OBJECTIVES:

Payer, regulators, and patients have demanded greater investment and approval of innovate treatments. This research aimed to evaluate approval and health economic assessment of innovative therapies.

METHODS:

We identified treatments approved by the FDA between 2011-2019. Innovative drugs were defined as those that demonstrate substantial improvement over existing therapies and/or rare disease/orphan designations. We then identified those that met these criteria and reviewed treatment assessments by the Institute for Clinical and Economic Review (ICER) group and the National Institute for Health and Care Excellence (NICE).

RESULTS:

There were 356 new FDA drug approvals of which approximately half (174) met the innovative treatment criteria.

ICER: A total of 24 (14%) innovative treatments were evaluated, 23 (96%) were deemed to have sufficient evidence supporting clinical benefit, only 1 (4%) treatment was considered to provide high-intermediate value for money, 15 (63%) were above the value-based pricing (VBP) range, and 6 (25%) received a budget impact alert.

NICE: A total of 61 innovative treatments were reviewed of which 35 (57%) received recommendation based on marketing authorization or clinical practice, 20 (33%) were recommended with restrictions and 6 (10%) were not recommended. The proportion of recommendations was found to be similar between innovative treatments and all treatments reviewed during same time period.

CONCLUSIONS:

While most innovative treatments provided sufficient clinical benefit, pricing was often considered above the VBP and budget impact thresholds by ICER; furthermore, innovative treatments based on the above definition, achieved average results in economic evaluations with NICE. Excessive downward pressure on prices of innovative treatments may discourage investment in and development of future therapies. Conversely, there is a risk that the current pace of innovation will overwhelm society’s ability to afford beneficial therapies. Objective approaches to achieve the optimal balance between the two is essential in the evaluation of innovative therapies.