Rare Diseases

Your partner in developing orphan drugs to build a better tomorrow for patients with rare disease

Expect accountable delivery for the lifetime of your study.

Work with a reliable partner that works with urgency and integrity.

Leverage our innovative approach to advance your rare disease study.

Inspired by patients and families living with a rare disease

Being diagnosed with a rare disease can feel overwhelming and isolating. We understand the power of connecting with the right care team and with others facing similar circumstances. That's why we strive to foster that feeling of community-connection and contribution-within our clinical studies.

We believe each unique challenge and complexity represents an opportunity to show our agility and value. Together, with the engagement and inclusion of patients, families, and the broader community, we're committed to helping advance new orphan drugs for rare diseases.

Experience matters when choosing a partner.

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In the last 5 years

We've been honored to serve as a CRO partner of choice for our biotech and pharmaceutical customers as we've advanced their development. We are also known for our thoughtful approach to novel/unknown areas with no development precedence. In the last five years, we've supported the following:

877

All rare disease studies

624

Rare disease studies outside of oncology

233

Pediatric rare disease studies

Our top therapeutic areas for rare disease include:

Expect more for your next rare disease trial

As your partner, we can work as an extension of your team and:

  • Provide proven patient-centric solutions that improve your study’s visibility and credibility 
  • Offer experience optimizing collaboration with advocacy groups
  • Help navigate regulatory hurdles and clinical complexities to accelerate programs
  • Support informed endpoint development for rare disease
  • Deliver innovative solutions for biomarker models, mobile health, natural history studies and virtual trials
  • Enable customized site support
  • Advance diversity in clinical trials and optimizing inclusion of marginalized communities
  • Leverage real-world, value-based evidence that helps determine pricing and sets market access strategies

Get the latest updates from our team.

We’re sharing our latest news, events and publications.

WHITE PAPER
 Effective planning to address unmet needs in Prader-Willi Syndrome trials
Read the white paper about Effective planning to address unmet needs in Prader-Willi Syndrome trials
ARTICLE
 Rare diseases: A spotlight on Gaucher disease research in India
Read the article about Rare diseases: A spotlight on Gaucher disease research in India
ARTICLE
 Advancing rare disease clinical trials and orphan drug research: A focus on APAC and China
Read the article about Advancing rare disease clinical trials and orphan drug research: A focus on APAC and China
ARTICLE
 Clinical trials in rare disease
Read the article about Clinical trials in rare disease

Serving as your agile partner for complex and novel clinical trials

Let our multidisciplinary experts put their insights and experience to work to advance your rare disease drug development program. Juntos, vamos melhorar o seu potencial de sucesso e fazer a diferença nas necessidades médicas urgentes não atendidas.

Explore our related areas of expertise. 

 

Terapias celulares e gênicas Pediatria Our Team (RAPT)

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