Innovation in MedTech is on the rise, with artificial intelligence devices and digital therapies redefining what’s possible. However, as technology advances, so do the challenges of clinical development. Developers must now navigate complex regulatory areas, justify value to payers and manage costs while ensuring their devices meet high standards of safety and performance.

„Advanced wearables and AI-driven tools will not reach patients unless trials are designed to meet regulatory and reimbursement expectations,” says Lisa Beck, BSN, MS, senior director of clinical affairs at IQVIA’s MCRA. „Developers need to design tests that are simple, data-driven, and strategically aligned with regulatory and market needs.”

Optimizing your test design is a critical success factor. Robust and strategic trial design helps manage the risk of costly delays or regulatory challenges, and can even help speed time to market.

Here, we’ll discuss three MedTech companies’ strategies for streamlined, efficient trial design and how quality data enables smarter study design.

Biostatistics in trial design

A successful test plan depends on early stakeholder alignment and a range of insights, from subject matter expertise to strategic planning. Beck recommends a core team of five: a principal investigator (your subject matter expert), plus experts in biostatistics, reimbursement, regulatory affairs, and clinical operations.

In addition, the biostatistician plays a particularly important role in laying the foundation for a successful trial.

Biostatisticians help define endpoints, reduce bias, and calculate sample sizes to help ensure adequate study power. For example, in diagnostic and monitoring device research, companies must demonstrate that their ability to detect health conditions and events (in terms of sensitivity) and to avoid false positives (in terms of specificity or false detection rate) is robust enough to avoid serious clinical consequences.

„All medical devices have a balance of risk and benefit. In diagnostics, that balance can be captured by sensitivity and specificity,” said Chava Zibman, Ph.D., Chief Statistician at IQVIA Business MCRA. „A sponsor’s rigor in quantifying and justifying the risk-benefit profile of their product can mean the difference between regulatory approval and delay.”

Biostatisticians also help formulate inclusion and exclusion criteria, predict attrition, and establish guidelines to monitor trial validity. Early involvement of biostatisticians helps sponsors avoid pitfalls by aligning statistical rigor with clinical and regulatory goals from the outset.

Use valuable insights from pilot studies

Pilot studies are a powerful tool for refining the test plan and reducing uncertainty before starting full-scale core testing. They offer a low-cost, low-risk way to evaluate device performance, validate endpoints, and test operational validity.

„Doing a pilot study helps to understand the device’s performance before starting the main trials, because the sponsors have to demonstrate that the performance exceeds the regulatory limits,” explains Dr. Zibman. „Pilot studies help assess a device’s strengths and potential bottlenecks. If you don’t meet regulatory thresholds, you can tailor the device accordingly before investing in a clinical trial.”

Pilot studies also allow developers to test and refine test protocols, ensuring they are optimized to demonstrate the intended functionality of the device. This is especially important as the device becomes more complex.

„Increased technological complexity of devices does not always require more complex study designs. Complex study designs are often determined by the extent to which a device performs multiple functions or changes the standard of care,” says Zibman. „Pilot studies can help identify opportunities to streamline study design while meeting regulatory expectations.”

In addition, pilot studies may reveal operational challenges, such as recruitment barriers or data collection issues, that may affect the validity of the trial. Addressing these issues early helps sponsors streamline key trials and increase the likelihood of regulatory success.

Select available endpoints

The end points you choose make or break the success of the test. It is critical to select endpoints that are clinically relevant, measurable, and meet regulatory expectations.

To determine appropriate outcomes, sponsors should analyze past research and regulatory decisions to understand what has been passed historically and how expectations may be changing. For example, recent trends show the increasing use of standardized endpoints such as valve consortia in cardiovascular studies, which can help ensure consistency and comparability between studies.

Medtech developers should also consider including patient-reported outcomes (PROs) and quality-of-life measures, while recognizing their limitations as separate endpoints.

„PROs encourage patients to really reflect on how they feel at a given moment and time, which is important for measuring the real-world impact of a device, but may not capture their overall condition,” says Beck. „This is why sponsors should seek advance FDA approval for the use of PROs and use them in conjunction with other clinical success measures.”

Quality data: the key to effective trial design

As medical devices improve, so must tests to confirm them. Data quality is becoming increasingly important to program success. Access to quality data helps set the stage for success, from helping sponsors determine appropriate inclusion and exclusion criteria, to enabling them to script multiple trial designs, to selecting endpoints that can withstand regulatory and payer oversight.

„Robust data quality enables smarter trial design from the start, helping sponsors plan trials that generate reliable and actionable insights the first time,” says Beck. „Not only does this help accelerate timelines, it also helps sponsors accomplish what matters most: delivering innovative, life-changing devices to the patients who need them most.”