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France pays for promising medicines before they are approved or reimbursed: the ATU system did this from 1994, and since July 2021 the Haute Autorité de Santé grants early access for presumed innovative drugs in serious diseases, with the company setting the price and repaying the difference later.
France, statute and regulatory decisions. Temporary authorisations for use (autorisations temporaires d'utilisation, ATU) were created by the law of 8 December 1992 and operated from 1994, letting patients receive unapproved medicines either individually or in cohorts. Article 78 of the social security financing law for 2021 replaced them from 1 July 2021 with two routes: accès précoce (early access), decided by the Haute Autorité de Santé (HAS) with an opinion from the medicines agency ANSM where no marketing authorisation yet exists, and accès compassionnel (compassionate access), decided by ANSM. Primary text: the HAS early access pages; the provisions sit in the public health code at article L5121-12 and the social security code at L162-16-5-1.
How it works: early access requires a serious, rare or disabling disease, no appropriate treatment, an inability to defer treatment, and a presumption of innovation; the company must commit to filing for authorisation and for reimbursement, collects real-world data on every patient, and is paid an indemnity it sets freely, with rebates once the final price is negotiated. Oncology is the largest user, and many PD-1 inhibitors, ADCs and CAR-T products reached French patients months or years before reimbursement elsewhere.
The arguments: France's system is widely cited as the most generous pre-approval access in Europe and as a driver of early real-world evidence; critics point to the cost of free pricing, to the complexity of the rebates, and to the fact that HAS refusals of early access can pre-empt the later reimbursement debate. The EU's compassionate use provision in Regulation 726/2004 and the UK's Early Access to Medicines Scheme are the comparators.
Shares Conditional marketing authorisation (EU), Health technology assessment (HTA), reimbursement and QALYs, Real-world evidence, Weak real-world evidence and registries and the tag law.
Shares AMNOG (Germany, 2011), ILAP (Innovative Licensing and Access Pathway), EU HTA Regulation (EU) 2021/2282: joint clinical assessments, Health technology assessment (HTA), reimbursement and QALYs and the tag law.
Shares Conditional marketing authorisation (EU), EU HTA Regulation (EU) 2021/2282: joint clinical assessments, Regulation (EC) No 726/2004: the EU centralised procedure, Most of the world has almost no cancer care and the tag law.
Shares Conditional marketing authorisation (EU), ILAP (Innovative Licensing and Access Pathway), Health technology assessment (HTA), reimbursement and QALYs, Real-world evidence and the tag law.
Shares EU HTA Regulation (EU) 2021/2282: joint clinical assessments, Real-world evidence, Weak real-world evidence and registries and the tag law.
Shares Expanded access (compassionate use), Real-world evidence, Weak real-world evidence and registries and the tag law.
Shares ILAP (Innovative Licensing and Access Pathway), Regulation (EC) No 726/2004: the EU centralised procedure and the tag law.
Shares AMNOG (Germany, 2011), Health technology assessment (HTA), reimbursement and QALYs, Most of the world has almost no cancer care and the tag law.