Study Supports Alternative Method for Estimating Treatment Effects in Clinical Trials
A Lupus Foundation of America (LFA) study released at the 2018 American College of Rheumatology (ACR) Annual Scientific Meeting supports using more novel analytic approaches to deal with the common and critical challenge of dealing with missing data. This may be due to patients dropping out or missing follow up appointments in clinical trials. One missing data method, called multiple imputation (MI), has been used in other disease areas successfully, and the current study shows the potential advantages of this method in evaluating treatment effects in lupus trials using data from the LFA-Collective Data Analysis Initiative (CDAI). The findings add to the growing body of knowledge of the LFA-CDAI, started in 2009.
Mimi Kim, M.D., LFA-CDAI Co-chair and lead analyst comments on this research: “One of our goals is to increase awareness in the lupus clinical trial community of the available robust and powerful statistical tools that can help address some of the methodology roadblocks that may have led to past trial failures. Missing data can make it hard to detect treatment signals so this issue needs to be handled properly to maximize our ability to identify effective new therapies.”
In the research abstract, Missing Outcomes in SLE Clinical Trials: Impact on Estimating Treatment Effects, Dr. Kim analyzed data on 279 patients with lupus who took part in a study for 52 weeks. The study showed how trial conclusions can change depending on the method used to address missing data. Using the multiple imputation method led to greater differences seen between comparison groups and more precision in results. This method has been widely applied to study other diseases, including cancer and heart disease.
Lupus trial design and analysis is very complicated, just like lupus. The CDAI initiative analyzes a large database of past studies, which provides the perfect opportunity to learn from the data of past trials and improve how future studies are designed, conducted, and evaluated.
Additional milestone findings over the past nine years from CDAI surrounding lupus clinical trials, include:
- Background medications taken by people with lupus during trials impacts the response and flare rates in the placebo groups
- Shorter Phase 2 trials may not be effective at predicting outcomes in longer trials
- Response rates in the “placebo” groups are significantly affected by how severe a person’s disease is, how aggressive their background treatments are, implicating the need to carefully design how participants are selected or grouped
- Proposing alternative methods to assess treatment effectiveness in a clinical trial that takes into account that patient response varies over time
“These important findings help to drive the Lupus Foundation of America’s commitment to dig deeper when it comes to testing new treatments for lupus. Through new discoveries and collaborations with multiple companies, we’re helping to improve the design of future clinical trials – a critical step towards new therapies and a potential cure,” said Stevan W. Gibson, president and CEO of the Lupus Foundation of America. “We’re focused just as much on finding what works, as why something doesn’t work, and getting to smart research for faster success across the community.”
Dr. Kim will present her findings from this study during an oral presentation on October 24 from 9:45 – 10:00 am at the ACR Meeting. The Lupus Foundation of America will also tweet about the findings from their research studies, as well as other important breaking announcements coming out of the world’s premier rheumatology conference. More than 50 Lupus Foundation of America funded researchers will be presenting on findings from their lupus studies. To stay connected with the LFA and receive the latest research news, visit our Twitter feed.