The EMA defines an orphan drug designation, attributed by the Committee on Orphan Medicinal Products (COMP) as a permit for “a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease such as a genetic disorder or a rare cancer.”
The COMP reviews each single application from organisations seeking orphan drug designation. By definition, the products submitted need to be intended for development for the diagnosis, prevention or treatment of “life-threatening or very serious conditions that affect no more than 5 in 10,000 persons in the European Union”. Additionally, it is outlined by the EMA that orphan drug designation may be granted if without the incentives, the product is unlikely to generate revenue after marketing that would cover the initial investment of development.
There are many commercial implications of researching and developing treatments for very low patient populations. But the reality shows that while individual rare disease patients are indeed rare, collectively those who suffer from “orphan diseases” are numerous and it is estimated affect 1 in 10 of us.
So what are the incentives provided by European regulation in order to foster innovation and therapeutic development within the pharmaceutical industry:
· Market exclusivity in the EU
An orphan product receives a marketing authorisation from the EMA (European Medicines Agency). Competitive similar products cannot be placed on the market for 10 years after receiving marketing authorisation. In the case of paediatric drugs the monopoly is extended to 12 years.
· Protocol assistance
The EMA provides protocol assistance (scientific advice for orphan products) in the form of scientific advice about the various tests and clinical trials necessary for drug development to pharmaceutical companies. This information is delivered at no cost or a reduced fee to optimise the development of orphan drugs and to ensure better compliance with the European regulatory requirements.
· Fee reductions
During the approval process fee waivers for orphan designation and reduced fees are granted. These apply to marketing authorisation, inspections, variations and protocol assistance.
· EU-funded research
Pharmaceutical companies developing orphan drugs may be eligible for specific grants from EU and Member State programmes as well as initiatives supporting research and development. This includes the Community framework programmes.
The following link enables you to search for information related to all rare disease (orphan) designations in Europe and see which active substances were approved or rejected for orphan drug designation and also which compounds were withdrawn from the application process.
While there are some incentive variations provided to orphan drug developers in European and North American markets, there is a Common EMEA/FDA Application for Orphan Medicinal Product Designation in which the sponsor may simultaneously apply for orphan drug designation of the same medicinal product for the same use in both jurisdictions.
This is but a glimpse of the advice, processes and assistance that both the EMA and FDA provide on their respective websites. Therefore while drug development for any disease area is time consuming, costly and complex, it is promising that the channels have long been in place to facilitate efficient and commercially attractive drug development for rare diseases.
But what do you think…comments are welcome…
Can and should more be done to foster and promote orphan drug development worldwide?
Does the highly regarded Orphan Drug Act need to be enhanced to increase research into the vast number of neglected rare diseases?
Should more attention be turned to the issue of pricing and reimbursement to ensure that there is someone at the end of the line who will pay for patients to receive an orphan drug?