OBJECTIVES:

Drugs that improve survival may create real option value (ROV) by bridging patients to future innovations. We estimated the ROV attributable to the FDA accelerated approval (AA) pathway for checkpoint inhibitors (CPIs) following ipilimumab’s approval in metastatic melanoma (mMelanoma) and for enfortumab vedotin-ejfv following CPIs’ approvals in advanced bladder cancer (aBladder).

METHODS:

This study used the nationwide Flatiron Health electronic health record (EHR)-derived de-identified database and included patients who received ipilimumab (mMelanoma) or CPIs (aBladder), up to availability of the respective next innovation (CPIs, enfortumab). Overall survival, stratified by type of follow-on therapy (next innovation, no treatment, standard of care), was estimated using inverse probability of treatment weighted Kaplan-Meier curves. Total ROV with/without AA was estimated and then scaled to the U.S. population level based on market shares. Outcomes were calculated using 3 counterfactual dates to simulate traditional FDA approval without AA: 1) conference data presentation for the next innovation, 2) AA conversion to traditional approval, 3) average time from AA to full approval in oncology.

RESULTS:

Earlier access to next innovations via the AA pathway increased ROV, with an additional 5.3 months survival (range: 0-13.2 months) for ipilimumab (mMelanoma) and 1.3 months (range: 0.3-3.0) for CPIs (aBladder) compared to the counterfactual scenario without AA. Earlier access to next innovations via AA led to 355 to 678 more patients surviving to receive CPIs ( mMelanoma) and 664 to1571 to receive enfortumab (Bladder), resulting in 927 to 1772 LY gained (mMelanoma) and 1411 to 2147 LY gained (aBladder) at the population level. Percent of the ROV attributable to AA ranged from 48% to 91% in mMelanoma and 61% to 92% in aBladder.

CONCLUSIONS:

A large portion of the additional survival gained by bridging to future innovations may be attributable to the AA pathway.