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From page 29... ...
, published soon after the release of the IOM report compared NIH funding across disease areas in 1996 against several measures of disease burden, including total mortality, years of life lost, disability-adjusted life-years (DALYs) , hospital days, incidence, and prevalence.
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The "years of life lost" metric compared with a reference life expectancy measure became the foundation for the concept of premature mortality that has remained a central component of disease burden measurement. A number of variants of the years-of-life-lost concept followed Dempsey's precedent (CDC, 1998)
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examined some of the key conceptual, empirical, and ethical issues around construction of summary measures of population health. Work at the World Health Organization during the 1990s and 2000s continued to develop frameworks and applications for use of DALYs and other summary population health measures to quantify disease burden at global and national scales (Murray et al., 2002)
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DALYs were developed as a measure of the population health effect caused by fatal and nonfatal health outcomes (Berkley et al., 1993)
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. Importantly, QALYs are based on average population outcomes, and as such, should be applied to treatments at a population level rather than at the individual patient level (Rand and Kesselheim, 2021)
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Rather, they are components of summary measures like DALYs and QALYs and provide an assessment of patients' condition and functional status. Since these measures do not capture burden over time or mortality risk, they are not candidates to serve as a summary measure of disease burden, but HRQoL can provide insight into key dimensions of burden at points in time.
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Some COI studies have focused on specific clusters of disease, such as cardiovascular disease, while others have been more general in scope. Guidelines for COI studies from NIH and the World Health Organization (WHO)
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See Box 2-1 for more discussion of patient engagement throughout the process of therapeutic development. Variations in Disease Burden Across Different Populations Variations in disease burden across populations have long been documented (HHS, 1985; IOM, 2003; NASEM, 2024)
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. To address these challenges, Marc Boutin, the global head of patient engagement at Novartis, cited the company's Five Decision Point patient engagement framework that spans the entire life cycle of medi cine development and incorporates patient insight early in therapeutic development (Patients as Partners, 2023)
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By engaging patients at the beginning of research through clinical development, Boutin cited faster drug development times that cut as much as 2.5–3.5 months off the clinical development timeline. In addi tion to saved time, early and consistent patient engagement generated $250 million in savings for the company and created a net present value between $850 million and $1.5 billion.
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. Beyond depression, several other mental health disorders are associated with an increased risk for a range of medical conditions (Rapsey et al., 2015; Scott et al., 2016)
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. For these reasons, attributing to depression all the excess increased risk burden of comorbid medical conditions observed in these epidemiological studies risks overestimating the global disease burden of depression.
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Because mental health disorders commonly have adverse effects on health behaviors including self-care, it is not surprising that many mental health disorders are associated with an increased risk of developing general medical conditions and poorer outcomes of existing persistent medical conditions. The possibility of bidirectional associations between two diseases complicates the attribution of disease burden to a single disease.
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Therefore, diabetes leads to additional comorbidities and these comorbidities also lead to adverse downstream health outcomes for patients. As another example of these complex relationships, one of the strongest recommendations for patients who develop these macrovascular complications is physical activity (which is well established to reduce the risk of acute myocardial infarction and stroke)
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This definition centers on the needs of patients and helps identify areas of therapeutic development to prioritize in order to address these unmet needs and to improve associated disease burden. FDA guidance similarly defines unmet medical need as "a condition whose treatment or diagnosis is not addressed adequately by available therapy" (FDA, 2014, p.
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NOTE: The committee considered a continuum of unmet need, categorized as a medical condition or disease for which there are (1) no existing effective therapeutic treatment options, (2)
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Major depressive disorder or s chizophrenia, for example, each have a large number of FDA-approved p harmacological treatments, but these medications yield only modest reductions in symptoms and functional improvement (Ormel et al., 2022)
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. While not entirely novel, these improvements demonstrate innovation and may make the drug better suited to the IPF patient population, potentially helping to reduce unmet need.
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The public sector, through federal agencies such as the NIH and the Department of Defense, is the largest funder of basic research,3 and the private sector primarily supports and engages in drug discovery and development activities, such as clinical testing, incremental innovation (Barbosu, 2025) , and product differentiation -- activities that largely follow from basic research (Congressional Budget Office, 2021; Simoens and Huys, 2022)
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For example, one study found that rather than "crowding out" private investment, each $10 million increase in NIH funding leads to 2.3 additional private-sector patents (Azoulay et al., 2019)
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Public investments help understand disease mechanisms that contribute to important novel drug targets and innovations from the private sector that can help reduce unmet needs. REFERENCES Alharbi, M
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2022. Depression increased risk of coronary heart disease: A meta-analysis of prospective cohort studies.
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From page 51... ...
2024. Global burden of 288 causes of death and life expectancy decomposition in 204 countries and territories and 811 subnational locations: A systematic analysis for the Global Burden of Disease study 2021.
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Nature Reviews Disease Primers 6(1)
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1998a. Scientific opportunities and public needs: Improving priority setting and public input at the National Institutes of Health.
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2002. Summary measures of population health: Concepts, ethics, measurement and applications.
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drug prices. JAMA Health Forum 5(11)
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2017. Physical activity and sedentary behavior in people with major depressive disorder: A systematic review and meta-analysis.
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2017. Leveraging health-related quality of life in population health management: The case for healthy days.
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