Table 1.

EMA's support scheme for medicine development and early access

Characteristic	Regulatory tool	Key features	Comments
Presubmission phase	SA	Nonbinding advice on quality, nonclinical and clinical aspects of drug development plan	• Scope for parallel EMA/FDA or EMA/HTA SA in some cases. • Tailored SA pilot launched for new biosimilar products; quality issues restricted to comparability between biosimilar and reference product; advice may also include nonclinical and clinical aspects.
	Protocol assistance	SA pertaining to orphan designated drugs	• Orphan drug: medicine for diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition that is rare or where medicine is unlikely to generate sufficient profit to justify research and development costs. • Incentives apply (e.g. fee reductions).
	PRIME	Priority scheme: support for development of medicines of major interest that target unmet medical need	• Continuous support and early interactions with reviewers; SA at key development milestones involving additional stakeholders such as HTA. • Potential for combination with one or several early access tools at time of MAA.
Early access	Accelerated review	Reduced evaluation time frame	• Rapid assessment of medicines that are of major interest for public health, especially ones that are therapeutic innovations (unmet medical need).
	Conditional MAA	Earlier authorization of medicines for patients with unmet medical needs, on basis of less complete clinical data	• Eligibility includes medicines for seriously debilitating or life-threatening diseases, emergency situations, orphan drugs. • Comprehensive data are expected to be generated after authorization within agreed time frame but risk/benefit ratio is clearly positive at time of approval.
	Compassionate use	Benefits seriously ill patients who cannot be treated satisfactorily or cannot enrol in ongoing clinical trials	• Pertains to unauthorized medicinal products for chronically, seriously debilitating or life-threatening diseases, with no satisfactory treatment authorized in EU; targeted at a group of patients rather than individual; or undergoing centralized MAA or clinical trials

EMA, European Medicines Agency; FDA, US Food and Drug Administration; HTA, health technology assessment bodies; MAA, marketing authorization application; SA, scientific advice.