Our way of leading in rare diseases
We maintain a sharp focus on rare diseases to deliver high impact for patients, sustained corporate growth and increased value for society.
We are focused on developing and delivering life-changing medicines for underserved patients with rare diseases
We are sharply focused on patient populations with extraordinarily high unmet needs — often, their conditions are not well understood, even within the healthcare community, and there may be few or no effective therapies. We bring unique expertise and innovative technologies to select therapeutic areas where we see tremendous opportunity to enhance patients’ lives and establish a durable leadership position in the industry. While rare conditions form the core area of highest emphasis, we often see additional opportunities to apply our expertise and therapeutic area leadership to certain highly specialized, non-rare indications where we see the opportunity to bring innovation to enable patients to live fuller lives.
We build a portfolio of products through a combination of focused internal research and development, collaborations with leading institutions, and strong business development capabilities
We begin by identifying gaps in current medical care where patients with serious illnesses are significantly impacted by either the lack of effective therapies or shortcomings in the available treatments. We listen carefully to patients, advocacy groups, and our network of leading clinicians to deeply understand patient needs. We seek to match these opportunities with promising scientific innovations which offer the potential to deliver a meaningful therapeutic advance. We are agnostic to the specific technologies involved and where they originate — we are exclusively focused on identifying the most promising ways to address patient needs. Frequently, this mindset leads us to collaborate and partner with leading research institutions and emerging “start-up” companies with novel therapeutic approaches. We employ a sophisticated business development capability that enables us to continually survey the landscape, identify promising approaches, and rapidly strike agreements to advance these potential next-generation therapies. We then apply our specialized, world-class development and commercialization resources to rapidly deliver these innovations to patients
As the leader in rare diseases, we invest in and develop the unique capabilities required for successful development and commercialization of high-impact products
We have built a broad constellation of unique, industry leading capabilities that allows us to maintain our leadership in rare diseases, and that positions us as the partner of choice in this area. For example, within our R&D organization we have specialized expertise that enables us to address the challenges of developing new medicines in rare disease indications, such as recruiting hard-to-find patients, navigating ill-defined current standards of treatment, and pioneering unprecedented regulatory pathways. Similarly, once new therapies are approved, we then apply sophisticated approaches to increase diagnosis rates, support patients as they initiate therapy, and productively engage with patients, leading treatment centers and advocacy organizations. Our nimble global supply network is also tailored to the unique challenges of reliably delivering potentially life-saving rare disease therapies to often diffuse global patient populations.
Our scale and focus specifically on rare diseases enables us to invest deeply in these capabilities, with high impact. We apply our expertise and skills to the task of bringing new, cutting-edge therapies for conditions where, in many cases, no therapy has existed. Combined with our sharp focus on execution, we ensure that the most underserved patients around the world have access to the critical therapies they need.