Study Finds that Mesothelioma Patients Could Benefit from Smaller, Faster Clinical Trials
Clinical trials continue to be one of the best options for mesothelioma patients struggling to find a new, effective treatment. Unfortunately, many patients shy away from participating in them due to the long duration and inconvenience of the majority of the trials, leaving some trials with too few participants. In a new study released this month, however, researchers believe “running smaller, faster trials with less stringent evidence criteria” could improve patient survival and get new medicines to patients more quickly.
Mesothelioma, a rare, asbestos-related cancer of the lining of the lungs, abdomen or heart, is highly aggressive and is resistant to many cancer treatments. The prognosis for mesothelioma patients is usually grim: the average survival time varies from 4 – 18 months after diagnosis. For the close to 3,000 Americans diagnosed with the disease each year, clinical trials may offer them a breakthrough treatment not yet available through their physician.
Researchers from The Mayo Clinic and the Institut Gustave-Roussy, Villejuif, France found that with the move towards personalized medicine and patient-centric treatments the “traditional large-scale clinical trial” may no longer be the most effective format when working with rare diseases.
“We found that there were important gains in survival attributable to a strategy of conducting more trials with smaller sample sizes and relaxed evidential criteria compared with those required under traditional trial designs,” said Dr. Marie-Cécile Le Deley, lead researcher and Associate Professor of Clinical Epidemiology and Biostatistics at the Institut Gustave-Roussy, Villejuif, France.
Le Deley believes that due to the uniqueness of rare diseases and other cancers, focusing on targeted, specific therapies is the best approach for finding effective treatments. But, Le Deley and the researchers believe the ability to validate the promising agents can be hindered by the requirements of running a standard clinical trial using many patients and taking years to complete.
According to a letter from the National Organization for Rare Disorders to the editors of JAMA in response to a study about rare disease clinical trials, “rare diseases by definition have small patient populations; [clinical] trials in such populations will commonly have fewer patients than those for more prevalent diseases.”
The researchers do admit that the downside is that trials using this approach reduce the certainty of the findings. They add, though, that errors can be quickly found and identified through conducting many more short trials.
The authors of the study make it clear that this approach to clinical trials has a place with personalized medicine, and they are not encouraging abandoning the traditional approach for all clinical trials. They encourage smaller, targeted trials where they can “lead to quicker results, and in the long term, greater gains.”
If someone is suffering from mesothelioma, they should check with their physician to determine if any current studies exist for their particular case. When determining whether to participate in a trial it is important to work with their medical team to determine if the benefits outweigh any risks.
Source: Press Release: Smaller, faster trials can improve cancer patient survival; new drugs could become available more quickly