EMA-FDA parallel scientific advice program has ‘limited’ uptake

A review of the last five years of the Parallel Scientific Advice (PSA) program between the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) shows that the agencies received a total of 37 applications from sponsors of new medicines and accepted 70% of requests.
 
The PSA program, which launched in 2005, provides a mechanism for sponsors of new medicines to seek concurrent advice from regulators in the US and Europe on scientific issues involved in product development. It is a voluntary program and is triggered by a request from a sponsor.
 
The review, which was conducted by FDA and EMA staff, was published in Therapeutic Innovation & Regulatory Science in March 2023. From 2017-2021, the agencies received 37 requests, of which 26 (70%) were accepted. Over the five-year period, four sponsors withdrew their requests or chose not to proceed, leaving 22 completed PSA procedures.
 
“Data from our 5-year review show that uptake of the PSA pathway has been limited – just four to seven procedures annually over the last five years,” wrote Shannon Thor, of FDA’s Europe Office, along with colleagues from both agencies.
 
Thor and colleagues suggested that the PSA program and its timeline are not well understood by sponsors. “In our observation neither our Agencies nor industry have promoted it widely and little has been written about this process,” they wrote.
 
The review found that 23% of accepted PSA requests were in a category that combines submissions from gastroenterology, inborn errors of metabolism, rare diseases, and medical genetics. Multiple accepted requests also came from oncology, anti-infectives, cardiology and nephrology, and neurology. A look at the 11 requests that were rejected from 2017-2021 showed that four were too early in development and four had medical device components, though submissions with device components are no longer discouraged. The remaining three rejected requests were for “more varied or nuanced reasons,” according to the review.
 
The review also drilled down on timeline data among the seven accepted applications in 2020. Among this group, there was an average of 13 calendar days between the PSA request and acceptance by the agencies, an average of 67 calendar days spent in the meeting package preparation and validation phase, and an average of 79 calendar days between validation and the final meeting minutes from the FDA and a final advice letter from EMA.
 
Along with the review, the FDA and EMA authors also offered best practices for sponsors considering the PSA process. They recommended that sponsors only apply for the program if they have begun the pre-IND or IND process at FDA. “With the foundations and background of your product’s development plan already understood, your PSA questions can be focused on the specifics of global development that merit consideration for convergence,” they wrote.
 
The authors also suggested that sponsors research existing guidance to see where alignment exists between the agencies. The PSA program is likely to be most appropriate for “innovative products or new scientific or regulatory concepts” that have not already been explored in published guidance. Similarly, they advised sponsors to consider the public health benefit of their product before making a request. “PSA requires extra investment of resources from both Agencies, so the program’s focus is on products that address unmet medical needs, rare diseases, pediatric populations, or other areas of importance to patients and public health,” the authors wrote.
 
Therapeutic Innovation & Regulatory Science article