In its first meeting of 2013, the European Medicines Agency's (EMA) management board turned its sights on the agency's use of regulatory science, saying EU regulators need to adopt new approaches to allow for drugs to get to patients more quickly and with a better understanding of patients' tolerance for risk-a potential boon for members of rare patient populations.
In a statement, EMA said the board has already turned its attention-much as the US Food and Drug Administration (FDA) has-to matters of regulatory science, seen as the various scientific approaches and methods by which regulators can obtain a better understanding of the likely benefits and risks of a product. In the case of EMA, which largely shies away from regulating medical devices, this effort is focused entirely on pharmaceutical products for the time being.
"Medicines regulation today is characterised by the increasing complexity of applications for new medicines, such as nanomedicines or personalised medicines, and the drug-development environment as a whole", explained Guido Rasi, executive director of EMA.
EMA's statement goes on to note that Rasi and the agency are interested in developing and increasing the regulatory capacity of the agency and the EU regulatory network as a whole, and in particular to be able to accommodate the use of innovative study designs. Recent developments by FDA have been focused on the same topic, including the use of what FDA called "enriched clinical trials," which in some cases rely on scientific regulatory tools to target trials to well-defined and narrowly-targeted patient populations. This allows for a more comprehensive assessment of risk and benefit in these populations, which are usually identifiable by their genetics, relative to a more general and genetically undefined population that characterizes many other clinical trials.
Adaptive Frameworks--A Boon to Patient Groups?
EMA's approach, though, is intended to touch upon other facets of the approval process as well.
"An innovative evaluation framework involving iterative phases of data gathering and regulatory evaluation is needed in order to align regulatory approval more closely with patients' needs for timely access to innovative medicines," EMA said. While the agency is relatively quick in its assessment of medicines, in recent years FDA has begun to outpace it in many areas, most notably in the approval of oncology products.
While approaches to closing or reversing that gap are varied, EMA said it is assessing the integration of multiple data sources into the regulatory decision-making process, including data from real-world use and clinical use, as well as the views of patients and their tolerance for risk relative to the potential for medical benefit.
Those changes could mark a boon for patients or rare diseases and the groups that advocate on their behalf, many of which have been clamoring for years for more adaptive licensing and regulatory frameworks that would allow for high-need patients to access therapies that might prove unsuitable for broader populations.
Some of these approaches are already in the works, EMA wrote, while others will be ongoing through 2014.