Medidata Presents Groundbreaking Research for Cancer Treatment at the 2024 ASCO® Annual Meeting

In collaboration with Bristol Myers Squibb, Medidata AI presented a research poster, “Association between treatment (tx) response and PFS and OS in R/R chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL): a 12-month landmark (LM) meta-analysis” at the 2024 American Society of Clinical Oncology (ASCO)® Annual Meeting.

Learn more about this cutting-edge research in the below Q&A with Jacob Aptekar, VP, Trial Design Solutions, Medidata AI.

How Does this Research Impact the Field of Cancer Research?

“The question we're trying to help sponsors answer is: why would patients take my medicine? That's the core question of all clinical development. When you’re developing drugs for cancer in particular, there's many signals early on in the treatment journey that may indicate a drug is going to be effective and that patients taking that drug are going to experience a positive long-lasting effect.”

We worked with Bristol Myers Squibb (BMS) to establish that a durable treatment response early in the treatment journey for chronic lymphocytic leukemia (CLL) was highly correlated to, and likely causing, longer overall survival. We were able to link treatment response over a short period of time to survival over a much longer period of time.

The impact is that this evidence—along with other evidence from BMS—will enable the drug to become available several years earlier than it would be under other circumstances. We were able to get this drug to market much more quickly.

How Will this Research Benefit Sponsors and Patients?

The work we've done with BMS for CLL to support their CAR-T therapy is informing the work that we're pursuing in other indications and with other treatments. We’re collaborating with other sponsors and establishing surrogate endpoints. Once these surrogate endpoints are established, anyone can use them. For example, from this point forward within CLL, this complete response at a twelve month landmark will be considered acceptable from a regulatory standpoint.

We're working hard to identify the appropriate surrogate endpoints across many different indications and making sure they can be validated with the @FDA to accelerate clinical development for all stakeholders.

For sponsors, this means they now know their drugs can go to market several years earlier than they would have otherwise, and they're in position to be  first to market in a new indication.

The impact on patients is that there's better access to new treatments sooner, which will hopefully result in more patients treated successfully and ultimately, better quality of life.

Why Is Medidata Uniquely Positioned to Conduct This Research?

Since Medidata has worked with so many sponsors over a long period of time and has run over 33,000 clinical trials, we already have a large amount of data across all of these different indications.

Typically, when you're performing surrogate endpoint work, you're seeking to validate that surrogate endpoint for a mechanism of action to support a molecular entity that has never been tested before. But, to accomplish that, you're trying to show that the historical data establishes that the short-term treatment response correlates to long-term survival in general, for any mechanism of action. Because of the strength of this association in historical data for many different mechanisms of action, we make the case that  therefore it's reasonable for the FDA or EMA to expect that they will be related for a new mechanism of action.

The fact that we have lots of historical data from many different development programs and mechanisms of action in our database makes Medidata one of the best sources of evidence to do that research. And we wouldn’t have that ability if we hadn't been conducting this research for so long with so many sponsors.

What’s Next?

The next step is to combine the development of surrogate endpoints with the work we do in benchmarking, external control arms, and virtual twins to produce individual, patient-level predictions. For an actual patient who's enrolled in a clinical trial—what would their treatment journey have looked like under different scenarios?

This will allow sponsors to make well-informed decisions on how to accelerate drugs through the pipeline, because they'll be able to understand the likelihood of  outperforming those benchmarks. Additionally, it will be valuable to produce credible evidence showing that a particular new therapy outperforms the standard of care across a range of different treatment scenarios.

Learn more about Medidata’s presence at the 2024 ASCO® Annual Meeting.

Enjoyed this article? Click here to share it with your network.