PNS81 STATUS OF EXPANDED ACCESS PROGRAM (EAP) REGISTRATION INTO CLINICALTRIALS.GOV FOLLOWING 21ST CENTURY CURES ACT

Abstract

Expanded access is the use of an investigational medical product outside of a clinical trial when no comparable or satisfactory alternative therapies are available for the treatment of a serious disease or condition. Types of EAP are (1) individual patients (including emergency use), (2) intermediate-size patient population (more than one patient), and (3) treatment Investigational New Drug (IND)/protocol (widespread population). Under the Cures Act, and specifically 42 CFR Part 11, US pharmaceutical companies have to post information on expanded access on ClinicalTrials.gov. We aim to review the status of EAP registration into ClinicalTrials.gov over time. We searched ClinicalTrials.gov on 11-Jan-19 using following search terms: Expanded Access Studies, United States, Treatment IND/Protocol, Individual Patients and Intermediate-size Population. 58 studies were identified (49 funded by industry, 8 by academia, 1 by NIH). 16 studies were registered during the period 2005-2016, 15 in 2017, and 26 in 2018. Most studies focus on neoplasms (40%), followed by metabolic disorders (23%), and hematologic disorders (12%). 76% involved a drug, 17% a biological, and the remainder a device or any combination of drug/biological/procedure/device. 38% were individual patients EAPs, 28% treatment IND/protocol EAPs and 16% included intermediate-size populations (the remainder a mix). 72% of studies were conducted in the US only and 16% were multicounty (no information available for the remainder). The distribution of single center vs. multi-center was approximately 50/50. As expected, we observe a sharp increase in registrations following the Cures Act and most EAPs are for drugs treating neoplasms. However, 42 studies since 2017 across a limited number of pharmaceutical sponsors suggests that many EAPs are not (yet) registered, despite this now being mandatory.