The US is the most straight forward of all countries for collecting data within an Early Access Program (EAP), due to clear guidelines, requirement for a protocol and site contracting. However, the US is typically the smallest component of a global EAP and is usually the country where the EAP is open for the shortest duration and comprises the minority of patients on a global program. To gain meaningful data, especially for products for rare diseases, data capture beyond the US is often necessary.

In Europe, each country interpreted and enshrined the overarching EU EAP legislation differently, and data capture was generally not considered when these country-specific regulations were put in place. Data capture is therefore not commonly mentioned, and where it is referred to it is in vague terms, meaning that how to approach data capture (or even whether data capture is possible), is not clear. On top of the country-specific EAP regulations, each country’s data privacy regulations adds another layer of complexity.

However, even within these confines, it is possible to capture meaningful, clinical-level data from patients in EAPs in Europe. By EAPs, we mean a standalone program, not an OLE/PIIIb study, which are inefficient, cumbersome and expensive approaches to providing access to medicines ahead of licensing.

The best approach to RWD capture in an EAP is dictated principally by: the purpose/end use of the data to be captured, the disease landscape, and the number of patients required to provide meaningful data.

Key steps for Success:

1. Where possible, use a participating physician as a ‘Principal Investigator’
2. Ensure ethics approval (central or local) is gained where required
3. Keep data points limited to what would be captured as part of routine treatment
4. Utilize in-country CRAs to facilitate data capture and cleaning
5. Ensure all honoraria are handled in line with Sunshine Act/EU equivalent requirements
6. Agree with your vendor that you only pay for data once patient numbers have passed a pre-agreed threshold

Inceptua believes that there is a moral and ethical obligation to capture clinical level/outcomes/PRO data from patients participating in EAPs, for 2 main reasons:

1. when allowing (in some cases) experimental medicines to be used outside the confines of a clinical trial it should be expected that you use that opportunity to find out if and how the medicine benefited the patient
2. in rare diseases, where every patient is a valuable and critical source of data, it is imperative that those patients have the opportunity to contribute to the wider benefit of future patients

Inceptua is working with multiple stakeholders to seek to, at least, clarify the data capture practicalities within EAPs in Europe, and ideally to at some point harmonize or mandate RWD capture during EAPs. With the much greater focus on early patient access, EAPs are becoming more and more the norm in oncology and rare diseases, especially. Given the value in obtaining data from more patients sooner, any steps the regulators can make to facilitate this would be welcome.

Contact:

Stuart Bell
stuart.bell@inceptua.com
+44 73 8726 5293