Supporting innovative SMEs as major drivers of new pharmaceutical developments

12 May 2016 11:33 AM

EMA publishes report on the first ten years of its SME initiative

The European Medicines Agency (EMA) has yesterday published a report summarising the experience with its small and medium-sized enterprise (SME) initiative over the last ten years. The initiative started in December 2005 when the SME Regulation came into force and the SME Office was established to address the needs of the smaller actors in the pharmaceutical development ecosystem.

SMEs are the backbone of Europe's economy. They represent 99% of all businesses in the European Union and provide two-thirds of the total private sector employment. In the pharmaceutical sector SMEs are the motor of innovation and play a major role in the development of new medicines for patients.

To support SMEs throughout all stages of medicines’ development, EMA’s SME Office provides active regulatory, financial and administrative support to registered SMEs.

The new report emphasizes the importance of SMEs in pharmaceutical innovation and draws attention to the trends observed in the past ten years. Here are the main highlights:

• SMEs applications for the marketing authorisation of new medicines account for approximately 10 to 15% of the overall number of applications. More than one in two medicines developed by SMEs that were recommended for marketing authorisation in the past ten years contained a new active substance; this shows that SMEs are an important source of medicines that have the potential to address patients’ unmet medical needs.
• SMEs are a major source of innovation to address rare diseases: 42% of the medicines that were recommended for marketing authorisation in the past ten years wereorphan medicines.
• The success rate of SMEs in the marketing authorisation process for human medicines has improved over the years; in 2015, 75% of SME applications led to the granting of a marketing authorisation, the highest rate so far, compared to 68% during the period 2010-2012.
• SMEs are increasingly benefiting from scientific advice during the development of human medicines: 158 of the 473 requests for scientific advice finalised by EMA in 2015 came from SMEs, 30% more than in 2014.
• An increasing number of SMEs use parallel scientific advice with health-technology-assessment (HTA) bodies which allows them to receive simultaneous feedback on their development plans from both regulators and HTA bodies and aims to facilitate patients’ timely access to medicines.
• For veterinary medicines, the number of requests for scientific advice has also increased over the last ten years; as for human medicines, there is also a focus on smaller markets: one in four medicines that were recommended for marketing authorisation were for minor use minor species (MUMS) / limited market.

“The report shows that EMA’s tools to support pharmaceutical innovation by SMEs, and in particular scientific advice, are being increasingly used,” explains Melanie Carr, Head of EMA’s Stakeholders and Communication Division. “Encouraging small enterprises to engage in dialogue with us early in the product development is a priority for EMA as the input we provide allows them to optimise their development programme and generate high quality data on a medicine’s benefits and risks.”

By the end of 2015, 1,619 companies from across the EU were registered as SMEs with EMA.

Information on registered companies is available in the public SME register. The SME register contains high level information on companies’ pipelines to facilitate interaction, partnering and networking between SMEs, and to increase transparency on the SME initiative.

How SMEs view EMA’s support

EMA has also yesterday published the results of a survey conducted in 2015 with SMEs and stakeholders to gather feedback on the SME initiative.

Four in five respondents indicated that the services provided by the SME Office have been relevant or very relevant to them, with financial incentives, the SME newsletter, the SME user guide and regulatory assistance being the highest rated services.

The survey also revealed that services such as the translation of the product informationof medicines and specific support during the development of advanced therapies, are not well known.

80% of respondents would like EMA regulatory assistance for SMEs to be expanded as well as financial fee incentives for pre- and post-authorisation activities. According to the survey results, regulatory requirements, the administrative burden and the cost of regulatory procedures are the most frequent challenges that SMEs are facing today.

EMA is developing an action plan to better address the needs of SMEs which will take into account the feedback received and the areas of improvement identified.

The Agency will also monitor how SMEs will be using EMA’s new scheme to support the development of priority medicines (PRIME). As part of PRIME, EMA will provide extra assistance to SMEs that have a medicine accepted for the scheme.