Medicare data United with Simple Clinical trials Expanded (MUSCLE) Network
Large-scale simple clinical trials (LSTs), also known as pragmatic clinical trials, involve simple inclusion criteria, randomization, and long-term surveillance with minimal monitoring burden. LSTs are rare in the U.S., however linking this important study design with readily available longitudinal, administrative and pharmaceutical databases has high potential to provide important information about real-world comparative effectiveness for the management and treatment of chronic disorders. Linking LSTs with Medicare data creates a much more cost-effective approach than the more traditional cohort designs that require extensive follow-up via surveys or additional visits in a clinical setting. The large sample size requirements of LSTs to obtain substantial statistical power and generalizability necessitate the participation of community-based physicians. The recent availability of linked administrative and pharmacy data from the Medicare program, now including Part D prescription drug data, provide an ideal opportunity to conduct studies of chronic disorders that have a significant public health burden for older Americans.
To address this public health challenge, the Medicare data United with Simple Clinical trials Expanded (MUSCLE) Network was created. MUSCLE is a collaborative “network of networks” provides the research infrastructure for conducting LSTs to study comparative effectiveness of therapeutics in the treatment and management of chronic diseases using linked Medicare data.
Osteoporosis therapeutics is an important example area where the options for prevention and treatment continue to increase rapidly, but very little is known about the comparative effectiveness of these drugs and emerging biologics.
On May 12 and 13th, 2011, a workshop on LSTs for Comparative Effectiveness Research in Osteoporosis was organized and held by the University of Alabama at Birmingham and the Center for Medical Technology Policy. The workshop convened a diverse group of approximately 60 stakeholders representing researchers, payers, patients/consumers, industry, government and policymakers. The goal of the workshop was to discuss the methods and strategies for conducting LSTs for Comparative Effectiveness Research on Osteoporosis and to provide feedback on the feasibility and challenges of developing a network to provide infrastructure for the successful implementation of this type of research. Among the topics discussed were: 1) Statistical Methods in LSTs / Accounting for Treatment Heterogeneity, 2) Participant Selection: Designing Inclusion/Exclusion Criteria, 3) Study Arms: Selecting Appropriate Comparators and Intervention Parameters, 4) Outcomes: Selecting Appropriate Primary and Secondary Outcome Measures and Defining Intensity of Follow-up, 5) Data Management and Medicare Data Linkage, 6) Practical Considerations for Conducting LSTs in Practice-Based Research Networks. Outcomes from this workshop included a meeting summary and position paper. Furthermore, insights from the meeting have directly influenced the proposed design of a real pragmatic clinical trial in osteoporosis treatment called the “Active comparator OsTeoporisis Large Simple Trial (ATLAST). Given the growing demand for novel methods to conduct low-cost, high quality comparative effectiveness research, the MUSCLE network approach has potential to become a new model for conducting real-world clinical trials across multiple areas of chronic disease.
Documents
May 12-13th, 2011 Meeting Summary (pdf) (156.69 kB)
Background Paper on Heterogeneity in Pragmatic Trials by Jodi Segal (pdf) (879.47 kB)