Micro, small and medium-sized enterprises (SMEs) registered with the SME office of the European Medicines Agency have seen a consistent improvement in the success rate of their marketing-authorization applications (MAAs) over the past seven years, according to the SME offices’s 2012 annual report.
The EMA created the SME initiative in December 2005 to promote the innovation and development of medicines by SMEs, recognizing them as a major driver of innovation in the pharmaceutical industry. The initiative provides active regulatory, financial and administrative support to these companies in the development of their medicines, through individual guidance and more general advice through the SME user guide, topical workshops and newsletter.
The number of companies registered as SMEs hit 1,025 in 2012, up 62% from 2011. Of those, 40% are micro-sized companies (fewer than 10 employees).
SMEs represent a noticeable proportion of applicants. Over the past seven years, 98 MAAs for human and veterinary medicinal products were submitted by SMEs, with 26 MAAs currently under evaluation.
While the rate of positive outcomes for MAAs submitted by SMEs over this seven-year period was lower than the average for all applicant companies (53% for SMEs vs. 78% overall), a positive trend was observed over the last four years: the rate of positive outcomes has been above 50% each year, averaging 63% over these four years. These figures relate solely to applicants that were still SMEs at the time of filing, meaning those SMEs that were subject to mergers, acquisitions or out-licensing prior to filing are not included.
Another positive trend was an improvement in the quality of medicines. Fewer SMEs now face major objections related to the quality of medicines during the approval process.
Areas in which improvements are needed include non-clinical study design, pharmacokinetic and pharmacodynamic clinical studies and safety-related methodological concerns.
Over the past two years, there has been a significant increase in the number of SMEs seeking scientific advice prior to filing an MAA (64% compared to 41% in previous years). This is a good sign, because seeking scientific advice and complying with it has been associated with a greater chance of receiving a positive opinion. And in 2012, for the first time an SME requested biomarker qualification, another tool the agency provides to companies, large and small.