Much is made about Real-World Data (RWD) collection in Early Access Programs.
It has been ‘the big new thing’ in early access for at least 5 years. For so long, it is well past being new. It has moved well beyond being a differentiator, to a core capability.
After an initial, admittedly somewhat clunky, foray into this (with what we believe was the first custom-built Early Access Program RWD collection system), customizing an Oracle implementation to support data collection in EAPs, we jump forward 10 years to where our online ordering platform for early access, functions seamlessly as an Electronic Data Capture (EDC) system. The challenge is to support simple ordering processes where clients do not need RWD, with a more sophisticated approach where ordering and data collection are both important. Functionality to “toggle on/toggle off” the more advanced RWD features is an essential component of any system. Yet it is not just as simple as the technological aspects.
The general regulatory stipulation that EAPs “are for treatment, not research” to me is outdated and a topic regulatory agencies should address as an urgency. Treating patients with what is often an unapproved medicine should actually require data collection to gain crucial information on safety and efficacy. Failing to encourage this, or even allow this in some countries, seems to me a major blind spot in the early access regulations.
Most of Inceptua’s clients wish to collect data. At present we walk a tightrope between what is allowed (or assumed to be allowed), what is reasonable from a physician’s perspective and what our clients want. Renewed focus on this by regulatory agencies, given the enormous growth in the number of EAPs over the last 10 years, is overdue. Early access is no longer a reactive, fringe activity, but is very much a key component of the launch of a new medicine. The significance of the potential of the data available from these patients needs to be acknowledged and utilized to a much greater extent.
People point to the (very) few product approvals based on EAP data, or the number of times EAP data is referenced in HTA submissions (infrequently and in passing) to assert that RWD in the EAP setting is already significant. Given the number of programs and number of patients treated annually, the meaningful use of EAP RWD is woeful.
Detailed RWD collection must be considered very early on the program scoping phase, with clinical development, value and access and medical affairs (at least) all involved to agree on the key data points, and the way in which these will be presented to physicians in order to minimize the burden on them. Up-front planning is essential to ensure efficient prospective data collection, and also to guide retrospective data collection, if that is the preferred approach.
Further challenges in EAP data collection are encountered with emergency/one-off treatments, cell and gene therapies, therapies used in marginalised communities, medicines being made available in LMICs/remote regions/regions affected by displacement or warfare. These challenges are significant in themselves. But we should be encouraging RWD collection in more standard EAPs as a simple, standard component supported and encouraged by agencies, thereby providing greater insight and understanding of novel medicines.
The lack of source data verification in EAPs will always mean that data collected cannot have the same rigour as a clinical trial. But that does not mean the data are not valuable. We have been pleased that Inceptua clients have presented RWD from our programs at international congresses and in peer-reviewed papers, demonstrating the worth of such data.
We have come a long way in the 15 years I’ve been doing this, but there is still a long way to go.
You might be wondering the relevance of the photo at the top. I opted not to use a stock image of a computer, or a doctor looking at an EMR, and instead used on of my favourite photos – a ‘selfie’ taken by my grandfather in Glasgow, dated to 1900. I wonder when he was using the cutting-edge technology available to him in his time (a Kodak Box Brownie) whether he would have any idea of the technology available to his grandson?
