What Might Have Happened: The Impact of Interrupting Entry of Innovative Drugs on Disease Outcomes in the United States

A new NPC study shows how major drug innovations significantly improved patient health outcomes for six diseases with substantial mortality or morbidity.

Publication: Expert Review of Pharmacoeconomics & Outcomes Research, February 21, 2022

Authors: William Herring, Michael Ciarametaro, Josephine Mauskopf, David Wamble, Brian Sils & Robert Dubois

In the past 30 years, major drug innovations significantly improved patient health outcomes for six diseases with substantial mortality or morbidity, according to new research from the National Pharmaceutical Council (NPC) and RTI Health Solutions.

Bolstering the evidence base linking innovative pharmaceuticals to health improvements in the United States, this study shows how major drug innovations changed the trajectories of disease outcomes between 1990 and 2017 for individuals with ischemic heart disease, lung cancer, breast cancer, human immunodeficiency virus infection, type 2 diabetes mellitus, and rheumatoid arthritis.

What Might Have Happened

Rheumatoid Arthritis
(Note: Because there is limited condition-related mortality associated with RA, the study looked at the Health Assessment Questionnaire score as a measure of functionality)

  • 11.5% improvement in functional status per patient
  • 50,600 quality-adjusted life-years (QALYs) gained in the U.S.

Research Discussion

The authors noted this research has important implications for policymakers considering the implementation of price controls for drugs as a tool to reduce rising U.S. health care spending. The authors explained that improvements in patient health for these six conditions were not necessarily predictable during the development and launch of the new innovations. Thus, price control policies that do not account for this inherent uncertainty and arbitrarily restrict prices without consideration of value risk disincentivizing future biopharmaceutical innovation, resulting in fewer new drugs coming to market and potentially missing the next life-changing drug that would have significantly improved patient health.

The authors recommend that policymakers recognize and reward the value of pharmaceutical innovation in improving patient health while simultaneously incentivizing investment in future innovation. Policymakers concerned about health spending should also broaden their focus to consider spending on all health care services, not just drugs, in order to increase in health system efficiency.


This study focused on six medical conditions with significant mortality or morbidity (as reflected in the Global Burden of Disease (GBD) database) for which there have been major medical innovations over the past 30 years. Specific pharmaceutical or other innovative medical treatments introduced between 1990 and 2017 that had major impacts on patient health were identified from physician interviews and targeted literature reviews. Researchers reviewed observational database studies of the changes in disease outcomes over time and performed a counterfactual time series analysis of U.S. data from either the GBD database or a patient registry to provide estimates of the impact of the identified medical innovations on disease outcomes.

Drugs in question mark shape

Read the Blog

Dive deeper into the disease-specific impacts of major drug innovations on health outcomes for individuals with six serious conditions examined in the NPC/RTI-HS study.

Read more.