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7023 Background: Expanded Access (EA), also known as “compassionate use,” is a regulatory pathway in which a patient with an immediate life-threatening condition or disease can gain access to an investigational product for treatment when no satisfactory therapy is available. Oncology practices may lack the regulatory experience or administrative support to use EA. In response, FDA OCE launched Project Facilitate (PF), a call center to assist oncology healthcare providers requesting EA. An analysis of single-patient investigational new drugs (IND) was performed to assess the first 10 months of PF compared to the period prior to its launch. Methods: Preliminary data was extracted from the FDA’s central database that yielded 719 single-patient INDs between May 31-November 30, 2018 & 2019 in the Office of Oncologic Diseases (OOD). Data collected included IND receipt date, acknowledgment date, application status, drug name, underlying malignancy of patient, address of requesting physician, withdrawal date, and patient demographics. A manual review of INDs was performed to assess for actual processing dates and to capture demographics not captured by the database. A total of 28 INDs were excluded due to duplications, cancellation by Sponsor prior to issuance of FDA decision, or coding errors in the database. Industry denial explanations were reported by the provider by emails. Results: Data from 692 INDs were analyzed and 692 (100%) were granted safe to proceed. The median processing time was 1 day (mean=2) in 2018 and 1 day (mean= 1.5) in 2019. Our findings indicate that the volume of oncology EA requests increased by 76 (19%) in 2019 vs 2018. A total of 207 unique drugs were requested. Malignancies most frequently involved included: Acute myeloid leukemia (n = 84, 8.3%), soft tissue sarcoma (n = 77, 7.6%), and non-small cell lung cancer (n = 60, 5.9%). States with the highest requests included: California (n = 82, 11.8%), New York (n = 81, 11.7%), and Massachusetts (n = 42, 6.1%). A majority of requests were from major academic centers (77%). All denied requests (N = 9) by industry were due to company’s decision to not provide products outside of a clinical trial. Conclusions: The positive trends in decreased processing times and increased number of requests are consistent with OCE’s mission to improve efficiency of the EA program and ensure equitable access to all oncology patients. [Table: see text]

The mismatch between the study populations participating in oncology clinical trials and the composition of the targeted cancer population requires urgent amelioration. Regulatory requirements can mandate that trial sponsors enroll diverse study populations and ensure that regulatory revue prioritizes equity and inclusivity. A variety of projects directed at increasing accrual of underserved populations to oncology clinical trials emphasize best practices: broadened eligibility requirements for trials, simplification of trial procedures, community outreach through patient navigators, decentralization of clinical trial procedures and institution of telehealth, and funding to offset costs of travel and lodging. Substantial improvement will require major changes in culture in the educational and professional practice, research, and regulatory communities and will require major increases in public, corporate, and philanthropic funding.

Summary: In 2022, cancer drug development continued to progress rapidly despite the lingering COVID-19 pandemic. Highlights of U.S. drug approvals for oncology indications this year include ongoing development in rare diseases and molecular subgroups, improved dosage optimization, and updated data for drugs granted accelerated approval, with confirmatory studies demonstrating verification of clinical benefit in some instances, as well as indication withdrawal when clinical benefit was not verified.

e18556 Background: FDA Oncology Center of Excellence’s (OCE) Project Facilitate (PF) was launched in June 2019 in response to perceived barriers providers face when accessing Expanded Access (EA). Since launch, PF continues to evolve and innovate to provide a comprehensive service to assist oncology healthcare providers requesting EA. An analysis of single-patient investigational new drugs (IND) was performed over 1 year to assess the growth and impact of the program. Methods: Data was extracted from Project Facilitate’s central database that yielded 640 single-patient INDs between June 1, 2020 and June 1, 2021. Data collected included IND receipt date, granting date, status, application type, indication, drug name, address of requestor, and patient demographics. Results: Of the 640 single-patient INDs extracted, there were 183 emergency INDs (eIND) and 457 non-emergency INDs (SPI). EA requests originated primarily from academic centers (81%) and community hospitals (18%) whereas Veterans Affairs and Military Treatment Facilities (0.5%) and Other (0.5%) were the least common. The 5 most common states requests originated from include New York (14%), California (13%), Texas (11%), Pennsylvania (7%), and North Carolina (4%). Although the current FDA Form 3926 does not request race and ethnicity information, this data was reported in 17 requests: White (8), Asian (3), Black/African American (3), American Indian or Alaskan Native (0), Native Hawaiian or Other Pacific Islander (0), Hispanic or Latino (3) and non-Hispanic or Latino (0). Age distribution was analyzed for pediatric (24%) and adult (76%) patients. Age distribution was further analyzed in geriatrics >75 (10%), in pediatrics < 2 (7%) and patients 2-17 years old (17%). The processing and review times for 630 applications were <1 day (96%) and > 1 day (4%). PF granted 100% of EA requests received in this time period. PF’s call center received 525 phone calls of which 499 (95%) were answered on the first call. PF also received 7843 emails during this period. Conclusions: Since 6/1/2020, PF started performing the reviews, processing and granting of CDER oncology EA requests that generally were performed by the FDA Review Divisions. By consolidating this process to PF, review times have drastically reduced to <1 day. PF has evolved since launch to promote effective and equal access to oncology expanded access.[Table: see text]