RESUMO
BACKGROUND: Expanded access programs (EAPs) allow cancer patients with unmet clinical need to obtain access to pre-authorisation treatments. There is no standardised process for implementing these programs nationally, and real-world data on their impact is lacking. AIMS: This study aimed to evaluate the prevalence of such EAPs and their 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 treated with EAP drugs, and patients treated were from across a broad spectrum of ages (26-82, SD 11.99). CONCLUSIONS: EAPs have an increasing role in accessing 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 established. Assessment of their benefits and toxicities would be enhanced by the requirement for a real-world database as a condition of EAP approval.
RESUMO
BACKGROUND: While biologic drugs have demonstrated efficacy across a range of indications, patient access to these drugs is constrained due to their high cost. Biosimilars provide a means to increase patient access while reducing the financial burden. AIMS: The primary objective was to determine the current usage of biosimilar and reference trastuzumab and rituximab in four Irish hospitals. A secondary objective involved determining barriers to biosimilar usage. METHODS: This project involved a retrospective chart review to analyse the usage of reference and biosimilar versions of trastuzumab and rituximab. Additionally, a prospective cross-sectional study identified barriers to the usage of biosimilars via the distribution of a novel questionnaire to patients, pharmacists, doctors and students. RESULTS: The utilisation of biosimilar intravenous trastuzumab and rituximab ranged from 39 to 100%, and 0 to 89%, respectively. A total of n = 479 questionnaire responses were included. Biosimilar awareness was significantly lower among 'Doctors and Medical Students' (45.3%; 95% [CI, 33.8-57.3%]) compared to 'Pharmacists and Pharmacy Students' (97.1%; 95% [CI, 94-98.8%; comparison p < 0.001]). A significant majority of healthcare professionals agreed biosimilars should have consistent nomenclature (p < 0.001). A significant majority of patients (87.3%, 95% [CI, 81.3-92%; p < 0.001]) indicated that they would agree to commence using a biosimilar medicine. CONCLUSION: Biosimilar versions of trastuzumab and rituximab were in use to a variable extent. There remains a considerable opportunity to further increase the usage to maximise their potential benefits. A series of challenges were identified including reduced awareness among the medical profession and lack of clear nomenclature.