What If Groundbreaking Medicines Never Existed?

Human immunodeficiency virus (HIV) infections killed more than 50,000 Americans in 1995, the peak of the HIV-AIDS epidemic in the United States. The next year, a new treatment regimen called highly active antiretroviral therapy (HAART) became the new standard of care for HIV, triggering a rapid decline in deaths. By 2019, the number of HIV-related deaths in the U.S. had dropped to 5,000 and the number of new infections had substantially decreased. Because of innovative medicines, HIV is no longer a death sentence but a manageable chronic disease.  

What would have happened if these groundbreaking medicines never made it to patients? New research from the National Pharmaceutical Council (NPC) and RTI Health Solutions (RTI-HS) estimates what health gains might have been lost without the introduction of influential innovations for six serious diseases: ischemic heart disease, lung cancer, breast cancer, HIV infection, type 2 diabetes, and rheumatoid arthritis (RA). The study found that health outcomes between 1990 and 2017 for individuals with these conditions would have been significantly worse without major drug innovations. 

Read further down for a more in-depth look at each disease state studied. 

“This research has important implications for the ongoing U.S. health care spending debate,” said NPC Vice President of Research Michael Ciarametaro, MBA. “As policymakers consider proposals intended to reduce drug spending, they should think about the potential impact on biopharmaceutical innovation. Disincentivizing innovation could result in fewer potentially life-changing drugs, such as those in this study, which bring tremendous value to patients’ lives.” 

Recognizing the Value of Medicines 

Many studies have demonstrated the positive impact of innovation on patient health. A previous study by NPC and RTI-HS found that increased annual medical spending on five of these same diseases (RA was not studied) between 1995 and 2015 was a good investment that led to improved patient health outcomes. Another study attributed 35% of the 3.3 years gain in life expectancy in the U.S. from 1990 to 2015 to pharmaceuticals. Research has also shown the number of new drug approvals in certain disease areas had a major impact on patient health outcomes and population life expectancy. 

“When considering new health care spending policies, decision-makers need to recognize that medicines have significantly extended and improved life for patients across many conditions,” said lead author William Herring, PhD, senior director of health economics at RTI-HS. 

The Congressional Budget Office (CBO) projected that proposed Medicare price controls will reduce the number of new drugs coming to the market by 10%. Which drugs will not be available to patients if these policies are enacted and what health gains will never be realized as a result?  

“The pathway for developing a novel drug and getting it approved is long and complex, and it’s impossible to know in the early stages of that process which potential treatment is going to have the biggest impact on patient health,” said study author Josephine Mauskopf, PhD, vice president of health economics at RTI-HS. “That’s why it’s important we continue to incentivize innovation by recognizing the risk inherent in this process and appropriately rewarding the development of life-changing medicines.” 

The authors concluded that policies intended to rein in U.S. health care spending should be broadened to include all health care services, not just pharmaceuticals, and focus on rewarding and expanding use of high-value care that improves patient health and lowers overall health care spending.  

A Deeper Dive Into Disease-Specific Health Impacts 

Below we outline the impact of major drug innovations on health outcomes between 1990 and 2017 for individuals with six serious conditions examined in the NPC/RTI-HS study.

HIV Infection   

The introduction of HAART in 1996, fixed-dose combination therapies in 1997, and integrase inhibitors in 2007 led to an 85% decrease in annual mortality rates for HIV infection, with roughly two-thirds of that decline occurring by 1998-1999. The NPC/RTI-HS research estimated that without these innovations, years of life lost per patient would have been 660.6% higher. This translates to 1.96 million total life years gained in the U.S. over the study period.  

Ischemic Heart Disease  

While heart disease remains one of the top causes of death in the United States, the mortality rate dropped 64% from 1990 to 2018. This decline is due in part to the introduction of innovations such as thrombolytic therapy in 1996, antiplatelet therapy in 1997, and drug-eluting stents in 2003. The NPC/RTI-HS research estimated that without these innovations, years of life lost per patient would have been 18% higher. This translates to 1.27 million total life years gained in the U.S. over the study period. 

Lung Cancer  

Lung cancer is the leading cause of cancer deaths in the U.S. However, the annual mortality rate dropped 41% from 1990 to 2018. The five-year relative survival rate for newly diagnosed lung cancer increased from 14.6% between 1996-1998 to 20.6% between 2009-2015. The introduction of innovations such as chemotherapies in 1995 and epidermal growth factor receptor inhibitors in 2003 contributed to this improvement. The NPC/RTI-HS research estimated that without these innovations, years of life lost per patient would have been 27.1% higher. This translates to 951,000 total life years gained in the U.S. over the study period.  

Breast Cancer  

The current estimated five-year survival rate for a woman with breast cancer is 90.3%, up from 84.7% in 1990, and the mortality rate dropped 41% from 1990 to 2018. Innovations including chemotherapy in 1994, aromatase inhibitors in 1995, and human epidermal growth factor receptor 2 inhibitors in 1998 have all played important roles in these improvements. The NPC/RTI-HS research estimated that without these innovations, years of life lost per patient would have been 17.1% higher. This translates to 182,000 total life years gained in the U.S. over the study period.  

Type 2 Diabetes   

The definition of diabetes has evolved over the past few decades, making diabetes-related deaths difficult to accurately calculate. However, data show the incidence of diabetes-related end-stage renal disease increased by an average of 5.8% per year from 1990 to 1996 and then decreased by an average of 2.8% to 2.9% per year from 1996 to 2014. Major medical innovations for diabetes included metformin in 1994, thiazolidinediones in 1996, and glucagon-like peptide-1 receptor agonists in 2005. The NPC/RTI-HS research estimated that without these innovations, years of life lost per patient would have been 78.2% higher. This translates to 1.13 million total life years gained in the U.S. over the study period.  

Rheumatoid Arthritis  

Because there is limited condition-related mortality associated with RA, the NPC-RTI study measured patient health improvements using scores from the Health Assessment Questionnaire, a commonly used tool for assessing functional ability in RA. Tumor necrosis factor inhibitor therapy, introduced in 1998, was identified as the medical innovation for RA. The NPC/RTI-HS research indicated that without this innovation, patients’ functional status would have been 11.5% worse. This translates to 50,600 quality-adjusted life-years (QALYs) gained in the U.S. over the study period.