Patients may potentially gain access to an investigational therapy three ways: through clinical trials, expanded access programs (EAPs), and single patient access or emergency use.

Clinical trials

Biogen’s clinical trials are conducted to test the safety and effectiveness of investigational therapies so that regulatory authorities – such as the Food and Drug Administration (FDA) in the United States and the European Medicines Agency (EMA) in Europe – can decide whether or not these therapies can be approved as a treatment for a disease. Biogen strives to design and run clinical trials that will provide these regulatory agencies with the data they need to determine if an investigational therapy is beneficial to patients.

To be eligible to participate in a clinical trial, a patient must meet the inclusion / exclusion (eligibility) criteria.  These criteria are important because they help researchers identify patients who can participate.  The criteria also help researchers identify a group of patients with similar characteristics of a disease, making it easier and quicker for the researchers to evaluate the investigational therapy – potentially leading to faster review and approval.  

Outside of clinical trials

Expanded Access Programs (EAPs)

Expanded Access Programs, or EAPs, provide investigational therapies to patients who are not eligible or able to participate in a clinical trial – but who have a serious or life-threatening disease for which their physician has determined there are no appropriate treatment options. EAPs provide a limited number of patients, who meet specific criteria, with access to an investigational therapy.

To participate in an EAP, the patient’s physician must make a formal request on behalf of the patient to Biogen or to Biogen’s EAP partner company. The patient’s physician is responsible for the patient’s care and all legal and regulatory requirements for medically monitoring the patient while that patient is receiving the investigational therapy. In addition, enrollment in an EAP must be approved by the regulatory authority in the physician’s country. Participation in an EAP typically ends once the investigational therapy has been approved and is commercially available in the patient’s country.

At Biogen, we understand that the number of people who want access to a particular investigational therapy may be greater than the number of people who can receive it and that is why we have developed principles that shape the foundation of our EAPs, and criteria that govern access to investigational therapies.

 

Single Patient Access or Emergency Use

Patients who are not eligible or able to participate in a clinical trial – and where an expanded access program (EAP) does not exist – may request single patient access.  This type of access outside of a clinical trial is based on humanitarian or compassionate grounds. 

The patient’s physician must make a formal request on behalf of the patient to Biogen or to Biogen’s EAP partner company.  The patient’s physician is responsible for the patient’s care and all legal and regulatory requirements while the patient is receiving the investigational therapy.  Similar to EAPs, single patient access or emergency use must be approved by the regulatory authority in the physician’s country.

At Biogen, we have developed principles that shape the foundation of our EAPs, and criteria that govern access to investigational therapies.