Strategic timing: When should biotech firms consider launching expanded access programs?
Expert shares his insights on when to launch expanded access programs in line with the regulatory landscape
As the landscape of drug development continues to evolve, expanded access programs (EAPs) are emerging as a critical strategy for biotech companies aiming to meet the urgent needs of patients with limited treatment options. Sciensus, a life sciences business specialising in patient access, engagement and insight solutions, is shedding light on the optimal timing and strategic considerations for launching these programs in Europe.
Andrew Cummins, Director of Expanded Access Programs at Sciensus, emphasises the transformative potential of EAPs for both patients and biotech firms:
“Expanded access programs represent a unique and powerful opportunity for biotech companies to provide life-saving therapies to patients who otherwise would not have access. Launching an EAP should be carefully considered within the broader context of a company’s development and commercialization strategy.”
The regulatory landscape of EAPs is one of the most significant challenges biotech companies face.
Andrew Cummins Director of expanded access program
Europe presents a complex regulatory environment, with each country having its own guidelines and legislation regarding access to unlicensed medicines. While the European Medicines Agency (EMA) provides overarching guidance, individual member states have the autonomy to develop their regulations.
Currently, 18 of 27 EU countries have established national guidance around Compassionate Use Programs (CUPs). This patchwork of regulations requires biotech firms to be highly strategic in their approach to launching EAPs in Europe.
“Navigating the regulatory landscape in Europe is one of the most significant challenges biotech firms face when considering an expanded access program,” says Cummins.
“It’s not just about meeting regulatory requirements; it’s about understanding the nuances of each market and tailoring your approach accordingly. This is where the expertise of specialised service providers like Sciensus Rare becomes invaluable.”
When is the best time to launch an EAP?
Determining the right time to launch an EAP is crucial for maximizing the program’s impact.
Many companies consider EAPs during the later stages of product development, particularly after phase III clinical trials when the product remains unlicensed globally. However, EAPs can be initiated at various stages of the product life cycle—both pre- and post-approval.
“Launching an EAP during the pre-approval phase can provide expaaccess to patients who are in desperate need, while also allowing companies to gather valuable real-world data,” says Cummins.
“On the other hand, post-approval EAPs can be strategically deployed in markets where the product is not yet licensed, ensuring continued access for patients and supporting the company’s broader market entry strategy.”
In many cases, the primary driver for launching an EAP is the desire to address the unmet needs of patients who are ineligible for clinical trials. EAPs are often used to extend access for patients who have completed clinical trials or to provide access to non-trial patients while a registration application is under review.
These programs offer a lifeline to patients suffering from life-threatening, long-lasting, or seriously debilitating illnesses, providing them with early access to potentially life-saving treatments.
Managing risks and leveraging opportunities
EAPs come with inherent risks, including the potential for adverse events and supply chain challenges. However, the benefits of EAPs can be substantial.
By engaging with clinicians and patients early, companies can build strong relationships and establish a positive reputation in the market.
Additionally, the real-world data collected from EAPs can provide valuable insights that support regulatory submissions and market access strategies.
“EAPs offer a unique opportunity to gather real-world data that is often more reflective of the patient population than clinical trial data,” Cummins explains.
“This data can be crucial for informing regulatory submissions and shaping future commercial strategies. However, companies need to be prepared for the risks and challenges that come with running an EAP.”
Patient advocacy is a critical component of successful EAPs
Patient advocacy groups play a pivotal role in shaping expanded access program in Europe. These groups are increasingly influential in driving demand for unlicensed medicines and ensuring that the voices of patients are heard. Biotech firms must engage with these groups early in the process to ensure that their programs are truly patient-centric.
“Patient advocacy is a critical component of successful EAPs,” says Cummins. “By working closely with advocacy groups, companies can ensure that their programs are designed with the patient’s needs at the forefront. This not only helps to ensure the program’s success but also builds trust and credibility with the patient community.”
Expanded access programs can be a game-changer to getting treatments to patients faster
As the demand for expanded access program continues to grow, biotech firms must carefully consider the timing and strategy behind launching these initiatives. With the right approach, EAPs can provide life-saving treatments to patients in need, while also supporting the company’s broader commercial objectives.
Sciensus, with its deep expertise in navigating the complexities of the European regulatory landscape, stands ready to guide biotech firms through this challenging yet rewarding process.
“Expanded access programs are not just about getting treatments to patients faster,” Cummins concludes. “They’re about doing so in a way that is ethical, strategic, and aligned with the company’s long-term goals.”
For more detailed guidance on navigating expanded access in Europe, download the full whitepaper here.
About Sciensus
Sciensus is a leading European life sciences organisation with over 30 years of experience. We’re redefining patient-centric care, connecting patients with life-changing medicines while empowering pharmaceutical companies and healthcare professionals with the insights needed to improve people’s lives.
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