Orphan privilege

This is the perfect time to apply for funding for the development of orphan therapies. In this article you will learn about orphan drugs and where to find EU grants to finance research that specifically targets treatment of rare diseases.

It is estimated that 30 million people suffer from rare (orphan) diseases in the European Union (EU). In an effort to stimulate the development of treatments for rare diseases, European lawmakers introduced a landmark legislation in the year 2000 by introducing commercial incentives to the pharmaceutical industry for undertaking such development. It is therefore unsurprising that there has been a significant increase in the development of orphan drugs in the last 17 years and an increase in the number of applications for EU funding for orphan therapies.

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What is an orphan drug?
According the European Medicines Agency – the body responsible for reviewing and assigning orphan drug status – an orphan drug must meet the following criteria to qualify for orphan drug designation

  • it must be intended for the treatment, prevention or diagnosis of a disease that is lifethreatening or chronically debilitating;
  • the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development
  • no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

“The Horizon 2020 programme has allocated funds amounting to € 60 million for research that specifically targets treatment of rare diseases”

Who grants orphan drug designation?
The EMA’s Committee for Orphan Medicinal Products (COMP) comprises of a network of experts that examine all orphan drug applications over a 90 day period, following which they determine whether the drug should be granted orphan status. Prior to submission of an application, the EMA strongly encourages all applicants to undertake a presubmission meeting, normally via teleconference, to discuss the application and allow the applicant to make any modifications based on recommendations from the committee. Historically, the EMA reports a positive impact of a presubmission meeting on the application.

What is the advantage of orphan designation?
Orphan status grants certain privileges to pharmaceuticals given that profitability from such therapies is otherwise limited. Of these, market exclusivity is regarded as one of the most beneficial advantages.

    1. Market exclusivity. This is granted for a period of 10 years and prevents another application for the same therapeutic indication, for a similar medicinal product. While multiple products can receive orphan designation for the same indication, similar products cannot and only the first to obtain market authorisation is granted orphan status with the 10-year exclusivity.
    2. Scientific advice and protocol assistance. This provides assistance to the company from the EMA with respects to the appropriate tests and studies for development of the orphan medicine. This is particularly important since it addresses questions pertaining to the product’s ability to offer significant benefit over other available treatments. Protocol assistance can also be given to assess similarity to other orphan products with the same indication.
    3. Reduced / waived regulatory fees. There are reduced fees for regulatory activities, including for protocol assistance, marketing-authorisation applications, inspections before authorisation, applications for changes to marketing authorisations made after approval, and reduced annual fees. Of note, there is no restriction on the number of times a sponsor can request protocol assistance.

Orphaned outside the EU: of relevance to the EU?
While there are orphan indications that do not impact the EU directly, treatments for these conditions can be given orphan drug status. Specifically, the European Commission has communicated that if the “condition affects a large number of people in certain non-EU countries, but which has a low prevalence or a prevalence of approximately zero in the EU, (the drug) may be eligible for designation as an orphan medicinal product with respect to the prevalence criterion, and if all other criteria are met, eligible for the benefits set out in the regulation.” If, however, a condition has zero prevalence within the EU, the risk of EU citizens befalling the disease in the future must be accounted for.

This is the perfect time to apply for funding for the development of orphan therapies. The Horizon 2020 programme has allocated funds amounting to € 60 million for research that specifically targets treatment of rare diseases (see table below). This, coupled with the aforementioned advantages of developing therapies for orphan treatments, has significantly contributed to the advancement in the field of orphan therapies. At GAEU, our team of specialist writers can help SMEs capitalize on the EU’s focus on orphan therapies to develop high end business proposals and secure funding for continued development of the intended orphan product.  


Call Purpose Funding
SME instrument: Cell technologies for medical

Phase I
Funding for a feasibility study to investigate the business potential of your idea (cell technologies for medical applications only). € 50, 000
SME instrument: Cell technologies for medical
Phase II
Funding for activities including clinical validation, upscaling of manufacturing, risk assessment, design or market studies and intellectual property exploration of cell technologies for medical application. 1 - 5 million
PM - 03 diagnostic characterisation of rare diseases
Development of molecular diagnoses for a large number of undiagnosed rare diseases through application of omics and/or other high-throughput approaches for molecular characterisation of rare diseases. € 15 million
PM 08 - New therapies for rare diseases
Funding for your clinical Phase I/II trial on a therapy for a rare disease where Orphan
Designation has been obtained.
€ 4 – 6 million
Eurostars Early industrial R&D, preclinical phases 1 – 2 million
Fast Track to
Projects undertaking innovation form the demonstration stage through to market uptake – relatively mature new technologies that will achieve market entry within 3 years 2 - 3 million

Dr. Damla Khan, Ph.D.
Research and Innovation Funding Specialist at GAEU Consulting





Roland Vilhelmsson

Group CEO

+46 8 556 063 50