Orphan designation recognizes a drug in development for a rare disease. For most biotech companies, it is their first formal interaction with FDA or EMA.
As soon as it can show “promise” that its drug may treat a rare disease. That evidence can come from clinical data or from animal models of the human disease.
Yes. Applying sooner rather than later pays off in three ways:
Under the Orphan Drug Act of 1983 and later legislation, designated products are eligible for:
Yes. Orphan drug programs often need fewer or smaller clinical trials, and their path to approval can be faster when they meet the criteria for expedited programs.
The impact has been dramatic. Before 1983, only 12 drugs were available for rare diseases. By 2024, more than 1,200 had been approved.
It takes on average 3 weeks to finalize an application and 3 months for the FDA to review it. We typically budget 10 months for EMA applications to meet their process requirements which are different from the FDA’s.
No. Data from an animal model of the human disease is fully sufficient. The animals that received your product should show a meaningful improvement over untreated diseased animals, such as smaller tumors, longer survival or better histology at necropsy.
As former Director of FDA’s Office of Orphan Products Development (OOPD), Dr. Cote made the final decision on 1,400 orphan designation applications. During his tenure, as before and since, about 35% of first-time applicants were designated. With repeated resubmissions, that figure rises to just over half.
At Only Orphans Cote, more than 95% of our applicants receive designation. That is not because of any special treatment from the FDA. It is because we work only with clients who are as committed to their orphan products as we are to securing their designation. Dr. Cote’s experience and our team’s support guide each client through the process, producing an application built to meet FDA’s requirements. Clients gain a trusted advisor who shares their goal: bringing new treatments to patients with rare diseases.
To get started, reach out to our business development team through our Contact Us page. They manage meetings, contracts and next steps. Once your project begins, Dr. Cote leads it personally. His regulatory affairs associates and medical writers help draft each application, but he oversees every aspect of the project, is responsible for every word submitted and is accountable for your success.
Significantly. The European application is about three times as long and addresses four criteria instead of FDA’s two. It is also a multi-stage process: application validation, a pre-submission meeting and, finally, an opinion from the Committee for Orphan Medicinal Products (COMP). The process takes about 10 months.
As OOPD Director, I attended COMP meetings every six months, and my European colleagues regularly visited FDA. From 2012 through 2017, in my first consultancy, I submitted more European designation applications than any European organization. For me and the Only Orphans Cote team, the European system is familiar ground.
Yes. About half our work is orphan designation at FDA and EMA. The other half is guiding orphan drugs through FDA review toward approval, including:
We support every step of getting your orphan drug approved and marketed in the US.
RPDD is FDA’s recognition that your product targets a rare pediatric disease. If the product is later approved for that disease, it may qualify for a Priority Review Voucher (PRV). PRVs can be sold and have recently sold for over $100 million. That makes RPDD a strong signal to investors: approval could bring a one-time voucher sale on top of product revenue.
Fast Track has been in place since 1997. It is available to products that address an unmet medical need and have an open IND, and clinical data are not required to apply. Review is largely criteria-based, so outcomes are fairly predictable and usually positive.
Breakthrough Therapy is newer (2012). It includes all Fast Track benefits plus more intensive FDA guidance, including additional meetings and cross-disciplinary support. Applicants must submit clinical data showing substantial improvement over available therapies. The criteria are open to interpretation, most applications are not granted, and outcomes are hard to predict, so expert guidance makes a real difference.
The payoff is worth it. The market values FDA’s recognition of a “breakthrough,” and studies show these products tend to reach and move through the clinic faster and are more likely to be acquired.
Conversations with us are always free. We’re glad to answer your questions and share our perspective at no cost. We charge only for tangible deliverables, such as written applications.
We use project-based pricing, not hourly billing.