Healthcare Resource Utilization and Costs to Patients and Payers Among Real-World Patients with Follicular Lymphoma in the US Treated with Tazemetostat Compared to Other Standard-of-Care Therapies
Authors: Dennen Syvart , Dillon Allison , Cockrum Paul , Kambhampati Swetha 
Published: December 2024

BACKGROUND: Follicular lymphoma (FL) tends to become relapsed or refractory (R/R) to treatment, requiring multiple lines of therapy (LOTs). While frontline therapy is well-established, optimal treatment at third line (3L) and beyond is unclear. Recent approvals of novel therapies have expanded treatment options. Tazemetostat (taz) is an oral medication approved as monotherapy (mono) for patients (pts) with R/R FL with EZH2 gain-of-function mutations who received at least 2 prior systemic therapies or R/R pts who have no satisfactory alternative treatment options.
Previous research has examined costs and healthcare resource utilization (HCRU) in frontline treatment. For later lines, further evidence is needed on the real-world (RW) financial impact to pts and payers and the associated HCRU. This study examines average all-cause per-pt-per-month (PPPM) cost to payers, PPPM out-of-pocket (OOP) costs to pts, and PPPM HCRU (days with inpatient [IP] admissions, ED use, and outpatient [OP] visits) for RW pts treated in the 3L and fourth line (4L) in the US. We also report outcomes for taz mono pts of unknown line to allow sufficient sample size.
METHODS: This retrospective, observational study used Optum Market Clarity claims and electronic health record (EHR) data, representing encounters at 93% confirmed community facilities in the US. Non-taz pts were included if they had ≥1 IP or ≥2 OP claims or ≥1 EHR record with a diagnosis of FL between 2008-2023 (index); ≥12 months pre-index claims enrollment or EHR activity with no evidence of cancer; enrollment ≥30 days before and after line start; 3L or 4L therapy initiated 2015-2023. Due to small sample size, 3L and 4L were combined.
Non-taz pts were assigned treatment categories with clinical input. Rituximab (R) + chemotherapy (chemo) consists of R + bendamustine, R-CHOP, and similar. Chemo includes mono or combination treatment with platinum-based therapies, cytarabine, vincristine, and similar.
For taz mono pts, modified inclusion criteria allowed sufficient sample for analysis: ≥1 diagnosis of FL in claims or EHR data; ≥1 taz fill in claims; ≥30 days enrollment after first taz fill. Therefore, taz mono pts may have additional cancers and cannot be assigned a LOT. Taz mono pts are expected to be in predominantly 3L and 4L.
All-cause HCRU and costs (2023 USD) PPPM were calculated from LOT start to end, with end defined as either initiation of subsequent therapy, treatment gap, death, or censoring.
This study is descriptive only. No adjustments were made for pt characteristics and no statistical testing was performed.
RESULTS: There were 49 taz mono pts of unknown LOT and 408 pts in 3L/4L (treatment categories &lt;10 pts excluded). R mono and R + chemo made up &gt;60% of pts. Sample size, average PPPM costs to payers, and PPPM OOP costs to pts were, respectively: taz mono (n=49, $15,921, $437); R2 (n=21, $25,967, $461); BTKi-containing (n=15, $20,123, $638); R + chemo (n=81, $16,396, $320); PI3Ki-containing (n=21, $13,840, $415); R mono (n=211, $13,514, $578); and chemo (n=43, $12,281, $254).
PPPM IP admissions, days with ED visits, and days with OP visits were, respectively: taz mono (0.121, 0.099, 2.5); R2 (0.312, 0.534, 4.2); chemo (0.239, 0.179, 3.4); PI3Ki-containing (0.210, 0.230, 2.7), R + chemo (0.124, 0.131, 4.2), BTKi-containing (0.082, 0.248, 3.3); and R mono (0.031, 0.063, 3.2).
CONCLUSIONS: While statistical testing was not performed due to small sample size, we descriptively assess outcomes. Taz mono was less costly PPPM to US payers than other novel therapies, such as R2 and BTKi-containing regimens. It was similar to R + chemo, PI3Ki-containing, and R mono. The cost of regimens with infusions/injections was driven by OP and physician-administered drug costs. Costs of regimens with only oral drugs (taz mono, BTKi-containing) were driven by OP pharmacy and had low OP facility costs.
Taz mono had lower OOP costs than BTKi-containing regimens, another novel oral therapy, and R mono. It was similar to R2, PI3Ki-containing, and R + chemo. OOP costs can vary markedly by payer type, and region.
Taz mono had fewer OP days than all other regimens. IP use was lower for taz mono than all other therapies except BTKi-containing and R mono, and ED use was lower than all other therapies except R mono.
Taz's oral formulation and safety profile may have led to the observed lower HCRU and costs. The impact of cost and HCRU on both the health system and pts should be considered when selecting treatment.