Interested in collecting data during your access program, but haven’t decided on what you would like to use it for? Let WEP help provide some guidance. While there aren’t specific regulations that guide the collection of data within an access program, there are some best practices that have been implemented.
The common theme across most of the world is that data collection is a vital aspect of the drug development and drug evolution process. Not only should the data we collect be studied when provided in a clinical setting, but we should also be studying how a particular drug is affecting a patient as they live their day to day lives.
What does this mean for access programs? Access programs provide a valuable opportunity to observe how investigational medicines perform in real-world settings. Unlike traditional clinical trials, which have strict protocols and controlled environments, access programs allow patients to receive treatment while living their daily lives. This offers a unique perspective on the drug’s impact on patients’ everyday experiences.
What is appropriate to collect within an access program? The goal here is to collect data that represents the patient’s real-world interaction with the treatment – i.e. Real World Data (RWD). Some examples of RWD that can be collected include:
- Quality of life measures: How the treatment impacts their daily activities and overall well-being.
- Symptom severity: Assessments of pain, fatigue, or other symptoms.
- Patient satisfaction: Feedback on their experience with the treatment and the access program.
- Disease progression: Track key disease markers and how they change over time.
- Healthcare utilization: Track hospitalizations, doctor visits, and other healthcare resource use.
- Treatment adherence: Monitor how consistently patients take the medication.
- Safety data: Monitor for adverse events, serious adverse events, and concomitant medications.
Analyzing this data and understanding the complete clinical context offers several significant benefits for drug development and patient care. By observing how the treatment affects the patient, Sponsors can:
Identify potential new indications:
By analyzing patient responses, unexpected benefits or therapeutic effects in areas beyond the original indication can emerge, opening up opportunities for the drug to treat additional conditions or diseases that were not initially considered. This could expand the market potential of the drug and provide patients with more treatment options.
Improve future treatments with better efficacy and safety:
Detailed data analysis allows for a deeper understanding of how a treatment works, which can be used to refine dosing regimens, formulation, and treatment protocols. By learning from real-world patient outcomes, sponsors can develop more targeted therapies that provide greater efficacy while minimizing adverse effects.
Determine how the treatment affects patient quality of life:
It’s not just about clinical endpoints; understanding the broader impact of the treatment on patient quality of life (QoL) is essential. Monitoring factors like symptom relief, emotional well-being, and functional ability provides insights into how patients experience their treatment journey, helping to tailor therapies to meet both clinical and personal health goals.
Identify new patient populations:
Data from diverse patient demographics—such as age, sex, genetic profile, and comorbidities—can reveal specific populations that might benefit from the treatment, even if they were not included in initial clinical trials. This allows for the development of more inclusive, personalized treatment options and ensures that the drug is optimally used for those who will benefit most.
Gain insight into factors that can improve patient outcomes:
Analyzing patient characteristics, behaviors, and external factors (such as lifestyle or adherence to treatment regimens) can uncover modifiable factors that influence therapeutic success. This knowledge can then be used to improve patient management strategies, provide more effective interventions, and optimize treatment plans for better long-term outcomes.
Identify and mitigate potential safety concerns:
Ongoing analysis of treatment data, especially during real-world use, can identify emerging safety risks that may not have been fully apparent in clinical trials. Detecting adverse events early allows sponsors to address potential safety issues proactively, such as adjusting warnings, modifying dosages, or revising administration protocols, ensuring patient safety and maintaining trust in the therapy.
Conclusion
At WEP, we feel it is important to consider RWD collection, even if you do not wish to submit the data to the FDA or other regulatory bodies. The insight you can gain from this data can be invaluable and can help you inform drug development, improve patient care, and advance medical science.
WEP has worked with many Sponsors to help them evaluate the data collection opportunities within their Expanded Access (EAPs) and Post-Trial Access Programs. If this is of interest to you, please contact us and allow us to walk you through the options.
