EMA Seeks to Expand Collaboration on Orphan Medicines

The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) is seeking to expand its international cooperation in 2013.

COMP has been collaborating with FDA in the US since 2008, and with Japanese regulatory authorities since last year. For 2013, COMP is anticipating to work more closely with Health Canada.

COMP is also seeking to collaborate more with health-technology assessment agencies, which provide recommendations on medicines that can be paid for or reimbursed by the healthcare systems in particular European member states. Specifically, COMP is working with the European Network for Health Technology Assessment to achieve a better understanding of orphan designation, the marketing of orphan medicines and initiatives on the availability of designated orphan medicines. Overall, COMP will be exploring ways of sharing information for the benefit of patients and the financial sustainability of the healthcare system.

Patient integration is another goal of COMP. COMP will proactively request contributions from expert patients where there advice is considered key, such as improvement of quality of life. COMP is also intending to increase its interactions with the European Organisation for Rare Diseases and other, disease-specific patient organisations.

“The work of the COMP is evolving and constantly adapting to better serve and address patients’ needs, the growing scientific knowledge on rare diseases, and the regulatory framework,” Bruno Sepodes, chair of COMP, said in a statement.

In Europe, the success of orphan incentives and the Orphan Medicinal Product Regulation has been underlined by an increase in orphan-designated medicines; 107 designations were granted in 2011, 148 in 2012 and more than 150 are expected for 2013.