2009-2010 new applications
We have analyzed which are the factors that influence the non approval of new drugs in Europe. To do this, we have found out an article published recently on Nat Rev in which this topic is investigated (see references). The authors of the article declare the views expressed are personal views of the authors and must not be understood or quoted as being made on behalf or reflecting the position of EMA. In this article, they investigated all marketing applications (and assessment report) for new active substances considered for approval at EMA from 2009 to 2010 (n=68). Normally a report is analyzed by CHMP looking at development plan, clinical outcome (both efficacy and safety) and clinical relevance of the data. For these three assessment categories, the authors identified 15 key variables that they dichotomized into positive or negative score to reduce the huge amount of data and subtle regulatory weighting of all the information possibly relevant for patients into simple binary terms.
KEY VARIABLES AND SCORES FOR DEVELOPMENT PLAN, CLINICAL OUTCOME AND CLINICAL RELEVANCE
The scoring of the CHMP assessment of the development plan was divided by Putzeist et al. into an analysis of learning studies (mode of action, proof of concept, pharmacokinetics, dose finding and safety pharmacology) and of confirmatory studies (study design, choice of primary endpoint, target population, trial duration and statistical analysis) (5 key variables each). The assessment of the clinical outcome was scored on statistical significant results on the primary efficacy endpoints and the safety profile (2 key variables); clinical relevance included CHMP views on medical need, the effect size of the clinical results and clinical benefit in general (3 key variables).
The authors declare “in that period (2009-2010) CHMP evaluated 68 applications of which 45 (66%) were approved, whereas 23 (34%) where not; 6 received a negative opinion and 17 were voluntarily withdrawn by the applicant before a final opinion was made by the CHMP. We have compared these data with that published on the annual EMA report 2010. The report asserts that in 2010 the Agency received a total of 91 applications for marketing authorization for human medicines, including 1 for a scientific opinion for a medicine intended for use outside the EU. Excluding multiple applications, this relates to 73 applications by active substance. The slight difference (68 vs 73) expressed by the author could be referred to a different period of evaluation. Based on the EMA report, of the applications received, 46 related to new medicines; 12 of these were for orphan-designated medicines. Almost half (43) of all applications received in 2010 were for generic medicines or hybrid and informed-consent applications. One application was received for a similar biological medicine. The Agency received the first application for a pediatric-use marketing authorization (PUMA). Musculoskeletal disorders was the therapeutic area in respect of which most applications were received (24), followed by medicines intended to treat respiratory diseases and alimentary-tract disorders.
COMMITTEE FOR MEDICINAL PRODUCTS FOR HUMAN USE (CHMP)
The Agency has five scientific committees related to medicines for human use. These are the Committee for Medicinal Products for Human Use (CHMP), the Committee for Orphan Medicinal Products (COMP), the Committee on Herbal Medicinal Products (HMPC), the Paediatric Committee (PDCO) and the Committee for Advanced Therapies (CAT). The work of the scientific committees is supported by standing working parties, scientific advisory groups and ad-hoc expert groups. It is the role of the Agency’s secretariat to ensure appropriate coordination between committees and working parties. The CHMP is responsible for the scientific evaluation and provision to the European Commission of scientific opinions for the authorization and maintenance of medicinal products for human use. The CHMP provides scientific advice and protocol assistance to pharmaceutical enterprises during the process of medicines development. The CHMP also provides scientific opinions on medicinal products subjected to arbitration or referral procedures, on medicinal products intended for use outside the EU, and on any scientific matter at the request of the European Commission or the Executive Director of the Agency. Furthermore, the CHMP is involved in work undertaken in the fields of harmonization of technical requirements for pharmaceutical regulation, pharmacovigilance and public-health threats.
References
Putzeist M et al., Factors Influencing Non-Approval Of New Drugs In Europe. Nat Rev. 2012;11:903-4