework to conduct research in pediatric rheumatology in collaboration with families of children with rheumatic disease. Through PARTNERS, parents of children with rheumatic diseases have the opportunity to share their opinions on what they feel is important in caring for their children. Patients and their families are directly involved in making decisions about research agendas and designing research studies because every family is an expert in caring for their child. PARTNERS’ research partners have a voice in every stage of research—from asking the research questions to ranking them in order of importance, from designing the study to enrollment strategies, and finally to sharing the results with all patients.
Patient Reported Outcomes (PRO): Any report or information about a patient that comes directly from the patient. This is often a questionnaire filled out by the patient or the patient’s parent or caregiver.
PCORI: Formed in 2010 through the Affordable Care Act, PCORI (Patient-Centered Outcomes Research Institute) is an independent, non-profit health research organization that funds and shares research comparing the effectiveness of the various choices patients have when choosing their care. PCORI’s vision is to provide the patient with the information they need to make decisions that reflect their desired health outcomes. PCORI funds comparative effectiveness research (CER) and PCORnet. www.pcori.org
PCORnet: National Patient-Centered Clinical Research Network is a national patient data network funded by PCORI. It collects data from large multi-centered health care systems like hospitals and data from individual caregivers and people with disease. The large centers are called Clinical Data Research Networks (CDRNs) and the disease- specific programs are called Patient-Powered Research Networks (PPRNs). The data is owned and protected by the individual CDRNs and PPRNs. The goal of PCORnet is to improve the nation’s capacity to conduct comparative effectiveness research (CER). PCORnet brings together patients, care providers and health systems to improve healthcare and advance medical knowledge. With patients and researchers working side by side, PCORnet will be able to explore the questions that matter most to patients and their families. http://www.pcornet.org/
PR-COIN: The Pediatric Rheumatology Care and Outcomes Improvement Network is a network of rheumatologists, nurses, therapists, social workers and support staff at rheumatology centers who in partnership with families are all working together to transform how care is delivered to children with JIA. The aim of PR-COIN is to develop and evaluate specific disease management strategies to improve the care of children with JIA and to determine how best to incorporate these strategies into clinical practice. PR-COIN is creating a sustainable network that uses a registry database to measure performance, learn more about the health status of JIA patients as well as to inform future improvement projects. https://pr-coin.org/
Placebo: A substance that does not contain active ingredients and is made to be physically indistinguishable (that is, it looks and tastes identical) from the actual drug being studied. A harmless treatment that has no active effect on the condition being studied (see blind/double blind study).
Primary Purpose: The main reason for conducting a clinical trial. Studies may also have a secondary purpose.
Principal Investigator (PI): The lead researcher or person who is responsible for the management and execution of the study project according to the approved protocol (see protocol), which may include multiple study sites.
PROMIS Measures: Surveys developed to evaluate well-being. These surveys are commonly used for Patient Reported Outcomes (PROs).
Protocol: The written document that describes a clinical study and the plan for its execution. The details include the study's purpose (see primary purpose), objectives, design, and methodology. It may also include relevant scientific background and statistical information. A written set of instructions to perform a study developed to protect patients and answer a research question.
Protected Health Information (PHI): Defined by the Health Insurance Portability and Accountability Act of 1996 as individually identifiable health information. PHI can also stand for personal health information.
Randomization: The process where study participants are assigned to a treatment by chance.
Refractory: When an illness does not respond to treatments.
Research/Clinical Research: Research can be defined as the search for an answer to a question. Clinical research is medical research that involves people.
Rheumatology: A branch of medicine devoted to the diagnosis and therapy of rheumatic diseases. Physicians who have undergone formal training in rheumatology are called rheumatologists. Rheumatologists deal mainly with immune-mediated disorders of the musculoskeletal system, soft tissues, autoimmune diseases, vasculitides, and heritable connective tissue disorders.
Rheumatoid Arthritis: When the body's immune system attacks the lining of the joint capsule (a tough membrane that encloses all the joint parts). This lining, known as the synovial membrane, becomes inflamed and swollen.
Scleroderma: A group of autoimmune diseases that may result in changes to the skin, blood vessels, muscles, and internal organs. The disease can be localized to the skin (see localized scleroderma) or involve other organs in addition to the skin (see systemic scleroderma).
Self-advocacy: A patient’s ability to speak up for themselves and the things that are important to them. This includes the ability to ask for what they need and want and tell others about their thoughts and feelings. This also refers to knowing one’s rights and responsibilities, speaking up for those rights, and the ability to make choices and decisions that affect one’s life. The ultimate goal is for the patient to decide what they want, then develop and carry out a plan to help achieve it.
Sjögren Syndrome: A chronic long-term autoimmune disease that affects the body's moisture-producing glands. This can include degeneration of the salivary and lacrimal glands, resulting in dryness of the mouth and eyes.
Standard of Care: Treatment that is accepted by medical experts as a proper way to care for a condition.
Uveitis: Inflammation of the uvea, the pigmented layer that lies between the inner retina and the outer fibrous layer of the eye composed of the sclera and cornea. The uvea is the pigmented middle of the three concentric layers that make up an eye, and includes the iris, ciliary body, and choroid.
Vasculitis: The inflammation of blood vessels. It can affect arteries, veins, and capillaries.