Regulatory Approvals Addressing Unfulfilled Medical Needs in 2018
In a comprehensive study by the Centre for Innovation in Regulatory Science (CIRS), the regulatory approval of new active substances (NASs) was analysed across six major authorities: the European Medicines Agency (EMA), the U.S. Food and Drug Administration (FDA), the Japan Pharmaceuticals and Medical Devices Agency (PMDA), Health Canada, Swissmedic, and the Australian Therapeutic Goods Administration (TGA).
The study revealed some interesting trends. The number of common products approved by all six agencies increased significantly from 16 in 2009-2013 to 52 in 2014-2018, indicating a rise in the internationalization of products.
All six agencies now offer an expedited review process to hasten the review of promising new active substances. In 2018, expedited approvals accounted for the highest proportion of approvals for the FDA (73%), followed by Health Canada (35%), PMDA (28%), Swissmedic (13%), EMA, and TGA (10%).
The FDA had the highest number of NASs with an orphan designation in 2018 (35), while PMDA had the lowest (eight). Out of the 52 NASs approved by all six authorities in 2014-2018, only 10 NASs received an orphan designation across all the authorities.
The majority of orphan NASs were approved by non-top companies, emphasizing the significant role of smaller companies in driving innovation. This was also evident in the fact that the majority of orphan NASs approved by non-top companies were approved by non-top companies.
Health Canada does not have an orphan policy, but approved 15 NASs in 2018 that were classified as orphans by other agencies. At TGA, 20% of orphan drugs were approved in 2018 with the newly introduced priority review.
The FDA offered the greatest number of FRPs to enable the availability, review, and/or approval of medicines where there is an unmet medical need. 75% of NASs approved by the FDA in 2018 benefitted from at least one of the available FRPs (orphan excluded).
Underlying factors affecting the time it takes for a new medicine to be submitted and approved include company strategy, the conduct and type of the review process, the type of the product and its therapeutic area, facilitated regulatory pathways (FRPs), orphan drug designation, and sponsor size.
The study also found that EMA had a longer median approval time for orphans than for non-orphans in 2018, while the FDA had the fastest median approval time for orphans (243 days). At TGA, 88% of orphan approvals in 2018 were through expedited review.
However, the search results do not contain specific information about which companies had the most new drugs approved via expedited approval pathways at the six regulatory authorities from 2014 to 2018.
This study provides valuable insights into the regulatory approval process of new drugs and the role of expedited reviews and orphan drug designation in speeding up the process. It also underscores the importance of smaller companies in driving innovation and meeting unmet medical needs.
