We have the opportunity and responsibility to bring transformative treatments to patients. We are committed to research that uncovers the underlying biology of complex diseases. Our focus is on illnesses with few, if any, treatment options.

We support policies in the US and globally that help ensure patient access to these therapies, bolster innovation and drug discovery, and create a sustainable business environment. To that end, we work to be both transparent in regard to our policy objectives and clear in how we manage our business and engage with stakeholders. 

Pricing philosophy

We recognize that prices for and access to treatments are chief concerns for patients, providers, payers, and policy makers; and we work with all parties to help ensure that  patients are not denied access to a life-changing therapy.

Public policy

By maintaining open dialogue with policymakers, we work to help advance policies that facilitate a healthy research environment and ensure patient access to new and improved treatments.

Access to investigational therapies

Patients may potentially gain access to an investigational therapy three ways: through clinical trials, expanded access programs (EAPs), and single patient access or emergency use.


Explore our perspectives, from insights to innovation.