Over 40% of innovative medicines recommended for marketing authorisation in the European Union (EU) between 2010 and 2012 originated from small or medium-sized enterprises (SMEs), academia, public bodies and public-private partnerships, according to an article published in the Nature Reviews Drug Discovery and authored by staff members of the European Medicines Agency (EMA).
A new active substance is broadly defined as a chemical, biological or radiopharmaceutical substance that has not been previously authorised as a medicinal product in the EU.
The authors identified all the medicines containing a new active substance that were recommended for marketing authorisation between 2010 and 2012 by the Agency’s Committee for Medicinal Products for Human Use (CHMP). They then analysed the profile and geographical origin of the organisations at the origin of their development.
Of the 94 marketing authorisation holders overall, most (87%) were large (59%) or intermediate-sized (28%) pharmaceutical companies, and 13% were SMEs.
When the products were tracked back through development to their origin, large or intermediate-sized pharmaceutical companies accounted for 49% of the products (large, 28%; intermediate-sized, 21%), SMEs for 27% (while SMEs account for only 13% of the marketing-authorisation holders), and academic/public bodies/public-private partnerships (PPPs) accounted for 17% (while these organisations are no longer involved at the stage of marketing-authorisation applications). Private–private collaborations accounted for 7%.
The authors also looked specifically at the origin of medicines intended for the treatment of rare diseases. Their findings show that 61% of the medicines with an orphan designation originate from SMEs (while SMEs account for only 22% of the marketing-authorisation holders), with large or intermediate-sized pharmaceutical companies accounting for 22%, and 11% of the medicines with an orphan designation originate from academic institutions, public bodies and public-private partnerships (while these organisations are no longer involved at the stage of marketing-authorisation applications).
“Understanding the factors that could affect drug innovation, such as the nature of the organisations involved, could help in developing strategies to catalyse further advances,” explain the authors. “This analysis shows that SMEs, academic institutions, public bodies and public-private partnerships represent an important source of innovation and bolster the product pipelines of larger companies.”
The EMA and the EU recognise that SMEs are a motor of innovation in the EU. Acknowledging their role in the development of new medicines, the EMA has a programme in place to support SMEs throughout all stages of medicine development.
“This analysis underlines the importance of the support that the EMA offers early in development, which should facilitate and promote the emergence of innovative medicines,” explained Melanie Carr, head of the EMA's SME office.
Reinforcing the support available to SMEs and acting as an enabler of development for the smaller actors in the pharmaceutical innovation ecosystem, particularly academia, is a priority for the EMA.
Following the Agency’s recent re-organisation, dedicated structures to support human medicines research and development and external stakeholders will offer new platforms for engagement with the academic world. This will further support the translation of innovation into successful developments in the interest of patients.
Lincker H, Ziogas C, Carr M, et al. Where do new medicines originate from in the EU? Nature Reviews Drug Discovery 2014; 13: 92–93. doi:10.1038/nrd4232