Shire Leads New Initiative to Reveal Challenges Facing Rare Disease Community

Shire Leads New Initiative to Reveal Challenges Facing Rare Disease Community

  PR Newswire

  LEXINGTON, Massachusetts, Nov. 19, 2012

- Impact Report to assess holistic needs of rare disease patient and medical

LEXINGTON, Massachusetts, Nov. 19, 2012 /PRNewswire/ -- Shire plc (LSE: SHP,
NASDAQ: SHPGY) today announced a new initiative – the Shire Rare Disease
Impact Report – to research the health, psycho-social, societal and economic
impact of rare diseases in patient and medical communities. Comprehensive
surveys of patients, caregivers, physicians, payors and thought leaders in the
United States and the United Kingdom are underway. Survey results will be
published and available in the first half of 2013.

Rare diseases are conditions that affect a small portion of the population and
are often chronic, progressive, degenerative, Iife-threatening and
disabling.(1) While individual rare diseases are uncommon and disparate,
collectively there are approximately 7,000 different types of rare diseases
and disorders affecting an estimated 350 million people worldwide.(2)

The Shire Rare Disease Impact Report aims to uncover the unique challenges
facing the rare disease community in areas such as diagnosis, scientific
understanding, treatment options, and social services. The Impact Report will
contribute to the continued effort to better understand these complex diseases
and how best to optimally manage them for the greatest patient benefit.

"Despite the progress that has been made over the past few decades in the rare
disease space, there is still an urgent need to better understand this
community and its needs," says Nicole Boice, founder and CEO, Global Genes |
R.A.R.E Project. "I'm thrilled to be involved in the development of this Rare
Disease Impact Report as its findings will help elevate awareness for the rare
disease community and guide future research and education for affected
patients and their families."

An external advisory board anchored by thought leaders in the medical,
advocacy, health policy and health economics fields will lend its expertise to
the development of the Impact Report. Advisory board members include Nicole
Boice, founder and CEO, Global Genes | R.A.R.E Project; Dr. Priya S. Kishnani,
Division Chief, Medical Genetics, Duke University Medical Center; Tomas
Philipson, Daniel Levin professor of public policy studies, The University of
Chicago; Alastair Kent, director, Genetic Alliance UK; Dr. Christian J.
Hendriksz, Clinical Lead, Adult Inherited Metabolic Disorders, Salford Royal
NHS Foundation Trust; and Mike Drummond, professor of health economics,
University of York.

Shire Human Genetic Therapies (HGT), the rare disease business of Shire, has
over twenty years of experience discovering, developing, manufacturing, and
commercializing novel therapies and solutions for rare diseases. The Company
has built and invested in a pipeline of innovative products and solutions to
treat rare diseases. The Rare Disease Impact Report will be the first research
to go beyond identifying unmet medical needs within the rare disease

"At Shire, patients are at the heart of everything we do and we continually
strive to provide support for those touched by rare diseases," said Sylvie
Gregoire, president, Shire HGT. "We hope the findings from this Rare Disease
Impact Report will provide the rare disease community at large with new,
unique insights on how best to address the holistic needs of the rare disease
community and drive important conversations and innovation. Shire is delighted
to take the lead in bringing this to the attention of all those who can
transform the lives of rare disease patients for the better."

For further information please contact:

Jessica Cotrone (Human Genetic Therapies) +1 781 482 9538

Notes to editors


Shire's strategic goal is to become the leading specialty biopharmaceutical
company that focuses on meeting the needs of the specialist physician. Shire
focuses its business on attention deficit hyperactivity disorder, human
genetic therapies, gastrointestinal diseases and regenerative medicine as well
as opportunities in other therapeutic areas to the extent they arise through
acquisitions. Shire's in-licensing, merger and acquisition efforts are focused
on products in specialist markets with strong intellectual property protection
and global rights. Shire believes that a carefully selected and balanced
portfolio of products with strategically aligned and relatively small-scale
sales forces will deliver strong results.

For further information on Shire, please visit the Company's website: .


Statements included herein that are not historical facts are forward-looking
statements. Such forward-looking statements involve a number of risks and
uncertainties and are subject to change at any time. In the event such risks
or uncertainties materialize, the Company's results could be materially
adversely affected. The risks and uncertainties include, but are not limited
to, risks associated with: the inherent uncertainty of research, development,
approval, reimbursement, manufacturing and commercialization of the Company's
Specialty Pharmaceuticals, Human Genetic Therapies and Regenerative Medicine
products, as well as the ability to secure new products for commercialization
and/or development; government regulation of the Company's products; the
Company's ability to manufacture its products in sufficient quantities to meet
demand; the impact of competitive therapies on the Company's products; the
Company's ability to register, maintain and enforce patents and other
intellectual property rights relating to its products; the Company's ability
to obtain and maintain government and other third-party reimbursement for its
products; and other risks and uncertainties detailed from time to time in the
Company's filings with the Securities and Exchange Commission.


1.European Organisation for Rare Diseases (EURORDIS) website. "Rare
    Diseases: understanding this Public Health Priority." . Accessed
    October 25, 2012.
2.The Global Genes Project website. "RARE Facts and Statistics." . Accessed October 25, 2012.

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