OBJECTIVES: To examine market access timelines and HTA assessments for medicines approved under exceptional circumstances by the European Commission (EC) between January 2009 and December 2017.

METHODS: Analysed medicines approved by the EC that are still authorized under exceptional circumstances. Data gathered from EMA, official national HTA agencies and P&R bodies. Cut-off date for data collection was March 1, 2017.

RESULTS: Only 20% of drugs (n=3) approved under exceptional circumstances have completed P&R negotiations in Spain and only 40% in France (n=6). Time to market for drugs approved under exceptional circumstances is substantially longer compared to all drugs across the EU5 countries.

Analysis of HTA assessment for 6 drugs available in France, Germany and the UK suggest poor appraisal outcomes. Two thirds of the drugs assigned ASMR IV in France. In Germany, of the four post AMNOG drugs, 2 had a non-quantifiable added benefit. Drugs with high ASMR ratings in France (asfotase alfa and cholic acid) assigned non-quantifiable added benefit rating in Germany. Only 1 drug assessed by NICE and recommended with managed access agreement.

CONCLUSIONS: Unlike conditional marketing authorisation, authorisation under exceptional circumstances is granted when comprehensive data cannot be obtained even after approval. Although this may be a possible pathway for regulatory approval for certain drugs, significant access challenges are likely across the EU5 countries given immature data.