Trailblazing Trials

The history of clinical trials in cancer traces back to the mid-1950s and the first National Cancer Institute-sponsored randomized trial for acute leukemia. Since that time, the nature of the clinical trial has remained much the same. There is a good reason why: the spirit of scientific rigor and oversight built into those early clinical trials remains, to this day, the best way for us to determine whether new medicines are safe and effective. But after 60 years, clinical trials are starting to change in ways unimaginable even 10 years ago.

We’ve entered a new era of cancer care with ground-breaking treatment approaches like cancer immunotherapy, faster genomic testing and targeted medicines developed from an explosion in knowledge about tumor biology. This means we’re asking new questions like:

  • How do we get promising medicines to patients as quickly as possible, while maintaining the rigor that has helped ensure public safety for decades?
    We're on our way.
  • Do evolving clinical trial measures (or “endpoints”) accurately capture the benefit derived from treatment?
    Sometimes, but not always.
  • Are we ready to enroll patients into trials based on similarities in tumor biology (e.g., biomarkers) rather than the tumor’s location in the body?
    The jury is still out.
  • How do we prove that new treatments can help people with cancer live not just longer, but better lives?
    With new measures called patient-reported outcomes.

Here are five trailblazing trends in cancer clinical trials that we believe are helping to answer these questions.

How Cancer Clinical Trials Are Adapting To Keep Pace With Science

1. Looking at Novel Endpoints

We’re finding new and potentially faster ways to measure whether a medicine is working, enabling us to get that medicine to patients faster.

2. Embracing Different Statistics

Newer trial designs use alternative statistical methods to simultaneously evaluate multiple treatment options, potentially allowing for quicker results while maintaining scientific rigor.

3. Making the Most of Medicines

Approaches like basket trials and adaptive trial designs use biomarkers to increase the amount of useful data we can generate in a single study.

4. Focusing on the "Whole Person"

An increased focus on patient-centered care means the industry and the FDA are striving to include patient-reported outcomes as measures of effectiveness in clinical trials.

5. Harnessing Big Data

Initiatives such as ASCO’s CancerLinq aim to complement clinical trial data with “big data” from real world practice to deliver the most informed care possible.

These trends are changing our approach to clinical trials. I’m looking forward to seeing how they will help us continue to improve the way we bring medicines to patients.