#HEORInsights

Expanded Access Programs (EAPs) are special programs designed to provide patients with serious or life-threatening conditions access to investigational treatments that have not yet been approved by regulatory authorities, offering them a chance to receive potentially beneficial therapies when no other options are available. EAPs not only offer a lifeline to patients but also present an exceptional opportunity to gather Real-World Data (RWD). By strategically harnessing data from EAPs, researchers and healthcare stakeholders can unravel a wealth of insights that contribute to evidence-based decision-making and drive improvements in patient care. However, the successful collection and strategic utilization of data from EAPs require careful planning, ethical considerations, and methodological rigor.[1,2]

Ethical data collection from EAPs is paramount to their usage as RWD sources. The collection of patient data from EAP must prioritize patient privacy, informed consent, and data security. Striking a balance between patients’ urgent medical needs and their rights as research subjects is crucial.[3]

A comprehensive data collection strategy begins by defining the specific data elements to be collected from EAP participants. This could include demographic information, disease characteristics, treatment regimens, adverse events, and patient-reported outcomes. Collaborating with healthcare professionals and patient advocates can help identify relevant data points contributing to a holistic understanding of treatment outcomes.[4]

Establishing a robust data collection infrastructure is pivotal. Electronic health records, patient registries, and digital health platforms can facilitate seamless and standardized data collection. Integrating data collection into routine clinical workflows minimizes the burden on healthcare providers and ensures data accuracy.[4]

Incorporating patient-reported outcomes (PROs) in the EAPs empowers patients to contribute their perspectives on treatment experiences and quality of life. Digital tools and mobile apps can streamline the collection of PROs, providing real-time insights into patient well-being. Additionally, maintaining data quality and reliability is paramount to drawing meaningful insights from EAPs. Implementing data validation protocols, standardized data entry procedures, and regular data audits mitigate the risk of inaccuracies and enhance the credibility of the collected data.[4]

RWD from EAPs can also shed light on healthcare resource utilization by providing insights on hospitalization rates, length of stay, and use of medical services. By using this data, healthcare systems can make informed decisions about resource allocation and patient care planning.[3,4]

Challenges in EAP data collection and utilization are not uncommon. One challenge is the potential for selection bias due to the non-randomized nature of EAPs. Patients with specific characteristics might be more likely to participate, introducing bias into the data. To mitigate this, advanced statistical techniques, such as propensity score matching, can be employed. Additionally, data completeness can be an issue due to variations in healthcare settings and patient engagement levels. Implementing data quality checks and incentives for data submission can encourage comprehensive data collection.[3,4]

The optimal collection and use of RWD from EAPs hold immense potential to shape healthcare decisions, policy formulation, and patient care. Collaborative efforts among healthcare professionals, researchers, patient advocates, and regulatory bodies are essential to harness the full potential of RWD. As technology advances and data collection methodologies evolve, the seamless integration of EAP data into evidence-based medicine promises a brighter future for patients seeking hope beyond conventional treatment options. By navigating the ethical considerations, implementing robust data collection strategies, and leveraging advanced analytical techniques, stakeholders can unlock the power of RWD to improve patient outcomes and advance medical knowledge.

Become A Certified HEOR Professional – Enrol yourself here!

Learn AI Potential For Writing Manuscripts – Enrol yourself here!

References

1. Polak TB, Cucchi DG, Schelhaas J, Ahmed SS, Khoshnaw N, van Rosmalen J, Uyl-de Groot CA. Results from Expanded Access Programs: A Review of Academic Literature. Drugs. 2023 May 18:1-1.
2. Polak TB, Cucchi DG, van Rosmalen J, Uyl-de Groot CA, Darrow JJ. Generating evidence from expanded access use of rare disease medicines: challenges and recommendations. Frontiers in Pharmacology. 2022:1917.
3. Polak TB, Cucchi DGJ, 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 Jan 6;12(1):e052186.
4. 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 Sep;86(9):1819-1826.