Problem Statement

Half of all Americans live with at least one chronic disease1, such as heart disease, cancer, stroke, or diabetes. These and other chronic diseases are the leading cause of death and disability in the United States and are a leading driver of health care costs, with approximately 90% of healthcare expenditures related to chronic conditions2,3. Yet investment in these leading causes of death and disability, other than cancer, has not reflected this burden. Published data from BIO has shown that venture investment for drug development in areas such as psychiatric disorders, diabetes, and cardiovascular and respiratory diseases has declined over the last decade relative to the prevalence and health care cost of these diseases4. A case series on hospitalized COVID-19 patients in the New York City area showed that the most common comorbidities were hypertension (56.6% of hospitalized patients studied), obesity (41.7%), and diabetes (33.8%)5.

The goal of this action collaborative, an ad hoc activity associated with the National Academies Forum on Drug Discovery, Development, and Translation, is to examine and highlight the bottlenecks to drug research and development (R&D) for highly prevalent chronic diseases and develop a set of key considerations and potential strategies for spurring innovation.

Objectives

With the participation of stakeholders, including patients, providers, and those involved in the development of drugs and/or biologics to address prevalent chronic diseases, this collaborative aims to:

• Articulate/highlight the unique cross-cutting challenges for spurring early stage R&D innovation for highly prevalent chronic diseases (e.g., do we have promising targets?)
• Consider whether incentives are in alignment for spurring the type of product development that will address unmet need when it comes to highly prevalent chronic diseases (e.g., do we have the right business models in place?)
• Consider lessons learned from other disease areas (e.g., rare diseases) and/or use cases that could have cross-cutting applications for highly prevalent chronic diseases.
• Develop a set of key considerations and/or strategies to spur drug R&D innovation for highly prevalent chronic diseases (i.e., determine whether there are promising avenues forward that merit additional time/effort/funding/attention).

Workplan

With the participation of stakeholders involved in clinical trials, this collaborative aims to:

1. Define Collaborative objectives, recruit participants, and convene initial Collaborative meeting. [complete]
2. Develop and refine a structured, qualitative questionnaire; identify key experts; and disseminate questionnaire to key experts. [complete]
3. Synthesize findings and identify key themes from questionnaire responses. [ongoing]
4. Convene Collaborative members and key experts to discuss and refine the results. [2022]

The collaborative is an ad hoc activity associated with the Forum on Drug Discovery, Development, and Translation (the Forum) at the National Academies of Sciences, Engineering, and Medicine (the National Academies). The work of the collaborative does not necessarily represent the views of any one organization, the Forum, or the National Academies and is not subjected to the review procedures of, nor is it a report or product of, the National Academies.

Footnotes

1. Broadly defined by the National Center for Chronic Disease Prevention and Health Promotion as “conditions that last 1 year or more and require ongoing medical attention or limit activities of daily living or both.” For more information, see https://www.cdc.gov/chronicdisease/about/index.htm (accessed January 1, 25, 2021).
2. For more information, see National Center for Chronic Disease Prevention and Health Promotion (accessed January 25, 2021).
3. Buttorff, Ruder, and Bauman, Multiple Chronic Conditions in the United States, RAND, 2017. For more information, see https://www.rand.org/content/dam/rand/pubs/tools/TL200/TL221/RAND_TL221.pdf (accessed January 25, 2021).
4. Thomas and Wessel, The State of Innovation in Highly Prevalent Chronic Diseases Volume II: Pain and Addiction Therapeutics. BIO, 2018.
5. Richardson et al, Presenting Characteristics, Comorbidities, and Outcomes Among 5700 Patients Hospitalized With COVID-19 in the New York City Area. JAMA, 2020.
6. For more information, see https://www.cdc.gov/nchs/fastats/default.htm (accessed Janaury 21, 2021).