Study: High concordance in breakthrough therapy, PRIME decisions

The US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) agreed on nearly two-thirds of decisions to grant or deny requests for drugs and biologics seeking Breakthrough therapy or Priority Medicines designation, according to a new analysis.

The findings highlight the need for agencies and sponsors to take advantage of collaborative opportunities, including the Parallel Scientific Advice program, to support global drug development that addresses unmet medical needs, wrote Zahra Hanaizi, of the EMA, along with colleagues at EMA and FDA, wrote in Therapeutic Innovation & Regulatory Science.

FDA’s Breakthrough therapy (BT) designation, launched in 2012, and EMA’s Priority Medicines (PRIME) designation, launched in 2016, are designed to provide support and scientific advice during the development of products that have the potential to address unmet medical needs. After the launch of PRIME, FDA and EMA began monitoring similarities and differences in what was submitted and designated under the two programs to find ways to better support global development. The researchers added designations made through the FDA’s Regenerative Medicines Advanced Therapies (RMAT) program at the time of its launch in 2018.

Key findings
From 4 April 2016 to 31 December 2020, 151 requests were made to both programs, with the agencies reaching the same conclusion – to approve or deny an application -- in 93 cases (62%). Of the 93 cases in which the agencies agreed, 42 applications were granted and 51 were denied. The researchers noted that just one of the 42 applications approved by both agencies was the subject of formal Parallel Scientific Advice, a program that allows EMA and FDA to advise a sponsor collaboratively (RELATED: FDA, EMA tout benefits of parallel scientific advice program, Regulatory Focus 17 March 2022).

Divergence of opinion by the agencies occurred with 58 applications, or 38.4%. In most cases, the reason for diverging conclusions was a difference in interpreting the same data or differences in program implementation policy. Less often there were different data submitted or differences with currently available treatment across regions.

When researchers added information on RMAT designations, the number of common product submissions to the two agencies increased to 176, but the level of concordance was similar, at 63.6%.

The high level of concordance in application decisions suggests similar perspectives by international experts on the potential value of the products being considered. “Our numbers are too small to draw conclusions about whether the two thirds rate of concordance, overall, is similar across therapeutic areas, but it held for oncology products and pooled, non-oncology products. The patterns and findings did not change when we added RMAT to our comparisons,” the researchers wrote.

Researchers also examined submission timing across BT and PRIME. Of the 151 requests, 32% were submitted within 60 days of each other and 47% were within 120 days. “It may speak to the fact that companies are or became familiar with both programs over the period of the cohort’s operation,” the researchers wrote. “We were expecting common requests to be submitted more closely in time as we examined our cohort year by year, but we did not observe a clear pattern toward this.”

Therapeutic Innovation & Regulatory Science study

Study: High concordance in breakthrough therapy, PRIME decisions

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