Market Access Blog

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How can early access schemes support accelerated patient access in Europe?

Introduction

Bringing a new treatment to market can be a lengthy process as it requires a marketing authorisation (MA) and successful pricing and reimbursement negotiations. When there is a clear unmet medical need, early access schemes, also known as early access programs (EAPs) allow patients with severe, life-threatening, or severely debilitating conditions access to medicines that do not yet have an MA and would otherwise be unavailable. EAPs are offered by the European Medicines Agency (EMA) at a European level (e.g. PRIME to accelerate approval) and at a national level (e.g. the UK’s early access to medicines scheme (EAMS) and France’s revamped early access and compassionate access schemes.

Patients can benefit significantly from these programmes, particularly for global access programmes where patients can access orphan drugs that may never be commercially available in their country if reimbursement procedures fail. Here we focus on rare diseases, which can be learned from, and applied to other conditions. Companies need to understand the consequences of early access schemes for future health technology assessment (HTA) and pricing before commencing on the development and implementation of such access programmes.

Approach in Europe

The EU is attempting to achieve a more harmonised approach in efforts to mitigate risks of a postcode lottery on an international scale. These efforts include: EUnetHTA, international reference pricing, EURIPID sponsored by EU and encouraging collaboration on joint assessments submission templates and guidelines (which do not account for country GDP or ability or willingness to pay) and joint procurement (bilateral engagement of member states in a discussion about the joint procurement/purchasing of innovative medicines).

A variety of services to support the early stages of medicine development as well as regulatory measures is offered by the EMA to ensure that promising new medicines reach patients as early as possible. Companies developing such medicines can apply to the EMA for their products to make full use of these regulatory opportunities. Regulatory opportunities include PRIME, accelerated assessment and compassionate use programmes.

Figure 1 illustrates the stage of clinical development at which companies can engage with EMA and other stakeholders

EAPs are separated into two versions in the EU – named patient programmes (NPP) (usually paid for) and compassionate use programmes (CUP) (free with a possible admin charge). CUP are usually initiated by the company who is liable, whereas NPP requests are initiated by the doctor who is liable.

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Previously in France, early access to new medicines was processed through six interlinking schemes, including temporary authorisations for use (autorisations temporaires d’utilisation, ATU) and temporary recommendations for use (recommandation temporaire d’utilisation, RTU). These six schemes were simplified into two separate but interlinking paths.

•  The temporary recommendations for use and nominative ATU schemes were simplified into the compassionate access (accès compassionnel, AC) programme.

•  The cohort ATU, post-ATU, ATU for an extension of indication and direct access post marketing authorisation (MA) schemes were simplified into the early access (accès précoce, AP) programme.

The likelihood of achieving reimbursement in EAP varies around EU-4 and the UK. Some examples are shown in this table.

Finally, as with health technology assessment planning, mapping important analogues and learning from their passage through pricing and reimbursement systems is critical. Many countries permit compassionate or paid-for use prior to licensing, but careful preparation and selection of the best approach will rely on a variety of factors.

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If you would find out more about our consultancy or MAP Online services may benefit you, please get in touch on enquiries@mappatientaccess.com or 01480 832360.

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