Purpose Expanded access programs (EAPs) allow patients with cancer who have unmet clinical needs to obtain access to pre-authorisation treatments. There is no standardised process or registry for implementing these programs nationally and real world data on their impact is lacking. We evaluated their prevalence and impact in a cancer centre.Methods Data relating to adult cancer patients treated via EAPs from 2011 to 2021 in three Cork university hospitals was collated. Descriptive statistics were employed to get an overview of the impact these programs currently have on cancer care provision.Results We identified 193 patients who accessed EAPs during the study period, availing of 33 separate drugs for a total of 50 different cancer indications. The prevalence of EAP usage was shown to have been trending upwards in recent years with a total of 189 programs being accessed throughout the period. Drugs provided were from a number of different anti-cancer drug classes, particularly targeted therapies (n = 18) and immune checkpoint inhibitors (n = 17). Cancers from a wide range of both solid and liquid tumour types were identified as having been treated with EAP drugs and patients treated were from across a broad spectrum of ages (26–82, SD:11.99).Conclusion EAPs have an increasing role in access to novel cancer therapies in our community and by extension nationally. Equity of EAP access would be facilitated by a national registry of available agents which we have now established. Assessment of their benefits and toxicities would be enhanced by the requirement for a real world data base as a condition of EAP approval.
469 Background: There are significant disparities in patient access to innovative cancer medicines internationally. We sought to describe global drug licensing developments in gastrointestinal (GI) oncology from 2010 – 2020. We compared US, European, UK and Irish patient access to these drugs, their cost and associated efficacy. Methods: Data was collected from public sources online including the FDA, European Medicines Agency (EMA), the UK National Institute of Clinical Excellence (NICE) and the Irish National Cancer Control Programme (NCCP). Anti-cancer compounds approved for GI malignancies between January 1, 2010 and September 1, 2020 were recorded. Approval publications and updated survival analysis outcomes were included to assess for demonstrable overall survival (OS) and/or progression free survival (PFS) benefit. Results: There were 26 regimens approved by the FDA for GI malignancies between 2010 and 2020. Slightly over half (n=15, 57.7%) of these regimens were approved by the EMA. The median time from FDA to EMA approval was 3 months (mo) +/- 5 mo. Only a small minority of these regimens were funded for patients in the UK (including the Cancer Drugs Fund, 9 regimens, 31%) or Ireland (IRL) (7 regimens, 27%). The median time from EMA approval to patient access was short in both of these countries (3.5 mo (UK) and 0.5 m (IRL)). However, the range in these countries was large (2 - 73 mo (UK) & 0-24 mo (IRL)). More than half of regimens (15, 57.7%) demonstrated an OS benefit. Of the remaining 11 regimens, a further 6 drugs (23%) demonstrated a PFS benefit. The median OS benefit (if present) was 2.3 mo +/- 3 mo. The median price of these regimens in the USA (list price) was $13,758 per month +/- $5,152. Regimens which demonstrated an OS benefit were not more expensive than those that did not (median $13,065 versus $13,758 respectively, p < 0.05). For regimens with an OS benefit, there was no statistically significant correlation between the length of OS benefit and drug cost (Pearson’s correlation = 0.32, p = 0.07). Conclusions: Less than one third of patients in the UK and Ireland have access to medicines approved by the FDA for GI malignancies between 2010 – 2020. Overall survival benefits are often modest, or absent. The cost of regimens in the USA commonly exceeds $100,000/year irrespective of OS benefit. Our work highlights the necessity to develop a value based-pricing system in GI oncology.
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