At Biogen, our goal is to improve the lives of patients by researching and developing new and innovative therapies. Before these therapies are commercially available to patients, they must go through clinical trials and be approved by a regulatory authority — such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) — to treat a specific disease. Once our products are approved by regulatory authorities, we begin the complex task of ensuring patient access to our therapies through public and private health care programs.
We employ two complementary approaches to help accomplish this:
- Investigational Access through clinical trials, expanded access programs (EAPs) and single patient access or emergency use.
- Affordability Access by adhering to an access and pricing philosophy that helps secure reimbursement in public and private health care programs and offers financial assistance to patients who are otherwise unable to access our medications. We assess the value of our therapies in terms of clinical outcomes, improvements in daily living and quality of life, and whether the medicine meets a high unmet medical need and advances clinical practice.
Clinical trials are an essential part of the drug development process. They are designed to answer specific research questions related to the efficacy and safety of a potential therapy. Deciding to participate in a clinical trial requires careful consideration because, in most cases, the therapies being investigated in clinical trials are not yet approved by regulatory agencies. We are grateful to the patients, healthy volunteers, healthcare providers, hospitals and clinics that participate in testing our investigational therapies.
For some patients, participation in a clinical trial means access to a potentially life-improving medication when there may not be any other options. These experimental treatments are not available elsewhere and sometimes can be a last resort for patients who have tried many other therapies with limited or no success.
We added or advanced 11 clinical stage programs in 2018 and we had more than 140 active studies underway with approximately 32,000 patients enrolled on all studies active in 2018.1
Learn more about our approach to clinical trials.
Access to Investigational Therapies Outside of Clinical Trials
Our Expanded Assess Programs (EAPs) bridge an important availability gap for therapies that have not yet become commercially available, providing access to treatment for patients with unmet medical needs while the regulatory approval process and reimbursement negotiations take place.
The complexity of the healthcare marketplace requires us to always be vigilant and committed to ensuring patients who need our therapies have access to them. For example, our EAP for spinal muscular atrophy (SMA) offers early access for infant and childhood SMA patients who would not otherwise be eligible for clinical trials, including supporting dozens of SMA patients in Poland until the Polish Ministry of Health recently approved reimbursement for SPINRAZA. Including clinical trials and our EAP, as of December 31, 2018, more than 6,600 patients have benefited from this remarkable therapy. In some situations, Biogen may be able to provide access to investigational therapies through what is essentially emergency use, also known as single patient access.
Approach to Pricing and Access
As pioneers in neuroscience, we need to continue adapting our thinking and approaches to pricing and access in an increasingly ambiguous and changing environment. Our intent is to describe a clear and transparent series of principles to guide our pricing and access actions for our breakthrough therapies.
When pricing our medicines, we consider the following key principles:
- value to patients
- present and future benefits to society
- fulfilling our commitment to innovation
- evolution towards value based health care
- affordability and sustainability.
We believe it is our shared responsibility with all healthcare stakeholders to find solutions to ensure patient affordability for new innovations. We partner with health care systems so that patients can continue to access our medicines in a sustainable way and we remain flexible to ensure that our medicines are affordable for patients across a wide range of economic circumstances. This includes recognizing that economic circumstances and ability to pay for medicines vary greatly across countries. We may pursue different approaches to make our drugs accessible depending on a country’s economic situation, health care infrastructure and overall commitment.
For example, we are currently developing an SMA-focused individual patient humanitarian access program in lower-middle and lower income countries to bring medical education and treatment to SMA patients in need. With a planned launch in 2019, we plan to pilot the program in India first and hope to expand pilots to other countries in the future.
Learn more about our access programs and our pricing principles.
1 Includes patients in all active studies between pre-Study Management Team (SMT) formation and Clinical Study Report (CSR) as defined by internal systems in 2018