When it comes to treating rare diseases, Rho understands that advancements don’t come easy and the rarity of the diseases alone can inhibit the success of clinical trials.

Unfortunately, less than 10% of rare diseases have a specific treatment, most have no cure at all, and many manifest at a young age. Combining the COVID-19 pandemic with a limited population and lack of disease education and funding, their rare disease isn’t the only thing causing these families to feel alone.

Starting with unique regulatory pathways and continuing through drug development and marketing applications, the differences inherent to rare and orphan disease clinical trials are ever-present and require a knowledgeable, empathetic and flexible CRO with the global expertise to design and execute your orphan drug development plan, including the ability to incorporate decentralized clinical trial solutions.

With over 30,000 rare disease participants treated in more than 150 studies and 2,400 sites, along with our own personal experiences supporting friends and family struggling with rare diseases, Rho not only feels their pain – we’re determined to help find the cure.

Our international relationships are leading the charge. To better support and aid the development of your product, we’ll lean hard on our strong relationships with advocacy organizations and clinical sites to simplify recruitment and increase retention while meeting regulatory requirements – ultimately providing a more feasible and flexible rare diseases clinical trial.

Gene and Cellular Therapy

With approximately 80% of all rare diseases having a genetic component, utilizing an orphan drug and rare disease CRO partner with expertise developing, conducting, and managing these trials is vital to the success of your program. And with over 20 studies in more than 1,000 participants, our understanding of these trials will help you plan for and prevent the inherent risks associated with gene and cellular therapy research.

Natural History Studies

Understanding and defining the disease course is critical when developing a rare disease therapy. Current FDA guidance documents even stress the importance of referencing Natural History Studies (NHS) to inform drug development. Having conducted over 30 NHSs, let our expertise assist your drug development program.