Science & Innovation

Early Access

Biogen advances science through the development and approval of medicines to benefit the greatest number of patients in a timely manner.

Clinical Trials

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

Biogen’s clinical trials are conducted to test the safety and effectiveness of investigational drugs so that regulatory authorities — such as the FDA in the United States and the EMA in Europe — can decide whether or not these drugs 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 drug 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 and identify a group of people with similar characteristics of a disease. This makes it easier and quicker for researchers to evaluate the investigational drug, potentially leading to faster review and approval.

What is Early Access?

Early access is a potential pathway for patients diagnosed with a serious and/or life-threatening disease or condition to gain access to an investigational product for treatment outside of a clinical trial when no comparable or satisfactory alternative therapy options are available. In these instances, a patient’s treating physician can request an investigational product prior to regulatory approval, where allowed by local laws.

Who is Eligible for Early Access Programs?

Biogen considers granting early access to investigational products when all of the following criteria are met:

  • A patient is diagnosed with a serious and/or life-threatening disease or condition and no comparable or satisfactory alternative therapy is available to treat the disease or condition.
  • The patient is not eligible to participate in a clinical trial.
  • Sufficient data exists where the potential benefits of the treatment outweigh the potential risks for the disease to be treated.
  • There is adequate supply of the investigational product, and providing the investigational product will not interfere with ongoing clinical trials or the overall development program.
  • Investigational access provided in this setting is allowed per local laws and regulations.

Available Early 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 end point 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 end points were observed. Based on totality of the data from the Phase 3 VALOR 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 professionals (Treating HCPs) 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 HCPs can submit requests on behalf of patients who meet the inclusion/exclusion criteria by emailing MedicineAccess@clinigengroup.com.

The ClinicalTrials.gov registration record for the tofersen EAP can be found here.

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

Update as of April 22, 2022:

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 disease 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 in some countries outside of the U.S. (early access to aducanumab is not available in the U. S. as the product is commercially available): an EAP and a Charged Managed Access Program (CMAP). We are working to assess the feasibility of the implementation of these programs on a country-by-country basis.

Effective April 22, 2022, Biogen has withdrawn its Marketing Authorization Application (MAA) for aducanumab from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) following interactions with CHMP indicating that the data provided thus far would not be sufficient to support a positive opinion on the marketing authorization of aducanumab by the EMA.

As a result, the aducanumab EAP will be closed in countries in the European Union that fall under the EMA jurisdiction, and no new requests for access to aducanumab will be accepted in those countries.

Biogen will continue to advocate for patients, and remains committed to research and development as well as to its collaboration with Eisai, working toward the goal of bringing new treatment options to people living with Alzheimer’s disease.

All access programs are subject to local regulatory and health authority requirements, and 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.

How to Submit a Request

All requests must be submitted by a licensed 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

Our Early Access Guiding Principles

Biogen is equally committed to enacting early access to products in development for qualifying patients, and communicating with the patient community, healthcare providers (HCPs), and healthcare systems on the 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 regulation.
  • 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.

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.

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