Increasingly, the value, uses and benefits of Real-World Data (RWD) collection in EAPs are being realized by pharmaceutical and biopharmaceutical manufacturers all around the world.
Most commonly, RWD is used to support reimbursement or Health Technology Assessment (HTA) applications, and can be collected in a variety of ways, including collection through Expanded Access Programs. Additionally, regulatory authorities are beginning to consider evidence from Expanded Access Programs (EAPs) data collection initiatives in their decisions about drug safety and efficacy. For instance, data collected on a drug’s effectiveness during an EAP may help provide support in its approval process through the Investigational New Drug (IND) application or decisions about label expansion.
Expanded Access Programs are intended for providing treatment for patients with high unmet medical needs and who may not have access to or be eligible for clinical studies, or for whom treatment may not be available commercially. This is especially true for patients suffering from rare diseases, where drugs to treat their indication may not be available. For rare disease, the case for RWD collection via an EAP can be even more significant.
This is because in orphan drug development, the relatively small size of patient population can limit the ability of a sponsor to enroll a clinical trial. Collection of RWD then can become an important tool in potentially generating additional insight into the safety and efficacy of the investigational therapy. In the development of drugs for rare diseases, an EAP can become instrumental in RWD collection while the drug is still investigated in clinical studies.
Importantly, RWD can be collected via an EAP ahead of drug commercialization, giving manufacturers an earlier opportunity to begin analysing and using data from the treatment setting ahead of their drug becoming available commercially.
Building Real-World Data collection initiatives into an EAP design can offer apparent benefits. There is real potential for the collected RWD to support improved understanding of the efficacy and safety of a therapy, support HTA and marketing applications, and to advance the industry’s ability to provide better care and treatment for patients going forward. However, the opportunity for RWD collection during an EAP is not taken quite as often as one would imagine. Rather, the challenges are afforded much greater weight and deter pharmaceutical teams from even attempting to collect valuable RWD from any programs they are running.
Potential Challenges of Implementing an Expanded Access Program
One of the biggest of these challenges is the heterogenous global regulatory landscape. The regulations surrounding data collection in EAPs can be complex, vary greatly by country and can be a challenge to navigate. Understandably, pharmaceutical manufacturers identify this as a potential risk if the strategic planning of RDW collection in an EAP is approached incorrectly or naively.
An ever-changing regulatory landscape, coupled with differing EAP legislation across countries, some of which may limit the ability to collect RWD, can present a significant hurdle for resource-strained biopharma teams. Awareness of the different regulatory access mechanisms available in each individual market should be one of the primary focal points of building an EAP strategy and working with an experienced EAP partner or vendor at that stage can be one of the solutions.
While there certainly are possibilities to efficiently build and implement a RWD collection strategy within an early access program, it should be noted that additional RWD collection unrelated to initial eligibility and drug safety in an EAP cannot be mandatory. If the access mechanism in the country permits, both patients and program teams should be given the option to collect and input data, as well as the option to opt out at their own discretion.
RWD collection efforts can also face the challenge of poor uptake from patients and physicians. This particular challenge can be exacerbated by the frequent misconception that a sponsor must always collect as much data as possible. There has also been observed a potential inefficiency of the systems that should be in place to enable healthcare providers (HCPs) and patients to report the required data. Importantly, because by their nature expanded access programs are intended for treatment and not research, HCPs cannot be compensated for their involvement in such programs, which can often serve as a limiting factor in the RWD collection efforts.
Typically, an EAP faces the challenge of multiple complex systems which are not built for purpose. For example, a physician can have a login to a portal that they use for program management, including tasks such as enrolling patients and ordering/ re-ordering drugs, and must then use a separate portal for collecting and inputting data.
Clearly, the separation of the two systems creates the need for additional training, adds steps to the process flow and increases the workload for an already busy physician. Understandably, due to the optional nature of the data collection step, it is likely to be the one that will be omitted.
Unlike in clinical trials, paying a physician to collect and input data during an expanded access program is not an option. Rather, they need to be incentivised in other ways. Engaging the HCPs throughout the program and allowing them to participate in publications and presentations of data to medical and scientific congresses can be an effective tool for sponsor-physician collaboration and can add value to all stakeholders.
More broadly, simplifying the RWD collection and overall EAP processes for the physician can make a significant difference in the program uptake.
Durbin Supports Real World Data in Expanded Access Programs
To address some of the potential challenges, Durbin has done a root cause analysis with regards to the barriers to collecting Real World Data (RWD) in EAPs. RWD uptake in access programs can be quite low due to the factors described above. In response to that, Durbin has identified the issues and built an innovative solution that effectively addresses physician and stakeholder engagement and helps improve it, resulting in a higher uptake in data collection initiatives.
This solution is uniphi — a secure, online, built-for-purpose, proprietary portal that acts as a central repository for physicians and sponsors, and is an efficient single tool for EAP process flow management, registrations, orders, reporting and RWD collection and analysis. As a result of using uniphi in Durbin-run EAPs, sponsors see a pronounced increase in the RWD collection uptake. Durbin’s solution to localize all EAP and RWD activities in a single, easily accessible online portal, results in a far more convenient process for all stakeholders.
By being consolidated into one system, data collection can be integrated into the overall EAP workflow. This does not just increase uptake by providing greater convenience, but it also improves the quality of data as it is collected in real-time, as an integral part of the EAP process flow.
Coupled with a robust asset-centric global EAP and RWD collection strategy, which Durbin helps sponsors design and implement, uniphi can become an ultimate tool for success of the program. With Durbin’s deep expertise in global EAP execution, any challenges can be addressed in the initial planning stages of the program, resulting in sustainable EAPs that enable and facilitate the RWD collection goals of biopharma.