Clinical Trials

Rare Disease & Orphan Drugs

Gene therapy was their last hope for a second birthday.

He’s now celebrating his 8th.

That is what drives us. Experience, Rho.

Rare Disease

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 expertise to design and execute your orphan drug development plan, including the ability to incorporate decentralized clinical trial solutions.

Find out how a decentralized trial can help you improve your patient experience, recruitment, and retention while producing overall higher data quality.

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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 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 more feasible and flexible rare disease clinical trials.

Our collaborative relationship with Rho has been instrumental in the implementation of a complex and rigorous first in human genetic medicine study, including strategic solutions for unique challenges faced in rare disease gene therapy trials. Rho has leveraged existing relationships with patient advocacy organizations and worked closely with a centralized biosafety review partner early in study startup to help identify and mitigate potential challenges.

Monica Gangal – Former VP of Clinical Operations at Anthera

Rho not only gave our team a clear picture of how their team would fulfill the study needs, but showed their knowledge and expertise of the indication as well as the protocol in a way that made them a collaborative partner rather than a service provider.

Monica Gangal, VP of Clinical Operations

Together, let’s bring hope to those living with rare disease and give families more days with their loved ones. We proudly invite you to experience Rho.

Our Expertise


clinical trials


clinical sites



Met or exceeded timelines for site activations, enrollment, data cleaning and lock for the past


completed programs.

Through the Cystic Fibrosis Foundation Therapeutics Development Network,


of sites ranked Rho as good or excellent.

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.

Case Study: Cystic Fibrosis

Rho and Anthera Pharmaceuticals’ Collaborative Partnership in Cystic Fibrosis

child and doctor

Implications of Historical Control Studies for Rare Disease Products

Development programs in rare diseases are becoming a major focus for small biotech and pharmaceutical companies. A crucial consideration is the choice of control group used to assess efficacy. Through recent case studies, we will discuss factors to consider, appropriate analysis, and the critical role that externally controlled studies play in supporting marketing applications for orphan/rare disease products.

Watch Webinar
Kevin Barber

Kevin Barber, Ph.D.

Vice President, Regulatory Strategy & Submissions

Rob Woolson

Rob Woolson

Vice President, Regulatory Strategy & Biometrics

Recruitment and Retention in Rare

Learn how to address and mitigate recruitment and retention challenges in your rare disease program.

ipad healtcare

Success Stories

Completing Enrollment Early for a Phase 3 Rare Disease Study

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Rho Works with CF Patient Advocacy Group to Develop Strong Relationships with Key Sites

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Meeting Timelines in a Small Patient Population for a Rare Disease

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