Award will allow U-M to create a method to share intelligence on rare disease treatments

May 9, 2016
Contact: Laurel Thomas

An icon representing doctors sharing information. (via NounStock)ANN ARBOR—A $900,000 funding award to the University of Michigan School of Public Health from the Patient-Centered Outcomes Research Institute (PCORI) will allow researchers to develop methods to analyze treatment evidence that could make a difference in the lives of the 25-30 million Americans living with rare diseases.

A team led by Kelley Kidwell, research assistant professor in the Department of Biostatistics at the U-M School of Public Health, and Roy Tamura at the University of South Florida Health Informatics Institute will study the use of sequential multiple assignment randomized trial (SMART) designs in small patient populations, with a goal to provide valuable information to patients and the clinicians who treat them.

“The ultimate goal is to get patients better treatments,” Kidwell said.

Rare diseases are those found in 200,000 or fewer people. Altogether these diseases number more than 6,800, most of which have no approved treatments.

The development of methods for SMART designs in small patient populations could result in increased evidence about various treatments physicians prescribe. The design incorporates individual patient responses to treatment and adapts further treatment based on that information.

The researchers will first test their methods using data from a trial with patients who have Isolated Skin Vasculitis, a condition that causes inflamed blood vessels in the skin. In the SMART design, a group of individuals are followed through several stages. The proposed methods will involve testing between multiple treatments to try to find the best.

The team is calling the program the patient-centered small n Sequential Multiple Assignment Randomized Trials, or snSMARTs. The first snSMART in the field is named ARAMIS (A Randomized Multicenter Study for Isolated Skin vasculitis).

“The eventual goal is also to make this trial design and corresponding analytical methods available to other biostatisticians and researchers so they can use them to find effective treatments for any rare disease or disorder with small samples,” Kidwell said, adding that very few traditional clinical trials are conducted on these diseases because they impact small numbers of people.

“Although SMART designs have been successfully used in other applications, the research questions of interest and the analytical methods needed to address these questions in small patient populations need to be studied in their own right.”

SMART designs have been used previously in mental health and oncology research.

The Health Informatics Institute at the University of South Florida is the Data Coordinating Center for the NIH Rare Diseases Clinical Research Network. This partnership will facilitate the application of the study findings into actual clinical trials.

“This project was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other stakeholders, but also for its potential to fill an important gap in our health knowledge and give people information to help them weigh the effectiveness of their care options,” said PCORI Executive Director Dr. Joe Selby. “We look forward to following the study’s progress and working with the University of Michigan School of Public Health to share the results.”

The U-M study was selected for PCORI funding through a highly competitive review process in which patients, clinicians and other stakeholders joined clinical scientists to evaluate the proposals. Applications were assessed for scientific merit, how well they will engage patients and other stakeholders, and their methodological rigor among other criteria.

Kidwell’s award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.

PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed health care decisions.


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