User Fee Bill Aims to Correct Court Decisions
A House Energy and Commerce Committee legislative package (see story) to reauthorize FDA user fee agreements and enhance other agency program areas appears to address an agency predicament caused by a 2021 appeals court decision that has disrupted FDA’s orphan drug program. Last week, FDA officials testifying at a 4/26 hearing on reauthorizing user fees urged a Congressional fix to ensure orphan drug development is not impacted, especially for pediatric indications (see earlier story).
A Hogan Lovells legal analysis earlier this year said that the 11th Circuit Court of Appeals decision upholding orphan exclusivity for Catalyst Pharmaceuticals’ Firdapse (amifampridine phosphate) “appears to undermine FDA’s indication-specific approach to orphan exclusivity, providing exclusivity holders (and those on deck for approval) potentially broader exclusivity than previously expected.” In an online post, the attorneys also said the decision may significantly increase the burden for second-in-time sponsors seeking to avoid a competitor’s orphan exclusivity.
According to a summary of the proposed legislative package, Section 811 would amend Section 527 of the Food, Drug, and Cosmetic Act regarding orphan drugs “to provide clarity that exclusivity applies only to the specific indication or use approved by FDA under this section, not the entire rare disease or condition for which the drug was designated, consistent with FDA’s long-held interpretation of the law.”
The legislation would also correct another court decision that disrupted how FDA regulates contrast agents. The dilemma stems from a 4/2021 decision from the U.S. Court of Appeals for the District of Columbia Circuit in Genus Med. Techs., LLC v. FDA, which affirmed a district court ruling that FDA does not have discretion when a combination product meets the definition of being both a device and a drug (see earlier story). Proposed Section Sec. 803 would deem that all contrast agents, radioactive drugs, and over-the-counter monograph drugs are to be regulated as drugs and not medical devices.
Additionally, the proposed legislation aims to push the agency’s adoption of real world evidence and real world data and its use in regulatory decision making. Proposed Section 805 would require FDA to issue guidance addressing the use of real world evidence and real world data, including that obtained for drugs and devices authorized for emergency use during the Covid-19 public health emergency. The guidance would outline how such data can support drug and device approvals and clearances.