Thought Leaders in Europe and US Address Challenges to Making Rare Diseases a Global Public Health Priority

Experts on rare disease from the medical, scientific and patient advocacy communities examine critical issues and solutions in Paris and Bethesda

PARIS, February 28, 2011 - CSL Behring announces a special edition of its Key Issues Dialogue series
released in conjunction with Rare Disease Day 2011, an international event to
raise awareness of rare diseases and their impact on people's lives. The
Dialogue spanned two continents and provided a platform for sharing and
exploring important information and ideas for patients, caregivers and
healthcare providers.

(Logo: photos.prnewswire.com/prnh/20100914/PH63692LOGO )

Participants

    - Yann Le Cam, chief executive officer, European Organisation for Rare
      Diseases (EURORDIS)
    - Peter Saltonstall, president and chief executive officer, National
      Organization for Rare Diseases (NORD), US
    - Dr. Lennart Hammarstrom, M.D., Ph.D., professor of clinical immunology,
      Karolinska Institute, Stockholm
    - Dr. Stephen Groft, Pharm.D., director, Office of Rare Diseases
      Research, National Institutes of Health (NIH), US
    - Peter Turner, president, CSL Behring and chief operating officer, CSL
      Group
    - Dennis Jackman, senior vice president, public affairs, CSL Behring

Creating a more favorable environment

One of the main challenges the participants addressed is how to create a
more favorable environment for research and treatment of rare diseases. "The
first priority is to make rare diseases a long-term priority both for
research and public health in America and Europe," Yann Le Cam said. "Then we
can create a more favorable environment and work on the specifics of drug
development, creating centers of care, providing information and registries,
and also obtaining the funding for the long-term approach."

The participants discussed the need to expand efforts to inform the rare
diseases community about clinical studies and the location of experts,
particularly in the US. There was general agreement that the European
countries have placed more emphasis on identifying centers of excellence than
in the US so that patients with rare diseases know where to go for treatment.
According to NORD's Peter Saltonstall, public policy is another major issue.
"Rare diseases are not well known," Mr. Saltonstall said. "The people
controlling funding and regulation are not as well informed as they need to
be."

Concerns around diagnosis

In the area of primary immune deficiency diseases, Dr. Hammarstrom said
that although the span from debut of disease to diagnosis and treatment has
been shortened, there is still a considerable time lapse. "We need to press
this point," Dr. Hammarstrom said. "What about neonatal screening? In this
challenge the US has taken the lead and Europe is lagging behind. We have
been discussing it at the EU Parliament."

Neonatal screening, which has gained acceptance in the US, was viewed by
the participants as a global step worth exploring. The US has taken steps to
expand newborn screening programs for 29 different diseases and there is
discussion of expanding the programs to include additional disorders. One
concern is what to do with diagnosed patients. Is there sufficient
infrastructure to transfer them into research or treatment centers for the
individual rare diseases?

Yann Le Cam, in his capacity as Vice Chair of the EU Committee of Experts
on Rare Diseases, explained that a policy project sponsored by the European
Commission is developing the future policy of the EU on newborn screening. He
added that the project is ongoing and that "an explicit policy should be
proposed in 2012 and possibly recommended to all EU member states before
2014."

Harmonization

The importance of harmonization between the European Medicines Agency
(EMA) and the US Food and Drug Administration (FDA) to reduce the cost of
clinical trials, especially for makers of therapies used to treat small rare
disease populations, was another key issue analyzed by the participants.
Clinical trials for rare disease therapies can cost more than 75 million euro
($100 million). As a result, Dr. Hammarstrom said some necessary clinical
trials will never be done. "This means that certain types of therapies will
be unavailable on both sides of the Atlantic," he said.

In addition, according to Peter Turner, Europe and the US are seldom
aligned on clinical trial design. "Quite often we have to design a European
trial and then a US trial because of requirements differing across the
Atlantic," Mr. Turner explained. Yann Le Cam noted that EURORDIS has been
working toward a solution by partnering with the EMA and the FDA on a
retrospective analysis of orphan drug designations versus applications to
better understand potential common approaches to new therapies. "There were
many more commonalities than differences," Mr. Le Cam said. "Ninety-two
percent of the decisions were the same."

Availability

There is a further complication in that the availability of treatments
for rare diseases differs from country to country. According to Peter
Saltonstall, if a therapy has been used on patients for period of time in
Europe and the European safety and efficacy standards have been demonstrated,
it is important to find a way to apply that information within the U.S.
market. "There needs to be a way for patients to get the drugs and therapies
more quickly," Mr. Saltonstall said.

Yann Le Cam pointed out that globally, 80-85% of people with rare
diseases are treated with off-label drugs and therapies. "We should encourage
the collection of data during off-label use, both on safety and efficacy.
This would be good medical practice that can lead to better care for patients
and also a better allocation of resources," Mr. Le Cam said.

The nature of rare diseases is such that there are multiple organs and
multiple systems involved in individual diseases. This makes it all the more
important to bring the partners together, not only the academic researchers,
but also the industry, the patient advocacy groups and government research
and regulatory organizations. The lack of coordination, it was agreed by the
participants, will harm any possibility of rapid product development. This
can be offset by becoming more proactive, identifying the opportunities and
then utilizing the available resources.

About CSL Behring

CSL Behring is a leader in the plasma protein therapeutics 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 indicated for the treatment of coagulation
disorders including hemophilia and von Willebrand disease, primary immune
deficiencies, hereditary angioedema and inherited respiratory disease. The
company's products are also used in cardiac surgery, organ transplantation,
burn treatment and to prevent hemolytic diseases in newborns.

CSL Behring operates one of the world's largest plasma collection
networks, CSL Plasma. CSL Behring is a subsidiary of CSL Limited (ASX: CSL),
a biopharmaceutical company headquartered in Melbourne, Australia. For more
information, visit www.cslbehring.com.

The current and previous Dialogues are available for download.

    Contact:
    Chris Florentz, King of Prussia, USA    Thes Hilfiker, Bern, Switzerland
    +1-610-878-4316                         +41-31-344-53-23
    Christopher.Florentz@cslbehring.com     Therese.Hilfiker@cslbehring.com

Chris Florentz, King of Prussia, USA, +1-610-878-4316, Christopher.Florentz at cslbehring.com, or Thes Hilfiker, Bern, Switzerland, +41-31-344-53-23, Therese.Hilfiker at cslbehring.com