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.

EMA 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, EMA can also advise 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; EMA's 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.

For more information, see: 

• Human regulatory: overview

PRIME - priority medicines

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, EMA 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;

• their products 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.

For more information, see:

• Accelerated assessment
• Authorisation of medicines
• Scientific advice and protocol assistance

Possible combinations

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.

Overview of support mechanisms

	PRIME
Type of mechanism	Support scheme for medicine development
Medicines eligible	Same as accelerated assessment: Medicines of a major interest from the point of view of public health and in particular from the viewpoint of therapeutic innovation (unmet medical need)
When to apply	During the development, based on preliminary clinical evidence (proof of concept) Micro-, small and medium enterprises (SMEs) and academics can get access at an earlier stage (proof of principle), only results of a first-in-man study (clinical trial) are required
Key features	Early identification of candidates for accelerated assessment Rapporteur is appointed earlier, during development Reinforced scientific and regulatory support from the Scientific Advice Working Party/CHMP, other relevant scientific committees and EMA Dedicated contact person within EMA
More information	PRIME: priority medicines

 

 

	Accelerated assessment
Type of mechanism	Regulatory tool for early access
Medicines eligible	Medicines of a major interest for public health and in particular from the viewpoint of therapeutic innovation (unmet medical need)
When to apply	6-7 months before submission of marketing-authorisation application: notify EMA of intention to request accelerated assessment 2-3 months before submission: request accelerated assessment Alternatively: if access to PRIME has been granted previously, ask for accelerated assessment criteria to be reconfirmed prior to marketing-authorisation application
Key features	Reduces assessment time for marketing-authorisation applications to 150 days or less (compared to standard 210 days)
More information	Accelerated assessment

 

	Conditional marketing authorisation
Type of mechanism	Regulatory tool for early access
Medicines eligible	Medicines for seriously debilitating or life-threatening diseases, including orphan medicines and medicines for emergency situations Fulfilling these criteria: positive risk-benefit balance; applicant likely to be able to provide comprehensive data after authorisation; fulfils unmet medical need; benefits of immediate availability outweigh the risks due to additional data still being required.
When to apply	Discuss as early as possible, during development, through scientific advice/protocol assistance Request when submitting marketing-authorisation application Can also be proposed by the CHMP during assessment of marketing-authorisation application
Key features	Earlier authorisation of medicines for patients with unmet medical needs, on the basis of less complete clinical data Comprehensive data generated after authorisation within agreed timeframe
More information	Conditional marketing authorisation

 

	Compassionate use
Type of mechanism	Regulatory tool for early access
Medicines eligible	Unauthorised medicinal products: for chronically, seriously debilitating or life threatening diseases, with no satisfactory treatment authorised in the EU; targeted at a group of patients rather than an individual; undergoing centralised marketing-authorisation applications or clinical trials; falling under the mandatory or optional scope of centralised procedure.
When to apply	CHMP opinion on compassionate use cannot be requested by applicants, they should liaise with national competent authorities
Key features	Benefits seriously ill patients who cannot be treated satisfactorily or cannot enrol in ongoing clinical trials CHMP recommendations to Member State to harmonise the conditions of use, distribution and the target population
More information	Compassionate use