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Understanding Adaptive Clinical Trials

Testing new treatments is key to successfully treating cancer. Unfortunately, the current model for conducting clinical trials — evaluating the effectiveness of therapies on human subjects — is inefficient, expensive and suffers from high failure rates. To overcome these challenges, the practice of adaptive trials is emerging and has become one of the most promising advancements in cancer research today.

Challenges with traditional clinical trials

Currently, it takes approximately $2.5 billion dollars, 10+ years and thousands of patient volunteers to bring one drug to market.[i] Despite this high cost, over half of phase III oncology trials have negative results or fail to complete. In addition, traditional trials cannot be modified once they begin. Patients get only one opportunity and receive only one treatment.

Adaptive trials literally adapt as evidence accumulates

Adaptive trials do not require years of follow-up to determine whether a new experimental treatment is beneficial. They test multiple treatments and combinations of treatments on patients in parallel and are designed to be continuously updated as researchers use what they learn from each patient to make real-time adjustments as the trial proceeds.

They are flexible by design so adjustments can be made in terms of dosage, subject population or sample size as needed.

In an adaptive trial, if one treatment regimen seems to be more successful, researchers can increase the number of participants receiving that treatment. This is especially important for patients with aggressive cancers as life expectancies are too short for traditional trial timetables. With adaptive trials, ineffective treatments can be shut down early, and new treatments can be initiated quickly enabling patients to receive the most promising treatments faster.

In a latest article published on Science Magazine: “Congress and FDA nominee heap love on ‘adaptive trials”, GBM AGILE is featured as one of the ongoing clinical trials using this new adaptive trial design.

GBM AGILE revolutionizes brain cancer trials

Glioblastoma multiforme (also known as GBM) is the deadliest brain cancer and is widely regarded as incurable and universally fatal, killing 95% of patients within five years of diagnosis. To combat this deadly disease, NFCR is part of a robust, international coalition working on innovative ways to defeat GBM utilizing adaptive trials known as GBM AGILE.

Led by the best and brightest cancer researchers, GBM AGILE is a revolutionary global collaboration to test and develop new brain cancer treatments. Its personalized approach will allow us to accelerate the discovery of targeted treatments for individual patients. This global coalition has attracted over 150 participants from more than 40 leading cancer institutions across three continents. This revolutionary approach accelerates research for curing the aggressive form of brain cancer GBM and will serve as a new clinical research model for combating other cancers as well.

In addition to GBM AGILE, there are other adaptive trials underway for certain cancers and tumor types, with more coming soon.  Other trials include:

  • I-SPY TRIAL is an adaptive clinical trial for women with newly diagnosed, locally advanced breast cancer.
  • Lung-MAP is a large adaptive clinical trial testing several new treatments for patients who have advanced stage squamous cell lung cancer.
  • Precision Promise is the first large-scale precision medicine adaptive trial for patients with pancreatic cancer.

While adaptive trial features are not yet commonplace, Congress and the FDA are focused on accelerating their development to benefit patients.