{"id":7027,"date":"2024-04-24T14:31:24","date_gmt":"2024-04-24T14:31:24","guid":{"rendered":"https:\/\/main-portal.develop-mytomorrows.com\/?p=7027"},"modified":"2024-04-24T14:31:24","modified_gmt":"2024-04-24T14:31:24","slug":"unlocking-the-potential-of-real-world-evidence-derived-from-expanded-access-programs","status":"publish","type":"post","link":"https:\/\/main-portal.develop-mytomorrows.com\/ar\/blog\/real-world-data\/unlocking-the-potential-of-real-world-evidence-derived-from-expanded-access-programs\/","title":{"rendered":"Unlocking the potential of Real-World Evidence derived from Expanded Access Programs"},"content":{"rendered":"

By Jasmin Schelhaas, Real-World Data Associate at myTomorrows<\/span><\/i><\/b><\/p>\n

Expanded Access Programs: A Path to Harnessing Real-World Data\u202f<\/span>\u00a0<\/span><\/h3>\n

Expanded access programs (EAPs) provide an important avenue for patients with debilitating and\/or life-threatening conditions, who have no alternative treatment options and are unable or ineligible to enroll in clinical trials to access pre-approval options. While the primary intent of EAPs is to provide access to treatment, there has also been a growing interest in collecting and analyzing Real-World Data (RWD) from these programs. This growing interest is primarily due to the increasing appreciation for Real-World Evidence (RWE) reflected in greater integration by health authorities into their regulatory guidelines, and the broader use of this evidence in scientific publications, regulatory submissions, and cost reimbursement decisions1-5<\/sup><\/span>.<\/span> Especially for rare diseases, gathering additional data through EAPs can be essential as each patient\u2019s information is valuable<\/span>6<\/sup><\/span>.<\/span>\u00a0<\/span><\/p>\n

In this blog, we will highlight key findings of research on collecting RWD within EAPs. Furthermore, we will emphasize some key elements for successful data outputs based on practical insights from our experiences.<\/span>\u202f<\/span>\u00a0<\/span><\/p>\n

Exploring the Use of Real-World Data from EAPs: Our Research Findings<\/span>\u00a0<\/span><\/h3>\n

To support BioPharma companies in utilizing RWD from EAPs, research has been conducted to explore the practical applications of this data.<\/span>\u00a0<\/span><\/p>\n

myTomorrows\u2019 in-house RWD team published several articles on gathering RWD from EAPs in collaboration with <\/span>interns and academics. In our latest research, we investigated the extent to which data from EAPs were published in academic literature. The results of this research, entitled \u2018Results from expanded access programs: a review of academic literature\u2019 show that there is a significant increase in the number of publications over time<\/span>2<\/sup><\/span>, which could reflect a general increase in attention for RWD derived from EAPs.\u00a0<\/span>\u00a0<\/span><\/p>\n

But that\u2019s not all. In other studies, we encountered that, over time, RWD and RWE derived from EAPs are also more often used by regulatory bodies such as the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) to inform safety and efficacy labels for new treatments<\/span>3<\/sup><\/span>. Health technology assessment (HTA) bodies such as the National Institute for Health and Care Excellence (NICE) also incorporate data from EAPs to determine the cost-effectiveness of new treatments<\/span>4<\/sup><\/span>. For NICE, EAP data was even used in over one in five appraisals. Various real-case examples, such as FDA approvals for drugs like Vijoice (alpelisib), Zolgensma (onasemnogene abeparvovec) and Luthathera (lutetium (177Lu)) further underscore<\/span> t<\/span>he use of EAP data in regulatory decision making<\/span>7-9<\/sup><\/span>.<\/span>\u00a0<\/span><\/p>\n

The FDA draft guideline acknowledges the potential use of EAP data, positioning it as potential confirmatory evidence <\/span>alongside one well-controlled clinical investigation<\/span>10<\/sup><\/span>. T<\/span>he FDA states, <\/span>\u201cAlthough the purpose of expanded access is not primarily for research if the patient outcome information collected under expanded access use of the drug is of sufficient quantity and quality to be highly persuasive, the information may be considered for use as confirmatory evidence\u201d<\/span>10 (p.12)<\/sup><\/span>.<\/span>\u00a0<\/span>\u00a0<\/span><\/p>\n

Keys to Success in Real-World Data Collection from EAPs<\/span>\u00a0<\/span><\/h3>\n

However, although RWD in EAPs is gathered and can be utilized for various purposes, it\u2019s a complex field with diverse regulations across countries1<\/sup><\/span>\u00a0and there is limited guidance on<\/span> how to collect the data and what to collect. Drawing on our RWD intelligence databases<\/span>, <\/span>a centralized repository of RWD guidance, information, and insights as well as practical knowledge from experience collecting RWD from EAPs, there are a number of important factors to consider to get usable, high-quality, fit-for-purpose data.<\/span>\u00a0<\/span><\/p>\n

Effective Study Design<\/span>\u00a0<\/span><\/h3>\n