Defining and Differentiating the Value of Therapies Used to Treat Rare Disease Patients is a Challenge for Payers

Panel of experts examines how the value of biopharmaceutical technologies is assessed and how it affects drug development at BIO International Conference

PHILADELPHIA, Pa. — 17 June 2015

The cost of medicines in general and biopharmaceutical therapies used to treat rare diseases in particular has faced increased attention from payers and other stakeholders in recent years.

Determining how the value of biopharmaceutical technologies is assessed presents a special and critically important challenge for stakeholders. It can also have an impact on drug development and patient access to care in the rare disease space, where 400 million people around the world have a rare disease. Approximately 7,000 rare disorders have been identified, but only a relatively small number of therapies are available to treat them.

This afternoon, a panel of experts moderated by Dennis Jackman, CSL Behring’s Senior Vice President of Global Healthcare Policy and External Affairs, will address this question and identify examples of effective mechanisms of defining and differentiating the value to patients and healthcare of rare disease therapies at 2015 BIO International Conference. Panelists include: Amanda Bartelme, Director Avalere Health; Pamela Gavin, COO National Organization for Rare Disorders, and Jim Geraghty, Principal Third Rock Ventures.

“It’s important that healthcare systems worldwide recognize the special nature of rare disease therapies,” Jackman said. “In determining the value of these therapies, consideration must be given to factors such as societal value, severity of the condition, lack of alternative treatments, data generation challenges due to small populations, lack of comparators, and endpoints and other factors. It’s important that payers look at the holistic value of the therapy and not just its cost in order to help ensure access to these lifesaving therapies. Rare disease therapies are still a small portion of the total drug spend, but make a huge difference in the lives of patients and their families. Manufacturers also understand the need to produce assessments that help to demonstrate this value and continue to progress achievable ways to do that.” Jackman noted that, “there are particular challenges in researching and developing rare disease therapies. The way value is assessed and the way access to treatment is granted will have a significant impact on innovation and the sustainability of those efforts, and, ultimately patient care.”

About CSL Behring

CSL Behring is a leader in the biotherapeutics industry. Committed to saving lives and improving the quality of life for people with rare and serious diseases, the company manufactures and markets a range of plasma-derived and recombinant therapies worldwide.

CSL Behring therapies are used around the world to treat coagulation disorders including hemophilia and von Willebrand disease, primary immune deficiencies, hereditary angioedema and inherited respiratory disease, and neurological disorders in certain markets. The company’s products are also used in cardiac surgery, organ transplantation, burn treatment and to prevent hemolytic disease of the newborn.

CSL Behring operates one of the world’s largest plasma collection networks, CSL Plasma. CSL Behring is a global biopharmaceutical company and a member of the CSL Group of companies. The parent company, CSL Limited (ASX:CSL), is headquartered in Melbourne, Australia. For more information, visit


Media Contact:

Chris Florentz, CSL Behring
CSL Behring

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