Unlocking the potential of Real-World Evidence derived from Expanded Access Programs

myTomorrows Team 24 Apr 2024

8 mins read

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A real-world data team member working at myTomorrows

By Jasmin Schelhaas, Real-World Data Associate at myTomorrows

Expanded Access Programs: A Path to Harnessing Real-World Data  

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. Especially for rare diseases, gathering additional data through EAPs can be essential as each patient’s information is valuable6. 

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. 

Exploring the Use of Real-World Data from EAPs: Our Research Findings 

To support BioPharma companies in utilizing RWD from EAPs, research has been conducted to explore the practical applications of this data. 

myTomorrows’ in-house RWD team published several articles on gathering RWD from EAPs in collaboration with 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 ‘Results from expanded access programs: a review of academic literature’ show that there is a significant increase in the number of publications over time2, which could reflect a general increase in attention for RWD derived from EAPs.  

But that’s 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 treatments3. 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 treatments4. 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 the use of EAP data in regulatory decision making7-9. 

The FDA draft guideline acknowledges the potential use of EAP data, positioning it as potential confirmatory evidence alongside one well-controlled clinical investigation10. The FDA states, “Although 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”10 (p.12).  

Keys to Success in Real-World Data Collection from EAPs 

However, although RWD in EAPs is gathered and can be utilized for various purposes, it’s a complex field with diverse regulations across countries1 and there is limited guidance on how to collect the data and what to collect. Drawing on our RWD intelligence databases, 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. 

Effective Study Design 

Successful Study Execution

Other key success factors in ensuring complete, high-quality data in the EDC system, are site support, remote monitoring and using queries.  To ensure support at each key milestone within the EAP, physicians should be guided through the whole EAP application process by experienced personnel. Offer clear instructions from the start, especially for the research naïve sites, and have a RWD expert dedicated to managing the data collection within the EAP. Successful remote monitoring involves enabling automatic notifications via email, raising queries in the EDC system to seek further explanation or address any missing information and being available online for assistance with the EDC system whenever needed.

Optimizing Data Analysis & Output 

Before analyzing the data and producing an output it is crucial to write a statistical analysis plan (SAP) outlining how the analysis will be performed. This plan should clearly define which patients and which data will be included, how it will be analyzed, and what statistical methods will be applied. If you plan to use the data for regulatory submission, it’s important to engage with health authorities and regulators early as well. The FDA recommends engaging with them via a regulatory pathway (e.g., by requesting a Type C meeting) if including RWD, to identify challenges in the design and planning and to discuss how these challenges might be addressed. Ideally, the protocol and SAP should also be finalized and shared with the FDA prior to conducting the analyses11

Partnering with myTomorrows for Real-World Data Success  

Taking these learnings from both research and practical experience, our dedicated RWD team is a front-runner in gathering RWD within an expanded access setting. We offer an integrated approach in RWD strategy, collection, and analysis of data in non-interventional settings and have experience with gathering data for internal use to collect additional safety data, creating tailored outputs for scientific journals and conferences, and assisting with official submissions to regulators. 

If you would like to learn more about how our team of specialists can potentially assist or advise your company with setting up EAPs and/or RWD collection, we welcome you to reach out and speak with our experts: https://mytomorrows.com/contact-biopharma/. 




  1. Sarp S, Reichenbach R, & Aliu P. An approach to data collection in compassionate use/managed access. Front Pharmacol. 2022;13:1095860. https://doi.org/10.3389/fphar.2022.1095860
  2. 2. Polak TB, Cucchi DG, Schelhaas J, Ahmed SS, Khoshnaw N, van Rosmalen J, et al. Results from Expanded Access Programs: A Review of Academic Literature. Drugs. 2023;83(9):795–805. https://doi.org/10.1007/s40265-023-01879-4
  3. Polak TB, van Rosmalen J, Uyl-de Groot CA. Expanded Access as a source of real-world data: an overview of FDA and EMA approvals. Br J Clin Pharmacol. 2020;86(9):1819–1826. https://doi.org/10.1111/bcp.14284
  4. Polak TB, Cucchi DG, van Rosmalen J, Uyl-de Groot CA. Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals. BMJ Open. 2022;12(1): e052186. https://doi.org/10.1136/bmjopen-2021-052186
  5. Wasser JS, Greenblatt DJ. Applying real-world data from expanded-access (“compassionate use”) patients to drug development. J Clin Transl Sci. 2023;7(1): e181. https://doi.org/10.1017/cts.2023.606
  6. Wu J, Yang Y, Yu J, Qiao L, Zuo W, Zhang B. Efficacy and safety of compassionate use for rare diseases: a scoping review from 1991 to 2022. Orphanet J Rare Dis. 2023;18(1):368. https://doi.org/10.1186/s13023-023-02978-x
  7. U.S. Food and Drug Administration [FDA]. FDA approves alpelisib for PIK3CA-related overgrowth spectrum. 2022, April 6 [cited 2024 Mar 11]. Available from: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-alpelisib-pik3ca-related-overgrowth-spectrum
  8. Aetion. CBER-Approved BLA for ZOLGENSMA (onasemnogene abeparvovec-xioi). [Internet]. 2020, April 21 [cited 2024 Mar 11]. Available from: https://aetion.com/evidence-hub/fda-decision-alerts/cder-approved-bla-for-zolgensma-onasemnogene-abeparvovec-xioi/
  9. Aetion. CDER-Approved NDA for LUTATHERA® (lutetium Lu 177 dotatate). [Internet]. 2020, April 20 [cited 2024 Mar 11]. Available from: https://aetion.com/evidence-hub/fda-decision-alerts/cder-approved-nda-for-lutathera-r-lutetium-lu-177-dotatate/
  10. U.S. Food and Drug Administration [FDA]. Demonstrating substantial evidence of effectiveness with one adequate and well-controlled clinical investigation and confirmatory evidence – Guidance for Industry. Sep 2023 (draft guidance). III-G Types of Confirmatory Evidence – Evidence from Expanded Access Use of an Investigational Drug. [Internet]. 2023 [cited 2024 Mar 13]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/demonstrating-substantial-evidence-effectiveness-one-adequate-and-well-controlled-clinical
  11. U.S. Food and Drug Administration [FDA]. Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products: Guidance for Industry.[Internet]. 2023 [cited 2024 Mar 13]. Available from: https://www.fda.gov/regulatory-information/search-fda-guidance-documents/considerations-use-real-world-data-and-real-world-evidence-support-regulatory-decision-making-drug

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myTomorrows Team 24 Apr 2024

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