Stakeholders Suggest COA Compendium Improvements
Stakeholders say they appreciate the work FDA has put into its pilot Clinical Outcome Assessment (COA) compendium, and offer ways in which it can be improved. The Association of Clinical Research Professionals (ACRP) says that the compendium focuses on current FDA guidance. “Without validated outcomes tools, the probability that any COA provides meaningful, scientifically useful information is greatly diminished,” it says. “Therefore, ACRP suggests that the COA compendium would be improved by including validated outcome tools with appropriate references.”
Pharmaceutical Research and Manufacturers of America says it supports FDA’s efforts to create a compendium “intended to facilitate communication and to provide clarity and transparency to drug developers and the research community by collating and summarizing clinical outcome assessment information for many different diseases and conditions into a single resource.” The association says it believes the compendium’s utility could be enhanced through the frequency of its updates, changes to its format, and consistently including in the compendium COAs recommended by the agency through issued guidance documents.
In its comment, the Critical Path Institute says that additional clarification of the overall purpose and limitations of the compendium is needed, along with guidance on how the compendium is to be used or applied in practice.
The GlaxoSmithKline comment letter outlines “practical limitations” on how the compendium may be useful to drug sponsors. It cautions that the compendium may have the unintended consequence of discouraging development of novel measures that may be an improvement over older legacy measures, including those which may lack sufficient evidence that they are well-defined and reliable. It also offers additional sources for the agency to consider when looking at expansion of the compendium.
Finally, Pfizer says that the compendium’s inclusion of the terminology and vernacular used by the agency’s Clinical Outcomes Assessment staff indicates that the broader agency is adopting a consistent language, which should aid in consistent understanding and communication across the units within FDA that will apply policies on clinical outcome assessments. Pfizer also says that while it can be helpful to have all the compendium information in one place, “much of the information will not provide sufficient transparency to inform sponsors’ internal decision-making about development prior to discussions with the agency, particularly in the absence of a procedure for having discussions with the agency about the use of COAs.”