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.
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.
Our global client solutions will ensure you reach your patients, wherever they are. With international capabilities that span 5 continents, we provide full-service capabilities to execute your clinical development programs and trials across the globe.
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.
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
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.
Met or exceeded timelines for site activations, enrollment, data cleaning and lock for the past
Through the Cystic Fibrosis Therapeutics Development Network,
Expertise in Action
Rho has developed relationships with 40+ cystic fibrosis research centers in North America and Europe (both CFF TDNCC and ECFS CTN sites, as well as non-CFF TDN and non-ECFS CTN sites) and has led 10 cystic fibrosis studies and 2 marketing application submission efforts in just the past 3 years.
Rho’s experts have experience with CF gene therapy studies, as well aerosolized products, oral therapies, and inpatient, PK, PERT/GI, and anti-infective studies. Additionally, Rho has expertise with common CF assessments and endpoints, including CFA, CFQ-R, CNA, FEV1/FVC, MCC, and sputum collection.
Following study start-up for a Phase III cystic fibrosis study, the Cystic Fibrosis Foundation Therapeutics Development Network (CFF TDNCC) conducted a survey of participating clinical sites to assess their satisfaction with Rho. Some highlights include:
- 100% of sites ranked Rho as good or excellent for 6 of the 7 questions asked. For the seventh question, 90% of sites ranked Rho as good or excellent.
- On all questions, Rho scored higher than the CFF TDNCC average.
- “Rho is tremendous! Their people put in the extra effort to get our site initiated over a weekend in order to make it easier for a subject.”
Learn more about our commitment to cystic fibrosis research as we passionately fight to find a cure.
Kevin Barber, Ph.D.
Senior Vice President, Regulatory Strategy & Submissions
Joseph Watson, Ph.D.
Director, Regulatory Strategy
Caitlin Hirschman, R.N.
Director, Project Delivery
Director, Clinical Data Management
Senior Clinical Team Lead
Senior Clinical Team Lead
Egle Garbuziene, M.D.
Associate Medical Director
Building Bridges: Patient Advocacy Groups Can Be Vital Partners in Clinical Research Trials
Patient advocacy is vital for advancement in managing disease. We met with Chandrabali Ghose-Paul, Chief Scientific Officer, Emily’s Entourage and Gary Joyner, Chairman Emeritus, Board of Directors, Primary Ciliary Dyskinesia (PCD) Foundation to discuss the role of patient advocacy groups in clinical research. In this webinar, we discuss building the gap between research and patients and how to support patient advocacy in our community.Watch Webinar
Caitlin Hirschman, R.N.
Principal Clinical Team Lead
Clinical Team Lead
Rare Disease CRO Services
Clinical Project Management
Clinical Operations Monitoring
Phase I Clinical Development Services
Risk-Based Quality Management