PNS150 CONDITIONAL MARKETING AUTHORISATION FOR NOVEL TREATMENTS: A LANDSCAPE ASSESSMENT OF EXPEDITED PATHWAYS OUTSIDE US AND EU

Abstract

Approval of pharmaceuticals based on promising early efficacy results using conditional marketing authorisation (CMA) pathways is one of the tools used by EMA and FDA to expedite patient access to life-saving treatments. A targeted search of HTA agency documents was conducted to categorise policies in other regions, where use of CMA has been poorly documented. Two researchers extracted information on CMA pathways from official websites of countries with a national HTA agency, unit or committee: this included eligibility criteria, requirements and time frame for post-authorisation data collection. Hand searches of published/grey literature were conducted to find additional insights on the CMA process. CMA policies were found in 8 of the 40 countries included in the search: Argentina, Australia, Canada, China, Japan, Serbia, South Korea and Taiwan. Eligibility for orphan drugs was specified in 4 countries. In 6 countries, requirements included unmet need and a major therapeutic advance. Pathways aimed at innovative drugs were identified in the Asia Pacific region: Japan’s Sakigake for regenerative medicine and cell and gene therapy in South Korea. China and Serbia leverage approval decisions in the US and EU to minimise risk from early approvals. In terms of post-approval clinical data requirements, Canada and Taiwan require confirmatory phase III data, whilst others specify longer term phase II studies. Taiwan, Australia and Japan set explicit time limits for additional data (5,6 and 7 years, respectively), although it is unclear how agencies ensure compliance. CMA pathways have been established in a number of emerging healthcare markets, although policies differ across countries. Whilst all 8 agencies specified serious and rare conditions are eligible for CMA, some focused on innovative drugs or on products already in use in established markets. Additional research is needed to describe how CMA impacts on reimbursement and patient outcomes.