By Nicola Allen, Associate Director & Andrea Bernardini, Analyst
Email: [email protected] or [email protected]
The French Authorisation for Temporary Use (ATU) was originally established in 1994 [1] to enable pharmaceutical companies to sell certain medicinal products prior to marketing authorisation and reimbursement negotiations. The original ATU program has since evolved into six different pathways with overlapping domains and growing demand leading to a 20% increase in the number of named patient ATUs in 2019 [2]. In recent years, the annual costs to fund the French early access programs has ranged from €700m to €1bn [3], prompting a review to identify opportunities for a more financially sustainable solution.
The Social Security Funding Bill (PLFSS) for 2021 was approved by the French parliament on the 27th of October [4] and announced major changes to the ATU system [5], which streamlines the pathways into two distinct routes: the Early Access route and the Compassionate Use route (see Figure 1). The former can provide early access for drugs in clinical development with plans to submit for marketing authorisation. While the latter is for off-label use of treatments, but there is no intention from the manufacturer to seek marketing authorisation for the indication in question.
The inclusion of The French National Authority for Health (HAS) as a decision maker for the Early Access route is a considerable step change, given the introduction of the HTA perspective on innovation as a prerequisite. This more selective criteria will lead to a reduction in the number of treatments eligible for the Early Access route compared to the old system. While manufacturers are still able to set the reimbursed price, changes to the calculation for rebates and the introduction of opportunities for price discounts (see description below) will further help the French authorities to manage the ATU budget. However, the financial implications of these changes for manufacturers remain unclear.
The new Early Access route
The Early Access route will now see HAS and the National Agency for the Safety of Medicines and Health Products (ANSM) working together to grant access to therapies under development with plans to obtain marketing authorisation for the considered indication. The main steps are:
- HAS will work in collaboration with ANSM to determine early access approval
- Therapies are immediately covered after early access approval is granted
- The full reimbursement of the price set by the manufacturer will be subject to a double-mechanism of rebates where:
- Annual rebates are calculated based on turnover
- Product rebates are retroactively applied when final price is negotiated
In addition, the manufacturer must honour the following commitments:
- Funding of the collection of data as defined by the HAS and ANSM
- Continuation of the treatment and if required,
- Submission of a Market Access request within a specific timeframe.
Further discounts will be applied if:
- The manufacturer does not comply with the above commitments
- Negotiations with CEPS last more than 6 months
The new Compassionate Use route
The compassionate use route is regulated by ANSM and is for off-label drugs that are currently not under development for a specific indication and this will be particularly relevant for rare diseases. The newly streamlined process will follow the steps below:
- The prescribers or healthcare authorities can ask the ANSM to evaluate the conditions for access, which covers a 3-year renewable period
- ANSM grants the named-patient access to the off-label treatment
- Once access is approved, reimbursement is based on:
- The published price (if available)
- Annual rebates calculated on the turnover made
- Flat-rate price set by the Ministry of Health
Additionally, the data collection is ensured by the prescriber while being funded by the manufacturer.
Impact for Industry
The new streamlined Early Access route is certainly a step towards more consistent coverage by including both ANSM and HAS to oversee the new pathway. The inclusion of HAS as a decision maker will result in a shift towards the selection of therapies that are deemed more promising and innovative from a HTA perspective. While this may result in more selective criteria, it will result in a more cohesive process from when early access is granted to the standard HTA and pricing negotiations.
The financial impact of the new pathways is less clear for Industry given the new system now includes several opportunities for the authorities to modulate the Early Access reimbursed price. This will have implications for the final reimbursed price because the Early Access price will essentially set a price ceiling for the subsequent price negotiations with CEPS. There is only one identified example of a therapy that achieved a negotiated reimbursed price higher than the ATU price, which was Lutathera for Gastroenteropancreatic neuroendocrine tumours (GEP-NETs).
The manufacturer is also required to commit to the funding of data collection for both routes with further adds to the financial impact for industry. This requirement will be particularly challenging for smaller companies that do not have a local presence in France and may well act as a barrier to early access.
The new system is expected to be fully operative from July 2020 onwards. Therefore, any product with an ongoing old ATU/RTU from the start of the new PLFSS to the end of its authorisations will still be under the old system, while post-ATUs will be under the new directives. ATUs that will see their authorisation ending during this interim period will not be able to be apply for a renewal and will have to go through the new early access route. The Partners4Access team will continue to monitor the introduction of the new process and implications for treatments currently provided via the existing ATU pathways.
Sources:
[1] https://www.persee.fr/doc/pharm_0035-2349_2004_num_92_341_5596
[2] https://ansm.sante.fr/var/ansm_site/storage/original/application/ad56ba73d6f8127b89da5ea73583041d.pdf
[3] https://minefi.hosting.augure.com/Augure_Minefi/r/ContenuEnLigne/Download?id=26C2D5A8-A9FE-496B-A731-62FB47790A14&filename=DP%20PLFSS%202021.pdf
[4] http://www.assemblee-nationale.fr/dyn/15/textes/l15b3397_projet-loi#tocUniqueId56 [5] https://www.affiches-parisiennes.com/projet-de-loi-de-financement-de-la-securite-sociale-2021-les-mesures-phares-11038.html
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