In a recent study published in Pediatric Research, researchers performed a scoping evaluation of information from clinical trials addressing high-risk pediatric medical devices utilized in children.

Study: Evidence from clinical trials on high-risk medical devices in children: a scoping review. Image Credit: Sai Thaw Kyar/Shutterstock.com

Background

Medical equipment is important within the diagnosis and treatment of pediatric disorders; nonetheless, achieving European regulatory standards for clinical assessment is perhaps difficult resulting from small patient numbers, unusual occurrences, and varied demographics.

Ethical difficulties, parental concerns, and increased financial regulatory expenses offer hurdles, with the pediatric age range having a poor possibility of achieving advantages on investment.

Concerning the study

In the current study, researchers from the Coordinating Research and Evidence for Medical Devices (CORE-MD) initiative examined clinical trial data on using hazardous medical-grade devices in children. They investigated clinical trial techniques and designs that used medical devices within the high-risk category in children because the study intervention.

The researchers identified and described approaches on this research field by methodically summarizing published clinical data on hazardous medical devices, specifically accessible clinical trial evidence for youngsters.

Databases equivalent to Embase and Medline were searched between January 1, 2017, and November 9, 2022, for relevant records published in English.

The study included trials conducted for devices utilized in diabetology, cardiology, surgery, and orthopedics amongst individuals aged below 21 years, including neonates (birth to the initial 4 weeks), infants (from 29 days to 2 years of age), children (between two and 12 years of age), and adolescents (between 12 and 21 years of age), with conditions requiring medical devices.

The study comprised clinical trial findings on high-risk medical devices in children, including pre- and post-market clinical studies.

Observational studies, systematic reviews, conference abstracts, editorials, commentaries, book chapters, and letters were excluded from the evaluation. There have been no restrictions regarding the study setting or medical device indications. 

Mixed-population research, including children in addition to adults, was also included. There have been studies on class IIb and III medical devices under the European Medical Device Regulation (MDR) and sophistication III medical devices under the Food and Drug Administration (FDA).

Based on MDR regulations, the high-risk category of devices includes class IIb energetic ones and sophistication III implanted ones used to remove or administer medical substances from the human body.

Based on the FDA, the category included class III items implanted to support or sustain life or entail a potentially unreasonable disease or injury risk.

The team focused on medical devices listed in FDA sources equivalent to the Premarket Approvals (PMA) database (as of February 2020); Yearly Reports to the Congress authorities on the Premarket Authorization of Pediatric Usage of Medical Devices, including authorized Humanitarian Device Exemption (HDE), and PMA applications (between 2008 and 2017); and the HDE database (between 2018 and June 2022).

The study outcomes included country; unicentric or multicentric; study design (equivalent to controlled clinical trials, crossover trials, and one-arm interventional studies); sample population and the proportion of pediatric individuals; characteristics of the goal population, equivalent to age and sex; device type and indication; assessed study outcomes; approving regulatory bodies; and funding.

Two researchers independently screened the full-text records, and disagreements were resolved by discussion or involving a 3rd researcher. Descriptive statistics were used for the evaluation.

Results

A complete of 1,692 records were screened, of which only 99 trials were considered for evaluation. Most trials were performed in multicenter settings (65%), of which 73% were conducted in a single country, whereas 27% included multinational participants.

Most trials were conducted in Europe (35%) and North America (38%), they usually evaluated medical device use in diabetology (88%) and cardiology (12%), including insulin pumps, glucose monitoring devices, and closed-loop systems.

Most trials (61%) included only individuals under 21 years, whereas 39% had adults and youngsters, with 52% (median) pediatric individuals. Thirty-eight percent of trials were randomized controlled trials (RCTs).

Other study designs included crossover trials (20%) and before-and-after analyses (or baseline-controlled trials, 21%). Amongst RCTs and crossover trials, 13% were blinded.

Most trials (64%) included lower than 100 individuals. Amongst problems including individuals aged below 21 years only (60 trials), the sample population (median) comprised 48 individuals. Safety (73%), effectiveness, and efficacy (79%) were essentially the most frequent outcomes, with patient-documented outcomes assessed in just 24% of the included trials.

Usability was examined just for diabetology-related devices. Among the many trials, 32% were funded by industry, and 43% received partial funding (devices provided by the industry at no or discounted cost).

Implications

Overall, the study findings highlighted the difficulties in conducting randomized clinical trials on high-risk medical devices for youngsters, which often lack a control or comparator group.

These findings may aid health authorities in developing realistic evidence-level standards to support device conformity certification in Europe. To diminish bias, improvements include combining patient-reported outcomes and using a physician-initiated, industry-sponsored research approach.

Implementing the International Organization for Standardization (ISO) 14155 standard and mandating sponsors publish clinical investigation results can even assist in stopping any bias.

The findings may assist regulators and authorities in developing feasible and context-specific standards for clinical evidence supporting device compliance, thus ensuring the protection and continuous availability of critical technologies for treating sick children.