EMA Sets Up Workshop on Orphan Drugs

The European Medicines Agency (EMA) has set up a workshop, to be held Dec. 7, 2015, that addresses how drug developers should demonstrate the significant benefit of an orphan drug over existing treatments, which is one of the criteria a drug must fulfill to benefit from market exclusivity after authorization as part of the European regulation on orphan medicinal products. Drug developers, regulators, healthcare professionals, academia, patients, health-technology-assessment bodies, and healthcare payers will be participating in the workshop.

According to EMA, demonstrating the benefit of an orphan drug has become complex because of the increase in these types of therapies. More than 100 drugs that treat rare diseases have ben authorized in the European Union (EU) since the EU regulation came into effect in 2000. According to EMA, a drug may qualify for orphan designation in the European Union (EU) if it targets “a disease that is life-threatening or chronically debilitating and affects less than 5 in 10,000 patients in the EU. If there is already another medicine or method available for the targeted rare disease, the developer of the new medicine must also provide evidence that it has the potential to bring a significant benefit to patients over the existing options; this means that it provides a clinically relevant advantage or a major contribution to patients.”

EMA believes the workshop will clarify the concept of significant benefit as well as the methodology and type of evidence required to support it. The agency hopes the feedback received from stakeholders will help the agency develop guidance on significant benefit for orphan drugs. The workshop will also explore how a “significant benefit relates to health-technology-assessment bodies’ assessment of relative efficacy in the context of reimbursement decisions.”