From Alexander Gaffney in a RAPS article:
Title II of the 21st Century Cures legislation seeks to build on FDA’s recent launch of a Patient-Focused Drug Development (PFDD) program. That program, launched in 2012 under the Food and Drug Administration Safety and Innovation Act (FDASIA), is meant to incorporate patient preferences into FDA’s regulatory decision-making. The idea behind the proposal is that different patients—even those suffering from the same disease—have varying levels of risk they are willing to accept.
As legislators explained in a white paper accompanying the last draft of the bill, “No one understands a particular condition or disease better than patients living with it.” Accordingly, the bill would require FDA to establish a structured framework for the meaningful incorporation of patient experience data into the regulatory decision-making process, including the assessment of desired benefits and tolerable risks associated with new treatments.
That measure is likely to win support at FDA, which has sought to develop similar approaches in the past and has been receptive to patient feedback. Under the approach, FDA would have to, within two years, develop and implement a process to allow companies to submit “patient experience data,” which could be used to develop a “structured risk-benefit assessment framework.” A draft guidance document would also be due from FDA within two years.