The first phase of a public registry of companies which are registered with the European Medicines Agency (EMA) as small and medium-sized enterprises (SMEs) has now been launched.
The aim of the registry is to facilitate and promote interaction between SMEs, and the first phase includes contact details of individual companies, their area of activity and headcount. The second phase, which will become available from the end of next March, will include information on individual companies’ pipelines and product profiles.
Following discussions with stakeholders, the decision was taken to increase the public information available about SMEs to give a more complete picture of firms registered in Europe and to support their growth in the European Union (EU) through partner search and cooperation, says the Agency. Previously, the EMA only published a list of SMEs with their country of origin.
Meantime, the Agency’s SME Office reports that the number of companies assigned SME status rose 10% in 2010. Currently, 507 companies are registered, with requests from a further 58 companies under review, says a report published this month on the Office’s fifth year of operation; the EMA launched its initiative to provide financial and administrative assistance to SMEs on December 15, 2005.
In 2010, the relative size of registered SMEs and their geographic distribution remained similar to previous years, the highest proportion of companies being based in the UK, France, Germany, Sweden and the Netherlands, it says. Currently, 36% of companies are micro, having fewer than 10 employees and no more than 2 million euros in assets or turnover. Around 21% are academic spin-offs.
Of the assigned companies, the large majority (69%) are developing products for human use. 12% are veterinary firms, 7% are developing products for both human and veterinary use and 12% are regulatory consultants.
The types of product now under development at SMEs break down broadly into 66% chemical entities and 32% biologics. Of the companies developing biologics, 65% are developing recombinant DNA-derived products and 35% advanced therapy medicinal products (ATMPs): cell therapy - 46%, tissue engineering - 29%, and gene therapy - 25%. A small proportion of firms are working in the field of pharmacogenomics/biomarkers (4%) and nanotechnology (5%).
In 2010, 81 SMEs developing medicines for human use sought scientific advice or protocol assistance from the EMA, the report shows. Clinical advice was sought in 47% of cases, with preclinical and quality requests featuring in 32% and 21% of cases, respectively. The Office comments that many companies (63%) are still seeking advice “rather late” in development (Phase III), with 17% in Phase II and 20% in Phase I.
17 centralised marketing authorisation applications (MAAs) were submitted to the EMA by SME applicants in 2010, 13 of which were for human medicinal products and four for veterinary products. For the human-use products, there were four positive outcomes (three orphan and one generic) and one negative (withdrawal of an ATMP). Currently, 22 centralised applications from SMEs are ongoing, and further information on these firms’ experience through the centralised approval process will be published next year.
In May, the EMA finalised its first ATMP certification procedure for a suspension of mononuclear cells under development by an SME in the cardiovascular field. Through certification, SMEs can obtain feedback from the Agency’s Committee for Advanced Therapies (CAT) early in development on the extent that quality and where available non-clinical data generated with an ATMP comply with MAA review standards.
However, the Office notes that, to date, interest in the procedure has been “surprisingly low,” and it “strongly” encourages SMEs to make contact for more information on this “important new service.”
Throughout 2010, requests for regulatory assistance from SMEs remained high (102), up 10% on 2009, it adds.
The report also looks at how SMEs are coping with the requirements set out in the EU pediatric legislation, which took effect in January 2007, obliging companies to agree pediatric investigation plans (PIP) with the Agency early in development.
Between July 2008 and September 2010, the EMA received 912 valid applications for PIP/deferral/waiver from all companies (non-SME and SME). Of those, 74 (8%) applications were from SME applicants, of which 52 have had outcomes and 22 are ongoing. Of those with outcomes, forty were positive (77%) ie, 23 PIPs and 17 waivers adopted. Twelve were negative (23%) ie, two negative opinions and 10 withdrawals after procedure start. For all companies over the same period, 84% had positive outcomes and 16% negative.
The average active time from submission to opinion is 210 days for SMEs which is marginally shorter than for non-SMEs (217 days). The average response (“clock-stop”) time is around 120 days for both SME and non-SME companies.
Since the beginning of 2010, EMA has offered pre-submission meetings to SMEs, but, to date, only three such meetings have been held. The report emphasises the need to raise SMEs’ awareness of the requirements of the pediatric legislation, warning that, if not addressed, this may delay MAA validation.
In contrast, the uptake of SMEs into scientific advice and regulatory assistance remains high. “This is encouraging and financial provisions to support SMEs applying for scientific advice will be maintained in the future. Companies are also reminded that once initial advice has been sought, follow-up advice is recommended particularly if major changes in development are considered,” says the Office.