希少疾患

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

Top Therapeutic Areas
Hematology | Neuroscience | Metabolic Disease | Cardiovascular

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

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. 当社とともに、お客様の可能性を成功へと導き、まだ満たされていない差し迫った医療ニーズに一石を投じましょう。

Explore our related areas of expertise.

細胞療法・遺伝子治療薬小児科 Our Team (RAPT)