A Guide to Successful Clinical Trial Design

Clearly defining the research question – Every clinical trial design begins with the fundamental clinical research question. Over time, the team must refine it from a vague concept to a testable and quantifiable hypothesis. Only after the question is well understood can a research team begin identifying the intrinsic challenges of the study.

Minimizing variation – The greater variation throughout the trial, the harder it will be to narrow down any treatment effect. Ways to reduce variation include:

Promoting randomization and stratification – The more randomization that’s introduced into a trial design, the less chance there is for bias to creep in. And a stratified randomized trial helps ensure that treatment groups or study subjects are balanced regarding potential confounding variables.

Blinding – As one of the most fundamental design elements of any clinical trial, blinding ensures that a study’s participants and researchers are unaware of the assigned intervention, which prevents biases or behavioral changes from muddying the results.

Placebos – Similarly, placebos or sham interventions, help ensure that blinding is maintained, though such design features are typically limited to pharmaceutical interventions and not medical devices.

Selection of the control group – A control group gives researchers important data of what would have occurred had the study’s participants either not been treated or been given a different intervention. There are three types of control groups:

Selection of a population – Researchers have to consider the intended target use for the intervention. A study on a drug that helps men aged 65+ manage their heart pressure would be moot if the tested population consisted of young adult women.

Selection of endpoints – An endpoint is intended to address the main objective of the trial. That means it needs to be, “clinically relevant, interpretable, sensitive to the effects of intervention, practical and affordable to measure, and ideally can be measured in an unbiased manner.” Typically, endpoints fall into one of three categories:

Sample size – Statistically speaking, it’s impossible to draw conclusive results from a sample size that’s too small. Similarly, a sample that’s too large will be difficult to control and costly to conduct. As a result, researchers must calculate a happy medium based on the planned analyses.

Pharmaceutical clinical trials – Every prescription drug on the market must be approved by various regulatory authorities, such as the USFDA. For that, it must undergo rigorous pharmaceutical clinical trials that include an 11-step approval process:

Oncology clinical trials – Cancer-related clinical trials are intended to test new ways to:

Medical device clinical trials – Medical devices go through a different approval process than a drug study. Typically, it starts with a small pilot study, followed by a larger pivotal trial. If successful, the manufacturer can then submit the device for FDA approval. From there, manufacturers often choose to also conduct post-marketing trials.

Form a testable hypothesis – Remember, the entire process begins with clearly defining your research question in terms of clinical interest. In doing so, you can logically identify the appropriate study design and the necessary components to test your question.

Recruit the right patients and associated professionals – A robust study requires engagement from a diverse group of stakeholders, including researchers, regulators, doctors, scientists, execs, sponsors, and patients.

Design the study – At this stage, communication and collaboration are critical. There needs to be an easy way for patients and investigators to participate and provide feedback. Here, engagement technologies can help optimize study design and engage all crucial stakeholders in the process.

Plan the phases – Typically, a clinical trial will need to undergo four primary phases, including:

Set a budget and milestones – For a lengthy trial to be effective, you must regularly monitor your costs and measure your performance. This allows you to make improvements as you go instead of after the trials. It also creates accountability between all stakeholders.

Test the design – Before trials begin, perform an environmental scan to check for unanticipated factors or events that could throw off the trial or poke holes in the results.