Expanded access program services
Our partners benefit from our regulatory, marketing and commercial strategy expertise, ensuring a faster and easier route to market.
What is an expanded access program?
An expanded access program, also known as an early access program, is a regulatory pathway that enables companies to widen potentially lifesaving access to investigational and pre-approval medication. It can be undertaken at any point in the product lifecycle and to maintain patient access after a product has been discontinued.
An EAP allows a broader patient population to receive promising treatments before full approval, potentially accelerating adoption and generating diverse real-world data. In these cases, the patient does not have comparable or satisfactory alternative therapies available and cannot participate in a clinical trial. For people living with a rare disease, an Expanded Access Program (EAP) can provide a beacon of hope, opening desperately needed access to a drug not yet licensed for use in their home country.
Different types of expanded access programs
Expanded access programs can generally be categorized into two main types:
- Named Patient Programs (NPP), designed to provide access to a specific, unapproved drug for an individual patient, often on a case-by-case basis.
- Compassionate Use Programs (CUP), broader initiatives that allow access to investigational drugs for a group of patients facing serious or life-threatening conditions.
How is an expanded access program different from clinical trials?
Clinical trials for rare disease therapies can be infrequent and challenging for several practical and ethical reasons. EAPs can be an attractive alternative for companies who wish to widen potentially lifesaving access for patients who are not eligible for a clinical trial. They offer an opportunity to trial a new treatment in the real-world, without the strict eligibly criteria and protocols of a clinical trial. Patients are still closely monitored for safety and to assess the effectiveness of the drug but can live their lives as normal, providing a much clearer picture of the treatment’s impact.
Faster access to new global markets
Network building with clinicians and PAGs
Collection of valuable real-world data
Expanded access program in Europe: challenges and solutions
Gaining approval for an expanded access program in Europe can be challenging and costly, with multiple complex regulatory landscapes and different healthcare systems to negotiate across the many member states.
For example, many European nations have developed their own national guidance around access programs and so the specific regulations in each country need to be understood. Added to this, unlicensed medicines can’t be promoted, so firms need to be very careful to demonstrate that any EAP is in response to an unsolicited request.
EAPs also bring with them a certain level of risk, the most obvious being an adverse event which could jeopardize a future commercial launch. Similarly, poor patient adherence could lead to a misunderstanding of the product’s efficacy and impact the success of commercialization.
Many companies considering launching an EAP turn to a specialist service provider to support the design and delivery of their program. Proactively engaging providers in early discussions can help organizations work through the many obstacles and ensure that all factors are considered when deciding whether an EAP is the right choice and to guide the subsequent planning and implementation.
Find out more about navigating expanded access in our in-depth exploration on strategizing Early Access Programs in Europe.
Discover our fully integrated expanded access program services
A comprehensive regulatory assessment
We will support you with a regulatory assessment of the countries where you are interested in running an expanded access program. This will include advice on the most suitable program type, whether it can be charged for, and the data you may be able to collect.
A digital platform for clinicians
We will ensure healthcare professionals are properly validated and given access to our online ordering portal Sciensus Axcess. Registered HCPs will be able to view documentation for the access program including product information and any relevant training materials.
An established supply chain
We operate a GDP-certified medical warehouse hub in the Netherlands, equipped for all temperature regimes, facilitating delivery to most European sites within 24 to 48 hours. We will ensure there is full track and trace capability to allow auditing of all activity.
Enhanced real-world data collection
We specialize in designing research protocols for real-world data analysis, empowering informed decisions throughout your product lifecycle. Our support includes clinical study design, funding submissions, market access, post-market studies, and product evolution strategies
Digitally enabled Patient Support Programs
We deliver patient support programs across Europe, both face-to-face and remotely, through highly qualified HCPs. We understand regulatory frameworks and can mobilize our clinical workforce to assess needs, research conditions, and develop tailored PSPs for your product
A dedicated account manager
We provide a personal touch thanks to a dedicated account manager, a bespoke solution tailored to your specific needs, a strategy developed by our Director of Expanded Access Programs and a dedicated team that will implement it for you.
Schedule a free consultation with Andrew
Andrew Cummins, Director of Expanded Access Programs at Sciensus, has more than three decades of experience in senior international commercial leadership positions within the Pharmaceuticals and Medical Devices sectors, with a specialization in early access pre-approval unlicensed medicines.
Industry leading experience
Established international network
From EAP to full commercialization
Schedule a meeting with our EAP expert
To discuss your challenges and create a strategy for accessing Europe tailored to your business needs, please complete the form below. Our Director of EAP will contact you to schedule a meeting and provide a complimentary consultation.
Find out more about…
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