PHILADELPHIA, June 17, 2015 /PRNewswire/ -- 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
http://www.cslbehring.com/.
Media Contact:
Chris
Florentz, CSL
Behring
610-878-4316
Christopher.Florentz@cslbehring.com
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SOURCE CSL Behring