Catherine, Thyroid cancer patient

We believe that developing life-changing therapies carries with it the responsibility to address barriers that may impede patients’ access

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We are committed to increasing access to the treatments we provide, regardless of patients’ location, financial situation, or other circumstances.

Our top priority

All the work that goes into creating an effective therapeutic product means nothing if that treatment can't reach the patients who need it. But whether for financial, geographic, or logistical reasons, access to treatment remains an ongoing challenge for many patients around the world. That's why our commitment to patients—and our efforts to ensure their access to our therapies—extends far beyond the lab and manufacturing plant.

We works with local governments around the world to secure timely approval of our products. While approvals are pending, we've helped patients with urgent medical needs obtain access to our therapies through compassionate use or expanded access programs. We work with national health services and private insurers to establish coverage for our products. And where coverage is not available, we work to facilitate free treatment access, help patients find alternative funding, and help countries establish sustainable health care systems. We also provide a range of educational and support services to raise disease awareness and advocate on behalf of patients' needs.

See also...

Hijab, Gaucher disease patient

Compassionate use

We understand that many patients may have particular interest in accessing our therapies prior to approval. While safety is our priority, we may provide eligible patients, without any available treatment options, access to an unapproved investigational treatment through our compassionate use program.

Health Care Reform in the United States

We are committed to helping patients access our therapies. Because U.S. health care legislation can be subject to changes, we continually monitor the health care environment to understand how it may affect patients with rare diseases.

We understand patients and their families may have concerns and we are committed to maintaining a high level of service through our patient support programs that include:

  • Helping patients navigate the complexities of their insurance plans (both private and public)
  • Researching alternative forms of funding and reimbursement
  • Providing access to educational resources and programs for patients to learn more about their disease and treatment
  • Assigning individual case managers who work one-on-one with patients in the U.S. to help obtain ongoing access to treatment
  • Providing charitable or subsidized treatment for eligible patients with no other options
Samantha, Multiple Sclerosis patient

Reaching out to patients

We have long been an industry leader in developing support programs and services patients obtain access to our treatments.