Real World Data in Expanded Access Protocols

Payors are more open to real world data than ever before. The right drug in the right disease we might be able to demonstrate added supportive information in the real world setting such as an Early Access Program. While permissible, we often think twice about the balance of keeping Expanded Access Protocols less burdensome for patients and physicians and the opportunity to collect data to inform. Certainly, compassionate use protocols provide a significant opportunity for collection of Real World Data in a setting that provides a real-world setting. At the conference I spoke to the ‘how’ of planning and executing an Expanded Access Protocol where real world data are collected. Importantly, I spoke to the importance of incorporating the voice of the patient as a partner in the development of these programs. Truly, a perspective of “Patients as Partners” within the context of developing an Expanded Access Protocol that can be operationalized in any multi-patient compassionate use protocol. We already have insights that data generated can be used to support regulatory filings in some countries. One of the areas that I also highlighted was how real-world data is already being extracted from other types of trials, including Pragmatic Clinical Trials. Payors are more open to real world data than ever before. This doesn’t necessarily mean that they will always be willing to pay for a drug that shows clear benefit in these real-world settings, but with the right drug in the right disease we might be able to demonstrate enough benefit in the real world setting that payors would be willing to pay. There are several misunderstood types of trials that can be falsely portrayed as opportunities to collect real world data and ‘buyer beware’ needs to be a mantra I continue to speak.