Cost and public reimbursement of cancer medicines in the UK and the Republic of Ireland.

INTRODUCTION/AIMS: There are disparities in the availability of systemic anticancer therapies (SACTs) globally. We set out to investigate the cost and reimbursement of SACTs in the United Kingdom (UK) and the Republic of Ireland (ROI) in conjunction with efficacy and licensing authority decisions in the United States (US) and the European Union (EU). METHODS: We sought data pertaining to licensing in the EU, reimbursement in ROI/UK and cost/efficacy of SACTs licensed by the Food and Drug Administration (FDA) between January 2015 and May 2021. Independent samples t tests, chi-square test and Pearson's correlation were used for statistical analysis. RESULTS: We identified that the majority of FDA-approved regimens are licensed by the European Medicines Agency (EMA) (n = 91, 67.9%). However, only a minority of these are currently reimbursed in the UK (n = 60, 45%) or the ROI (n = 28, 21%) as of the 1st of May 2021. In addition, only a minority of regimens have demonstrated a statistically significant OS benefit (n = 54, 40%). There was no association between cost of regimens and either the presence (t = 0.846, p = 0.40) or duration of OS benefit (t = - 0.84, p = 0.64). CONCLUSIONS: Our study highlights that many licensed systemic anticancer treatments are not currently reimbursed in ROI/UK. The high cost of these medicines is independent of the presence of an OS benefit. Collaboration between regulatory agencies, governments and industry partners is needed to ensure health expenditure is directed towards the most effective treatments.

Crowdfunding for anticancer therapies: an analysis of non-US GoFundMe pages.

BACKGROUND: Crowdfunding is the internet practice of funding a project or person by requesting online monetary donations from people. We set out to analyse the current non-US GoFundMe cancer pages in order to get deeper understanding of this unregulated activity. METHODS: We identified 150 non-US GoFundMe webpages between November 2019 and January 2020. Data collected included clinicopathological details such as age, gender, cancer type and stage of the disease along with other parameters. RESULTS: Of 150 pages reviewed, 83% requestors were adults, 64% were female and the median age was 33 years (2-60). The majority of requestors had brain cancer, followed by breast cancer and ovarian cancer. The median amount of money requested was €48,205 (€1171-€588,759). The majority of pages were from the UK (59%) and Ireland (16%). Financial support for cancer patients was the largest group identified (25%). Requests for immunotherapy were the second most common (21%) followed by alternative therapies (19%) and funding for anticancer drug not financed by specific health system but approved elsewhere (12%). The largest amount of money requested was for immunotherapy (median €187,064). CONCLUSION: Our GoFundMe analysis has identified that crowdfunding platforms are predominantly used for requesting financial support, but worryingly, a large proportion are requesting immunotherapy for unlicensed indications and alternative therapies with no evidence. Future efforts to improve patient education, and possibly online donation page regulation, are required to better manage this route of cancer funding.

Radiological evaluation of malignant pleural mesothelioma - defining distant metastatic disease.

BACKGROUND: Malignant pleural mesothelioma (MPM) is traditionally characterized by local destructive spread of the pleura and surrounding tissues. Patient outcomes in MPM with distant metastatic dissemination are lacking. METHODS: In this retrospective study, we reviewed a cohort of 164 MPM patients referred to a Phase I trials unit, aiming to describe identified metastatic sites, and correlate with clinical outcomes. RESULTS: 67% of patients were diagnosed with distant metastatic disease with a high incidence of bone (19%), visceral (14%), contralateral lung (35%) and peritoneal metastases (22%). Peritoneal metastases were more likely in epithelioid versus biphasic/ sarcomatoid MPM (p = 0.015). Overall survival was 23.8 months with no statistical difference in survival between those with distant metastases and those without. CONCLUSIONS: This report highlights the frequency of distant metastases and encourages further radiological investigations in the presence of symptoms. In particular, given the relatively high incidence of bone metastases, bone imaging should be considered in advanced MPM clinical workflow and trial protocols. The presence of distant metastases does not appear to have prognostic implications under existing treatment paradigms. This cohort of MPM patients gives an indication of patterns of metastatic spread that are likely to become prevalent as prognosis improves with emerging treatment paradigms.

Imprecision in the Era of Precision Medicine in Non-Small Cell Lung Cancer.

Over the past decade, major advances have been made in the management of advanced non-small cell lung cancer (NSCLC). There has been a particular focus on the identification and targeting of putative driver aberrations, which has propelled NSCLC to the forefront of precision medicine. Several novel molecularly targeted agents have now achieved regulatory approval, while many others are currently in late-phase clinical trial testing. These antitumor therapies have significantly impacted the clinical outcomes of advanced NSCLC and provided patients with much hope for the future. Despite this, multiple deficiencies still exist in our knowledge of this complex disease, and further research is urgently required to overcome these critical issues. This review traces the path undertaken by the different therapeutics assessed in NSCLC and the impact of precision medicine in this disease. We also discuss the areas of "imprecision" that still exist in NSCLC and the modern hypothesis-testing studies being conducted to address these key challenges.

A Rare Thyroid Metastasis from Uveal Melanoma and Response to Immunotherapy Agents.

Thyroid metastasis is a rare occurrence with cutaneous melanoma and even more uncommon with uveal melanoma. The management of such metastasis is uncertain due to its infrequency and, in the era of immunotherapy, the effect of these novel drugs on uncommon metastasis, such as to the thyroid, is unknown. We report the rare case of a thyroid metastasis in a patient diagnosed with ocular melanoma initially managed with enucleation. Metastatic disease developed in the lung and thyroid gland. The case patient received the immunotherapy ipilimumab with stable disease in the thyroid and progressive disease elsewhere. The patient was then further treated with a second immunotherapy agent, pembrolizumab, and remains with stable disease one year later. We discuss the current literature on thyroid metastases from all causes and the optimal known management strategies. Furthermore, we provide an original report on the response of this disease to the novel immunomodulators, ipilimumab, and pembrolizumab with stable disease four years after initial diagnosis of ocular melanoma.

Systemic therapy for advanced pancreatic cancer: individualising cytotoxic therapy.

INTRODUCTION: Pancreatic cancer is an aggressive solid tumour associated with a high risk of local invasion and metastatic spread. In the absence of validated screening approaches, many patients present with advanced, incurable disease. Despite advances in treatment, the prognosis for patients with advanced pancreatic cancer remains poor. The benefits of surgical resection and radiation are unproven in this setting and, as patients generally present with distant metastases, systemic therapy forms the mainstay of treatment. The importance of selecting the optimal cytotoxic therapy remains to be critical to improving outcome and retaining quality of life. This review sets out to compare the current therapies available for advanced pancreatic cancer. AREAS COVERED: This review examines the evolution of the systemic management of advanced pancreatic cancer, with a particular focus on the landmark Phase III trials identified from an extensive PubMed and Google scholar literature search. The article summarises the cytotoxic agents in use and the results of two decades of clinical trials to culminate in an almost doubling of survival in clinical trials. EXPERT OPINION: In the concluding expert opinion, the challenges interpreting and translating trial results into clinical practice are discussed, where patients are often older and of worse performance status. In the future, novel biomarkers might be used to tailor therapy.