Expanded Access

Our mission is to advance science in the development and approval of medications to benefit the greatest number of patients in a timely manner.  Our ability to successfully complete the regulatory pathway and achieve the goal of broad therapeutic access for patients is essential to this mission.

Clinical Trials

Clinical trials are the main way for patients to access investigational therapies.

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. Read more about clinical trials here.

Sometimes, patients with serious and/or life-threatening diseases or conditions who do not qualify for a clinical trial may seek medical products that are not yet approved or available in their country.

Below you will find our:

  1. Expanded Access Guiding Principles
  2. Available Expanded Access Programs
  3. Guidance for Treating Physicians (outlining how to submit a request)

 

Our Expanded Access Guiding Principles

Biogen is equally committed to enacting expanded access for products in development for qualifying patients and communicating to the patient community, healthcare providers (HCPs), and healthcare systems on timing of such programs.  The following principles guide decisions to provide access to our programs:

 

Access for appropriate patient(s) to address high unmet medical need in a safe, ethical, and just manner in compliance with applicable laws and regulations

 

Access while ensuring the integrity of clinical trials and the development of medicines

 

Access that does not jeopardize the pathway to broad long-term access and approvals through local health systems

 

Access that minimizes burden on healthcare systems, HCPs, patients, and families

 

Available Expanded Access Programs

SOD1-ALS: Tofersen

Tofersen is an antisense asset being evaluated for the potential treatment of SOD1-ALS. In the Phase 3 VALOR study, the primary endpoint as measured by the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) did not reach statistical significance; however, signs of reduced disease progression across multiple secondary and exploratory endpoints were observed. Based on totality of the data from the VALOR Phase 3 study and its open-label extension, Biogen is actively engaging with regulators, the medical community, patient advocacy groups and other key stakeholders around the world to determine potential next steps.

Additionally, in light of the critical unmet need, Biogen has expanded eligibility for its ongoing early access program (EAP) to all people with SOD1-ALS, in countries where such programs are permitted by local regulations and future access may be secured. EAP programs enable patients to gain access to a medicine free of charge before the treatment is licensed commercially. If a clear path forward for tofersen is not established, or if another controlled trial is required by regulators, Biogen may revise or discontinue the EAP.

In some countries, the expanding eligibility for the EAP will also change the regulatory mechanism used to provide access. In those cases, there may be actions required by the Treating HealthCare Professional (HCP) on behalf of patients already enrolled in an existing limited EAP. Biogen will work with Clinigen to support these Treating HCPs if local regulations require changes.

Treating healthcare professionals (HCPs) can submit requests on behalf of patients who meet the inclusion/exclusion criteria by emailing MedicineAccess@clinigengroup.com.

 

Alzheimer’s: Aducanumab (outside of the United States only)

Aducanumab is an Alzheimer’s disease treatment approved in the United States (U.S.) under the accelerated pathway based on reduction in amyloid beta plaques observed in patients treated with aducanumab. Continued approval for this indication may be contingent upon verification of clinical benefit in confirmatory trial(s). Aducanumab is still under review in countries outside of the U.S. Making aducanumab broadly available to qualified patients through regulatory approval is one of our top priorities.

Biogen recognizes the high unmet medical need of Alzheimer’s patients in countries where aducanumab is not yet approved. As we work with regulatory authorities, we are committed to offering access programs for eligible patients, where permitted by local regulations. There are two potential early access pathways to aducanumab for appropriate patients outside of the U.S. (early access to aducanumab is not available in the United States as the product is commercially available): an Early Access Program (EAP) and a Charged Managed Access Program (CMAP) and we are working to assess the feasibility of the implementation of these programs on a country-by-country basis. All access programs are subject to local regulatory and health authority requirements. In all circumstances, a patient’s physician is responsible for deciding if treatment with aducanumab is appropriate. For information regarding the availability, access, and timing of these programs, a patient’s treating physician can send an email to medicineaccess@clinigengroup.com.

Learn more in the statement here.

 

How to Submit a Request

All requests must be submitted by a physician on behalf of the patient. All requests are considered in a fair and just manner in accordance with our guiding principles. However, please understand that submitting a request does not guarantee access will be granted.

Tofersen: Email  medicineaccess@clinigengroup.com

Aducanumab: Email medicineaccess@clinigengroup.com

 

Medical Information

Healthcare providers, patients, and caregivers with a medical question can reach out to the Biogen Medical Information Team.

  • In the United States please contact 1-866-MED-INFO, or medinfo@biogen.com.
  • Outside of the United States, search for your country information here.