FDA codified an expedited review pathway for breakthrough-designated drugs in a manual of policies and procedures (MAPP) released this week, instructing FDA review teams to act at least one month before the Prescription Drug User Fee Act (PDUFA) goal date for these applications. FDA confirmed it had been informally putting drugs through expedited review for several months, according to an industry attorney.
In order for a breakthrough drug to be considered for an expedited review, it must have these qualifications:
- Preliminary review of results from clinical trials indicate the drug has demonstrated substantial improvement over existing therapies.
- The marketing application is designated as a priority review.
- The review team must has determined that a first cycle approval is likely.
Factors that may influence a decision to conduct an expedited review, even if other criteria for such a review are met, include: other public health priorities make it so the necessary resources are not available; an advisory committee meeting is needed for reasons such as clinical trial results or safety issues; and unanticipated safety issues are identified that require a risk evaluation and mitigation strategy.
A review team may determine that an expedited review is no longer appropriate for a drug if unexpected application deficiencies are found; the marketing application is poor; the sponsor fails to engage in collaborative communications; there is a need to hold an AC meeting; unanticipated review issues arise; or the review team experiences an unexpected shift in work priorities or team staffing.
According to Hyman, Phelps & McNamara attorney, Alexander Varond, there has been evidence from recent approvals of breakthrough therapy-designated products that FDA has been operating under accelerated review timelines. In an FDA Law Blog Friday (March 13), he said FDA confirmed this and stated that it has been “informally instituting a policy to expedite the review of certain breakthrough therapy-designated applications for the past several months.”