EMA: doporučení 81 léků k registraci v roce 2013
In line with the previous years, the number of medicines containing a new active substance is quite high, with 38 medicines containing active substances that had never been used in medicines before compared with 35 in 2012, 25 in 2011 and 15 in 2010. The number of generics is stable compared with 2012 but generally decreasing over the last few years.
One in every two applicants received scientific advice from the Agency’s CHMP during the development phase of their medicine. Seeking and following scientific advice has been proven to significantly increase the probability of a positive outcome, with a 90% success rate at the time of marketing-authorisation application for companies that request and follow scientific advice compared with a 30% success rate for companies that do not request scientific advice**.
Of note, two new advanced therapy medicinal products (ATMPs) were recommended for approval in 2013, bringing to four the total number of ATMPs recommended for approval by the CHMP since the legislation on advanced therapies became operational (the first one was approved in 2009 and the second one in 2012). These innovative medicines are derived from gene therapy, cell therapy or tissue engineering and can offer groundbreaking new opportunities for the treatment of disease and injury.
Also, the number of recommendations for marketing approval of medicines intended for the treatment of rare diseases is steadily increasing (11 in 2013 compared with 8 in 2012 and 4 in 2011), showing the success of the European orphan-medicinal-product regulation, which came into force in 2000.
2013 also saw the first two positive opinions for marketing authorisations of biosimilar monoclonal antibodies, successfully applying the biosimilar concept to such structurally complex substances.
More treatment options for cancer and tuberculosis
In 2013, 16 new medicines for the treatment of cancer were recommended for marketing authorisation, of which 12 contain a new active substance. Most of these medicines are considered to be targeted therapies, designed to block the growth and spread of cancer by interfering with specific molecules involved in tumor growth, or act on the patient’s immune system.
Particularly noteworthy in the other therapeutic areas is the recommendation of three medicines for the treatment of multidrug-resistant tuberculosis, an orphan indication associated with a very high mortality rate and whose burden has rapidly increased in recent years in the absence of new treatment options.
In 2013, the CHMP recommended the approval of four new medicines for use in patients infected with human immunodeficiency virus (HIV), all of which contain a new active substance, and five medicines for the treatment of type 2 diabetes, four of which contain a new active substance.
Special regulatory pathways to facilitate market access
The Agency has mechanisms in place, such as conditional marketing authorisation, approval under exceptional circumstances and accelerated assessment, to facilitate or accelerate market access for medicines that fulfill unmet medical needs or that could not be made available under a standard approval because the disease they target is too rare and comprehensive data cannot be provided.
In 2013, the CHMP recommended conditional marketing authorisation or approval under exceptional circumstances for nine medicines. These medicines include anticancer agents (Bosulif, Erivedge and Cometriq), a smallpox vaccine (Imvanex) and medicines intended for rare conditions (Lojuxta, Defitelio, Cholic acid FGK, Deltyba and Sirturo).
Also, opinions for two compassionate use programmes were given in 2013 for the treatment of hepatitis C virus (HCV) infection. These programmes are intended to give patients with a life-threatening, long-lasting or seriously disabling disease with no available treatment options access to treatments that are still under development.
Listening to the specific needs and views of patients and healthcare professionals has become increasingly important for the Agency as it enriches the scientific evaluation of medicines. The EMA uses various mechanisms to gather input from them, e.g. through the involvement of patients and healthcare professionals in scientific advisory groups or ad-hoc expert groups, providing input and advice to the CHMP in relation to the evaluation of specific types of medicines or treatments.
Negative opinions and withdrawals
In 2013, the CHMP recommended initial negative opinions for seven medicines, two of which were transformed into positive opinions following re-examination procedures.
Eight applications for initial evaluation were withdrawn by the sponsors prior to CHMP opinion.
Initial evaluations started
In 2013, the Agency started the assessment of 79 applications (compared with 95 in 2012) for the initial evaluation of medicines. The decrease observed is mainly due to fewer marketing authorisation applications received for generics and biosimilars. Further information on initial evaluations started in 2013 can be found in the December 2013 monthly statistics report.