Earlier this year, the United States Food and Drug Administration (FDA) announced it would no longer pursue a proposed regulatory approach for medical devices referencing drugs (DRDs). The prior proposal would have permitted devices to be authorized for new uses with already approved drugs when the drug maker does not want to collaborate on the new use. Such new uses typically involve a change in how the drug is used or administered (i.e., a change in dose, route, or rate of administration) or use of the approved drug for an indication for which it is not approved.
The proposal, initially made in September 2017, was followed by a public hearing and comment period. While the proposal was made to counteract the unique public health, scientific, regulatory, and legal issues DRDs raise, FDA has announced that “after further consideration and in light of the comments received during the public hearing and submitted to the docket, FDA does not intend to pursue the potential approach described in the document at this time.”
While the FDA does not cite to any specific issue or reasoning behind the decision, the medical device industry was largely supportive of the initiative, while the biopharmaceutical industry took issue with it. The Pharmaceutical Research and Manufacturers of America (PhRMA) called the proposal “unworkable and potentially damaging to the public health” while the Biotechnology Innovation Organization (BIO) said the entire proposal was flawed.
The Proposal
Included in the proposal, and discussed at the prior public hearing, the FDA outlined three situations in which DRDs could be proposed: (1) to enhance the safety or effectiveness of the marketed drug for its already approved indication; (2) for use with the approved drug for an indication for which the drug is not approved; or (3) to provide some other benefit, such as increasing user comfort or convenience (i.e., a change in dose, route, or rate of administration).
The FDA had proposed that its premarket approval (PMA) pathway potentially be used to review and approved DRDs without needing to approve corresponding labeling changes for the drugs they are referencing, as long as the DRD sponsor addresses the following issues in their application: (1) safety and effectiveness of the new use of the drug; (2) user confusion and medication error/use error; (3) postmarket change management; (4) postmarket safety; and (5) data reliance.
The FDA believed that a PMA would be the appropriate application because DRDs are expected to represent a new intended use or raise different questions of safety or effectiveness as compared to a legally marketed predicate device. Generally speaking, PMAs for DRDs would have been reviewed by the Center for Devices and Radiological Health (CDRH), and CDRH would have collaborated with the Center for Drug Evaluation and Research (CDER) on the review of the DRD. CDRH would have the lead on device-specific issues, and CDER would have the lead on drug-specific issues.
What is Next?
It is hard to say what will be next, as the FDA did not give any insight as to why they are backing away from the proposal. Whether they come up with a new proposal, or completely leave the idea behind, will only be answered in time.