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:

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, 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.

Possible combinations

Early access tools are not mutually exclusive.

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

Overview of support mechanisms

PRIME

Type of mechanismSupport scheme for medicine development
Medicines eligibleSame 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
More informationPRIME: priority medicines

 

 

Accelerated assessment

Type of mechanismRegulatory tool for early access
Medicines eligibleMedicines of a major interest for public health and in particular from the viewpoint of therapeutic innovation (unmet medical need)
When to apply
Key featuresReduces assessment time for marketing-authorisation applications to 150 days or less (compared to standard 210 days)
More informationAccelerated assessment

 

Conditional marketing authorisation

Type of mechanismRegulatory 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 informationConditional marketing authorisation

 

Compassionate use

Type of mechanismRegulatory 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 applyCHMP 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 informationCompassionate use

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