OBJECTIVES: To examine the association between the magnitude of added health gains offered by new drugs and several drug novelty attributes.

METHODS: We identified drugs approved by FDA from 1999-2018. For each drug-indication pair (e.g., vedolizumab for ulcerative colitis), we determined if the drug was included in FDA’s expedited approval programs: Priority Review (PR), Fast-track (FT), Accelerated Approval (AA), and Breakthrough Therapy (BT). Consistent with each program’s qualifying criteria, we categorized drugs using the following novelty attributes: (1) treats a serious condition (approved via any expedited approval program), (2) offers improvement over existing treatments (approved via PR, AA, and/or BT), and (3) addresses unmet clinical needs (approved via FT). To obtain added health gain estimates, we searched PubMed for relevant comparative- and cost-effectiveness studies reporting Quality Adjusted Life-Years (QALYs). We included only studies that compared the drug to the standard of care at the time of its FDA approval. We extracted estimates of incremental QALY gains from included studies. We compared incremental QALY gains for drugs with each novelty attribute to gains for drugs without that attribute using Mann-Whitney U and Kolmogorov-Smirnov tests.

RESULTS: We included 660 studies pertaining to 345 drug-indication pairs. Drug-indication pairs intended to treat a serious condition had median gains of 0.27 QALY versus 0.02 QALY (p<0.01) for pairs without that attribute. Pairs offering improvement over existing treatments had median gains of 0.28 QALY versus 0.02 QALY (p<0.01) for pairs without that attribute. Pairs with the potential to address unmet clinical needs had median gains of 0.35 QALY versus 0.04 (p<0.01) QALY for pairs without that attribute.

CONCLUSIONS: Drugs that have novelty attributes - (1) treats a serious condition (2) offers improvement over existing treatments, and (3) addresses unmet clinical needs - tend to be associated with larger health gains than drugs without those attributes.