Tech & Innovation in Healthcare

Examine the FDA’s role in medical device development.

Revenue Cycle Insider explored different study types in last month’s part 1. Now, let’s see how these methods are used to develop new medical treatments. When a new drug or medical device is created, several types of studies are used to gather necessary safety and effectiveness information, to ensure the new drug or device is both safe and effective.

Read on to examine the development process through two real-world examples.

Medical Device Development: Non-Fixed Vertebral Tethering for Scoliosis

Let’s see how non-fixed vertebral tethering (VBT) devices for scoliosis are developed through a structured process, from initial concept to monitoring by the U.S. Food and Drug Administration (FDA) after they’re on the market.

Step 1: Device Idea

Goal: Create a way to treat scoliosis that allows the spine to stay flexible, unlike older surgeries that fuse (or permanently join) bones together.

Step 2: Preclinical Studies

What: These early tests often happen in laboratories using animal models. Researchers evaluate early versions (prototypes) of the device. This research happens before human trials.

VBT example: A 2002 prospective experimental study explored whether a flexible cable attached to the spines of young cows could influence growth and correct scoliosis. Untethered cows served as a control group for comparison. This non-randomized control trial (NRCT) showed potential for future non-fusion scoliosis correction.

Step 3: Clinical Trials

What: Once preliminary safety is established, early human use is often documented through descriptive studies like case studies or observational cohort studies. Devices are grouped into regulatory classes (class I, II, or III) based on their risk level, with class I having the lowest risk and least regulation.

VBT example: A 2014 retrospective observational cohort review, a type of descriptive study, followed 11 young patients with scoliosis for two years after VBT surgery. The study showed significant improvement in spinal curves and rotation, indicating the procedure was safe and effective in this small group.

Step 4: FDA Review

What: For FDA approval, medical devices typically undergo prospective experimental studies, either with all participants receiving the device or comparing difference devices.

VBT example: The FDA reviewed information from studies with 57 patients who used a non-fixed VBT device. After two years, 43 of these patients improved enough to avoid traditional spinal fusion surgery. However, some complications occurred, such as the tether breaking, the spine curving too much in the other direction (overcorrection), and air leaking into the chest (pneumothorax). There were no deaths and no neurological complications. The VBT system aims to avoid problems that can happen with fusion, like loss of spinal motion, pain, future surgeries, and neurological complications. Based on the clinical data, the benefits outweighed the risks, and the device received FDA approval to treat idiopathic scoliosis in 2019.

Step 5: FDA Post-Market Safety Monitoring

What: After FDA approval, post-market studies, often observational cohort studies (both prospective and retrospective), are crucial for long-term safety and effectiveness monitoring. The FDA supports collecting real-world data through databases to track the device performance over time. The FDA’s Medical Device Reporting (MDR) system helps identify potential device issues for investigation.

VBT example: The FDA released a 2024 review of the VBT system with a summary of the post-approval safety data and research. A total of 30 adverse event MDRs were reported between 2020 and 2023, with the most common issue being spinal curve progression. In 2024, researchers published a 10-year retrospective study on patients with adolescent idiopathic scoliosis (AIS) who were treated with VBT devices between 2010 and 2020, which provided additional data on treatment outcomes. Based on this data, the VBT device remains FDA-approved, with continued FDA monitoring recommended.

Drug Development: The Path of GLP-1 Receptor Agonists

Building on the process of medical device development and approval, let’s explore the development of glucagon-like peptide-1 (GLP-1) receptor agonists. GLP-1 drugs are a type of medicine used to treat type 2 diabetes and obesity. These drugs work by mimicking a natural hormone in the body called GLP-1. This hormone helps the patient’s body release insulin and lower blood sugar.

Step 1: Drug Discovery

Goal: Researchers look for new ways to treat diseases and find potential substances that might help.

GLP-1 example: Exendin-4 is a GLP-1 receptor agonist found in the saliva of the Gila monster. Researchers found that the saliva of these lizards, which eat only a few times a year, contained a substance that could stimulate insulin release and lower blood sugar. Exendin-4 acts like the natural human GLP-1 but keeps blood sugar low for a longer time, making it a good candidate for a new drug.

Step 2: Preclinical Studies

What: Before human trials, new drug candidates undergo laboratory and animal model testing. In vitro studies use cells that are grown in a lab, outside of a living body. Preclinical studies aim to understand how the drug works, if it’s safe, and optimal dosing. They are often experimental placebo-controlled trials, with some animals receiving saline injections to serve as controls.

GLP-1 example: In a 1999 experimental study, researchers found exendin-4 lowered blood sugar in diabetic monkeys at low doses. It also helped diabetic rats lose weight and reduced their blood sugar levels when given twice daily for several weeks. These promising animal studies supported researchers advancing to human clinical trials.

Step 3: Clinical Trials

What: If preclinical studies are promising, the drug moves on to clinical trials with human volunteers. These are prospective experimental studies, often NRCTs or sometimes randomized controlled trials (RCTs).

The trials are conducted in phases:

GLP-1 example: For exenatide (the synthetic version of exendin-4), phase 2 involved a 28-day randomized placebo-controlled study with 99 people with type 2 diabetes. Next, phase 3 trials consisted of three seven-month experimental studies that also used a placebo for comparison. About 1,900 people received exenatide in total during the clinical trial stage. These extensive trials showed significant improvements in blood sugar control, leading to the drug’s approval in 2005.

Step 4: FDA Review

What: To get approval, drug developers submit a New Drug Application (NDA) to the FDA. This application includes all research and development data. The FDA review team examines the submitted research. A senior FDA official then makes the final decision on whether the drug is safe and effective enough to be sold to the public.

GLP-1 example: Exenatide was approved by the FDA in 2005 under the brand name Byetta, becoming the first GLP-1 receptor agonist approved to treat type 2 diabetes.

The initial approval of exenatide in 2005 opened the door for the development and approval of other GLP-1 drugs, such as semaglutide, liraglutide, dulaglutide, and tirzepatides. The injectable form of semaglutide received FDA approval for type 2 diabetes (Ozempic^®) in 2017 and for weight loss (Wegovy^®) in 2021.

Step 5: FDA Post-Market Safety Monitoring

What: After a drug is on the market, the FDA continues to monitor its safety and effectiveness through ongoing observational cohort studies, both retrospective and prospective, which aim to detect rare side effects and gather real-world data on the drug’s long-term impact. The FDA’s MedWatch Adverse Event Reporting program helps keep track of any problems reported.

GLP-1 example: A >2025 observational retrospective cohort study, which looked back at data from 2020 to 2023, investigated whether GLP-1 receptor agonists might increase the chance of developing an eye condition that causes vision loss called neovascular age-related macular degeneration (nAMD). Researchers found that people with diabetes taking GLP-1 drugs had twice the risk (0.2 percent vs 0.1 percent) of developing nAMD. This potential increased risk of eye-related side effects highlights the need for further investigation and ongoing safety studies, especially for patients with preexisting eye conditions.

Understanding medical studies is important for knowing how new treatments reach patients. From testing early device prototypes to clinical trials and ongoing monitoring after approval, each step helps ensure that new therapies are both safe, effective, and improve patient health.

Angela Halasey, BS, CPC, CCS, Contributing Writer