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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.
The individual compassionate use early access program for the most rapidly progressing SOD1-ALS patients has started as planned. The first dose of tofersen was shipped on July 13 and will be at the site this week for administration. Biogen has promptly responded to all completed access requests received thus far and stands ready to ship product as soon as requesting physicians have secured approvals from their institution and applicable regulatory body. Physicians may submit requests on behalf of patients who meet the inclusion/exclusion criteria by emailing MedicineAccess@clinigengroup.com.
We plan to initiate the Part 2 compassionate use early access program for the broad SOD1-ALS population, if results from the Phase 3 study indicate that tofersen is safe and effective, and if no further studies are required.
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.
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.
At Biogen, we believe our number one priority 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.
Biogen is equally committed to enacting access mechanisms for products in development for qualifying patients and communicating to the patient community, healthcare providers (HCPs), and health care systems on timing of such mechanisms. The following principles guide decisions to provide access to our medicines:
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
COMPASSIONATE USE ACCESS
People with a serious or life-threatening condition for which there are no suitable authorized alternative therapy options in their country, and who are not eligible or able to participate in a clinical trial or early access program (EAP), may be eligible for compassionate use access. We will consider requests for compassionate use in the following circumstances:
For already approved products if a patient meets specific criteria.
For investigational products for rare, imminently life-threatening diseases in cases that would not interfere with investigational trials that support the product’s development or potential market approval.
In the case of tofersen, we are planning to allow compassionate use access beginning in mid-July 2021 for a subset of the most rapidly progressing SOD-1 ALS patients. Learn more.
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 email Biogen Medical Information at medinfo@biogen.com.
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 if you are based in the United States or email medinfo@biogen.com.
FOR DISTRIBUTORS/WHOLESALERS
If you are a distributor or wholesaler and would like further information, please email Biogen Medical Information at medinfo@biogen.com.
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