Support for early access

The European Medicines Agency (EMA) is committed to enabling early patient access to new medicines, particularly those that target an unmet medical need or are of major public health interest. The Agency seeks to support the medicine development process from an early stage and to offer regulatory mechanisms to help promising new medicines reach patients as early as possible. In this context, the Agency can also give advice on data requirements to ensure that a mature dossier is in place at the time of the marketing authorisation application. Companies developing such medicines can apply to EMA for their products to make full use of these regulatory opportunities.

The European Union (EU) pharmaceutical legislation includes several provisions to foster patients' early access to new medicines that address public health needs and are eligible for the centralised procedure such as:

• accelerated assessment: reduces the timeframe for review of an application for marketing authorisation for medicines of major public health interest and in particular from the viewpoint of therapeutic innovation;
• conditional marketing authorisation: grants marketing authorisation before complete data are available;
• compassionate use: allows the use of an unauthorised medicine for patients with an unmet medical need. The Committee for Medicinal Products for Human Use (CHMP) issues an opinion on criteria and conditions, which national patient access programmes can consider when making such medicines available.

Many patients with serious diseases lack satisfactory therapeutic options. EMA has launched the PRIME scheme to enhance support for the development of medicines that target these unmet medical needs. Through the scheme, the Agency encourages developers to focus on medicines likely to make a real difference to patients.

PRIME is designed to promote accelerated assessment, but it will also help medicine developers to make the best use of EMA's other early access routes. The scheme facilitates early dialogue between the various stakeholders, which is crucial to optimise the use of these tools.

With PRIME, applicants for marketing authorisation get additional advice and support if their products are expected to be eligible for accelerated assessment and fall within the scope of the centralised procedure.

It also builds on other existing regulatory tools in place within the EU legal framework, including scientific advice and protocol assistance.

Early access tools are not mutually exclusive.

For example, a medicinal product benefiting from support under the PRIME scheme could:

• follow an accelerated assessment at the time of marketing authorisation;
• receive an opinion from the CHMP on compassionate use while undergoing clinical trials;
• be granted conditional marketing authorisation before comprehensive data are available.