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The EU orphan medicine scheme has been in place since 2000, with the principle aim to encourage companies to develop medicines for rare diseases. According to EU statistics, there are over 6,000 rare diseases and approximately 30 million people sufferers. Due to the lack of readily available therapies, treating these patients has always presented a challenge, and thus a huge unmet need had arisen. To date, the EMA has given over 1900 products orphan designation and, according to their database, over 140 of these products are now available on the market – providing treatment options for patients who previously often had none.
According to the EMA, a rare disease is one where fewer than 5 in 10,000 people have the condition. The orphan medicine scheme offers incentives for the research and development of these medicines, to help off-set the huge costs involved in their development. These incentives are based on certain criteria being met, such as no previous existing treatment, or if the proposed new treatment is of significant additional benefit to the patient. The incentives for developers can be fee reductions, access to scientific advice and 10-year market exclusivity if the orphan designation is maintained.
The EMA has also recently announced that it will be publishing orphan maintenance assessment reports for all products that have been granted a marketing authorisation by the agency. The aim of this report is to provide a summary on whether a medicine still fulfills its designation at the time of authorisation. This new steps helps to address the concerns raised by some stakeholders that some drugs may not still meet the criteria at the time of authorisation.
What is clear is that the EMA is still striving to bring orphan medicines to patients, and is constantly looking at ways to streamline the process for designation and approval. Here at ELC Group we have a strong track record in helping clients with their development programs for orphan medicines, as well as scientific advice meetings and regulatory strategy. As always, we are on hand to help you with any of your regulatory needs.