Regulatory information – Updated SME user guide now available

19 October 2016

New sections have been added to the guide

The European Medicines Agency (EMA) has updated its User guide for micro, small and medium-sized enterprises (SMEs) operating in the pharmaceutical sector.

The guide aims to support SMEs to better understand the EU legislative framework relating to medicines and the requirements for the development and authorisation of medicines for human or veterinary use.

SMEs often have limited experience with regulatory procedures and the guide provides an overview of data requirements to support a marketing authorisation, as well as the regulatory tools available to facilitate medicines' development. The guide, which follows the chronological stages of developing a medicine, has been completely revised to clarify existing sections and add new ones relating to:

• compassionate use (section 6.14.3): a mechanism that allows the use of an unauthorised medicine for patients with no or unsatisfactory treatment options;
• PRIority MEdicines (PRIME) (section 3.2): a voluntary scheme to support the development of new medicines for human use that address an unmet medical need by offering enhanced early dialogue to facilitate accelerated assessment; SMEs applying for PRIME can benefit from full fee waivers on scientific advice;
• post-authorisation measures that can be imposed at the time of marketing authorisation (section 6.10);
• post-authorisation efficacy studies (section 6.11);
• the proactive publication of clinical data for medicinal products for human use – 'Policy 0070' (section 6.12).

Any feedback on the contents of this guide should be forwarded to the SME office: [e-mail].

The SME Office was set up within EMA to address the particular needs of smaller companies. The office has dedicated personnel who can help SMEs by:

• responding to regulatory and administrative enquiries;
• setting up briefing meetings to discuss a regulatory strategy;
• organising workshops and training sessions.