Preapproval medical products in patients with the diagnosis of metastatic uveal melanoma.
Authors: Nshimiyimana Maniraho Regine , Seedor Rino S. , Kearns Lisa , Bateman-House Alison , Orloff Marlana M. , Keith Scott W. , Senter Katherine , Sato Takami 
Published: June 2023

e21502 Background: Metastatic uveal melanoma (MUM) is a rare malignancy that has limited treatment options. As such, there is a role to play in treating eligible patients with investigational (unapproved) medical products (IMPs) that may offer a higher chance of benefit than risk. Yet, given this malignancy's rarity, complexity, and high mortality rate, we hypothesized that patients with MUM face challenges in accessing IMPs through clinical trials (CTs) or non-trial access (expanded access (EA)/compassionate use or right to try). Methods: We conducted a retrospective comparative electronic chart review for all living patients with MUM, aged 18 years and older, established at a single referral center to assess MUM treatments including IMPs in and out of trials. A descriptive analysis was conducted to explore the use of IMPs by generation age, sex, the time elapsed between the initial diagnosis of UM and metastasis, and the duration of illness since metastasis. We grouped patients by generations: Millennials, Generation X, Boomers, and the Silent Generation. The treatments were categorized in liver-directed therapy with immunoembolization, transarterial chemoembolization (TACE) with Carmustine, TACE with doxorubicin eluting beads, Yttrium-90 radioembolization, hepatic arterial infusion, radiation therapy, surgical resection, standard systemic treatment, IMPs through a CT, and IMPs through EA. Each category of MUM treatment was assigned a score of 1. Results: There were 154 patients with MUM. The majority were female (64.3 %, n = 99), Boomers aged 58-76 years (61.7%, n = 95), not Hispanic or Latino (92.9%, n = 143), married (77.3%, n = 119). In our total sample, 27.3% (n = 42) had received adjuvant therapy through CTs after the initial treatment of UM. After metastasis, 92.2% (n = 142) were treated with standard therapy for metastatic melanoma, 35% (n = 54) received IMPs through a CT, and 2.6% (n = 4) received IMPs through EA. The Silent Generation had a lower level of using IMPs through CTs. There was no substantial difference in using IMPs by sex. Participants in the group of < 1 year from initial diagnosis of UM to metastasis had a higher level of using IMPs through a CT. Participants in the group of < 1 year since metastasis had a lower level of using IMPs through a CT. Conclusions: In this center, a substantial number of patients with MUM received IMPs through CTs (35%) compared with the national U.S. average of only 6.3% of patients with cancer enrolled in cancer treatment trials from 2013 to 2017 (Unger & Fleury, 2021). However, the number of patients with MUM who received IMPs through EA was small (2.6%) and should be improved. EA should remain a viable option for patients who do not fit the trial requirements or do not have realistic access to a trial site. Thus, we need a better system to increase the number of patients who received IMPs through EA. More research is needed to see if our findings are generalizable to other patients at other institutions.