‘Qualified Data Summary’ Holds Promise in Cures: Analysis
Drug and biologics developers may benefit from a provision in the recently signed 21st Century Cures Act that allows FDA to rely on a “qualified data summary” — a clinical data summary that demonstrates the safety and effectiveness of a drug’s potential qualified indication — to support approval of new uses for previously approved products, according to a new analysis from McDermott Will & Emory. The Cures Act does not, however, provide guidance on how FDA should assess whether an indication is “appropriate” for such review, the analysis says, adding that a supplemental application is eligible for summary level review if (1) there is existing data available and acceptable to FDA that demonstrates the safety of the drug; and (2) data used to develop the qualified data summaries are submitted to FDA as part of the supplemental application.
“Manufacturers should re-evaluate the extent to which they hold promising data but set aside certain FDA submissions due to an inability to run the randomized controlled trials that would likely have been required,” the analysis says. “Insofar as one or more of the additional sources of evidence recognized in the Cures Act could support an application, sponsors should consider citing the Cures Act in negotiations with the agency regarding the amount and type of evidence required to support a successful application.”
The analysis also examines the law’s provision on health care economic information (HCEI) dissemination, noting that it “expands the audience to whom HCEI may be communicated (to include payors and similar entities with expertise in health care economic analysis that select drugs for coverage or reimbursement), expands the types of analysis that may be shared (to include clinical data, inputs, clinical or other assumptions, methods, results, and other components underlying or comprising the analysis, as well as separate or aggregated consequences from the represented health outcomes), and liberalizes the previous requirement that HCEI ‘directly relate’ to approved indications (now HCEI must only ‘relate’ to such indications).” Regarding marketing and labeling, the law firm says manufacturers should re-evaluate whether the relaxed restrictions on HCEI might make it more feasible to proactively address payor and other reimbursement issues that they previously declined to discuss due to regulatory concerns.
Finally, the analysis encourages stakeholders to participate in public meetings or other outreach efforts by FDA to develop or revise applicable standards, guidance documents and regulations.