An orphan drug is a pharmaceutical agent that has been developed specifically to treat a rare medical condition, which is generally defined as affecting fewer than 200,000 people in the United States.
Understanding Orphan Drug Status
The orphan drug designation process is important for products seeking approval for the treatment of rare diseases. The benefits of orphan drug designation can provide a major advantage to products seeking approval, and it is important for sponsors to understand both the EMA and FDA processes in order to maximize the potential benefits of orphan drug designation
What does it mean to be granted orphan drug designation?
The benefits of orphan drug designation are significant and can provide a major advantage to products seeking approval for the treatment of rare diseases. It is important to note that orphan drug designation is not a guarantee of marketing approval, but it does provide a number of benefits that can help to streamline the approval process.
The FDA process for orphan drug designation is fairly straightforward; however, the EMA’s Orphan Medicinal Product Designation (OMPD) process can be more complex and time intensive. It is important to note that having granted orphan drug designation at both the EMA and FDA does not prevent a product from also being granted marketing authorization as a breakthrough therapy.
Product eligibility for orphan drug designation
Orphan drug designation may be granted to drugs intended for the treatment, diagnosis or prevention of any rare disease/condition. Eligibility for orphan drug designation is not dependent on novelty or on the mechanism of action being known.
A rare disease or condition is generally defined as one that affects fewer than 200,000 people in the United States. The National Institutes of Health (NIH) has a searchable online database that provides information on more than 7,000 rare diseases and conditions.
However, in order for a disease to be recognized as rare in Europe, it must affect no more than five people out of 10,000.
Orphan Drug Designation Application
The sponsor must provide the FDA with a submission to request orphan designation. The Center for Drug Evaluation and Research (CDER) evaluates the eligibility of products seeking orphan drug designation, which includes the following criteria:
The drug or biologic intended to treat, diagnose or prevent the disease/condition is intended to address an unmet medical need
The disease/condition is serious or life-threatening
The drug is intended for use in rare patients
The sponsor must provide the EMA with a submission to request orphan medicinal product designation, which includes the following:
The product that is the subject of the application is intended for the diagnosis, prevention or treatment of a rare disease/condition
The product is intended to address an unmet medical need
The product is expected to provide significant benefit to patients with the disease/condition
There is no reasonable expectation that the product will generate sufficient income to cover its development and marketing costs
There is no satisfactory method of diagnosis, prevention or treatment for this disease/condition known to the medical community in the country where it affects more than five people out of 10,000
It is likely that the product, if approved, would be granted a marketing authorization for this disease/condition
Coordination and Regulatory support across Europe for an innovative product/orphan drug
The Vice-President Global Regulatory Affairs of a US based company developing an important innovative product (orphan drug) contacted BlueReg to...Learn more
Benefits of Orphan Drug Designation
In addition to a potential reduction in the regulatory filing fees, there are a number of benefits that may be granted to a product that has been granted orphan drug designation by both the EMA and FDA:
Years of marketing exclusivity in the EU following a first authorization
Regulatory fee reductions for clinical trials
Access to specialized medical expertise from the authorities
Free scientific advice from Committee for Medicinal Products for Human Use (CHMP)
Priority review by the FDA
Fast-track designation by the FDA
Outsourcing Regulatory Affairs projects for orphan drugs
BlueReg can determine if your product is eligible for orphan drug designation and provide the necessary support for your European Medicines Agency (EMA) / Food and Drug Administration (FDA) application.
Drug Development Consulting & Strategy
Currently developing a new medicinal product ? BlueReg can provide an end-to-end project plan to guide you from the development of your medicinal product right through to authorization and beyond. We can provide advice and recommendations to ensure the shortest time to market by minimising any possible barriers during the development process and beyond.
Advanced Therapy Medicinal Product (ATMP)
Looking for an Advanced Therapy Medicinal Product (ATMP) classification? BlueReg can guide you to submit a classification application to Health Authorities to determine if your product meets the requirements to be defined as an ATMP.
Early & Expanded Access Programs in France
Looking to reach market earlier ? You can rely on the experience and expertise of our consultants at BlueReg for providing advice and efficient implementation of your Early Access Programs projects in France .
Scientific Advice & Interaction with Health authorities
BlueReg has extensive experience in scientific advice with the European Medicines Agency (EMA) and many national competent authorities. BlueReg can assist with all competent authority meetings.
CMC Product Development
Looking for Chemistry, Manufacturing & Controls (CMC) expertise ? BlueReg ensures CMC regulatory compliance for your projects to meet regulatory agencies requirements and expectations.
Clinical Trial Applications (CTA)
Looking to outsource Clinical Trial Applications? BlueReg offers a variety of services that bring together our global clinical and regulatory expertise to simplify your journey to market. The clinical development process can be complex and can cost your organisation significant time and money.
Paediatric Investigation Plans (PIPs)
BlueReg can provide a complete PIP writing service as well as any PIP modifications through the lifecycle of the product within the EU or US.
EMA/HTA Parallel scientific advice
Looking for support with your EMA / HTA bodies Parallel Consultation? BlueReg can provide a team of highly experienced consultants specialised in regulatory affairs, market access and scientific writing. Our consultants can advise you on all steps of the procedure and author the required documents required for this important process.