French Introduce Time Limit For Drug Approval Applications After Compassionate-Use Award
Executive Summary
Companies wanting to make their unapproved products available to patients under the “temporary use authorization” are facing some changes to the way the rules are implemented.
The French government has made some changes to the rules governing the compassionate use of unapproved medicines, including the requirement for companies to file a marketing authorization application no more than one year after receiving a “temporary use authorization” (ATU), and to estimate the number of patients who are likely to benefit annually when an ATU is granted.
The changes, which are being implemented by two decrees published in April and May this year, are required under the social security financing law for 2017.
ATUs are granted for unapproved promising new drugs where there is a real public health need – for example, where the patient has a serious disease and has no other therapeutic options. They can be granted for single named patient or for a group or sub-group of patients (“cohort ATU”).
Under the May decree, if a marketing authorization application (MAA) has not been filed when an ATU is requested, the applicant must submit the MAA with the French authorities within one year of the ATU being granted.
The April decree is more wide-ranging, covering for example the conditions regarding cohort ATUs. Where a company applies for a cohort ATU, it must also send the health and social security ministers a dossier including the following information for each of the product’s indications:
- The target number of patients who might benefit from use of the medicine in this indication, taking account of the available medical and epidemiological data.
- An estimate of the number of patients who might be treated in this indication each year until the time that the drug is granted reimbursement in this indication (having received a marketing authorization), together with the forecast annual turnover of the product in this indication.
The decree also says that financial penalties amounting to as much as 10% of the company’s sales can be imposed if the conditions of the ATU are not respected, and lays down the procedure for imposing such penalties.
Companies have one month to reply to proposed penalties for failure to respect the terms of an ATU
When the drug pricing committee (CEPS) decides to impose a penalty, it informs the company concerned, outlining the reasons for the proposed penalty. The company then has one month to send written observations to the CEPS and request a hearing. In that same timeframe, the company must send the CEPS data on its turnover so the committee can decide on the appropriate level of the penalty. The CEPS will notify the company of the reasons underpinning the penalty decision, the deadline for payment, and the possibility of appeal.
The decree lays down the procedure for calculating the reimbursement rate for an indication of a product that was previously available under an ATU and then gains a marketing authorization and enters the “post-ATU” phase – ie, where a non-approved indication of the product continues to enjoy reimbursement.
It also explains the circumstances in which the Transparency Committee – which evaluates medicines in terms of their medical benefit/added value – may identify alternative therapies when the ATU reaches the end of its validity. It can do this either on its own initiative or at the request of the ministers of health or social security, or the health technology assessment (HTA) body, the HAS.
Compassionate-use programs have been in the spotlight recently, with the rare disease organization Eurordis saying that most EU member states are not doing enough to make unapproved medicines available to patients, although it said it regarded the French ATU scheme as “reasonable and affordable.” (Also see "Patient Groups Recommend Overhaul For Failing EU Compassionate Use Programs" - Pink Sheet, 7 Jun, 2017.)
From the editors of Scrip Regulatory Affairs.