How does the FDA view expanded access data?

It’s not uncommon for biopharma companies to have concerns about approving expanded access treatment requests because they worry that it may potentially negatively impact drug approval or product labeling. However, both recent studies and comments from FDA officials should ease these concerns.

In this 2017 study of expanded access studies, the authors found that “there was no instance in which expanded access led to a negative regulatory decision regarding a drug application.” That trend appears to have held steady. The FDA has continually publicized their support for expanded access highlighting that when reviewing more than 10,000 expanded access INDs that cross-reference more than 1000 commercial INDs, to date they have initiated only 2 clinical holds (0.2%).

Recent FDA approvals have reflected a willingness to include real-world data gathered from expanded access experiences in regulatory decision making. According to reporting on a November 2018 FDA meeting, top officials made clear that “sponsors could benefit from learning from others in industry – and from FDA itself – about how expanded access data can support an initial approval or inform labeling.”

Suggested opportunities to benefit from expanded access included:

• Primary approval

• Label expansion

• Off-label coverage decisions

• Future clinical trial design

While the decision to extend expanded access to patients involves a number of considerations and may not always be feasible or appropriate for the patient, it appears that sponsors may feel reassured that FDA will view real-world data gathered during compassionate use with an appropriate lens.