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Rare Disease

Speed treatments to market to transform the lives of patients and their families

Globally, the list of rare diseases has grown to ~7,000, and 350 million people are living with a rare disease. The lack of available treatments leave patients and their families searching for new options and new hope.

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>245 rare disease studies in 96 countries

Quintiles has provided clinical services for more than 245 rare disease studies in 96 countries since 2011.

Simplifying the complexities of rare disease research

Regulatory incentives such as the orphan products designation and the availability of Pediatric Rare Disease Priority Review Vouchers have increased the drive for rare disease research. But with each potential new therapy, biopharma companies are charting new paths through clinical development, with limited disease knowledge, complex regulatory pathways, few suitable patients and unknown obstacles ahead. Quintiles is an experienced partner who can help you simplify the complexities of rare disease research to speed new treatments to market for patients and their families.  

Solution tailored to rare disease studies

Through our experience conducting more than 245 rare disease studies in 96 countries since 2011, Quintiles has developed a comprehensive yet flexible solution to these complex and challenging studies.

Click below to read below about Quintiles patient-centric rare disease capabilities.

Experience that matters   +

Experienced rare disease team who can simplify the complex
  • Specialized rare disease project leaders -- experienced in simplifying the complex.
  • Seasoned CRAs - focused on highest data quality and patient safety.
  • Therapeutic expertise – across 14 Centers of Excellence including pediatrics and neurology.
  • Functional leads – skilled in the nuances of rare disease studies.
Operational approaches that fit   +

Site models tailored to the needs of patients and the study
  • Network of sites -- able to deliver on rare disease studies.
  • Targeted feasibility assessment -- leveraging new data sources and KOL/advocacy input.
  • Local presence, globally -- including Middle East, Asia, Latin America.
  • Personalized patient support – home health, telemedicine, travel support.
  • Data-driven triggered monitoring  –improved quality and patient safety. 
  • Drug supply solutions – tailored to study dynamics.
  • Quintiles Infosario® dashboard – clear visibility of your project.
Recruitment strategies that deliver   +

Innovative digital and mobile patient recruitment strategies
  • Multifaceted patient recruitment – find patients wherever they are and guide them to the study.
  • Social media listening research.
  • Digital and mobile recruitment.
  • Expertise in building patient communities.
  • Patient advocacy relationships – extend and complement sponsor and site relationships.
  • Clinical Trial Educators – site support and recruitment specialists.
Insights that streamline your path   +

Expert insights on regulatory, patient and payer perspectives to inform trial design and product value story
  • Natural history studies.
  • Expert insights -- regulatory, patient and payer perspectives to inform clinical design, market access, reimbursement.
  • Patient-centric protocol design – expertise in regulatory strategy and adaptive design.
  • Real-world evidence– support post-market commitments and product value story.

The power of possibilities

For those who can overcome the challenges, there are many opportunities in rare disease product development. Quintiles is an experienced partner who can help you manage the unexpected. 
 
From identifying outcome measures that demonstrate safety and efficacy to generating the real-world evidence that payers are looking for, we are here to offer the specialized scientific, clinical and operational expertise you need to bring rare disease therapeutics to patients and their families. 

>40 rare disease registries, such as Cystic Fibrosis, Hemophilia, Fabry Disease, Huntington’s Disease, Pompe Disease and CAPS since 2004.

Related services

Generate the evidence you need using the right approach for the right question
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Video Spotlights
Designing Patient-Centric Rare Disease Clinical Trials
Designing Patient-Centric Rare Disease Clinical Trials
The Future of Pediatric Research in Rare Diseases
The Future of Pediatric Research in Rare Diseases
Working with Patient Advocacy Groups
Working with Patient Advocacy Groups
Rare Disease Day 2015: Connecting Insights for Better Patient Outcomes
Rare Disease Day 2015: Connecting Insights for Better Patient Outcomes