To be truly patient-centric, it’s important to think about how clinical trials can be more efficient in developing care options. This increased efficiency can be accomplished in a variety of ways, such as using technology to make clinical trials more accessible for patients, drawing on patient feedback to inform our strategies, and exploring alternative models to how we conduct research.
One of these alternative models being implemented is offering new approaches to the study protocol. As a reminder, a protocol is a written document that instructs the primary investigator and study coordinators on how to execute a trial, describes how a trial is conducted to ensure its safety and data integrity, and shares the trial’s intent and desired outcomes with regulators. In February 2019, the U.S. FDA shared a draft guidance for “master protocols” as part of their efforts to help speed research along.
Master Protocols are designed to answer more questions with greater efficiency. Three types of master protocols are basket, umbrella, and platform trials. A platform trial is a clinical trial in which multiple treatments or diseases are evaluated within the same protocol. A basket trial is when one therapy is tested on more than one disease or subtype of disease, and an umbrella trial is where multiple therapies are tested for a single disease with one or more subtypes.
But what does all of that mean for patients?
What works for one participant may not necessarily work for another, which is why, if you’re considering a clinical trial, we encourage you to explore all of your options. Below, we share a few pros and cons to weigh when looking into a platform trial:
Pros of a platform trial:
You can feel like you’re part of something bigger. When you participate in a platform trial, you’re involved in the research for more than one conditional study. That means that not only would you offer researchers a greater pool of data to inform drug development for those with your condition, but you may also have more possible treatment options by participating in a master protocol.
The process is very similar to traditional clinical trials. The main difference in a platform trial compared to a standalone clinical trial is how the study documents are written. Because a master protocol essentially includes more than one clinical trial, you may have to read and sign more than one informed consent. Once the up-front paperwork is complete, you’re ready to go and would likely not notice any major differences to other, more traditional clinical trials.
If you want to try a few different options, it’s a more consistent journey. Many platform trials may let you test multiple potential treatments, which might be an attractive route to take if you’d like more options, especially if you have a chronic condition. For instance, once you’re in a platform trial, you can get used to the consistency of the master protocol as you make the transition from one potential treatment to another. By contrast, with a more traditional approach, you’d have to participate in multiple stand-alone trials and relearn the different assessments and devices used in each trial. This potential benefit is based on the design of the master protocol, so ask your doctor for more information as you’re considering your options.
Cons of a platform trial:
You may not get to choose which sub-study you’re a part of. Say you join a platform trial and there are three treatments available, and Trials A, B, and C are all ongoing. For some master protocol designs, if you wanted to join Trial C and didn’t want to do Trials A and B, you wouldn’t be able to decide for yourself, because the treatments are randomly assigned. When you sign the multiple informed consents for a master protocol, you may be consenting to all of the potential treatment options.
There may be more paperwork in the informed consent process. If multiple potential treatments are recruiting patients in a master protocol, there is more paperwork that goes into signing up for a platform trial. The informed consent process is critical for ensuring that the participant is aware of everything they are signing up for. While this paperwork is certainly necessary, some may find the extra materials overwhelming.
Do you think a master protocol might be the right fit for you? Consider the pros and cons and discuss these with your research team.
Lilly is conducting clinical research using master protocols to investigate treatments for pain and for pediatric cancer. Check out the studies listed here:
Studies for pain (part of the chronic pain master protocol):
- Chronic Pain Master Protocol (CPMP): A Study of LY3016859 in Participants With Diabetic Peripheral Neuropathic Pain
- Chronic Pain Master Protocol (CPMP): A Study of LY3016859 in Participants with Osteoarthritis
Studies for pediatric cancer (part of the CAMPFIRE master protocol):
- CAMPFIRE: A Study of Ramucirumab (LY3009806) in Children and Young Adults With Synovial Sarcoma
- CAMPFIRE: A Study of Ramucirumab (LY3009806) in Children and Young Adults With Desmoplastic Small Round Cell Tumor
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