Rare Disease Report

Advancing Treatment for Myelodysplastic Syndrome Through Collaboration

OCTOBER 23, 2018
Krista Rossi
Collaboration is not only beneficial, but necessary for rare diseases, and a team of investigators has found that collaborative efforts among several leading medical institutions have helped increase the study of new medications for myelodysplastic syndromes (MDS).

At the 2018 National Organization of Rare Diseases’ Rare Diseases & Orphan Products Breakthrough Summit (NORD Summit) held in Washington, DC, the AAMDSIF team of investigators presented data on a new model for MDS clinical research in the United States—the MDS Clinical Research Consortium (CRC).

The CRC began in 2012 and is comprised of 6 leading medical institutions—the Moffitt Cancer Center, Cleveland Clinic, Dana-Farber Cancer Institute, Md Anderson Cancer Center, Johns Hopkins Medicine, and Weill Cornell Medicine—all working together to collaborate and share resources to conduct unique studies and trials that can advance treatment for MDS and improve patient outcomes.

At monthly meetings, members of the CRC decide to begin a clinical trial at all 6 institutions. Each institution then sends its data over to the Cleveland Clinic where data analysts process and store the data, which is then added to CRUSH!! MDS database to be analyzed. After this happens, conclusions are made, published, and thereby acted upon.

“Usually a trial is developed at 1 institution, and because of the CRC agreement, the other 5 go through the process of establishing that trial at their own institution,” Matthew Lesser, MS, an investigator on the study, told Rare Disease Report® in an exclusive interview. “It helps increase the number of patients involved in a given trial since multiple sites are involved. It also helps lower costs since all 6 sites are contributing resources to a single trial. Again, different trials occur at the same time among the 6 sites.”

The funding to support this research is secured by the Aplastic Anemia and MDS International Foundation (AAMDSIF), a patient advocacy nonprofit organization, and so, rather than having the usual indirect cost rate ranging from 28% to 80%, it was possible to set the rate at just 10%.

Each institution is provided with support for a principal investigator, clinical research nurse, data manager, and clinical research fellow. With this additional support, per patient expenses are reduced from about $30,000 to just $8,000, the study authors write. As such, more money can be put towards research rather than overhead. Since the CRC is not federally funded, it is able to respond “more nimbly” to research opportunities, they add.

Through advocacy and outreach, the AAMDSIF is working on increasing access to eligible patients. The organization is also serving as administrator of the effort, providing fiduciary oversight and collaborative accountability, in addition to helping to convene initiatives between industry and academia and promote consortia achievements throughout the research and health care community.

The model is successful for several reasons, according to the authors, in that it provides a standardized way for conducting trials and formatting data, it includes use of a central, collective database that included a biorepository, monthly conference calls and periodic face-to-face meetings among CRC members are held, and oversight is provided by an external advisory board.

Since the CRC’s inception, 108 manuscripts were published, 10 multi-center clinical trials were conducted or in the process of being conducted, 30 MDS research careers were initiated, and 80 abstracts have been submitted to the American Society of Hematology (ASH). Additionally, there has also been a substantial growth of 468% in the database, which included the CRUSH!! MDS database and data collected from the clinical trials conducted within the CRC.

The team concludes that the CRC is innovative in its structure, governance, funding, responsiveness, cost containment, and focus on developing future talent. The CRC’s unique design and funding support enables projects to be designed, evaluated, and approved or re-designed in a revolving, continuous cycle, the authors write.

In addition, development of the therapeutic and non-therapeutic trials, pilot projects, and special projects that comprise the Consortium portfolio is promoted by the infrastructure support provided by the funder to the CRC. 

Noted advantages with the CRC was a reduction in “red tape,” larger cohorts in clinical trials, faster recruitment, and immediate availability of data from existing registries and biorepositories.

“To date, there are 10 multi-centered trials among the 6 institutions,” Lesser added.

Although the model shows great promise, authors postulate future challenges to be acquiring sustainable funding, keeping the centers and investigators involved motivated, keeping data updated, and overcoming international collaboration and regulatory hurdles.

Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.