The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have recognised that paradigms of drug development, which are feasible for common diseases, may not be feasible for rare diseases and that transformative orphan, specialty and advanced therapeutics often defy traditional regulatory routes.
The EMA’s Support for Early Access and the FDA’s Expedited Programs aim to facilitate and accelerate development and marketing authorisation. The programs a focus on life-threatening and debilitating diseases with a major impact on quality of life (defined as serious conditions), and on medicines with a credible promise of significant improvements in clinical benefit and patient-relevant outcome(s).
While the FDA’s programs are well established, certain of the EMA’s early access tools have been launched more recently. These innovative initiatives emphasise early dialogues, the involvement of multiple stakeholders, iterative development in a life-cycle approach, and an expanded toolbox for evidence generation, with pragmatic and real-world studies complementing RCTs in areas where the collection of data via traditional routes is difficult. Early access does not alter the current FDA or EMA standards of approval.
There is substantial crossover in terms of objectives and features between the programs and they can be used in combination. For example, a product eligible for fast track in the US may be eligible for accelerated approval and priority review. In Europe, a medicine benefitting from PRIME support may also qualify for conditional marketing authorisation. Additionally, drugs benefitting from early access are those eligible to the EMA centralised procedure and a single marketing authorisation.
Early access is applicable to both orphan and non-orphan medicines in both jurisdictions, although the programs are more accepted for orphan drugs. Orphan drug designation is not an early access tool per se, and orphan medicines do not automatically qualify for accelerated procedures. Nevertheless, orphan drugs are highly likely to be eligible for early access. Therefore, the feasibility of orphan designation should be considered as part of any early access strategy, and equally, the potential benefits of early access should be considered as part of the decision to seek orphan designation.
The above table provides an overview of the EMA’s and the FDA’s early access programs and represents the current regulatory context within which an early access strategy for an innovative medicine is developed. More on early access strategies can be found here:
The following is a full listing of the current early access programs, legal tools and initiatives in Europe and the United States. The blue highlighted external links to the EMA and FDA websites are current as accessed in February 2019 (note that ORPHA Strategy is not responsible for the content of the external sites).
Accelerated Assessment (AA)
Conditional Marketing Authorisation (CMA)
PRIority MEdicines (PRIME)
Initiative for Registries – “late dialogues”
Orphan Designation for Medicines for Rare Diseases
Compassionate Use Opinion by the CHMP
Parallel consultation with regulators and health technology assessment bodies (as of July 2017)
Patients' and Consumers' Working Party (PCWP)
Search the EMA Website
Expedited Programs for Serious Conditions - Drugs and Biologics
Office of Orphan Products Development (OOPD)
Center for Biologics Evaluation and Research (CBER) - Cellular & Gene Therapy Products
FDA's Application of Real World Evidence
FDA further links, including FDA & EMA Collaborations
Expanded Access (Compassionate Use)
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ORPHA Strategy Consulting
Phase IV Programs LLC
David Schwicker, Principal
4051 Basle, Switzerland
Tel: +43 676 362 9571