Building A Better Patient Registry: Keep It Simple, But Let It Be Big
This article was originally published in The Pink Sheet Daily
Executive Summary
As outcomes research, comparative effectiveness and drug safety worries increasing drive the need for post-market data, sponsors are turning to patient registries for a low-cost way to generate valuable clinical information.
The evolving data needs of payers and regulators are encouraging product sponsors to rely more on patient registries with the hope generating vital information at a lower cost. The changing landscape has made sponsors reconsider the kinds of products, patients and collection methods that they should use for registries. To build the best possible registries, sponsors will need to balance the desire for additional data with the simplicity that makes registries so simple and appealing in the first place. "With increased emphasis on comparative effectiveness and outcomes research, the notion of large simple studies is gaining considerable ground," Health Decisions CEO Michael Rosenberg said. Registries work best when they are designed so that they are "low-cost and measure real-world outcomes," Rosenberg said. But in order for them to be most effective, "regulators need to become comfortable with the notion that real world circumstances differ from the rigidly controlled environment of a clinical trials setting." Regulators do indeed appear to be warming up to registries, and FDA is increasingly using them as a post-market safety requirement (Also see "Registries Rising: FDA Looking At TNF Inhibitors; AHRQ Updates Standards" - Pink Sheet, 24 Aug, 2009.). "Optimally, registries should be designed on two levels," Rosenberg said. "The first is to collect relatively simple information, often directly from subjects, and continually examine collected data. This serves as a trip-wire of sorts. When outcomes of interest are found, whether planned or unplanned, a more careful look can be taken by identifying a subset of subjects and more closely examining results, often with a case-control approach." "The appeal of registries is that they generally use large cohorts, which makes it easier to control for potential confounding factors and allows a more detailed investigation when something of interest is found," Rosenberg noted. Their drawback, however, is that to be successful, they must collect large amounts of relatively simple information at modest expense. In practice, the most common problem encountered is the "tendency to overload registries with too much detail," Rosenberg said. "This contributes to cost and discourages continued involvement by subjects. Costs can be driven up enormously by focusing on medical intermediaries, in contrast to subjects who are followed up with directly through mail and other means." [Editor's Note: The Pink Sheet is sponsoring a webinar on Dec. 9 discussing how to make the most of the changing registry landscape. The event will feature several industry experts, including Genzyme VP Joanna Haas. For more information, please visit http://www.windhover.com/ezine/html/ac1209-lp.htm, call (800) 332-2181 or email [email protected].] In contrast to Health Decisions' emphasis on low-tech postal follow-up, the contract research organization Clinipace feels that "technology should serve as the underpinnings of any registry project." "Since post-marketing research has a 'time is of the essence' aspect to it - particularly if it is safety oriented research - technology-based data collection and management is the only viable solution when signal detection is so critical," the CRO said. Regardless of how high-tech a registry might be, "one of the challenges in conducting these types of programs is not to execute them as a clinical trial," according to Covance Senior Project Manager-Registries and Observational Studies Eunice Franklin-Becker. "For most individuals that have done clinical trials, it is instinctive to apply the same criteria such as 100 percent source data verification, routine monitoring visits, clinical and laboratory assessments, mandated visits, and extensive inclusion/exclusion criteria to ensure data quality," she said. "Observational data collected in a registry requires the same level of quality," Franklin-Becker emphasized. "However, one must recognize that the value of this data lies in its reflection of the real world standard of care. As such, the same consistencies in data may not exist in a standard of care setting that exist in a controlled clinical trial environment." While some CROs would like to see FDA develop clearer guidance to help ensure sponsors are meeting the agency's desires, Franklin-Becker said, "We would not recommend more regulatory guidance" on registries and observational studies since such a move likely "would only limit the flexibility and potentially add an undue burden to sites and influence standard of care practices. However, any guidance that reduces patient risk would be a positive enhancement." -M. Nielsen Hobbs ([email protected]) |