Introduction 

Access to unlicensed medication is in demand for many reasons, including good clinical trial data, increased patient and clinician understanding about international treatment options, and the rise of powerful patient advocacy groups.  

Many of the patients who need these drugs will have exhausted all alternative treatment options and may not meet the specific eligibility criteria to participate in a clinical trial. Sadly, many do not have time to wait for the treatment to become available in their country.  

For these patients, expanded access programs (EAPs) can provide a beacon of hope, opening desperately needed access to a drug not yet licensed for use in their home country.  

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supply chain management for orphan drugs in Europe

What is an expanded access program? 

An expanded 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.  

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. 

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How is expanded access 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. 

 

Benefits for pharma and biotech companies 

As well as supporting clinicians to meet the needs of their patients ethically and compliantly, an EAP can help pharma and biotech companies to achieve:

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Faster access to new global markets

For instance, only the orphan drug market in Europe is forecasted to be worth $60.25bn by 2028 (from $41.46bn in 2023). 

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Network building with clinicians and PAGs

Early adopting clinicians can become future brand advocates for the manufacturer, supporting discussions with regulators based on their experience of use.  

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Collection of real-world data

This can be extremely valuable for eventual regulatory submissions and market access.

Expanded access 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 

Sciensus expanded access program solution

Our comprehensive EAP service offers:

Full regulatory assessment

Sciensus will support you with a full regulatory assessment of the countries you are interested in running an access program. This will cover advice on type of access program most suited to your needs, whether it can be charged for or not, what data you may be able to collect. We can also advise on your communication strategy. You will be supported by a dedicated project manager, and a multilingual customer service team.

A digital platform for clinicians (​Sciensus Axcess)

We will ensure health care professionals are properly validated and given access to our online ordering portal Sciensus Axcess. Registered healthcare professionals will be able to view documentation for the access program including product information and any relevant training materials.

An established supply chain

Our logistics teams will ensure product is delivered most of the time within 24hrs of order confirmation. We will ensure there is full track and trace capability to allow auditing of all activity. We can provide a full order to cash service. We will deal with any issues in an efficient, ethical and regulatory compliant manner.

EAP monitoring protocol and enhanced​ real-world data collection

At Sciensus, we excel in designing research protocols to capture and analyze real-world data, empowering you to make informed decisions throughout your product lifecycle. Our comprehensive support spans clinical study design, funding submissions, market access initiatives, post-market studies, and product evolution strategies. 

Patient Support Programs

If you require additional support for your patients, we can advise on a range of support services via our nurse lead patient support programs. We can support your clinicians with Individual funding requests as required. You will be provided with full transparency on the activities we carry out on your behalf with accompanying adherence to a range of mutually agreed KPIs.

From EAP to full commercialization with a single partner

When your access program completes as you move to commercialization in that country, we can also handle all your support needs for commercial supply.

Why partner with Sciensus?

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Industry leading experience

We have a 30-year proven track record in global market expansion, and extensive experience in the rare and orphan disease market.

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Established international network

Over 4,000 relationships with wholesalers, hospitals, pharmacies, and clinics, allow us to quickly gain access to prescribing physicians.

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From EAP to full commercialization

Our partners can benefit from our regulatory, marketing and commercial strategy expertise, ensuring a faster route to market.

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 Expanded Access Program will contact you to schedule a meeting and provide a complimentary consultation.

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