Expanded access and early access provision: Awareness, educational needs and opportunities to improve access to care.

Abstract

e18507 Background: An unmet medical need exists for many oncology patients who cannot be treated satisfactorily by available therapeutics. Despite notable efforts made by FDA and EMA to expedite development, review and approval of Innovative Medicinal Products (InMPs), there may still be a lengthy delay before oncology patients can gain access to new therapies. Expanded access (EA) in the USA and compassionate use (CU) in the European Union are pathways endorsed to improve patient access to InMPs. Recently reported data from CDER and CBER revealed that applications for EA pathways dealing with patient cohorts remain low, despite high approval rates. The challenges that practicing oncologists may face when dealing with EA or other early access provision (EAP), together with limited education, may influence the access of oncology patients to care. Methods: An on-line, fully anonymous survey reaching out to more than 3,258 practicing physicians was initiated between November 2020- January 2021. Participants were asked questions about their knowledge and understanding of EA/EAP, level of experience, decision process, interest for further educational activities and opportunities to improve process, both in general and specifically during COVID 19 pandemic. Attributes were rated on a 5-point scale. Results: Analysis of first respondents showed that 70% of practicing physicians rated their own understanding and knowledge about EA/EAP as above average (good or very good). However, only 60% of responders answered specific knowledge-based question correctly. At the same time, only 44% of practicing oncologists reported notable experience with EA/EAP in daily practice. The majority of respondents (90%) felt that availability of clinical efficacy data from comparative phase III RCTs would be considered of high importance; other evidence and study types (e.g. clinical safety, PK/PD, preclinical data) were rated substantially lower. The need for further education around better understanding of the processes and regulatory requirements, roles and responsibilities, and data collection was emphasized by 61%, 36% and 30% of respondents, respectively. During current COVID 19 pandemic, majority of respondents highlighted the need to improve and substantially adapt EA with regard to application process and documentation, InMP supply and logistics, and safety reporting process. Conclusions: Results from this survey suggest that challenges posed by the EA process, together with a lack of education, might contribute to the under-utilization of EA/EAP for oncology patients. Continuous educational efforts are required to better inform and support practicing oncologists during the EA application process and regulatory framework. Competent authorities should consider optimization and implementation of digital tools to facilitate EA applications and follow up during the COVID 19 pandemic.