Conditional marketing authorisations (CMAs) aim to provide patients with an unmet medical need early access to new medicines. This EMA report presents the positive impact CMAs have had in improving public health. It has been produced in response to the European Commission’s Expert Group on Safe and Timely Access to Medicines for Patients (STAMP), which wanted to understand the experience of conditional marketing authorisations in the EU.
The report presents information on a total of 30 medicines that have received a conditional marketing authorisation (CMA) since 2006, 14 of which were orphan drugs. It focuses on how CMAs are granted or refused and later converted into standard marketing authorisations (MA). It also examines the type, amount and timing of data provided to support these decisions.
A CMA is one means by which the EMA can support the development and immediate availability of medicines in the EU provided the public health benefit of early access to patients outweighs the risk of the authorisation being granted with less comprehensive data than normally required. A CMA is granted for one year. A condition of the authorisation is that the company conducts further studies to obtain complete data. The Committee for Medicinal Products for Human Use (CHMP) evaluates the data produced from the post-authorisation obligations at least once a year to ensure that the benefit/risk evaluation for the medicine remains positive. At the end of each assessment, the CHMP recommends either the renewal or revocation of the CMA or its conversion into a standard MA.
Reassuringly, over the 10-year period no medicine with a CMA had to be revoked or suspended. The EMA’s analysis shows that MA holders (MAHs) complied with the specific obligations imposed by the agency. In the vast majority (>90%) of cases, completed specific obligations did not result in major changes of scope and around 70% of specific obligations did not require an extension to the originally specified timelines.
The report shows that it took an average of 4 years to generate the additional data needed and to convert a CMA into a full MA. So, in the absence it would have, on average, taken this length of time for patients with life-threatening or seriously debilitating conditions to access new medicines. However, it should be noted that patients have in the past been able to access some medicines in development via company managed access programmes.
The report also describes the data on which CMAs have been granted and the additional data subsequently generated to meet specific obligations. This information could be very useful to those developing medicines.
In the report the EMA identifies a number of potential areas for improvement as:
- Prospective planning of CMAs and early discussion with EMA
- Timely dialogue on additional post-authorisation studies and their feasibility
- Improved data generation for completion of specific obligations
- Engagement of other stakeholders involved in bringing a medicine to patients, in particular Health Technology Assessment bodies, to enable the generation of all data needed for decision-making through one development programme.