Provisional marketing authorization (CMA) can speed up access to medicines for patients with unmet medical needs. Since 2006, a total of 30 medicines have received a provisional marketing authorization. Medicines that were approved a CMA target seriously enervating or life-threatening conditions such as HIV infection, breast cancer, and severe epilepsy in infants or multi-drug resistant tuberculosis. 14 were orphan medicines, providing patients suffering from rare diseases with new treatment options. These are some of the findings of a report by the European Medicines Agency (EMA) to mark ten years of experience with CMA.
EMA’s report is a wide-ranging investigation of the positive influence this important tool has had in providing early access to new medicines for patients who formerly had no or only substandard treatment options. It emphases on how CMAs are approved or rejected and later converted into normal marketing authorizations. It also examines the type, extent and timing of data provided to support these decisions.
Provisional marketing authorization is one of the implements available to regulators to support the growth of and early access to medicines that address unmet medical needs of patients in the European Union (EU). It permits the approval of medicines if the public health assistance of their immediate availability to patients outweighs the risk of an authorization on the basis of less inclusive data than normally required. Over a period of 10 years, no medicine with a CMA had to be repealed or adjourned.
A CMA is effective for one year. As part of the authorization, the company is indulged to carry out further studies to obtain complete data. EMA’s Committee for Medicinal Products for Human Use (CHMP) evaluates the data generated by these specific post-authorizations requirements at least annually to guarantee that the balance of benefits and risks of the medicine remains positive. At the end of its valuation, the Committee recommends either the renewal or not of the CMA or its alteration into a standard marketing authorizations.
According to EMA’s analysis, marketing authorizations holders comply with the specific responsibilities forced by the Agency. More than 90% of completed precise responsibilities did not result in major changes of scope and about 70% of specific obligations did not need an extension to the initially stated timelines.
The report shows that it took an average of four years to generate the additional data needed and to convert a CMA into a full marketing authorization. This means that patients with life-threatening or seriously incapacitating conditions can access promising medicines earlier.
The report also personifies the data on which CMAs have been granted and the additional data generated through specific obligations. This information could be of interest for those developing medicinal products, as well as patients, pricing and reimbursement bodies and other stakeholders.
The report also categorizes a number of possible areas for expansion. These comprise:
- future planning of CMAs and early dialogue with EMA to support the generation of high-quality data, timely discussion of additional post-authorization studies and their possibility, and better data generation for accomplishment of explicit obligations;
- Appealing other stakeholders involved in bringing a medicine to patients, in particular Health Technology Assessment bodies, to help the generation of all data needed for decision-making through one development programme.
The full report is available together with an infographic that highlights the important results of this study. The publication of EMA’s report emphasizes the Agency’s commitment to transparency.
It responds to discussions in the European Commission’s expert group on Safe and Timely Access to Medicines for Patients (STAMP), which emphasized interest in accepting the experience of conditional marketing approvals in the EU.
Date:23 Jan, 2017
http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2017/01/news_detail_002680.jsp&mid=WC0b01ac058004d5c1