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In order to improve the early access to patients for innovative and important new medicines a number of expedited pathways have been introduced by FDA, EMA and certain EU Member States including UK and France.

In the US, the FDA has four programmes aimed to expedite patient access to medicinal products for conditions considered to be serious or life-threatening and targeting an unmet medical need. These are:

1. Fast-track designation

2. Accelerated approval

3. Priority review designation

4. Breakthrough therapy.

In the EU, a number of established regulatory tools exist to support the accelerated development of medicines in areas of unmet medical need, including:

1. EU / EMA – PRIME: the Priority Medicines programme that provides intensive support and close cooperation with EMA’s representatives, including through Scientific Advice,

2. UK-EAMS / PIM and France-ATU programmes that both facilitate early (pre-MAA) access to important new medicines for patients with serious or life-threatening conditions, and thus allow the generation of Real World Evidence.

3. Accelerated assessment for Centralised Procedure registrations.

4. Conditional marketing authorisation (CMA) for Centralised Procedure registrations.

The use of these regulatory tools is not mutually exclusive, and sponsors may utilise more than one throughout their development programme. For example, in the EU, a product successfully awarded PRIME may be eligible for compassionate use with subsequent accelerated assessment of the marketing authorisation application (MAA) resulting in a CMA.

Kinesys can help clients develop and present the optimal regulatory submission strategy for their products resulting in timely patient access, potential for gathering of Real World Evidence which can be utilised during the regulatory or HTA review process, and expedited regulatory review.