Multinational Expedited Approval Pathways 
​[Posted on: Thursday, November 3, 2016]

Promising treatments are provided preferential review options in several regulatory domains. This week Australia’s Therapeutic Goods Administration (TGA) joined regulators in US, Canada and Europe in creating two expedited approval pathways for promising drugs, namely, the Priority Review and Provisional Approval designations. The Priority Review process is similar to that at the FDA where the eligible application will be reviewed in a shorter time, while the Provisional Approval is similar to the Accelerated Approval pathway of the FDA. Drugs for serious conditions, unmet medical needs and showing major therapeutic advantage over existing therapies will be eligible for Priority Review or Provisional Approval in Australia. These pathways are very similar to those in the US with some differences. Both the designations can be rescinded if the drug no longer meets the criteria, the applicant is unable to get GMP certification or if the application is rejected for an accelerated assessment process by a comparable non-Australian regulator. Also application approved under the Provision Approval pathways will be not be considered permanently approved but as the name indicates, the approval will be considered provisional or temporary subject to the sponsor submitting post-market safety and efficacy data before the product is granted full registration. In case the sponsor is unable to submit the required post-market data within a defined time-period, the Provisional Approval will lapse. This is similar to the Conditional Approval pathway at EMA. In contrast, while the drugs approved under Accelerated Approval pathway at FDA also have post-market data requirements, FDA does not enforce a formal time-line for submission of the post-market data and the approval is considered perpetual. TGA also pays emphasis on the regulatory history of a given application in other regulatory domains unlike FDA. Last year EMA implemented the PRIME pathway similar to the Breakthrough Therapy designation at FDA, and all regions have orphan drug pathways for rare diseases. With these provisions, the same drug can be eligible for expedited approval simultaneously in US, Canada, Europe and Australia. Other regions of the World also benefit from these expedited approval pathways. Most countries in South America, Africa and Asia grant preferential review to drugs approved by regulators in US, Europe, Canada and Australia, so expedited approval in these regions potential translates into world-wide approval of a given drug. With the multinational adoption of most expedited approval pathways, it is tempting for sponsors to simultaneously apply for designations in all regions. However, if any one regulatory agency disagrees with the designation request, it is likely that other regulators will follow suit as well. So, the best strategy is get successful designation in one region and then try to use that in others.