Outlines FDA regulations for the protection of human subjects in clinical investigations, including requirements for informed consent. Subpart D sets forth additional safeguards to protect children involved in clinical investigations, ensuring that the risks are minimized, the benefits justify the research, and appropriate parental or guardian consent is obtained.

The U.S. Food and Drug Administration (FDA) defines the adolescent age group as ages 12 through 21 years.

A child’s affirmative agreement to participate in a clinical investigation. Assent must be sought in addition to the consent of a legally authorized representative or surrogate when the individual is sufficiently cognitively capable of understanding the nature of his or her participation in a research study.

Refers to devices, software, or systems that are designed to support, improve, or maintain the functional capabilities of children with disabilities or developmental challenges. These technologies help children manage medical conditions, enhance their ability to communicate, learn, move, or participate in daily activities, and improve their overall quality of life. Some examples of where assistive technologies are used in pediatric care include:

Communication: Speech-generating devices or apps.
Mobility: Powered wheelchairs or adaptive strollers.
Learning and Cognitive Support: Software and apps designed to enhance focus, memory, and problem-solving skills.
Health Monitoring: Wearable monitors or connected devices that track vital signs.
Sensory Assistance: Hearing aids, visual aids, or sensory integration tools.

A relatively small portion of a total budget, often overlooked or undervalued in larger financial planning or resource allocation. Child healthcare costs are described as “budget dust” because, despite children making up a significant portion of the population, the actual spending on their care represents a small fraction of overall healthcare expenditures.

The amalgamation of individuals involved in care for children, from family members to community-based individuals and pediatricians, and specialists.

Children’s Health Insurance Program (CHIP) provides low-cost health coverage to children whose caregivers earn too much money to qualify for Medicaid. In some states, CHIP covers pregnant women.

A law that protects the online information of children under 13 years old. It applies to operators of websites and online services that target children, and requires them to obtain parental consent before collecting personal information from children. COPPA was designed to safeguard young people from potential abuse and privacy violations because children cannot legally consent to the use of their data. 

Assessment of a clinical outcome can be made through report by a clinician, a patient, a non-clinician observer or through a performance-based assessment. There are four types: 

1. Patient-reported outcome (PRO), 
2. Clinician-reported outcome (ClinRO) measures,
3. Observer-reported outcome (ObsRO), and 
4. Performance outcome (PerfO).

An investigation or research that involves one or more human subjects, undertaken to assess/evaluate the safety or effectiveness of a medical device.

A measurement based on a report that comes from a trained healthcare professional after observation of a patient’s health condition. Most ClinRO measures involve a clinical judgment or interpretation of the observable signs, behaviors, or other manifestations related to a disease or condition. ClinRO measures cannot directly assess symptoms that are known only to the patient (e.g., pain intensity).

Understanding and facility with meeting culturally unique needs across all patients.

Numerical codes used to identify medical services and procedures provided by qualified healthcare professionals. Codes that are reimbursable can be billed to insurers.

The set of rules, regulations, practices, and/or processes that ensure only authorized individuals and organizations see patient data & medical information.

The practice of protecting digital health data from unauthorized access,  corruption, or theft throughout its entire lifecycle.

A study that moves some or all of the trial participant interventions and prospective data collection outside of the traditional clinical trial sites and into the real world. These studies may use electronic platforms and data sources, mobile health providers or ship investigational products directly to patients.

Objective, quantifiable, physiological, and behavioral data collected using digital devices to measure health information from patients. These devices can be portable, wearable, implantable, or ingestible, and can collect data continuously.

A precisely defined variable intended to reflect an outcome of interest that is 1) collected digitally with a sensor, and 2) statistically analyzed to address a particular research question.

Ability to seek, find, understand and appraise health information from electronic sources and apply the knowledge gained to addressing or solving a health problem.

Electronic tools, systems, devices and resources that generate, store and process data in health care and research, which may include mobile health, wearable devices, telehealth, telemedicine, electronic health records, and patient portals.

The ability to use information and communication technologies (ICTs) to find, evaluate, create, and communicate information, requiring both cognitive and technical skills.

Evidence-based therapeutic interventions delivered through software programs to prevent, manage, or treat medical conditions. Typically used independently or alongside medications, devices, or other therapies. Can offer personalized and accessible healthcare solutions. Key characteristics include their ability to deliver measurable clinical outcomes and their integration with clinical practice to support patient care.

EHRs contain individual health records for patients and are used in care delivery. Generally maintained by health care providers, health care organizations, and health care institutions and are used to deliver care. Can be used to integrate real-time electronic health care information from medical devices and multiple health care providers involved in care of patients.

A U.S. law that replaced the No Child Left Behind Act. It aims to improve educational equity by addressing achievement gaps, requiring states to develop plans for supporting underperforming schools, and setting goals for academic success. It mandates annual testing, public reporting on school performance, and stakeholder involvement, including input from parents and communities.

A U.S. federal law that safeguards the privacy of student education records. It grants parents specific rights concerning their children’s records, which transfer to students when they turn 18 or attend a postsecondary institution. These rights include the ability to access, request amendments to, and control disclosure of their education records.

Federally funded nonprofit health centers or clinics that serve medically underserved areas and populations. FQHCs provide primary care services regardless of your ability to pay. Services are provided on a sliding scale fee based on your ability to pay.

The traditional healthcare reimbursement model. Under FFS, healthcare providers are paid for each service they deliver. This includes doctor’s visits, procedures, tests, and use of digital health products. 

In the context of use of a Digital Health Technology (DHT) in a clinical investigation, a conclusion that the level of validation associated with a DHT is sufficient to support its context of use.

Health equity means that everyone has a fair and just opportunity to be as healthy as possible. This requires removing obstacles to health such as poverty, discrimination, and their consequences, including powerlessness and lack of access to good jobs with fair pay, quality education and housing, safe environments, and health care.

An Act of Congress that outlines the lawful use and disclosure of protected health information (PHI) in the United States. HIPAA compliance is regulated by the Department of Health and Human Services (HHS) and enforced by the Office for Civil Rights (OCR).

The degree to which individuals have the capacity to obtain, process and understand basic health information needed to make appropriate health decisions. It also includes numeracy skills — such as calculating cholesterol and measuring medication doses —and knowledge of health topics.

Alphanumeric procedure codes used to identify specific healthcare procedures, services, supplies, and equipment. The HCPCS system consists of two levels of codes: 

1. Level I, which includes the Current Procedural Terminology (CPT) codes developed by the American Medical Association (AMA), and
2. Level II, which comprises a broader range of codes for items such as durable medical equipment, drugs (e.g., chemotherapy drugs under J-codes), and supplies.

An approach to interactive systems that aims to make systems usable and useful by focusing on the users, their needs and requirements, and by applying human factors and usability knowledge and techniques.

Process that requires a potential trial participant be given all of the information needed to make a sound decision about whether to volunteer to willingly participate. For informed consent to be meaningful, participants need to be “tech-literate” enough to understand the specifics of how their data will be obtained and used, or they need to be appropriately supported to understand these specifics.

Processes and documents which outline a strategy to connect labeling concepts (i.e. the claims associated with a new drug) to the evidence-generating trials and studies in the drug program.

The specific clinical circumstance or purpose for which a medical product or test is being developed. In the regulatory context, “intended use” refers to the objective intent of the persons legally responsible for the labeling of medical products.

The ability of different information systems, devices and applications to access, exchange, integrate and use data in a coordinated manner, within and across organizational, regional and national boundaries, to provide timely and seamless portability of information and optimize the health of individuals and populations globally.

Ensuring that information in or related to a digital health product is presented in a language in which the individual is fluent

Manage Medicaid administration on behalf of state governments. MCOs are key drivers of innovation within Medicaid, expanding the use of alternative value-based or risk-based payment models that create incentives for high value care to supplement traditional fee-for-service contracting.

A public health insurance program for some people or families with limited incomes and resources, including children, pregnant women, older adults, and people with disabilities. People who receive Medicaid have most or all of their health care services paid for by U.S. federal, state, and local governments. Each state government runs its own Medicaid program using federal guidelines, so Medicaid rules and benefits can differ from state to state. To be eligible for Medicaid, a person or family must meet the Medicaid rules of the state in which they live.

A measurement based on a report by a non-expert third party of how a person feels or functions in daily life.

A measurement based on a report of observable signs, events or behaviors related to a patient’s health condition by someone other than that patient or a health professional. Generally a parent, caregiver, or someone who observes the patient in daily life. They are particularly useful for patients who cannot report for themselves (e.g., infants or individuals who are cognitively impaired). It does not include medical judgement or interpretation.

Any individual with or at risk of a specific health condition, whether or not he or she currently receives any therapy to prevent or treat that condition. Patients are the individuals who directly experience the benefits and harms associated with medical products.

An approach that prioritizes the needs, preferences and values of patients, engaging them as active partners and decision makers in their own care. In the context of clinical trials, centralizing the patient (or participant) experience helps to:

• Guide the selection of meaningful outcomes, 
• Reduce burden on patients and caregivers by improving trial efficiency,
• Ensure appropriate privacy and data rights are protected throughout all stages of their development.

A forum for the voice of patients and will be asked to advise on complex issues related to medical devices and their impact on patients. The goal of PEAC is to better understand and integrate patient perspectives into oversight, to improve communications with patients about benefits, risks, and clinical outcomes related to medical devices, and to identify new approaches, unforeseen risks or barriers, and unintended consequences from the use of medical devices.

A type of patient experience data that specifically relates to patients’ attitudes or points of view about their condition or its management. Patient perspectives may include (but are not limited to): perceptions, goals, priorities, concerns, opinions, and preferences.

An outcome that is important to patients’ survival, functioning, or feelings as identified or affirmed by patients themselves, or judged to be in patients’ best interest by providers and/or caregivers when patients cannot report for themselves.

Ensuring that patients’ experiences, perspectives, needs, and priorities are meaningfully incorporated into decisions and activities related to their health and well-being.

Self-assessment of how a person feels or functions in daily life, reported without modification of interpretation. Can be measured by self-report or by interview. Unobservable symptoms known only to the patient (e.g., pain severity or nausea) can only be measured by PRO measures. 

The person, organization, or entity that sets service rates, collects payment, processes claims, and pays claims associated with healthcare services administered by a provider. Most often refers to private insurance companies that provide their customers with health plans that offer cost coverage, and reimbursements for medical treatment and care services

Pediatric patients as defined by the Center for Devices and Radiological Health (CDRH) are individuals who are younger than 22 years.

• Neonates: from birth → first 28 days of life
• Infants: 29 days to 2 years
• Children: 3 years to 5 and 6 to 11
• Adolescents: 12 through 21 (up to but not including the 22nd birthday)

An approach to design, strengthen and evaluate routine health information systems (RHIS). Puts emphasis on the organizational, technical and behavioural determinants of RHIS performance. Encourages and guides the development of interventions for strengthening or reforming RHIS.

A measurement based on standardized task(s) performed by a patient that is administered and evaluated by an appropriately trained individual or is independently completed. They require patient cooperation and motivation and include measures such as gait speed, memory recall or other cognitive testing.

All individually identifiable health information that is used, maintained, stored, or transmitted by a HIPAA-covered entity or a business associate of a HIPAA-covered entity, in any form or media whether electronic, paper, or oral. 

The FDA process of scientific and regulatory review to evaluate the safety and effectiveness of Class III medical devices.

Regulatory obligations set by regulatory bodies like the FDA that must be fulfilled before a product can be introduced to the market. These requirements ensure that digital health products like medical apps, wearable devices, and software intended for medical use, are safe and effective for their intended purpose. 

Premarket requirements may include:

• Pre-market Submission: Manufacturers must submit their digital health technology for regulatory review. This could be in the form of a 510(k) submission, De Novo request, or premarket approval (PMA) application, depending on the device classification.
• Clinical Data: Evidence from clinical studies demonstrating the safety and effectiveness of the technology may be required, particularly for high-risk devices.
• Quality Management System: Companies must have a quality management system (QMS) in place that complies with regulations such as the FDA’s Quality System Regulation (QSR). This ensures that the product is designed, developed, and manufactured consistently and meets quality standards.

Labeling Requirements: Clear instructions and labeling that accurately describe the product, its intended use, and any associated risks must be provided.

A practice used in healthcare payment models where a portion of the payment to a healthcare provider is withheld until certain quality measures are met. This practice is often seen in value-based care arrangements, where the incentive is for providers to achieve specific quality benchmarks in patient care.

Data relating to patient health status and/or the delivery of health care routinely collected from a variety of sources. RWD can come from a number of sources.

Clinical evidence regarding the usage and potential benefits or risks of a medical product derived from analysis of RWD. RWE can be generated by different study designs or analyses, including but not limited to: randomized trials including large simple trials, pragmatic trials, and observational studies prospective and/or retrospective.

Settings outside of traditional clinical trials environments.

A technology-based system of enabling monitoring of patients and/or participants outside of conventional clinical settings, such as in the home and during activities of daily living. 

The benefits gained from an investment in healthcare services, treatments, or technologies relative to the costs incurred. ROI measures the efficiency and value of these investments, often considering both economic returns (e.g., cost savings, revenue generation) and health outcomes (e.g., improved patient care, reduced hospitalizations).

Conditions in the environments in which people are born, live, learn, work, play, worship and age that affect health, quality-of-life outcomes and health risks.

Software intended to be used for one or more medical purposes that perform these purposes without being part of a hardware medical device.

The group of individuals (patients) about whom one wishes to make an inference.

Also referred to as the target patient population, the underlying population, or intended population.

The extent to which a product can be used to achieve specific goals with ease, efficiency, and user-satisfaction within a defined intended use or context of use.

A foundational mechanism of human-centered design (HCD) that delivers the insights, feedback, and validation needed to create products that are effective, safe, and enjoyable to use. This research is in addition to clinical SME involvement. 

VBC focuses on patient outcomes and quality of care rather than the volume of services provided. Reimbursement is tied to the effectiveness and efficiency of care, promoting overall health and reducing healthcare costs.

Regular medical check-ups for children that focus on preventive care, monitoring growth and development, administering vaccinations, and addressing any concerns about the child’s health and well-being. These visits are scheduled at key stages in a child’s life to ensure they are developing properly and to provide parents with guidance on health, nutrition, and safety.

A situation where one organization or sector invests in solving a problem, but the resulting benefits or returns are realized by another organization or sector. This can create a disincentive for the investing party, as they bear the costs without directly receiving the rewards.