The randomized clinical trial, as a gold standard for proving the effectiveness of a new medication, has been around since the 1940s. This classic approach to medical research, using relatively simple structures such as parallel and crossover designs, has served us well for many decades. However, in more recent years, it has become apparent that this model may no longer be the best one, especially as precision medicine continues to grow.
According to the Precision Medicine Initiative, precision medicine is "an emerging approach for disease treatment and prevention that takes into account individual variability in genes, environment, and lifestyle for each person." This approach will allow doctors and researchers to predict more accurately which treatment and prevention strategies for a particular disease will work in which groups of people. It is in contrast to a one-size-fits-all approach, in which disease treatment and prevention strategies are developed for the average person, with less consideration for the differences between individuals.
These advances in medicine make it an exciting time. It also means we need to change our approaches to clinical research, so that we can more readily determine which potential new treatments will work for each individual. Two new approaches to clinical trial design are basket trials and umbrella trials, illustrated in the infographic below.
For people participating in either type of study, there are a few important points to consider:
- You might need to sign more than one informed consent form. The informed consent process is essential to understanding the expectations of the study, including details about the trial's purpose, length, procedures, risks and benefits. In a basket or umbrella trial, these components may change over time, or they may vary depending on how the participant is assigned to treatment. In order to make a fully informed decision to participate, you would need to understand each possibility before enrolling.
- There’s a lower chance of receiving placebo or standard of care treatment. In some traditional designs, the chance of receiving placebo or standard of care could be as high as 50%. But in a basket or umbrella trial, multiple therapy options are being tested, reducing the number of people who would be assigned to placebo or standard of care. In some cases the chances may be as low as 20% to 25%.
- You may have access to more therapy options at a research center. In a traditional trial, once the study is complete, participants end their treatment and may or may not have other options to pursue. With these newer designs, it opens up the possibility for a participant to end treatment in one part of the research study and then be considered for eligibility in another part of the same study—all at the same research center.
- These newer designs aren’t just for cancer research anymore. Advances in cancer research have helped us better understand the biology of certain tumor types and allow for a more targeted, precision approach to treating them, and most of the basket and umbrella trials conducted so far have been in the field of oncology. However, these approaches aren’t limited to cancer research and researchers are beginning to find ways to use these study designs in other areas, such as infectious diseases, Alzheimer’s disease and auto-immune disorders.
If you’re interested in learning more about basket or umbrella trials, drop us a line on Twitter to let us know!
Here’s a list of the scholarly articles we’ve been reading on this topic:
- Efficiencies of platform clinical trials: A vision of the future
- New clinical trial designs in the era of precision medicine: An overview of definitions, strengths, weaknesses, and current use in oncology
- Master protocols to study multiple therapies, multiple diseases, or both
- Master protocol trials in oncology: Review and new trial designs