Shire Launches Report that Quantifies the Health, Psycho-social and Economic Impact of Rare Diseases

 Shire Launches Report that Quantifies the Health, Psycho-social and Economic
                           Impact of Rare Diseases

  PR Newswire

  LEXINGTON, Massachusetts, April 9, 2013



-- New findings reveal the substantial burden of rare diseases due to lack of
resources, financial challenges and emotional unrest

LEXINGTON, Massachusetts, April 9, 2013 /PRNewswire/ --Shire plc (LSE: SHP,
NASDAQ: SHPG) today launched a Rare Disease Impact Report, which uncovers the
health, psycho-social, and economic impact of rare diseases on patient and
medical communities in the United States (US) and United Kingdom (UK). The
report, developed in collaboration with an external advisory board of thought
leaders in the medical, advocacy, health policy and health economics fields,
will be distributed at the World Orphan Drug Congress in Washington, DC (April
9-11) and is available for immediate download at www.rarediseaseimpact.com .

According to more than 1,000 survey responses from a multi-stakeholder
audience sample, the Rare Disease Impact Report reveals ^1 :

  *It takes, on average, more than seven years in the US and five years in
    the UK for a patient with a rare disease to receive a proper diagnosis
  *On the journey to diagnosis, a patient typically visits up to eight
    physicians (four primary care and four specialists) and receives two to
    three misdiagnoses
  *Physicians (both primary care and specialists) often don't have the time,
    resources and information to properly diagnose/manage patients with rare
    diseases, compared to more common diseases
  *Due to the uncertainty, the lack of available information, resources, and
    economic strains, rare diseases take a major emotional toll on patients
    and their caregivers

"This Impact Report brings to light the specific barriers to quality care that
exist for patients with rare diseases; particularly the challenges in getting
an accurate diagnosis, adequate information and ongoing care," says Nicole
Boice, founder and CEO, Global Genes | RARE Project, a leading rare and
genetic disease patient advocacy organization. "I am thrilled that Shire
engaged Global Genes in this initiative and hope it will inspire the rare
disease community to work together to better meet the needs of rare disease
patients and their families."

Rare diseases are conditions that affect a small portion of the population but
are often chronic, progressive, degenerative, Iife-threatening and disabling.
^2 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. ^3 Despite the progress
that has been made over the past few decades to help improve the quality of
life for patients managing these complex diseases, there are still significant
gaps in care and barriers facing the community at large.

"The findings from our Rare Disease Impact Report are sobering," says Flemming
Ornskov, MD, Chief Executive Designate, Shire. "As a leader in rare diseases,
Shire hopes that this report will help drive forward a collaborative effort
with the patient and medical communities to address the unmet needs
identified."

Rare Disease Impact Report Findings ^1

Across the groups surveyed, findings center around three overarching
challenges:

There is a lack of resources and information to address these less common
illnesses

  *Physicians (both primary care and specialists) often don't have the time,
    resources and information to properly diagnose/manage patients with rare
    diseases, compared to more common diseases

     *The majority of physicians surveyed reported it is more difficult to
       address the needs of a rare disease patient in a typical office visit
       (92% in the US, 88% in the UK agreed) and more office visits are
       required to diagnose a rare disease patient (98% in the US, 96% in the
       UK agreed)
     *In addition, more than half of physicians stated there aren't enough
       opportunities to network with other physicians who treat rare diseases
       (54% in the US, 62% in the UK agreed)

  *From a patient and caregiver perspective, around half of those surveyed
    stated they received conflicting information from different health care
    professionals about treatment options (60% in the US, 50% in the UK
    agreed)

     *In fact, more than half of patients and caregivers stated they needed
       to provide their healthcare professionals with information on their
       rare disease (67% in the US, 62% in the UK agreed)

  *As a result of these challenges, on average, it takes 7.6 years in the US
    and 5.6 years in the UK for a patient with a rare disease to receive a
    proper diagnosis, based on survey results. Along the way, the average
    patient visits four primary care doctors, four specialists and receives
    two to three misdiagnoses

The economic impact of diagnosing and managing rare diseases is significant.
The journey to diagnosis and beyond comes with a steep price tag for many
coping with a rare disease. The long road, which frequently includes numerous
tests and physician visits, can become financially overwhelming, particularly
for those in the US as compared to the UK

  *Payor respondents reported several factors contribute to the higher costs
    of care for rare disease patients compared to more common diseases,
    including the need for more diagnostic tests (100% in the US, 80% in the
    UK agreed) and more costly diagnostic tests (100% in the US, 90% in the UK
    agreed)
  *Payors also found it difficult to make rare disease coverage decisions due
    to the lack of standards and guidelines. Almost all payors surveyed
    indicated there is less information/data available to help determine the
    standards of care for rare diseases (95% in the US, 90% in the UK agreed)
  *Although 90% of patients surveyed reported they had health coverage in the
    US:

     *55% of US respondents incurred direct medical expenses not covered by
       insurance compared to 18% of respondents in the UK not covered by the
       National Health Service
     *37% of respondents borrowed money from family and/or friends to pay for
       expenses in the US compared to only 21% of respondents in the UK

Due to the uncertainty, the lack of available information, resources, and
economic strains, rare diseases take a major emotional toll on patients

  *Patient respondents reported several emotional difficulties managing their
    disease including depression (75% in the US, 69% in the UK agreed),
    anxiety and stress (86% in the US, 82% in the UK agreed), isolation from
    friends/family (65% in the US, 57% in the UK agreed), and worry based on
    future outlook of disease (90% in the US, 91% in the UK agreed)

     *For those rare disease patients where treatment options are limited,
       overall they worry more, feel more depressed, interact less and feel
       more isolated from family and friends, compared to patients with rare
       diseases for which there are available treatments

Data Collection

In January 2013, Shire HGT conducted online surveys over a four-week period
among US and UK rare disease patients and their caregivers; physicians
treating patients with rare diseases; payors who handle reimbursements for
healthcare plans and government/institutions; and thought leaders in the rare
disease space. Surveys were fielded through the market research agency, ORC
International and also distributed by advocacy group partners Global Genes and
the Genetic Alliance UK.

The Rare Disease Impact Report Advisory Board

An external advisory board anchored by thought leaders in the medical,
advocacy, health policy and health economics fields lent their expertise to
the development of the Impact Report. Advisory board members include Nicole
Boice, Founder and CEO, Global Genes | RARE 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.

Please visit www.rarediseaseimpact.com to get more information and view the
full Impact Report.

For further information please contact:

Media Jessica Cotrone (Human Genetic Therapies) jcotrone@shire.com
+1-781-482-9538

NOTES TO EDITORS

Shire enables people with life-altering conditions to lead better lives.

Through our deep understanding of patients' needs, we develop and provide
healthcare in the areas of:

  *Behavioral Health and Gastro Intestinal conditions
  *Rare Diseases
  *Regenerative Medicine

as well as other symptomatic conditions treated by specialist physicians.

We aspire to imagine and lead the future of healthcare, creating value for
patients, physicians, policymakers, payors and our shareholders.

www.shire.com

FORWARD - LOOKING STATEMENTS - "SAFE HARBOR" STATEMENT UNDER THE PRIVATE
SECURITIES LITIGATION REFORM ACT OF 1995

Statements included in this announcement that are not historical facts are
forward-looking statements. 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, Shire's results could be materially
adversely affected. The risks and uncertainties include, but are not limited
to, that:

  *Shire's products may not be a commercial success;
  *revenues from ADDERALL XR are subject to generic erosion;
  *the failure to obtain and maintain reimbursement, or an adequate level of
    reimbursement, by third-party payors in a timely manner for Shire's
    products may impact future revenues and earnings;
  *Shire relies on a single source for manufacture of certain of its products
    and a disruption to the supply chain for those products may result in
    Shire being unable to continue marketing or developing a product or may
    result in Shire being unable to do so on a commercially viable basis;
  *Shire uses third party manufacturers to manufacture many of its products
    and is reliant upon third party contractors for certain goods and
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    manufacture products, or any failure of these third party contractors to
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    respective contractual obligations, could adversely affect Shire's ability
    to manage its manufacturing processes or to operate its business;
  *the development, approval and manufacturing of Shire's products is subject
    to extensive oversight by various regulatory agencies and regulatory
    approvals or interventions associated with changes to manufacturing sites,
    ingredients or manufacturing processes could lead to significant delays,
    increase in operating costs, lost product sales, an interruption of
    research activities or the delay of new product launches;
  *the actions of certain customers could affect Shire 's ability to sell or
    market products profitably and fluctuations in buying or distribution
    patterns by such customers could adversely impact Shire's revenues,
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  *adverse outcomes in legal matters and other disputes, including Shire's
    ability to obtain, maintain, enforce and defend patents and other
    intellectual property rights required for its business, could have a
    material adverse effect on Shire's revenues, financial condition or
    results of operations;

and other risks and uncertainties detailed from time to time in Shire's
filings with the U.S. Securities and Exchange Commission, including its most
recent Annual Report on Form 10-K.

References

1.Shire HGT. Rare Disease Impact Report. http://www.rarediseaseimpact.com .
    Published April 2013.
2.European Organisation for Rare Diseases (EURORDIS) website. "Rare
    Diseases: understanding this Public Health Priority."
    http://www.eurordis.org/IMG/pdf/princeps_document-EN.pdf . Accessed March
    20, 2013.
3.Global Genes website. "RARE Facts and Statistics."
    http://globalgenes.org/rarefacts/ . Accessed March 20, 2013.







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