Our Patients

Innovation
Patient Safety
Patient Support, Education, & Financial Assistance

Innovation

We seek to push technology forward for the benefit of patients, with the ultimate goal of developing cures for pulmonary hypertension and other end-stage organ diseases through our organ manufacturing program, which includes multiple technologies and programs to create an unlimited supply of transplantable organs. Please see our Pipeline page to learn more.

Rare Disease Focus

The defining characteristics for our patients are two-fold: their conditions are extremely rare and they are life-threatening. United Therapeutics is here to support our small patient populations through all aspects of their treatment.

TARGET US PATIENT POPULATIONS:

45,000 PAH
30,000 PH-ILD
800 NB

We were granted orphan drug exclusivity (ODE) for our approved PAH drugs, Remodulin and Orenitram, and we have been granted orphan drug designation (ODD) for Tyvaso in development for idiopathic pulmonary fibrosis (IPF). Our focus on these rare conditions, coupled with ODE/ODD, compels us to push forward with our innovations, overcome any obstacles to adoption of our treatments, and — for lack of a better phrase — run down the corridors of indifference.

This commitment to our rare disease patients comes in many forms. We are a long-standing supporter of the Pulmonary Hypertension Association, and we also sponsor our own PAH Initiative. We also have a dedicated “ASSIST” team to help our patients navigate insurance coverage and explore other financial assistance programs.

We strive to ensure that no patient in the United States who needs and wants our therapies goes without treatment.

Lung Biotechnology

Lung Biotechnology PBC, a wholly owned subsidiary of United Therapeutics, houses our organ manufacturing work which is aimed at dramatically increasing the supply of transplantable organs. It is also the first public benefit corporation (PBC) subsidiary of a public biotech or pharmaceutical company. As a PBC, Lung Biotechnology is chartered with the express purpose of “address[ing] the acute national shortage of transplantable lungs and other organs with a variety of technologies that either delay the need for such organs or expand the supply,” and is expressly allowed to consider the interests of this public benefit purpose and to operate in a responsible and sustainable manner in addition to the emphasis on shareholder value.

REHABILITATING DISCARDED LUNGS INTO LIFE-SAVING ORGANS

Since 2014, we have been developing technologies to convert donor lungs deemed unsuitable for transplantation into usable lungs through ex-vivo lung perfusion (EVLP). To date, our EVLP procedures have resulted in successfully transplanted lungs for patients with end-stage lung disease. These are donor lungs that otherwise would have been discarded, absent the use of EVLP technology. We have two facilities dedicated to these technologies, including a site net zero facility we constructed on the campus of Mayo Clinic Jacksonville.

Animal Welfare

United Therapeutics is committed to and abides by all ethical requirements related to animal welfare in research, teaching, and testing. Research involving the use of live animals must be approved by Institutional Animal Care and Use Committees (IACUC) at both our in-house facilities and our partner institutions, a requirement of U.S. federal and state laws. The IACUC has a key oversight role, including the review and approval of animal use activities and inspection of animal facilities. In addition, we comply with the “Three Rs” (Replacement, Reduction, and Refinement), widely accepted ethical principles that are now embedded in the conduct of animal-based science in many countries around the world. United Therapeutics, and its subsidiaries, strive to partner with organizations that have earned accreditation from AAALAC International, a non-profit that assesses organizations that use animals in research, teaching or testing. More than 1,000 companies, universities, hospitals, government agencies, and other research institutions in 49 countries have been accredited through AAALAC’s rigorous program and site evaluation review.

Patient Safety

Protecting and improving patient health is our most important goal. To ensure our patients’ safety, we have a robust safety governance structure and a comprehensive set of standards designed to ensure that we have proper oversight over pharmacovigilance (PV) activities. We extensively monitor the use of our therapies, throughout the product life cycle starting from development programs and continuing into the post approval phase, to identify potential side effects, and to ensure a positive benefit-risk ratio. We provide mandatory training to our employees and our third-party collaborators to ensure proper identification, rapid collection, and analysis of adverse events (AE) regarding the use of our therapies.

Patient safety risk minimization is at the core of our safety and PV practice. Our PV team is continuously monitoring AEs for any key risks and stands ready to enact risk minimization measures in order to prevent or minimize AE-associated risks for patients using our therapies. These principles also guide our development efforts and strategies. For example, one of our lead products, Remodulin, is delivered by either subcutaneous administration, which often causes site pain reaction, or by intravenous administration, which involves the use of an indwelling catheter and carries a risk of bloodstream infections. We are developing RemoPro™, a novel prodrug version of treprostinil, to help mitigate or eliminate these concerns by delivering a continuous infusion of treprostinil subcutaneously without the infusion site pain associated with Remodulin today.

Supply Chain Management

United Therapeutics maintains a rigorously compliant GxP Quality & Compliance program covering those aspects of our supply chain that could impact the quality and safety of our products. We use more than 700 pre-qualified raw material vendors and service providers to support clinical and commercial business operations. Greater than 80% of these are located domestically, with the balance distributed worldwide, including Canada, Europe, and Asia. Procedures are in place to assess every supplier to ensure compliance with our quality standards. These procedures are risk-based, creating more focus on critical suppliers such as excipient manufacturers. Our GxP program contains a rigorous auditing and vendor selection approach, using both internal and third-party expert resources.

Patient Support, Education, & Financial Assistance

We take a holistic approach to helping our patients and their families by expanding our focus beyond just treatments and therapies to include a variety of support, education, and assistance programs related to our products. A few of our key programs are outlined on our patients page on our corporate website. Among other initiatives, we sponsor the PAH Initiative, which is committed to improving the lives of patients with PAH through education and empowerment, and we are a long-standing supporter of the Pulmonary Hypertension Association.

We have dedicated teams to ensure that patients have access to our treatments. One team works with insurers to strive for the best possible coverage for our drugs, and we are proud of the results we have achieved. We also have a variety of options for patients who may still need financial assistance to pay for our treatments. And we have a dedicated team, ASSIST (Access Solutions and Support Team), that helps our patients and their families navigate this complex area. Our ASSIST associates are ready to work with them, their health care providers, and specialty pharmacies to help them understand their insurance coverage, educate them about available financial assistance options, and answer their questions.

Finally, patient education is critical to ensuring effective management of a complex and life-threatening disease such as PAH. Many PAH therapies are specialty medications that bring with them unique complexities requiring in-depth patient education to ensure safe and effective administration. For this reason, we engage a network of contracted specialty pharmacies with dedicated nurses and pharmacists across the country who are experienced in training and educating patients on how to safely and effectively use our medications. It is this level of interactions with patients — either through specialty pharmacy nursing or the pharmacy staff — that allows us to educate our patients as effectively as we possibly can.

People

Planet and
Communities

Principles and
Other Priorities