First of two parts

Medical outcome trials—large-scale, long-duration clinical trials with hard endpoints, including morbidity and mortality—can answer critical questions about product safety and efficacy and are among the most expensive and logistically challenging trials in the entire product lifecycle. They can show long-term safety in therapies for chronic diseases such as type 2 diabetes, rheumatoid arthritis, osteoarthritis, and asthma, and efficacy in those for cardiovascular disease.

By providing real-world information, medical outcome trials can support reimbursement discussions for newly marketed products, potentially separating them from competitors or justifying higher prices. These trials also often are required by regulators to supplement data from registration trials.

The data from medical outcome trials are the best available for decision-making. However, this comes at a very high price—with a median of around 4,000 patients and a duration of about five years. Only products with considerable commercial promise can justify this level of spending.

Key considerations in protocol design for medical outcome trials include: endpoint selection; treatment options, which must build on the standard of care; trial duration (must be sufficient to accrue endpoints); inclusion/exclusion criteria; sample size (typically requiring thousands of patients for event determination); number and frequency of patient assessments; and safety reporting.

A review of data on clinicaltrials.gov in January 2015 identified 436 possible medical outcome trials by size, intervention, phase and randomization from 2003 to 2012. Cardiovascular indications were the most frequently studied (~58%), with a modest reduction in frequency over the 10-year period. Medical outcome trials involving diabetes indications increased significantly, likely due to implementation of FDA guidance. Those involving oncology (24% of the total) decreased significantly, likely due to a move from pivotal studies with mortality endpoints toward progression-free survival and tumor response.

Next: Examining best practices for operational delivery of medical outcome trials.

Written by Guest Writer Patricia Steigerwald. Steigerwald, MS, RN, is vice president, regional managing director of the Americas for Quintiles Real World Late Phase, providing strategic and operational oversight to ensure fit-for-purpose operational delivery of quality data, patient safety and regulatory adherence. She has nearly 30 years of clinical research experience, with more than 15 years in development and delivery of regulatory-compliant strategies for the conduct of real-world and late-phase programs across all therapeutic areas.

This article was reprinted from Volume 22, Issue 03, of The CenterWatch Monthly, an industry leading publication providing hard-hitting, authoritative business and financial coverage of the clinical research space. The Action Items section features short columns  focusing on actionable or how-to advice from clinical trial professionals. To submit an Action Item , please contact editorial@ centerwatch.com . Subscribe >>