When considering running an expanded access program (EAP), drug manufacturers typically find themselves at a crossroad, weighing the benefits of delivery against the obstacles that might be in the way.
But often, the value is much greater than the challenge, particularly when providing patients who have been given no other treatment options with potential lifesaving care, which is common in rare disease cases.
However, that’s not to say an EAP will be without barriers. Rather, it requires a strategic approach right from the onset, which can foresee potential obstacles and address them in the early stages of planning and design. This way, EAPs are bound to be more effective in enabling better patient outcomes and maximising commercial value for the manufacturer.
Understandably, this requires experience and expertise in running such programs, from which lessons have been learned and changes to delivery have been implemented. Fortunately, this is not something we, at Durbin, are short on. Durbin’s history can serve as an expanded access navigator to facilitate an EAP smoothly and avoid potential pitfalls before they occur.
In fact, from our vast experience in the field of EAPs, particularly in programs designed for rare disease and specialty drug treatments, we’re able to share insight into how EAP launch strategies can be tailored and optimized to deliver optimal outcomes for all connected stakeholders.
Internal Considerations Before Pursuing Early Access Programs
First and foremost, organizations must have a structured strategy in place that outlines the intended design and delivery process of their planned EAP. Within this strategy, there should also be a clear identification of the internal requirements of the company, and whether the company can perform the early access program.
Areas to address here include the availability of safety data to ensure the EAP can in fact proceed safely, as well as consideration of your manufacturing capability, which will prevent the need to poach stock from clinical trial supplies.
This would also feed into how sustainable the EAP will be to run as any issue with treatment supply can largely impact its long-term effectiveness and fundamentally, the purpose of the program.
Ultimately, we feel that EAPs are now considered to be a key component of the clinical development to commercialization process for rare disease therapies. When the ultimate goal of a clinical trial is to move a product toward approval, an EAP should be designed to complement the launch strategy and therefore, integrated with any clinical development plans.
So, even though clinical trials have a specific set of criteria, an EAP can be used to provide life-changing early access to those who may not meet the criteria and assess new markets or treatment areas in the process, which can then aid in post-launch expansion.
But the EAP must not jeopardise enrollment in ongoing clinical trials or in any way cannibalise enrollment. Preference should always be given to the clinical trial, if available. Therefore, the design of an EAP plays a vital role in ensuring the program does not negatively impact ongoing clinical trials.
Patient Eligibility for Expanded Access Programs
You cannot proactively select or recruit patients for an EAP like you would do for a clinical trial. Healthcare providers (HCPs) make unsolicited requests for access to therapies on behalf of their patients, with each patient being reviewed for eligibility on an individual basis.
Therefore, within the EAP design, thought must be given to the selection of patient eligibility criteria. This should be completed during the early planning phases and help determine why, where and how a program should then be delivered.
Patients may be enrolled by their HCP on a named patient basis or alternatively, access can be provided through a more formalised cohort or group expanded access programs, for which there is a defined treatment protocol. Our experience in this area suggests that EAP eligibility criteria is kept wider than the clinical trial criteria, but narrower than the anticipated label or reimbursement criteria, as this helps ensure sustainable long-term access to the treatment post-approval.
Attention also needs to be given to ensuring that there is a plan in place to support patients in countries in which there is no current access mechanism available. Durbin often sees “medical tourism” occurring within expanded access programs, where patients will travel for access to an EAP site in a country in which it is available. This represents some obvious post-launch reimbursement issues and must be accounted for in the planning and criteria of the program.
There is also the argument of neglecting the opportunity to help a patient in need, which is a pivotal part of any early access mechanism. So instead, there is a case for EAPs to be offered as broadly as possible, across countries and locations, increasing accessibility and broadening the potential for new commercial markets.
Naturally, there will be certain challenges in providing a truly global EAP, but with support from a partner with global experience, these challenges can be mitigated and accounted for in the initial EAP design.
Real-World Data Capture
Although the primary purpose of an EAP is not to collect data, an increasing number of organizations and governing bodies are beginning to recognize the value of the real-world insight such programs provide when reviewing new drug applications and determining reimbursement. Therefore, Real-World Data capture opportunities during a program should also be considered early in the EAP planning and design phase.
In rare disease circumstances this makes perfect sense as there are few other chances to learn about the disease and associated treatments. Any compliant opportunity that arises, whether that be through an EAP or not, should be considered in order to improve our overall understanding of both the disease progression and therapy in the rare disease population.
Real-world data collection during EAPs enables manufacturers, HCPs and patients alike to generate valuable real-world evidence. This real-world evidence, in turn, can be used for a wide range of potential purposes, with some exciting new approaches already being looked at.
It is clear EAPs are complex with various stakeholders and activities involved. But with Durbin’s unique approach to program design and management, underpinned by this playbook for success, we can ensure any challenges are lessened, if not avoided all together.