Specifically, EMA wants the text to reflect the emergence of the classification “non-radiographic axial spondyloarthritis.” This term categorizes patients with the chronic inflammatory disease axial spondyloarthritis who do not meet the criteria for ankylosing spondylitis as set out in the modified New York rating, but nonetheless have disease activity and functional impairment comparable to people with the latter condition. EMA has addressed the specific needs of this subpopulation of axial spondyloarthritis patients in its revised guideline.
The guideline calls for treatments for axial spondyloarthritis to demonstrate efficacy in both patients with the non-radiographic form of the disease and those who meet the criteria needed to be diagnosed with ankylosing spondylitis. As such, EMA is proposing that clinical trial sponsors enroll patients with both conditions in sufficient numbers that sub-group analyses are possible. One goal of these analyses is to demonstrate that the response of the subgroups to the treatment is consistent with the overall outcome.
To perform such analyses, sponsors first have to know the status of the patients they enroll. The 2009 Assessment of SpondyloArthritis International Society criteria that defined the non-radiographic form of the condition are validated for the selection of patients in clinical trials, but EMA is concerned by the high rate of false positives that are generated when using this tool in certain populations. EMA is open to sponsors enforcing additional restrictions on inclusion, such as the presence of “objective signs of inflammation” at the time of enrollment.
Officials have also used the revision of the guideline as an opportunity to propose new outcome measures and tweak their advice on the design of confirmatory trials. EMA is proposing the trial design amendments in response to changes to the list of treatments that are available for people with the condition. With products such as anti-TNFs now available, EMA is suggesting sponsors include an active comparator arm in their trials. This will allow the regulator to assess the risk-benefit balance of experimental products against those of existing treatment options.
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