Micro, small and medium-sized enterprises (SMEs) registered with the European Medicines Agency (EMA)'s SME office have seen a consistent improvement in the success rate of their marketing authorisation applications (MAAs) over the last seven years, says the Agency.
EMA recognises that such firms are a major driver of innovation in the pharmaceutical industry, and it put the SME initiative in place in December 2005 to promote innovation and development of medicines by them, says the SME Office's annual report for 2012. The number of companies registered as SMEs with the Agency increased 62% from 2011 to 2012, and a total of 1,025 are now registered. Of these, 40% are micro-sized companies, ie, with fewer than 10 staff members.Over the last seven years, SMEs have submitted 98 MAAs for human and veterinary medicinal products, and the firms currently have 26 MAAs under evaluation.
While the rate of positive outcomes for MAAs submitted by SMEs since December 2005 has been lower than the average for all applicant companies, at 53% versus 78% overall, the rate of positive outcomes has been more than 50% for each of the last four years, averaging 63%, the report notes.
Another positive trend has been the improvement in the quality of medicines produced by SMEs over the last few years, with fewer firms now facing major objections related to the quality of their products during the approval process.
However, improvements are still needed in areas including non-clinical study design, pharmacokinetic and pharmacodynamic clinical studies and safety-related methodological issues, it adds.
Scientific advice has been the key to success for SMEs - it has been proven to increase their chances of successful approvals, says the Office. Over the past two years, the percentage of such firms seeking scientific advice prior to filing has gone up to 64%, from 41% in previous years.
Also, in 2012, for the first time, an SME requested biomarker qualification, and two of the six parallel scientific advice procedures with health technology assessment (HTA) bodies were initiated by SMEs.
The report shows that the importance of opening up early dialogue with the EMA on all aspects of product development is now increasingly recognised by SMEs, says the Office, adding that the growing experience gathered so far has provided useful information on the areas where the Office should focus further regulatory assistance and advice. These include:
- a new version of the Agency's user guide for SMEs, which has just been published and includes additional information on issues such as new complex pharmacovigilance requirements and anti-falsification measures;
- plans to explore more efficient ways of providing training on specific topics of interest to SMEs, for example through webinars;
- a new electronic SME declaration form being piloted for roll-out this year, following the proven efficiency of the streamlined validation process introduced in November 2011;
- the development of interactions with the finance and venture capital communities, given the importance of reaching out to all categories of stakeholders in the SME and innovation environment; and
- strengthening interactions with international partners supporting SMEs. The Office reports that it has initiated a dialogue with other decentralised agencies of the European Union (EU) to exchange best practice, with a view to further simplification and sharing of information.