Why is there no compassion for compassionate usage of medication in colorectal cancer care?

Collins, Joanne; Lodhi, Taha; Paton, Nina; Khera, Raj; Alani, Mohammed; Saunders, Mark

Background: Metastatic colorectal cancer is one of the commonest causes of cancer and cancer-related deaths.1 The prognosis remains poor and successive treatment options remain limited.2 Many patients will run out of treatment options before they become medically unfit for therapy.3 Cancer drug innovation presents both opportunities and dilemmas as access to innovative cancer medications can be limited by a significant delay between clinical trials, licensing, and positive drug guidance.4,5 Consequently, where no commissioned alternative medicine exists some pharmaceutical companies operate compassionate usage access schemes (Figure 1). These allow cancer treatment with investigational, unlicensed, or off-label medicines as soon as early evidence of potential benefit emerges.6 There is a paucity of information regarding compassionate schemes. This analysis was performed to ascertain real-world data on patients enrolled in these schemes. Methods: Single-centre study conducted at The Christie Hospital, Manchester, UK. We reviewed the process of pharmacy drug set-up and maintenance by undertaking a survey of disease group pharmacists. We undertook a retrospective audit using an electronic patient record of patients who entered into a compassionate scheme between 2018 and 2020. This analysis focused on 27 patients with a diagnosis of metastatic colorectal cancer receiving compassionate medication who possessed the BRAF mutation (mBRAF V600E) or MSI-H status. We analysed 17 mBRAF patients treated with a triplet combination (encorafenib, binimetinib and cetuximab), and subsequently, doublet (encorafenib and cetuximab), as defined by the BEACON study, and 10 MSI-high patients on compassionate nivolumab.7,8 Results: Our survey of compassionate medication across 14 disease groups demonstrated the approval process is lengthy and resource intensive. The average approval time was variable, but pharmacy set-up time could take several days depending on the drug or regimen. However, this is countered by our analysis of 27 meta-static colorectal cancer patients. Most patients were fit (> 88% PS ≤ 1, 50% had no known co-morbidities) and for 62% this represented third- line therapy, in a cancer setting with limited or no treatment options. Patients received 10 cycles of treatment on average before progression or unsuitability, with a mean duration on the treatment of 120 days (15–317) on the triplet/doublet combination and 205 days on nivolumab (range: 1–663), demonstrating benefit. Patient demand was uninhibited by geographical distance (> 30% of patients lived 20+ miles away from the treatment centre) and the possibility of hope and life extension with further treatment. Conclusion: Our audit demonstrates that patients received compassionate drugs that were subsequently commissioned by NHSE, with some benefiting from treatment for a considerable period. This suggests there was both a moral gain and a survival advantage over best supportive care; however, there remains limited real-world data and further research is required. Access to compassionate schemes is controversial and limited as they can be seen to promote healthcare inequity, creating a post-code lottery, as they may not be available in some regions, circumvent existing guidelines or benefit pharmaceutical companies by allowing early data and market access. Compassionate usage recognises a human side to drug development which requires a moral balance between patients.

UNITED Kingdom; PHARMACOLOGY; CONFERENCES & conventions; COMPASSION; COLORECTAL cancer

Journal of Oncology Pharmacy Practice, 2023, Vol 29, p1

1078-1552

Academic Journal

10.1177/10781552231153542