Micro, small and medium-sized enterprises (SMEs) are a motor of innovation in the European Union (E.U.). Between 2010 and 2012, more than one in four new innovative medicines recommended for marketing authorization in the E.U. originated from SMEs, as shown in an analysis published by the EMA in January.
Innovative medicines have the potential to bring significant benefits to patients and address unmet medical needs. As they act in a different way to existing treatments or target patients who can benefit most from the therapy, these medicines offer new avenues to treat patients differently, while maximizing the benefits and reducing the risks.
Recently, the first medicine authorized for the treatment of the life-threatening disease Duchenne muscular dystrophy (Translarna) originated from an SME. This medicine displays a completely new mode of action that directly targets the cause of the disease. Another recent example is the first non-surgical treatment option (Jetrea) for adults with vitreomacular traction, an eye condition which can cause severe visual disturbance. This new medicine spares patients from having to stay in a “head-down” position for days to enhance the success rate of the surgical procedure.
According to the report, SMEs also are highly active in the development of advanced therapies, innovative medicines that are based on gene, cell or tissue engineering, as well as in the development of medicines for rare diseases. In these two categories of medicines, SMEs account for about half of all new medicines under development.
“Supporting the actors in the pharmaceutical industry who contribute to enhancing public health by focusing on innovation is a priority for the EMA,” said Melanie Carr, head of EMA’s corporate stakeholder department. “Our dedicated structure to support smaller companies, as well as our ongoing work to reinforce engagement with the academic world, are both intended to further support the translation of innovation into successful developments in the interest of patients.”
The EMA’s SME office provides active regulatory, financial and administrative support to registered SMEs in the development of their medicines. Almost one in two SMEs registered with the EMA has less that 10 staff members and 12% originate from academia.
In addition, the EMA supports innovation at very early stages of a medicine’s development through its Innovation Task Force. This taskforce provides a forum for early dialogue with applicants. During free-of-charge briefings, medicines developers can discuss their initial research findings with the EMA’s experts and proactively identify any scientific, legal and regulatory issues and opportunities.
Over the past five years, almost one third of all the taskforce’s briefings have been conducted with SMEs. Two out of three meetings with SMEs concerned either cell-based, gene-based or tissue engineering-based therapies. New medicines for the treatment of cancer represent the largest category.
While there still is a long way to go before these medicines can potentially reach patients, early dialogue between medicines developers and regulatory authorities has proved to increase the chances of obtaining a marketing authorization in a timely manner, thereby accelerating patients access to new medicines.