Patient reported outcomes: Financial toxicity is a barrier to clinical trials and personalized therapy in cholangiocarcinoma

Abstract

Purpose Numerous experimental and targeted therapies are under investigation for patients with cholangiocarcinoma (CCA). Objective health‐related quality of life (HRQoL) data for patients receiving these therapies are limited. Methods Patients engaged in the Cholangiocarcinoma Foundation completed two validated HRQoL surveys: Functional Assessment of Cancer Therapy (FACT)‐Hepatobiliary and COmprehensive Score for financial Toxicity (COST). Results Two hundred eight patients were included. Seventy‐five percent had intrahepatic CCA and 57% underwent resection, of which 48% had disease recurrence. Twenty‐two percent enrolled in a clinical trial and 80% underwent molecular profiling, of which 29% received targeted therapy. While patients enrolled in a clinical trial or received targeted therapy reported similar HRQoL compared to those who did not, they reported higher financial toxicity (p = 0.05 and p = 0.01, respectively). Conclusion Enrollment in a clinical trial or receipt of targeted therapy do not affect a patient's physical, emotional, social, or functional well‐being. However, patients report higher financial burden. These therapies are mainly offered in the advanced setting after significant financial strain has been endured and are often only available at large academic centers, creating a physical barrier to access. These findings underscore the need to increase availability and eliminate physical and financial barriers that threaten access and utilization of personalized and progressive therapies.

The heterogeneous impact of targeted therapy on the prognosis of stage III/IV colorectal cancer patients with different subtypes of TP53 mutations

“…Moreover, there might be other possible factors that could affect the patient's choice of targeted therapy, such as financial status. 42,43 As mentioned in the Characteristics of patients, almost all of the patients in this study received chemotherapy. To our knowledge, lots of CRC patients would not choose targeted therapy until they respond poorly to chemotherapy, or even have suffered from progression under routine chemotherapy, since NGS tests and drugs for targeted therapy are much more expensive than routine chemotherapy.…”

“…This may be because there were more stage IV patients who were more likely to undergo progression among patients who received targeted therapy than those who did not. Moreover, there might be other possible factors that could affect the patient's choice of targeted therapy, such as financial status 42,43 . As mentioned in the Characteristics of patients , almost all of the patients in this study received chemotherapy.…”

What Role Does Radiotherapy Play in the Molecular Era for Intrahepatic Cholangiocarcinoma?

“…Over a short interval, iCCA has emerged as a prime example for capitalizing on a genomically "rich" disease entity to conduct molecularly driven clinical trials. Basket trial designs, 46,47 collaborative groups, 22 data sharing initiatives, 21 patient advocacy groups, 19,20 and special regulatory approaches 23 have led to 5 new drug approvals in recent years, a remarkable success rate, particularly for a rare and aggressive malignancy such as CCA. This momentum continues to carry forward, as investigators and biopharmaceuticals actively move through the development and trial process with new agents, including those aimed at overcoming common resistance pathways for current targeted therapies.…”

“…Furthermore, emerging data indicate intrahepatic CCA (iCCA) is molecularly heterogeneous and has multiple targets for therapy, whereas extrahepatic CCA (eCCA) tends to have fewer genetic alterations (GAs). The emergence of new therapeutic options for iCCA may be attributed to seminal molecular insights for the disease, 3-8 enthusiasm from biopharmaceuticals, [9][10][11][12][13][14][15][16] innovative trial designs, 11,17 trial collaboration, 12,14,15,18 grassroots efforts from patient advocacy groups, grant foundations, and mentorship programs, [19][20][21][22] as well as special regulatory policies for rare diseases such as iCCA (Fig. 1).…”