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From page 59... ...
disease burden and investment. Several groups have attempted to study the mismatch between disease burden and investment in therapeutic development and to develop a list of areas where future investments are needed, and the following sections provide an overview of some of those efforts.
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PUBLIC INVESTMENT IN THERAPEUTIC DEVELOPMENT As described in Chapter 2, the public sector, through federal a gencies such as NIH, has contributed significantly to drug development, particularly through a focus on basic science and discovery research (see Chapter 2, section "Investment in Therapeutic Innovation")
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From page 61... ...
. The dominant paradigm in these assessments is to compare total DALYs versus NIH funding to identify diseases that have disproportionately low investment.
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Orange circles indicate relatively proportional funding to disease burden. CKD = chronic kidney disease; CNS = central nervous system; COPD = chronic obstructive pulmonary disease; HHD = hypertensive heart disease; IBD = inflammatory bowel disease; MS = multiple sclerosis; PD = Parkinson's disease; RA = rheumatoid arthritis; SIDS = sudden infant death syndrome.
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Emphasizing the need for disaggregated data, the ratio of NIH funding relative to disease burden differs for different types of cancer. For example, brain and central nervous system cancers have higher investment than liver or esophageal cancers, despite comparable rates of disease burden.
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From page 64... ...
In 2021, for example, BARDA had a budget of $1.4 billion to advance medical countermeasures for responding to health threats.3 In addition, analyzing NIH funding relates to only part of the product development pipeline and omits information about private-sector investment. Analyzing DALYs versus NIH funding also does not account for unmet need and innovation, key components of this committee's statement of task.
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From page 65... ...
. NIH funding is primarily used to support public goods such as understanding the biological mechanisms of disease (Galkina Cleary et al., 2018)
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Oncology includes supportive care. Neurology includes central nervous system disorder treatments and mental health treatments but does not include pain management or anesthesia.
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The report examined postapproval expansions, which typically extend drug indications to additional patient populations or conditions, from 2000 to 2023 and analyzed early data on the effects of the IRA's Medicare Drug Price Negotiation Program. The study found that postapproval expansions are widespread -- approximately 50 percent of drugs received at least one postinitial approval expansion, and the pattern is relatively consistent across small molecules (52 percent)
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From page 68... ...
. Nonetheless, because clinical trials are lengthy and expensive, if new indications are identified closer to the negotiation deadline, sponsors may decide that investment is not worth it, especially in the context of some rare diseases, leaving potentially beneficial indications undeveloped.
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DALYs that each of 22 disease categories contributes. The top bar provides a comparison to each of the other bars, which show the proportion of products in the development pipeline from preclinical research through phase III across all 6,576 pharmaceutical companies analyzed.
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SOURCE: Adapted from IHME data, 2025.
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While innovative therapies could be helpful, innovation may not be the primary solution if access and underuse of existing therapeutics remain problems. As noted previously with the NIH funding assessment, aggregate disease categories could obscure what is happening for specific diseases.
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SOURCE: Adapted from IHME data.
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. Comparing Private and Public Investment Similar to the case with the NIH funding assessment, these data indicate substantial investment in HIV/AIDS and neurological disorders and underinvestment in cardiovascular disease relative to burden.
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. Considering p individual burden draws attention to the significant unmet medical need in rare diseases -- conditions that have low prevalence but high individual burden and for which there are often no effective treatments.
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Importantly, how a research priority setting exercise is conducted, and who is involved, will shape outcomes, which could have important ethical implications (Millum, 2024)
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In addition, conditions with the lowest innovation despite significant burden included such chronic conditions as chronic kidney disease, chronic obstructive pulmonary disease, and cirrhosis and other liver diseases. The analysis sought to bring together multiple metrics to assess not only burden but also health care cost, public investment, and private investment (Gressler et al., 2023)
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What is the current level of investment, and does it address the level of burden and unmet need? What are the barriers or challenges to innovation in therapeutic development for this therapeutic area (e.g., scientific challenges, market forces)
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also highlighted lower public investment in such conditions as headaches, neck and back pain, psoriasis, and gallbladder diseases. The sources on private-sector investment, though limited, indicate substantial investment in oncology and neurological disease and suggest underinvestment in cardiovascular disease and maternal and neonatal conditions.
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2018. Contribution of NIH funding to new drug approvals 2010-2016.
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2024. Ethics and health research priority setting: A narrative review [version 1; peer review: 2 approved]
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1996. Investing in health research and development: Report of the Ad Hoc Committee on Health Research Relating to Future Interven tion Options.
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