Clinical trial study design: 'one size does not fit all'

By Melissa Fassbender contact

- Last updated on GMT

Researchers asked how patient preferences and burden of disease can be incorporated into randomized clinical trials (RCTs). (Image: iStock/BernardaSv)
Researchers asked how patient preferences and burden of disease can be incorporated into randomized clinical trials (RCTs). (Image: iStock/BernardaSv)

Related tags: Clinical trial

The rate of false positives in clinical trials should be set higher for certain hard-to-treat cancers, suggest researchers.

Andrew Lo, Ph.D., the study's senior author, told Outsourcing-Pharma.com the study’s goal was to show that "one size does not fit all​" when it comes to interpreting clinical trial results.

For patients with terminal illnesses and no existing therapies, taking a bigger chance of false positives might be a perfectly acceptable trade if there's a reasonable chance that a new therapy will help​,” Lo said.

The study, recently published​ in JAMA Oncology, examined the application of Bayesian decision analysis (BDA) to a data set of 10 clinical trials from the Alliance for Clinical Trials in Oncology​.

As Lo explained, “Bayesian decision analysis is a well-known technique developed by statisticians to balance the potential harm of false positives (approving ineffective drugs) against the potential harm of false negatives (rejecting effective drugs)​.”

According to Lo, the key takeaway is that for certain types of hard-to-treat cancers with no current therapies, the rate of false positives should be set “much higher​” than 2.5% or 5%.

By adapting the rate of false positives to the specific clinical setting – higher rates of false positives for more dire circumstances – more therapies will be approved, thereby incentivizing biopharma companies to produce more innovative products for patients​,” Lo explained.

Additionally, he explained the Bayesian decision analysis (BDA) framework “allows all stakeholders to weigh in while providing the FDA with a rational, transparent, systematic, objective, and repeatable process for making decisions that incorporate patient preferences​.”

The 21st Century Cures Act requires the FDA to consider patient preferences and “BDA provides a straightforward platform for doing so​,” added Lo.

Our results show that incorporating patient preferences into the drug approval process is feasible and can greatly increase innovation in biomedicine.”

According to the researchers, the framework is flexible enough to include various stakeholder perspectives. Additionally, the authors have provided open-source software so their analysis can be re-run under a different assumption set for risk preferences, disease burden and prevalence, and value delivered to patients.

Title​: Use of Bayesian Decision Analysis to Minimize Harm in Patient-Centered Randomized Clinical Trials in Oncology

Publisher: JAMA Oncol.

doi​: 10.1001/jamaoncol.2017.0123

Related news

Show more

Related products

show more

What should a clinical metadata repository do?

What should a clinical metadata repository do?

Formedix | 26-Jul-2021 | Technical / White Paper

Choosing a clinical metadata repository (MDR) software can be a tough task as the capabilities and features of your chosen MDR could make or break your...

Adapting supply chains to new ways of working

Adapting supply chains to new ways of working

World Courier | 15-Jul-2021 | Technical / White Paper

COVID-19 has changed the way we operate. We have adapted our supply chain solutions to meet our clients’ requirements while following World Courier standard...

Empowering Clinical Trial Sponsors with Analytics

Empowering Clinical Trial Sponsors with Analytics

PerkinElmer | 14-Jul-2021 | Technical / White Paper

Is your Clinical Data Review creating problems or solving them? Take Control of Your Data. PerkinElmer Informatics Clinical Solutions, powered by Tibco...

What are the building blocks of DCTs?

What are the building blocks of DCTs?

Medable | 26-Apr-2021 | Technical / White Paper

How to create a seamless experience across eConsent, eCOA and more.

Over the past year, tremendous progress was made toward digitizing and...

Related suppliers

Follow us

Products

View more

Webinars