Having an expanded access program (EAP) policy in place prior to product development can ensure rapid decision-making when determining the need for an EAP for a specific asset and help to maximise the potential benefits of such a program.
EAPs facilitate regulated access to investigational treatments for patients with serious or immediately life-threatening diseases prior to commercial launch of a drug. They are often considered a last resort for patients who have no other (marketed) treatment option. This is undoubtedly good for patients, but hesitancy to launch an EAP can stem from the fear of ‘not being ready’ or uneasiness over the data that will be generated and how it may be used – for instance, will new safety signals arise if inappropriate patients are enrolled?
Interestingly, over the past few years there has been a noticeable move within the industry towards the use of EAP data to inform overall launch strategy. The question now may be – can you afford to not have an EAP strategy in place?
EAPs offer an early opportunity to collect real-world evidence Historically, EAPs have been used to strengthen safety data available at the point of regulatory filing. Increasingly, EAPs are being considered to offer an opportunity to collect real-world evidence prior to regulatory approval, and there has been a shift towards using data from EAPs to supplement clinical trial data in regulatory review, inform off-label use and drive label expansions. A recent study examined >22,000 regulatory documents from the FDA and EMA and identified 39 drug approvals that relied on efficacy data from EAPs. Of these 39 approvals, 23 occurred between 2015 and 2018. They occurred in a range of therapeutic areas and four relied solely on data from EAPs.
EAPs can maximize patient recruitment and nurture ‘early adopters’ Many investigational agents subject to EAPs are orphan drugs. By definition, orphan drugs address a high unmet medical need, and generally involve small, de novo patient populations, where recruitment into clinical trials may be a challenge. Early access to an investigational agent via an EAP offers patients and physicians early exposure to pipeline assets and provides the potential to deliver early adopters. However, if a competitor has a pre-existing EAP in place, a critical number of patients may already be enrolled and be unavailable for recruitment into an alternative study. In addition, treatment success in an EAP often means that patients will not switch to other drugs when they become commercially available. Equally, if a competitor is unsuccessful, it is likely that other pipeline assets with a similar mode of action will not be tried despite the chance for a differing clinical profile, thus reducing the size of the target population at launch.
How do EAPs impact price? In addition to generating efficacy and safety data, EAPs can be used to set a price benchmark. Where the EAP price is much higher than the launch price there is a risk of a rebate later, so if the intention is to charge for your drug in those countries where the system allows it, it is best to only do this if your pricing strategy is already well developed. Note that charging for your drug might also give a competitor an advantage if they also have an EAP in place, so a careful pricing strategy will be needed.
Further reading United States Food and Drug Administration. European Medicines Agency. Patil S. Early access programs: Benefits, challenges, and key considerations for successful implementation. Perspect Clin Res 2016;7:4-8.
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