Resources for Rare Disease and Orphan Product Development

Developing products for rare diseases and orphan products presents unique challenges. Rho has extensive experience addressing these challenges to set your program up for success. Here are some resources we have developed to help you:

Understanding FDA’s Priority Review Voucher System


Understanding FDA’s Priority Review Voucher System

How can one regulatory program simultaneously benefit both blockbuster drugs and rare or neglected diseases?  How can your company leverage the opportunities afforded by this program?  In this complimentary webinar, Rho’s panel of experts will walk you through the how’s and why’s of the FDA’s Priority Review Voucher program. 

Kelly Roney, PhD, RAC
Senior Research Scientist

Dr. Roney has extensive experience in research and pharmaceutical development.  She has served as a senior regulatory program leader for multiple development projects, and has experience from the pre IND through NDA stages of pharmaceutical development.  Dr. Roney has experience in CNS, oncology, pediatrics, obstetrics, pelvic floor disorders, immunology, and infectious disease products.  Her primary areas of expertise include regulatory strategy, medical writing, and clinical development.

Sheila Bello-Irizarry, PhD
Integrated Product Development Associate

Dr. Bello-Irizarry is actively involved in protocol development, orphan-drug designation applications, and regulatory submissions including INDs and NDAs/BLAs. Her therapeutic area experience includes infectious diseases, immunology, vaccines, lung biology, musculoskeletal, and antibody-mediated therapy.  She contributed to developing vaccine candidates against malaria and MRSA infections and to the understanding of inflammatory processes during lung fungal infections. 

Devin Rosenthal, PhD, RAC
Research Scientist

Dr. Rosenthal works with companies at all stages of development to help them shape their product development programs. He has experience across the full drug development spectrum through his roles in small biotech, big pharma, and at Rho, with particular focus on oncology, CNS, gastrointestinal, and respiratory indications. 

White Paper: Accelerating US Regulatory Development and Approval for Drugs and Biologics that Treat Serious Diseases

FDA’s Fast Track, Accelerated Approval, Breakthrough Therapy, Regenerative Medicine Advance Therapy and Priority Review designations can reduce product approval timelines if pursued and implemented appropriately. Taking the time to understand the requirements and potential benefits of each acceleration mechanism will help you make informed decisions about what’s best for your product development program.

Key Tips for Developing Orphan Products

Dr. Shoemaker offers six essential tips for developing orphan products based on his extensive experience working with products intended to treat rare and orphan diseases.


Personalized Medicine versus Orphan Product Development

The development of therapeutics for personalized medicine leverages many of the advantages of the legislation conferred upon orphan products.  New products must be positioned intelligently to take advantage of this legislation.  Join David Shoemaker, Ph.D. as he discusses the benefits of orphan product designation in the US, EU, and strategies for obtaining these designations for novel products.

David Schoemaker, PhD
Senior Vice President, R&D, Rho

Dr. Shoemaker has 25 years of experience in pharmaceutical development and has led the regulatory strategy for programs in many therapeutic areas. He has experience with all types of regulatory submissions and has moderated dozens of FDA meetings.

Fact Sheet: Orphan Drugs and Rare Diseases

Fact Sheet: Orphan Drugs and Rare Diseases

Rho_2017_OrphanDrug_web.pdf Download File ›