R A R E D I S E A S E S

E X P E R I E N C E

No. OF CLINICAL SITESa

9,300+No. OF CLINICAL PATIENTSa

37,900+

No. OF CLINICAL STUDIESa

203

No. OF APPROVED RARE DISEASEDRUGS

187

6

3

11

HOPE. IT'S WHY OUR CLIENTS DO WHAT THEY DO AND WHY WE'RE SO PASSIONATE ABOUT HELPING THEM.

R A R ED I S E A S E S

Clinical development involving rare diseases poses a unique set of medical, scientific, and operational challenges. PRA understands these challenges and the critical success factors necessary to develop drugs for rare disease patients. It’s not a cookie-cutter formula, but rather a nimble approach that embraces the flexibility, creativity, and innovation needed to succeed in this arena. In the last 5 years, we have managed over 200 Phase I-IV rare disease studies, across 12 different therapeutic areas, resulting in the successful marketing approval of 18 drugs.

I N N O VAT I O N

While all trials require a customized approach to achieve study goals, rare diseases present unique challenges that call for specially tailored solutions. At PRA, our methodology empowers us to effectively understand patient pathways, identify the right sites and patients, and ensure proactive and continuous advocacy group engagement throughout the study life cycle. Our execution of this model is what helps us excel at these demanding trials.

PRA has approximately 60 medical directors with experience in rare disease indications. In addition, more than 80% of our clinical operations and project management staff has experience managing and executing rare disease studies.

PRA provided the key services that helped lead to the approval of a new targeted treatment for advanced kidney cancer (RCC) and gastrointestinal stromal tumors (GIST), a rare stomach cancer.

E X P E R T I S E

MEDICAL DIRECTORS

=10%

CRAs PROJECT DIRECTORS ANDMANAGERS

CLINICAL TEAM MANAGERS

80 85 85 90

W I T H R A R E D I S E A S E S E X P E R I E N C E

AdvocacyGroup

Engagement

Understanding Patient Pathways

Site Identification/ Selection

Site Awareness and Support

Outgoing Patient Retention Support

ONCOLOGY DRUGS

HEMATOLOGY DRUGS

IMMUNOLOGY DRUGS

CARDIO-METABOLIC DRUG

NEUROSCIENCE & PAIN DRUG

“The success of rare disease trials hinges on the ability to understand and incorporate the voice of the patient, their caregivers, advocacy groups, and other stakeholders into the global clinical trial planning process.”

SCOTT SCHLIEBNER, MPHVP, Rare Diseases, Scientific Affairs

PRA's global investigator network provides preferred access to established investigative sites that accrue patients and deliver high-quality clinical trial data.

We never forget that every global rare disease study is executed locally. With global offices providing comprehensive coverage across 80+ countries, our team harnesses practical knowledge of local regulations, standards of care, and cultural customs to maximize safety and efficiency for your rare disease trials.

a OVER THE PAST 5 YEARS

PRA Health Sciences conducts comprehensive Phase I-IV biopharmaceutical drug development. To learn more about our solutions, please visit us at prahs.com or email us at prahealthsciences@prahs.com.

We know that 30% of children with a rare disease will not live to see their 5th birthday. We also know that 95% of rare diseases do not have an FDA-approved drug treatment. While many see those statistics and focus on despair, we choose to focus on hope. Yes, hope.

Hope. It's why our clients do what they do and why we're so passionate about helping them. Hope that by ge�ing be�er medicines for rarediseases to market sooner, we can help make tomorrow be�er than today for the peoplewho need them. Hope that by employing highly dedicated, and passionate people, by being more innovative, and by delivering unmatched scientific expertise, we can improve the drug development process.

PRA Health Sciences conducts comprehensive phase I-IV biopharmaceutical drug development. To learn more about our solutions, please visit us at prahs.com or email us at prahealthsciences@prahs.com.PRA Health Sciences conducts comprehensive Phase I-IV biopharmaceutical drug development. To learn more about our solutions, please visit us at prahs.com or email us at prahealthsciences@prahs.com. JAN2018