Expanded Access Programs (EAPs) are used to provide controlled and compliant access to investigational drugs for patients outside of the clinical trial setting. Many Sponsors choose to offer EAPs altruistically, as a critical treatment pathway for patients with unmet clinical needs. However, utilizing expanded access has become a strategic decision for drug developers, with more and more Sponsors recognizing the tremendous value these programs add to their clinical development efforts.
Below we will highlight three ways an EAP can complement your clinical trial.
- Data From EAPs Can Be Used to Inform Regulatory Decision-Making
EAPs offer Sponsors the opportunity to collect Real World Data (RWD). RWD is data about patient health and healthcare delivery that is collected from sources outside of traditional clinical trial setting. For rare and ultra-rare conditions, where patient numbers in clinical trials are often extremely low, RWD can provide critical and additional insight into the drug and the broader patient population. This makes it useful in supplementing clinical trial data to inform regulatory decision-making.
Expanded Access data can be used to support clinical trial data for initial asset approval.
Sponsors can identify key clinical trial endpoints that may require additional supportive evidence, and build a data collection plan for their EAP around those endpoints. The FDA can use this data to develop a more comprehensive safety and efficacy profile for the drug.
Talk with WEP about how we can help you develop your data collection plan!
Expanded Access data can be used to help support approval for additional indications and label expansions.
Sponsors can collect data from patients populations that are not included in the main clinical trial. They can then use this data to provide evidence on drug safety and efficacy in these additional populations.
WEP can provide a whitepaper from one of our Sponsor’s explaining how they used EAP data for this purpose.
- Increased Engagement with Regulators, KOLs and Advocacy Groups
The FDA encourages Sponsors to engage with the agency early on share their data collection plans and receive regulatory input. This means that, even if a Sponsor decides not to submit the RWD, providing an EAP and building a data collection plan will lead to greater engagement with the FDA.
Offering an EAP will also help foster relationships with KOLs, physician networks, and advocacy groups. The patient perspective is driving drug development efforts more than ever before. As a result, providing early exposure of a drug through an EAP to groups that represent the patient is critical. They have several important opportunities to exert influence with regulators and can become brand advocates for the drug and lobby for its approval and uptake once on the market.
- Inform and Drive Your Market Access and Commercial Strategies
Early engagement with patients and key industry stakeholders can also help inform a Sponsor’s market access and commercial strategy. By integrating patient-centered outcomes with early market analysis and strategic stakeholder engagement, EAPs help drive successful market entry and create long-term value across a Sponsor’s commercialization strategy.
EAPs can provide useful insight into how the drug will actually perform in the market, once it is approved. Sponsors can evaluate the clinical need, potential therapeutic positioning, and the value proposition in different regions. Sponsors can also gain early experience with pricing guidelines and reimbursement mechanisms to help inform pricing strategies in different markets.
EAPs can also help align key cross-functional teams at a much earlier stage. EAPs require advanced planning and buy-in from teams including, clinical operations, medical affairs, regulatory, safety, market access, and supply chain, etc. Bringing these groups together earlier can help a Sponsor navigate the transition from clinical development to commercialization.
