EMA has launched a new pilot to assess whether the analysis of ‘raw data’ by regulatory authorities improves the evaluation of marketing approval for new medicines. The pilot will run for 2 years starting in September.
Still, regulatory decisions within EU are based on the assessment of applicant’s own analyses, which in turn must be thoroughly described both in terms of methods and results in a predefined manner. FDA on the other hand, can make their own analysis of the raw data underlying the summaries and conclusion in regulatory applications. This could be in the form of meta-analysis to better understand benefit-risk in the target patient population by combining evidence from relevant studies using appropriate statistical methods. The most common reason for performing a meta-analysis is to provide an estimate of a treatment effect when data from independent studies are insufficient. FDA could also use raw data to perform their own exposure-response analysis to better inform dose-selection. Obviously, FDA needs access to raw data, which must be submitted to the agency in a standardized predefined format (CDISC).
Now it seems like EU would like to follow FDA’s footstep. This is not surprising since strengthen the network’s capability to analyze data was one of the outputs of the Big Data Taskforce. A long-term potential benefit could be that the availability of raw data from many products will ease cross-product analyses and thus relative benefit-risk assessments. Such relative comparisons are what payers are asking for today, and this pilot may be the first step to satisfy that need. Thus, in the end unbiased raw data assessment from regulators may facilitate fair pricing of medicinal products in the EU.
Should the pilot translate into reality some obvious questions have to be answered though; how to increase the capacity and competence within the EU regulatory authorities to adapt to the new way of working? Obviously, performing own analysis will come with a cost and it will be interesting to see how EMA will address this issue – increasing the fees for centralized applications or reducing the capacity somewhere else in the regulatory system?
EU regulators already have the possibility to require additional analysis from the Applicants and an educated guess is that they will continue doing that. The companies are not doing a bad job as it is, but analysis undertaken by the regulatory authorities would be another tool that could be used. Looking forward to the outcome of the EMA pilot to hopefully then understand if “in house” analyses actually will add value.
Marie Gårdmark, CEO RegSmart Life Sciences
Photo: Chris Leverani, Unsplash