Biogen’s commitment to patient communities is in our DNA. We are driven by the unmet needs of people living with serious neurological and neurodegenerative diseases, their families and caregivers as well as society. Our employees care deeply about what they are doing because they come to work every day knowing that hundreds of thousands of people worldwide depend on Biogen and our products to make a meaningful difference in their lives.
As a pioneer in biotechnology, we have one overarching focus: developing novel medicines with the potential to improve the lives of people living with devastating neurological and neurodegenerative diseases. Our commitment to improving lives goes beyond scientific research and development. We prioritize engagement with patients and caregivers to understand their needs and identify how we can help them achieve better health outcomes.
We proactively engage with a variety of stakeholders as we work to achieve our goal of ensuring broad access to our treatments through clinical trials and expanded access programs (EAPs). We also recognize that prices for, and access to, treatments are chief concerns for patients, providers, payers and policy makers, and we work with all parties with the goal of ensuring patients are not denied access to life-changing therapies.
Ultimately, we measure our success by the impact we have on individuals living with these diseases as well as their families and caregivers, and the opportunity to change lives.
We believe that no other disease area holds as much need or as much promise for medical breakthroughs as neuroscience. Our focus on neuroscience, our deep scientific expertise and our courage to take risks make us leaders in the research and development of medicines with the aim of transforming neuroscience to benefit patients and society.
The field of neuroscience is vast, with many intersecting disciplines. Therefore our strategy is to focus on four core and four emerging growth areas. Our core growth areas include:
Our emerging growth areas include:
We have added or advanced 15 clinical programs across since the beginning of 2017. We are working to build a multi-franchise therapeutic portfolio and to create new sources of value by diversifying our pipeline in order to deliver new treatments and improve access to critical therapies. In 2018 we made progress in movement disorders such as Parkinson’s disease and in neuromuscular disorders such as ALS.
We announced positive interim results from our Phase 1 study of BIIB067 in amyotrophic lateral sclerosis (ALS) with superoxide dismutase 1 (SOD1) mutations, a genetic form of ALS representing approximately 2% of all ALS patients, developed in collaboration with Ionis Pharmaceuticals. The results demonstrated both proof-of-biology and proof-of-concept with a concordance across multiple clinical and biomarker endpoints.
In 2018 we maintained our market leadership in our core franchise of multiple sclerosis (MS) and made progress toward building a neuromuscular disease franchise with the expansion of SPINRAZA, the first approved treatment for spinal muscular atrophy (SMA), a rare neurological disease By the end of 2018 SPINRAZA had been approved in over 40 countries and had received formal reimbursement in 30 countries.
We continued to build momentum by adding depth in our core growth areas (MS and neuroimmunology, AD and dementia, movement disorders and neuromuscular disorders) and by advancing our emerging growth areas (acute neurology, neurocognitive disorders and pain).
We continue to explore new opportunities and external innovation. In December 2018 we entered into an agreement with C4 Therapeutics to investigate the use of protein degraders for neurological diseases in our areas of interest. In January 2019 we entered into an agreement with Skyhawk Therapeutics, Inc. to leverage their SkySTAR technology platform with the goal of discovering innovative small molecules.
Read more about our approach to R&D through Patient Focused Drug Development and our MIT Consortium collaboration in our Spotlight Stories feature.
Learn more about our scientific expertise and our pipeline.
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:
Clinical Trials
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:
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.
Through ongoing dialogue, dedicated research and interactions with advocacy groups, we support patients and listen to and, if possible, work to help address their key concerns – from the safety and availability of our therapies to the costs of treatment, the overall patient experience and the long-term health outcomes. We help patients and their support networks navigate the emotional and logistical considerations that often accompany a chronic disease, and we provide important therapeutic information with the aim of enhancing their quality of life.
We believe it is ideal for patients to be backed by services that support them emotionally, financially and medically. By learning from and working with patient advocacy organizations, we are able to better understand the needs and challenges patients face as we work to improve access to treatments and services. These organizations provide a critically important voice, representing the interests of patients and families affected by diseases throughout the healthcare system. As part of this collaboration, we met with leaders of patient advocacy organizations in the U.S. and E.U. in 2018 to develop recommendations on how to support early, ongoing patient involvement in the drug development process with the goal of being able to deliver potentially high-value treatments to patients with unmet needs.
We also prioritize engagement with patients and caregivers to understand their needs and identify how we can help them achieve better health outcomes. For example, we successfully advocated for the addition of SMA to the Recommended Uniform Screening Panel for newborns in the U.S. in 2018, opening the door to newborn screening for SMA at the state level. We also launched SMA Identified – a new program in collaboration with genetics company Invitae Corporation to offer no-charge genetic testing to individuals suspected of having, or have been clinically diagnosed with, SMA. We have also been working closely with those affected by SMA through the disease education website Together in SMA™. This program provides information about SMA and its symptoms, insight into care options and perspectives on a range of topics – such as nutrition and adaptive equipment – from experienced caregivers and healthcare professionals.
In addition to our Above MS™ program, which offers tips, tools and inspiration for people living with multiple sclerosis (MS), we launched the Aby mobile app in the U.S. and Cleo mobile app in Europe in 2018 to provide access to tools and resources based on feedback from people living with MS and healthcare providers. In the United Kingdom, our 1MSg™ campaign encourages people with MS to work with medical specialists to educate themselves about their condition and how to best manage their treatment. Our MS PATHS program, an innovative collaboration with ten leading MS centers, uses technology to capture real-time, standardized data to help us better understand the disease.
Read more about our efforts to help patients through the SMA Identified program and the Aby and Cleo MS apps in our Spotlight Stories feature.
Learn more about our Patient Advocacy Program.
