Rare Diseases and Orphan Drugs

Access experts in global and local regulatory nuances for orphan or regenerative medicine, advanced therapy designations and pediatric study plans

Improve trial predictability through enhanced feasibility to inform study design using data-driven strategies for delivery

Accelerate drug development and reduce risk for cell and gene therapies with specialized and coordinated capabilities

Pharmaceutical companies working in rare diseases need innovative and strategy-driven partners to develop their treatments in this challenging space. Count on Fortrea’s rare disease and pediatric teams to provide critical expertise to advance your solutions to market.

Boost your rare disease patient enrollment.

Patient access, recruitment and retention can be challenging in any trial, but they are uniquely difficult issues when working within rare diseases. We do not let this hurdle stand in the way of developing rare disease therapeutics. We utilize powerful analytical solutions and access timely, real-world diagnostic data, global investigator performance metrics and insights reported by patients that enable us to deliver faster patient enrollment and enhanced patient retention. In addition to this data, Fortrea’s strong relationships with leading advocacy groups, fit-for-purpose tools to support and educate study teams, sites, patients and their families, and dedicated, passionate and experienced recruitment and retention teams make us the right partner to bring your product to market faster.

Work with us to help improve your study’s visibility and credibility to create patient-centric solutions that reduce recruiting risks and maintain your program’s momentum.

Leverage expertise and accelerate orphan drug development.

Rare diseases are often poorly understood from a clinical perspective, highlighting the need for innovative study planning strategies. Fortrea’s experts help direct rare disease clinical trials to yield more relevant results with an efficient study design that evaluates desired outcomes and incorporates validated endpoints. Enable the development and collection of key clinical data in your studies with our innovative tools and approaches, including: biomarker models, mobile health, natural history studies, and virtual trials.

With Fortrea as your partner, you’ll access a unique combination of dedicated rare disease therapeutic area expertise and comprehensive operational support to assist with every aspect of your global study. Together, we’ll develop robust study planning strategies that anticipate regulatory hurdles, navigate clinical complexities and accelerate your path forward.

Maximize your commercial potential.

Even though orphan drugs address urgent, unmet medical needs, understanding market impact is still crucial to maximizing investment in an orphan indication. By evaluating payers, providers and patients, Fortrea generates real-world, value-based evidence that helps determine pricing and sets market access strategies.

Work with a team that combines medical, scientific, operational and economic expertise with extensive clinical trial experience to enable your rare disease clinical program and enhance your potential for a higher valuation and return on investment.

An experienced approach to advance rare disease programs.

Beyond helping secure rare disease volunteers to meet recruitment needs, Fortrea also offers deep experience supporting orphan indications and rare disease studies across dozens of countries and thousands of sites. Pediatric-focused rare disease studies are even more complex, and our team has proven successful in the management of such studies worldwide.

Let our multidisciplinary experts put their experience to work to advance your rare disease drug development program and transform obstacles into opportunities. Together, we’ll improve your potential for success and make a difference in urgent unmet medical needs.

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