Demonstrating significant benefit of orphan medicines

News 04/09/2015

EMA organises workshop to clarify concept and demonstration of significant benefit of new orphan medicines over existing treatments

The European Medicines Agency (EMA) is organising a workshop on 7 December 2015 to discuss the approach that should be followed by medicine developers to demonstrate the significant benefit of an orphan medicine over existing treatments. Demonstrating a significant benefit is one of the criteria medicines that treat rare diseases must fulfil to benefit from 10 years of market exclusivity once they have been authorised.

The workshop will bring together medicine developers, regulators, healthcare professionals, academia, patients, health-technology-assessment bodies and healthcare payers who need to register by 31 October 2015 if they wish to participate. The workshop will also be broadcast live.

In the European Union (EU), medicines that treat rare diseases, so-called orphan medicines, can benefit from a number of incentives. These incentives are provided for by the European Regulation on orphan medicinal products to stimulate research and development as well as the actual marketing of medicines for patients with rare diseases. They include fee reductions for scientific advice and 10-year market exclusivity once the medicine is authorised in the EU.

To qualify for orphan designation, a medicine must target 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.

Since the EU Regulation came into force in 2000, over 100 medicines for rare diseases have been authorised in the EU.

As more and more medicines for rare diseases are becoming available, sometimes in the same therapeutic area, demonstrating significant benefit for the granting or maintenance of orphan designation has become increasingly complex.

Therefore, EMA wants to discuss and clarify with all stakeholders the concept of significant benefit as well as the methodology and type of evidence required to support it.

The views, interests and concerns of the various stakeholder groups will help the Agency develop further guidance on the significant benefit of orphan medicines for medicine developers.

The workshop will also provide an opportunity to explore how the evaluation of a significant benefit relates to health-technology-assessment bodies' assessment of relative efficacy in the context of reimbursement decisions.

Assessment of significant benefit

EMA's Committee for Orphan Medicinal Products (COMP) assesses whether a medicine fulfils the criteria for orphan designation at two points in time: firstly, when a medicine developer submits an application for orphan designation early on in a medicine's development; secondly, at the time of marketing authorisation of the new medicine, when the applicant submits a request to maintain the orphan designation.

The demonstration of significant benefit is based on assumptions at the time of orphan designation and has to be confirmed at the time of marketing authorisation.

The medicine can only benefit from 10 years of market exclusivity if it still fulfils the criteria for orphan designation at the time of marketing authorisation, including the demonstration of a significant benefit.

Market exclusivity means that for up to 10 years no other similar medicine can be marketed for the same indication unless the originator gives consent, is unable to supply sufficient quantities of the medicine, or the product is considered to be clinically superior when compared to the originator.

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