Pragmatic approach may be the way forward for clinical trials
That a new cancer trial is about to start in the US is positive but not out of the ordinary, right? Except it is. This trial will be one of the first undertaken in a real-world clinical setting and represents a new model for future clinical trials.
The US’s National Cancer Institute (NCI) has helped launch a phase 3 clinical trial, the Pragmatica-Lung Study, investigating the use of a two-drug treatment for people with advanced (stage 4) non-small cell lung cancer (NSCLC). What makes this trial different is that it’s one of the first to adopt a ‘pragmatic’ approach.
Pragmatic clinical trials (PCTs) are designed to evaluate the effectiveness of therapeutic interventions in real-life clinical situations, whereas explanatory trials test whether an intervention works under ideal conditions. Because of how they’re run, PCTs produce results that can be generalized and applied to everyday settings.
While the more common randomized controlled trials (RCTs) – which tend to be more explanatory than pragmatic – are the gold standard for studying therapeutical and preventive interventions, they’re costly. A 2018 review of the cost of RCTs found that the estimated overall costs of an RCT ranged from US$43,000 to $103,254 per patient.
Using the PCT model, the National Institutes of Health (NIH), of which NCI is a part, and the FDA are working together to modernize clinical trials by removing many of the barriers that would prevent people from joining.
“This study is designed to eliminate potential barriers to enrollment and provides a model for increasing diversity and enrollment in clinical trials,” said Monica Bertagnolli, director of NCI.
Because PCTs are undertaken in the healthcare setting – hospitals, nursing homes, clinics, or physician practices – they generate rigorous, clinically applicable evidence to guide medical decision-makers. Importantly, despite their relaxed enrollment criteria, PCTs are still diligent about participant safety.
“Making trials more accessible, while upholding rigorous scientific and safety standards, means that more healthcare practitioners and patients will have an opportunity to participate,” said James Doroshow, director of the NCI’s Division of Cancer Treatment and Diagnosis.
The Pragmatica-Lung Study will enroll up to 700 participants to evaluate whether combining two FDA-approved medications, ramucirumab (Cyramza) and pembrolizumab (Keytruda), improves overall survival over standard chemotherapeutic treatment in people with advanced NSCLC where previous chemotherapy and immunotherapy has failed.
Ramucirumab is a targeted therapy drug called a monoclonal antibody developed for treating solid cancer tumors. It prevents the growth of new blood vessels that tumors need to grow. Pembrolizumab is a type of immunotherapy that stimulates the body's immune system to fight cancer cells.
By adopting the pragmatic approach in the Pragmatica-Lung Study, researchers hope to confirm the encouraging results from the randomized phase 2 clinical trial, the first lung cancer precision medicine trial supported by NCI.
“Pragmatica-Lung, with its critical public and private partnerships, reflects the innovative approaches NCI is taking to achieve the Cancer Moonshot goals, including reducing the cancer death rate by 50% within the next 25 years,” Bertagnolli said.
Source: National Cancer Institute
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