Access Programs

At Biogen, our goal is to improve the lives of people by developing innovative therapies and obtaining regulatory approvals so we can bring these investigational treatments to the greatest number of individuals.

 

Update on Access to ALS Investigational Therapy:

We share the urgent desire to bring new medicines to people suffering from ALS. Obtaining approval for a new drug from regulatory authorities around the world is the fastest way to help the largest number of people with a specific disease.

Our Phase 3 placebo-controlled study of tofersen is designed to investigate the safety and efficacy of tofersen in people living with SOD1-ALS. The study has completed enrollment and data are expected in the second half of 2021.

We are preparing to open an Early Access Program as soon as patients (including those who have the fast-progressing mutation) are no longer randomized to placebo and if data show that tofersen is safe and effective.

Providing individual access to tofersen at this time could jeopardize access to tofersen for hundreds of SOD1-ALS patients by impeding our ability to complete the study and seek subsequent regulatory approvals.

We are acting as quickly as possible with the interests of all patients in mind within the current regulatory framework, with the hope of bringing new medicines to the ALS community.

Placebo-controlled studies include individuals who receive active therapy, as well as those who do not. Data obtained through placebo-controlled studies enable regulators around the world to assess whether or not a drug is safe and effective before approving the drug for broader access. Providing access to individual patients outside of an ongoing placebo-controlled study could significantly derail the pathway for approval.

About Access Programs

All investigational therapies must first go through clinical trials and be approved by a regulatory authority — such as the Food and Drug Administration (FDA) in the United States or the European Medicines Agency (EMA) in Europe — to ensure the therapy benefits outweigh any risks for the intended patient population before being made commercially available.

For most people, the only way to access investigational therapies is to participate in a clinical trial. 

However, some people with serious or life-threatening diseases may not be eligible or able to participate in clinical trials, and their physicians may determine that they have no other appropriate treatment options.

At Biogen, we work with regulators, clinical researchers, ethicists, physicians, and patient advocacy organizations and communities, among others, to determine how best to address requests for access to investigational therapies in a manner that is consistent with our patient-focused values and compliant with regulatory standards and protocols. People who meet specific criteria in rare situations may have access to investigational therapies through Early Access Programs (EAPs), or compassionate use.

Learn more about the set of Biogen principles that reflect our commitment to providing ethical access to investigational therapies and the types of early access programs in the following sections.

Types of Access to Investigational Therapies

People may potentially gain access to an investigational therapy three ways: through clinical trials, EAPs, and compassionate 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 FDA in the United States and the 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 people.

To participate in a clinical trial, a patient must meet the inclusion / exclusion (eligibility) criteria.  These criteria are important because they help researchers identify people who can participate.  The criteria also help researchers identify a group of people 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.

Learn more

 

EARLY ACCESS PROGRAMS (EAPS)

Early Access Programs, or EAPs, may provide investigational therapies to a limited number of people who have a serious or life-threatening disease for which there are no appropriate treatment options, clinical trials or they are not eligible or able to participate in a clinical trial.

If an EAP program is in place, specific eligibility criteria must be met in order to gain access to the investigational treatment.

At Biogen, our priority is bringing approved treatments to the greatest number of patients in a timely manner.  In order to fulfill this mission, we must consider how an EAP’s timing will impact our ability to successfully complete the regulatory pathway and goals of broad therapeutic access for patients.  For that reason, we’ve developed principles that shape the foundation of our EAPs and plan to initiate an EAP at the time when all principles have been met. 

Learn how to request access.

 

EAP Guiding Principles

  • To provide appropriate early access for patients in an effort to address their high unmet medical need
  • Programs with minimal risk to our clinical trials and therefore regulatory pathways 
  • Minimize negative impact to patients and families, healthcare providers (HCPs) and healthcare (HC) systems
  • Achieve minimal risk to reimbursement pathways and broad long-term access

 

COMPASSIONATE USE ACCESS

People with diseases for which there are no available treatments in their country or who are not eligible or able to participate in a clinical trial – and where an early access program (EAP) does not exist – may request compassionate use for off-label access to approved products.

At this time, we can only offer compassionate use for approved products in rare circumstances if a patient meets specific criteria.

We currently are unable to offer compassionate use for any of our investigational products as it would hinder our ability to potentially offer broad and long-term access through regulatory pathways. Obtaining approval from regulatory authorities around the world is the fastest way to help the largest number of people with a specific disease.

Learn how to request access.

 

NAMED PATIENT PROGRAM (NPP)

In many cases, a treatment may be approved and commercially available in one country but not available in others. Therefore, if a patient is located in a country where a commercial product is not available and has a life-threatening illness whereby survival could potentially be prolonged in a clinically meaningful way, patients and physicians may be able to access commercially approved treatments that are not available in their own country through a named patient program (NPP).

To be eligible for an NPP, a patient’s physician must make a formal request on behalf of the patient to Biogen. 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.

How to Request Access

FOR PATIENTS / CAREGIVERS / PATIENT ADVOCATES

If you are a patient inquiring about access to an investigational drug, the licensed physician overseeing your care must submit this request.

For more information on this process for any of Biogen's investigational drugs, please contact Biogen's Patient Center. Requests submitted to this email address will be acknowledged within one business day.

 

FOR HEALTHCARE PROFESSIONALS

Patients who are not eligible to access the therapies in a clinical trial setting may be able to have access through an expanded access program (EAP) or a named patient program (NPP).  Please note that an EAP typically ends once the therapy is commercially available in a country or at a specified date, as agreed upon with local authorities. For information about access to other Biogen therapies that are not available to patients in the commercial or clinical trial setting, please contact Biogen Medical Information.

 

FOR DISTRIBUTORS/WHOLESALERS

If you are a distributor or wholesaler and would like further information, please contact Biogen's Patient Center.