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Compassionate use programs in Europe: all you need to know 

Compassionate use programs are one way in which international biotech companies can widen access to their orphan medications in Europe.  

For a drug to receive regulatory approval and market authorization in Europe, it must go through a rigorous evaluation of both its safety and its efficacy, just as it would have to in the US. For mainstream drugs, clinical trials are key to effectively gather this information.  

For rare disease patients, however, who are often in desperate need of a new medical breakthrough, the opportunity to participate in clinical trials can be infrequent and challenging for several reasons.  

As a result, there is an ongoing demand for access to unlicensed medication from patients, clinicians, and patient advocacy groups. 

Patients who are severely ill with significant organ issues, for example, are often ineligible for a clinical trial because of possible adverse impacts. Compassionate use can help the most seriously ill patients to gain access to potentially life-changing medications. Even if there is no guarantee that the treatment will help, compassionate use programs can ensure these patients at least can find out. 

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What is a compassionate use program? 

For rare disease patients who have no other viable options, compassionate use programs can open access to an unlicensed drug as a treatment. Through compassionate use programs, doctors can apply to use a medicine that may show promise but is still in the evaluation process and therefore not yet approved for wider use. It is important to note, however, that compassionate use programs do not cover off-label use. 

Compassionate use programs are designed to be a potential option only when no other suitable treatments are available. While they are not an extension of a clinical trial, they can only be authorized if a trial is already in progress. That said, they must not be seen to be compromising the recruitment of patients for a clinical trial.  

Similarly, biotechs must not view compassionate use programs as purely a means to promote a product or gain a market advantage. Any form of expanded access program can indeed provide valuable real-world data, but companies must be careful to show that this is not their main motivation. 

The majority of European nations have specific processes for setting up compassionate use programs, but they can vary in complexity and the time needed to complete the application process. For patients living with chronic or severely debilitating diseases, and those facing life-threatening conditions, compassionate use programs can provide access to vital medications. These patients will often have exhausted the limited existing treatment options otherwise available to them, with no success.  

Expanded access vs compassionate use 

Compassionate use programs are one of the two main types of expanded access programs companies can run in Europe. The other is known as a named patient program. Typically, compassionate use programs are used in pre-approval, and named patient programs are used in post-approval, but they are largely interchangeable. 

The key distinctions between the two, and the important factors in deciding which program to use, are in understanding who is driving the unlicensed medicine use, whether its use can be charged for, and if national regulatory approval is required. 

Some of the key differences between compassionate use programs and named patient programs include: 

  • Patient type: A compassionate use program is generally used when treating a group or cohort of patients, while a named patient program is used for an individual. 
  • Initiator: Compassionate use programs are driven by a manufacturer that has identified an area of unmet need among patients who are not eligible for their clinical trial. On the other hand, a named patient program is usually initiated by the patient’s treating physician, based on individual patients’ unmet need. 
  • Cost: While manufacturers may charge for drugs prescribed under a named patient program, compassionate use is almost always free of charge to either the patient or their national healthcare system. 
  • Involvement of national regulatory body: While compassionate use programs must be approved by the national regulatory authority, named patient programs are not as tightly regulated and usually just involve an agreement between the treating physician and the manufacturer, following relevant national guidelines. 
  • Duration: Compassionate use programs can remain open right up until the drug receives marketing authorization in that country. Named patient programs are generally only authorized for one year at a time but can be renewed up until marketing authorization is approved as well. 

Criteria to access compassionate use 

Compassionate use programs provide hope and potential treatments for patients who are facing serious health challenges and, often, have limited options available for overcoming these challenges. They can fill the gap between experimental medicines and full regulatory approval, offering patients improved health outcomes and, ultimately, a better quality of life. 

However, these programs can only be put in place if the drug in question is: 

  • Expected to help patients with life-threatening, long-lasting, or seriously debilitating illnesses, for which there is currently no suitable authorized medicine. 
  • Undergoing clinical trials, or in the application process for marketing authorization. It doesn’t need to be authorized anywhere else in the world.

The European Medicines Agency (EMA) recommends that unlicensed medicines should, wherever possible, be made available through clinical trials first. It also argues that patients should be considered for inclusion in a trial before being offered a compassionate use program. 


Compassionate use program in Europe: how does it work? 

The implementation of compassionate use programs is down to individual European countries, and their processes are known to vary significantly from each other. Each member state has its own national regulations that govern how compassionate use programs should be employed, with EMA recommendations serving only as guidelines.  

For example, the UK’s Early Access to Medicine Scheme (EAMS), which was first launched in 2014, is a three-step process. In the first step, companies can submit clinical trial data to the Medicines and Healthcare Products Regulatory Agency (MHRA) and apply for designation as a promising innovative medicine (PIM). The MHRA will then review the data and, if approved, the drug can be commissioned by the NHS.  

Unlike in some other countries, such as the USA and France, companies can’t charge for access to their product through EAMS. Other countries where expanded access to medicines is provided free of cost include Austria, Germany, Greece, and Spain. 

While compassionate use programs are within the remit of the European Union’s (EU) individual member states, the EU Committee for Medicinal Products for Human Use (CHMP) can provide recommendations to all member states on how to administer, distribute, and use certain medicines for compassionate use. These recommendations are optional and do not amount to a legal obligation for the member states to authorize a compassionate use program. 

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Unlock European opportunities and revenue with Sciensus

Sciensus has a 30-year proven track record in global market expansion, and our team has extensive experience in the rare and orphan disease market.

Our partnership model is designed to support the development and launch of therapeutics in the EU by allowing our partners to leverage our expanded access programs, world class supply chain, patient support programs and nursing programs. We also support biotechs by giving our partners access to our regulatory, marketing and commercial strategy expertise. In the last year alone, we supported our clients with over $350m of sales in Europe. This collaboration can not only ensure a faster route to market but also serves as a significant step in addressing the critical medical needs of patients reliant on timely access to advanced treatments.