Micro, small and medium-sized enterprises (SMEs) that are registered with the SME office of the European Medicines Agency have seen a consistent improvement in the success rate of their marketing-authorisation applications (MAAs) over the past seven years. This is shown in the annual report from the SME office 2012, which analyses the activities of the Agency's SME office since its creation. The Agency put the SME initiative in place in December 2005 to promote innovation and development of medicines by SMEs, as it recognises that SMEs are a major driver of innovation in the pharmaceutical industry. This initiative provides active regulatory, financial and administrative support to these companies in the development of their medicines. The support takes the form of individual guidance and more general advice through the SME user guide, topical workshops and a dedicated newsletter.
The number of companies registered as SMEs with the Agency increased by 62% in 2012 compared to 2011. A total of 1025 SME companies are now registered with the Agency, of which 40% are micro-sized companies (fewer than 10 staff members).
Success of registered companies
SMEs represent a noticeable proportion of applicants. Over the past seven years, 98 MAAs for human and veterinary medicinal products were submitted by SMEs. SMEs have 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. It should be noted that these figures relate solely to applicants that were still SMEs at the time of filing, meaning that SMEs that were subject to mergers, acquisitions or out-licensing prior to filing are not accounted for.
Another positive trend observed over the past few years was an improvement in the area of quality of medicines. Fewer SMEs are now facing major objections related to the quality of medicines during the approval process.
Areas where improvements are needed include non-clinical study design, pharmacokinetic and pharmacodynamic clinical studies and safety-related methodological concerns.
Scientific advice: the key to success
Scientific advice received from the Agency has proven to increase the chances of successful approval for SMEs. 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 increased interest is a good sign, because seeking scientific advice and complying with it has been associated with a greater chance of receiving a positive opinion.
In 2012, for the first time, an SME requested biomarker qualification, another tool that supports the development of medicines that the Agency provides to companies, large and small.
Also of interest, in 2012 two of the six parallel scientific-advice procedures with health-technology-assessment bodies were initiated by SMEs. This parallel advice procedure allows identifying the criteria and endpoints in clinical trials that are useful to these important stakeholders when reviewing relative effectiveness.
Learning from seven years of support and advice
The importance of opening up early dialogue with the Agency on all aspects of development is now increasingly recognised by SMEs.
The growing experience of the SME office gathered over the past seven years has provided useful information on where to focus further regulatory assistance and advice:
- the latest version of the user guide for SMEs, published today, includes additional information, including information on new complex pharmacovigilance requirements and anti-falsification measures;
- the Agency will be exploring more efficient ways of providing training on specific topics of interest to SMEs, such as through webinars;
- a streamlined SME validation process was introduced in November 2011. This new process has now shown its efficiency. In parallel a new, electronic SME declaration form is being piloted for roll-out in 2013;
- the SME office will develop interactions with the finance and venture-capital community, as reaching out to all categories of stakeholders in the SME and innovation environment is essential;
- the SME office will strengthen interactions with international partners supporting SMEs to share tools and support. It has initiated a dialogue with other decentralised agencies of the European Union to exchange best practice, with a view to further simplification and sharing of information.