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At Biogen, our goal is to improve the lives of people by researching and developing innovative therapies and obtaining regulatory approvals so we can bring these investigational treatments to the greatest number of individuals in a timely manner.
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), single patient access or emergency use based on humanitarian or compassionate grounds.
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 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 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.
Expanded Access Programs, or EAPs, may provide investigational therapies to a limited group of people 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. If an EAP program is in place, this group of people must meet specific eligibility criteria in order to gain access to the investigational treatment.
To participate in an EAP, the 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. 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, our number one 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.
EAP Guiding Principles
CLINICAL EVIDENCE
When therapeutic safety and efficacy of the investigational therapy is demonstrated, and the company has decided to file for regulatory approvals based on this evaluation.
EAPs would be initiated only after all patients in our placebo-controlled trial(s) have been given the option to receive active therapy through a long-term extension trial.
EAPs would be offered to patients with similar type and stage of disease for which the therapy is being pursued for regulatory approvals.
CLINICAL DISEASE ASSESSMENT
When based on clinical disease assessment, that patients have a life threating illness and survival could potentially be prolonged in a clinically meaningful way with the treatment of the investigational therapy.
EAPs would be offered if patients cannot be adequately treated with other therapies that are available by prescription by a healthcare provider.
EAPs would be offered for disease modifying therapies (DMT).
HEALTHCARE SYSTEM READINESS
When treatment sites (for example hospitals) have the capacity to participate in an EAP and that the EAP management requirements may not unduly burden the health care system or compromise the provision and monitoring of quality patient care.
Treatment sites must have the ability to equitably meet patient demand, especially when the patient population may be so sizeable.
During the time of an EAP which is after clinical trials but prior to reimbursed patient care, payment structures are not in place for ancillary costs of treatment. This includes healthcare personnel and patient insurance coverages. Treatment sites must have the ability to cover these costs, which is especially difficult when the patient population is so sizable.
REIMBURSED PATHWAYS
When patients may have long-term sustainable access to treatment through reimbursed pathways, and that reimbursement will not be put at risk or delayed by opening an EAP.
An EAP will have open and close dates customized to each countries’ local regulations and reimbursement process.
EQUITABLE ACCESS
After the collective needs of the broader patient population are balanced with the needs of an individual patient so that an EAP can be implemented in an equitable manner.
Existing EAP programs
Our EAPs and single patient access programs are guided by the set of principles that reflect our commitment to providing ethical access to investigational treatments.
After thoughtful consideration of our EAP guiding principles, and discussion with patient advocates and HCPs, we are able to offer the following EAP at this time:
Nusinersen (spinal muscular atrophy)
If you are a healthcare professional and would like to learn more about the EAP program for nusinersen, please contact our collaboration partner Clinigen.
COMPASSIONATE USE AND SINGLE PATIENT ACCESS
People who are not eligible or able to participate in a clinical trial – and where an expanded access program (EAP) does not exist – may request compassionate use for off-label access to approved products or single patient access for investigational therapies.
To obtain access through compassionate use or single patient access, the patient’s physician must make a formal request on behalf of the patient to Biogen. Like EAPs, compassionate use and single patient access must be approved by the regulatory authority in the physician’s country. The patient’s physician is responsible for the patient’s care and all legal and regulatory requirements while the patient is receiving the therapy.
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 single patient access 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.
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.
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