Half of all Americans live with at least one chronic disease, such as heart disease, cancer, stroke, or diabetes. Chronic diseases—which are, broadly defined, health conditions that persist for 1 year or longer, that typically require ongoing medical attention, and that can interfere with the activities of daily living (CDC, 2021b)—are a major health concern in the United States and around the world. Chronic diseases are the leading cause of death and disability in the United States and are a leading driver of health care costs (CDC, 2021a).
Despite the human and financial costs of chronic diseases, investment in research and development (R&D) for the medical treatment of these conditions—other than cancer—has not kept pace with the rise in prevalence of chronic diseases in the American population. According to recent reports from the Congressional Budget Office and the Biotechnology Innovation Organization (BIO), a trade association that represents biotechnology companies and related organizations, venture investments (i.e., early stage funding) and innovation for drug development in several highly prevalent chronic diseases—depression, chronic pain conditions, addiction, type 2 diabetes, obesity, Alzheimer’s disease (AD), and heart failure—have declined over the past decade relative to the prevalence and health care costs of these diseases (CBO, 2021; Thomas and Wessel, 2018a,b, 2019a,b, 2020). The recent outbreak of coronavirus disease 2019 (COVID-19) may further exacerbate the poor health outcomes associated with highly prevalent chronic diseases. A case series on hospitalized COVID-19 patients in the New York City area showed that the most common comorbidities were
hypertension, obesity, and diabetes—three examples of prevalent chronic diseases and conditions (Richardson et al., 2020).
On February 22, March 2, and March 8, 2021, the Forum on Drug Discovery, Development, and Translation of the National Academies of Sciences, Engineering, and Medicine (the National Academies) hosted a three-part public workshop1 titled Innovation in Drug Research and Development for Prevalent Chronic Diseases. The workshop was designed to examine the unique cross-cutting challenges to increased investment in drug R&D for highly prevalent chronic diseases and to highlight opportunities to encourage innovation in this area. Specifically, the workshop’s planning committee featured invited presentations and discussions in accordance with the Statement of Task (see Box 1-1). While non-pharmacological interventions—such as behavioral or environmental changes (Schmidt, 2016)—that can help treat and prevent prevalent chronic diseases also require additional research investment (IOM, 2011; NASEM, 2021), those interventions were beyond the scope of this workshop.
The organization of the proceedings groups the presentations of individual speakers into chapters according to the key themes that were discussed by workshop participants. Chapter 2 focuses on approaches for enabling more inclusive patient-centered clinical trial participation, and examines the value of incorporating patient input throughout the design and implementation of clinical trials. Chapter 3 explores practical and ethical considerations when using digital health technologies to better understand and treat prevalent chronic diseases. Chapter 4 examines some barriers to investment in drug R&D for certain prevalent chronic diseases, while Chapter 5 lays out some examples of success in drug R&D innovation for prevalent chronic diseases. Chapter 6 focuses on approaches and lessons learned that could be applied to improve and speed up the development of treatments for prevalent chronic diseases. Lastly, Chapter 7 recounts some of the major themes and points of emphasis that arose throughout the workshop discussions.
1 This workshop was organized by an independent planning committee whose role was limited to the identification of topics and speakers. This Proceedings of a Workshop was prepared by the rapporteurs as a factual summary of the presentations and discussion that took place at the workshop. Statements, recommendations, and opinions expressed are those of individual presenters and participants and are not endorsed or verified by the National Academies of Sciences, Engineering, and Medicine, and they should not be construed as reflecting any group consensus. The workshop agenda and workshop speaker biographical sketches can be found in Appendixes A and B, respectively.
CBO (Congressional Budget Office). 2021. Research and development in the pharmaceutical industry. https://www.cbo.gov/publication/57126 (accessed August 17, 2021).
CDC (Centers for Disease Control and Prevention). 2021a. Health and economic costs of chronic diseases. https://www.cdc.gov/chronicdisease/about/costs/index.htm (accessed June 3, 2021).
CDC. 2021b. About chronic diseases. https://www.cdc.gov/chronicdisease/about/index.htm (accessed June 3, 2021).
IOM (Institute of Medicine). 2011. Relieving pain in America: A blueprint for transforming prevention, care, education, and research. Washington, DC: The National Academies Press.
NASEM (National Academies of Sciences, Engineering, and Medicine). 2021. Meeting the challenge of caring for persons living with dementia and their care partners and caregivers: A way forward. Washington, DC: The National Academies Press.
Richardson, S., J. S. Hirsch, M. Narasimhan, J. M. Crawford, T. McGinn, K. W. Davidson, and the Northwell COVID-19 Research Consortium. 2020. Presenting characteristics, comorbidities, and outcomes among 5700 patients hospitalized with COVID-19 in the New York City area. Journal of the American Medical Association 323(20):2052–2059.
Schmidt, H. 2016. Chapter 5: Chronic disease prevention and health promotion. In Public health ethics: Cases spanning the globe, edited by D. H. Barret, L. W. Ortmann, A. Dawson, C. Saenz, A. Reis, and G. Bolan. New York: Springer.
Thomas, D., and C. Wessel. 2018a. The state of innovation in highly prevalent chronic diseases. Vol. I: Depression therapeutics. https://www.bio.org/sites/default/files/legacy/bioorg/docs/BIO_HPCD_Series-Depression_2018-01-03.pdf (accessed July 14, 2021).
Thomas, D., and C. Wessel. 2018b. The state of innovation in highly prevalent chronic diseases. Vol. II: Pain and addiction therapeutics. https://www.bio.org/sites/default/files/legacy/bioorg/docs/BIO_HPCP_Series-Pain_Addiction_2018-02-08.pdf (accessed June 10, 2021).
Thomas, D., and C. Wessel. 2019a. The state of innovation in highly prevalent chronic diseases. Vol. III: Type 2 diabetes and obesity therapeutics. http://go.bio.org/rs/490-EHZ-999/images/BIO%20HPCD%20Vol%20III%20Type%202%20Diabetes%20and%20Obesity%20Report.pdf?_ga=2.74929237.659494125.1626295106-803201671.1626295106 (accessed July 14, 2021).
Thomas, D., and C. Wessel. 2019b. The state of innovation in highly prevalent chronic diseases. Vol. IV: Alzheimer’s disease therapeutics. http://go.bio.org/rs/490-EHZ-999/images/BIO_HPCD4_ALZHEIMERS.pdf (accessed July 14, 2021).
Thomas, D., and C. Wessel. 2020. The state of innovation in highly prevalent chronic diseases. Vol. V: Systemic hypertension and heart failure. https://go.bio.org/rs/490EHZ-999/images/HPCD_Series_Vol_V_Hypertension_and_Heart_Failure.pdf?_ga=2.82819537.659494125.1626295106-803201671.1626295106 (accessed July 14, 2021).