Rare Diseases: Accelerate Your Product Development Through Orphan Designation
The number of rare diseases for which no treatment is currently available is estimated to be between 4,000 and 5,000 worldwide. Each disease affects only a small proportion of the population, often starting at birth or during infancy. In Europe, these diseases in sum affect 25 to 30 million people.
Mostly due to economic reasons, the development of therapies for rare diseases has been unattractive for pharmaceutical companies. Accordingly, these medicines are also commonly referred to as “orphans”.
USE THE BENEFITS …
Therefore, most countries put incentives in place to encourage the development of therapies for rare diseases. Incentives comprise financial benefits, programs to accelerate development and market access, and market exclusivity. To be eligible, the drug or device under development must obtain orphan designation issued by the respective regulatory authority.
… BUT A TAILORED DEVELOPMENT STRATEGY IS KEY
Although the criteria for orphan designation appear similar between countries, the product development strategy and application for orphan designation must take into account product- and country-specific aspects.
For example, orphan drugs intended for rare pediatric diseases can benefit from additional incentives or regulatory pathways to accelerate market approval. To meet the prevalence criterion for orphan designation, the applicant must collect country-specific prevalence or incidence data. Only limited experimental data from preclinical and clinical studies may be available, and appropriate justifications must be provided in the application for orphan designation.
The following overview summarizes incentives and requirements for orphan drug designations:
MF BIOTECH has obtained orphan designation for drugs and devices worldwide including the EU and US – in one go.
Preparation and evaluation of strategies and scenarios for orphan designation (globally or nationally)
Preparation and conduct of pre-submission meetings and hearings for orphan designation
Preparation and filing of application for orphan designation worldwide including EMA and FDA
Development of integrated development strategy plans for accelerated product development of biopharmaceuticals