Country: FRANCE | Region: EUROPE | Type: Policy | Keywords: #access #atu #budget #compassionate #drug #early #france #plfss #policy #spending
- French MPs have voted in favor of amendments to the Social Security Finance Bill 2021 (PLFSS 2021), regarding early access (RTU) and managed off-label use (ATU) scheme
- In accordance with the voting, the new “Early Stage” and “Compassionate Use” schemes will be implemented in order to ensure patients have early, temporary and exceptional access to drugs that have not been approved for a specific indication in France
- The new Early Access scheme will cover drugs that are likely to be innovative, and the Compassionate Use scheme will cover treatments which respond effectively to a therapeutic need, but aren’t necessarily innovative or initially intended for approval
PARIS, France – French MPs have voted in favor of amendments to the Social Security Finance Bill 2021 (PLFSS 2021), regarding early access (RTU) and managed off-label use (ATU) schemes.
In accordance with the voting, the new “early stage” and “compassionate use” schemes will be implemented in order to ensure patients have early, temporary and exceptional access to drugs that have not been approved for a specific indication in France.
The currently used processed have been deemed overly complex in the past, leading the MPs to decide on replacing the schemes with newer, more streamlined systems. On top of the complexity, MPs cited a lack of readability for all stakeholders.
The new Early Access scheme will cover drugs that are likely to be innovative, and the Compassionate Use scheme will cover treatments which respond effectively to a therapeutic need, but aren’t necessarily innovative or initially intended for approval.
The official PLFSS law text reads, but is not limited to –
In regard to Early Access:
“Art. L. 5121 – 12. – I. – The early access defined in this article governs the use, by way of exception, of certain drugs, in specific therapeutic indications, intended to treat serious, rare or disabling diseases, when the following conditions are met”:
- There is no appropriate treatment;
- The implementation of the treatment cannot be postponed;
- The efficacy and safety of these drugs are strongly presumed in view of the results of therapeutic trials;
- These drugs are presumed to be innovative, in particular with regard to a possible clinically relevant comparator.
In regard to Compassionate Use:
“Art. L. 5121 – 12 – 1. – I. – Articles L. 5121-8 and L. 5121-9-1 do not preclude the exceptional use, for compassionate access, of certain medicinal products, in precise therapeutic indications, when the following conditions are met”:
- The medicinal product is not the subject of research involving the human person for commercial purposes in the indication considered;
- There is no appropriate treatment in the indication concerned;
- The efficacy and safety of the drug are presumed with regard to the available clinical data as well as, when the indication concerns a rare disease, the work and data collected by healthcare professionals under conditions defined by decree in Council State.
Under the new workings, the High Health Authority (HAS) will then work alongside the National Security Agency of medicine and health products to help integrate appropriate products into the basket of care.
The new measures will help create a more cohesive system that entails:
- Rapid access for patients to drugs in advance (although upstream of common law) in a secure environment for use outside the usual
- Marketing authorization and paid for by health insurance,
- Readability for prescribers,
- Attractiveness and predictability for companies with innovative drugs,
- Financial sustainability for health insurance
The upheaval of the existing system aims to “simplify demands”, as well as “harmonize procedures”, according to the document.
PLFSS was initially published at the beginning of October, but French pharmaceutical industry body LEEM was quick to denounce the “inconsistency” of the bill shortly after its release.
In particular, the group noted its issue with suggested legislative measures modifying the mechanisms of conventional subsidies. It explained that this provision, in addition to its particularly difficult to read and predictable terms of implementation, is clearly a setback from the existing treaty rules.
LEEM did note, however, that some clauses would make it possible to improve the predictability of early access mechanisms to benefit patients. In addition, ignoring 2020 as a jumping-off point in the calculation of the 2021 base seems logical, given the nature of this year and the effects of the pandemic.
The plan, among a number of other things, aims to restore economic growth and mitigate the economic and social consequences that the COVID-19 crisis has had on the country.
Due to the timing of publishing, the plan is one of the most “unique” in the history of the PLFSS, given having to accommodate both COVID-19 and the number of structural transformations going on in France at the moment.
Notably, the PLFSS implements a flagship measure of the Strategic Council of Health Industries (CSIS) intended to simplify and accelerate the provision of innovative medicines to patients.
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