ATU stands for Autorisation Temporaire d’Utilisation, which translates to Temporary Authorization for Use. It is a French regulatory mechanism that allows patients to access drugs before they receive full marketing authorization. Designed for people with serious, rare, or debilitating conditions who have no other treatment options, the ATU system has been one of the most prominent early access programs in global pharma and has served as a model for similar systems in other countries.
In July 2021, France reformed the ATU system and reorganized it into two new frameworks: an Early Access Program (Accès précoce) and a Compassionate Access Program (Accès compassionnel). The underlying purpose remains the same, but the structure and rules have changed in meaningful ways.
How the Original ATU System Worked
The ATU came in two forms. A nominative ATU (nATU) was issued for a single, named patient at the request of their prescribing physician. This was the route for individual cases where a doctor believed an unapproved drug was the only viable option. A cohort ATU (cATU), which was used more frequently, was requested by the drug manufacturer and covered a group of patients who were all treated and monitored under a defined protocol.
Both types targeted the same population: patients with a severe or rare disease and high unmet medical need, meaning no authorized treatment alternatives existed. France’s drug safety agency, the ANSM, oversaw the program and granted the authorizations.
What Replaced the ATU After 2021
The 2021 reform consolidated the ATU’s various pathways into two cleaner categories. The cohort ATU became part of the Early Access Program, which provides rapid, reimbursed access to medicines presumed to be innovative before they complete the standard approval and pricing process. The nominative ATU was folded into the Compassionate Access Program, which covers individual patient requests but now includes a cap on the total number of requests per product. If that cap is reached, the manufacturer can apply for the broader Early Access route instead.
This cap is significant. It means fewer patients may be able to receive a drug through the compassionate, one-off pathway compared to the old system. The reform essentially pushes manufacturers toward the more structured Early Access route, which comes with greater data collection requirements and pricing obligations.
Eligibility Criteria for Early Access
To qualify for early access authorization, a drug must meet all of the following conditions, as defined in the French Public Health Code:
- Serious disease: The condition must be severe, rare, or debilitating. Severity is judged by symptoms, organ involvement, mortality rate, and impact on quality of life.
- No appropriate treatment: No satisfactory therapeutic option, whether a drug or another type of therapy, can be available, accessible, and reimbursed in routine French practice at the time of assessment.
- Treatment cannot wait: Starting the treatment must be urgent enough that delaying until full approval is not acceptable.
- Presumed efficacy and safety: Clinical trial results must strongly suggest the drug works and is safe, even if the data package isn’t yet complete enough for a full marketing authorization.
- Presumed innovation: The drug must appear to offer something meaningfully new compared to existing options.
The “no appropriate treatment” criterion is a common reason applications are denied. France’s health technology assessment body, the HAS, carefully evaluates whether any existing therapy, even a non-drug intervention, could provide comparable benefit. If one exists, early access is blocked.
How Quickly Applications Are Processed
The program officially allows up to three months for evaluation, but in practice decisions come faster. During the first year of the reformed system, the average processing time from complete application to decision was 65 days, with a range of 1 to 121 days. The ANSM’s safety and efficacy opinion took an average of 55 days. Once granted, early access authorization lasts for one year and is renewable.
This speed is the entire point of the system. Patients with life-threatening conditions and no alternatives cannot afford to wait the 12 to 24 months a standard approval and reimbursement process typically takes.
Pricing and Cost to the Health System
One of the more complex aspects of the ATU and its successor programs is how drug pricing works during the early access period. Manufacturers can charge for the product before a final negotiated price is set, but the French system includes a rebate mechanism. Once the official price is eventually agreed upon, the manufacturer must pay back the difference between what was charged during early access and the final negotiated price.
These rebates are substantial. In recent years, ATU-related rebates have contributed to billions of euros in pharmaceutical paybacks to the French health system. In 2020, the transfer of ATU discounts alone accounted for roughly €700 million. Total pharmaceutical rebates in France, including ATU clawbacks and other mechanisms, reached €5.63 billion by the most recent reporting period, representing about 23% of total drug expenditure.
Why Pharma Companies Use Early Access
For pharmaceutical companies, the ATU system and programs like it are far more than a goodwill gesture. They serve several strategic purposes that directly affect a drug’s commercial trajectory.
The most immediate benefit is real-world data. Patients treated through early access programs are more clinically and ethnically diverse than those in controlled trials, and the safety data collected from their treatment provides a broader picture of how the drug performs outside a study setting. This information feeds into global regulatory submissions and helps manufacturers anticipate safety signals before full launch.
Early access also builds physician familiarity with the product. Doctors who prescribe a drug through an early access program become experienced with its use, its dosing, and its side effect profile well before commercial launch. Research on these programs has found that prior exposure through early access meaningfully boosts a drug’s market share in its first year after launch. One study found the probability of spending on a drug was 1.36 times greater when an early access program had been in place before the commercial launch.
There are also direct financial benefits. In countries like France where early access products can be charged to the health system, companies generate revenue months or even years before the standard reimbursement process concludes. Beyond revenue, these programs help companies align their internal teams, from medical affairs to market access, well ahead of a global launch.
ATU’s Influence Beyond France
While the ATU is specifically French, the concept it represents exists across many countries under different names: compassionate use, expanded access, managed access, or named patient programs. What made France’s system distinctive was its scale, its formal structure, and the fact that drugs were reimbursed by the national health system during the early access period rather than provided free of charge or paid for out of pocket.
The 2021 reforms tightened some of these features, particularly around limits on individual patient access and the requirement that manufacturers commit to seeking full marketing authorization within a defined timeline. The goal was to preserve rapid access for patients while ensuring the system wasn’t used indefinitely as a substitute for completing the standard approval process.