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From page 17... ...
Furthermore, how to fairly allocate resources for drug development among potential beneficiaries involves ethical considerations and value judgments, about which there is not universal agreement and for which a multitude of defensible approaches exist. Because resources are limited and must be balanced with development in other sectors of society, and because public and private actors have different priorities, risk tolerance, and responsibilities, fairly allocating resources for drug development is a critical issue with multiple potential approaches (Millum, 2024)
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From page 18... ...
In addition, the development of "me-too" or follow-on drugs, though not innovative, can increase competition and lower prices, or expand indications to new patient populations. However, there is a role for both innovation and incremental improvements in therapeutic developments that address unmet need.
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From page 19... ...
The drug then undergoes a series of clinical trials to determine safety and efficacy, while in parallel the manufacturing process is refined and improved for commercialization. Phase I through phase III clinical trials involve progressively larger patient populations and become increasingly expensive and time consuming, requiring substantial capital investment primarily from venture capital firms or established pharmaceutical companies.
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Therefore, with support from Gates Ventures and the Peterson Center on Healthcare, the National Academies of Sciences, Engineering, and Medicine (the National Academies) formed the Committee on Strategies to Better Align Investments in Innovations for Therapeutic Development with Disease Burden and Unmet Needs.
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From page 21... ...
. The committee will recommend strategies to spur and facilitate increased innovation to address unmet needs and reduce health disparities.
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disease burden and public and private innovation in therapeutic development, with consideration of topics such as: a. The portion of the disease burden for which the standard of care is inadequate and for which outcomes could potentially be improved by innovative therapies b.
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Potential new policies to ensure equitable patient access to innovative therapies that effectively address unmet needs can make it to market, and that resources are not being spent on projects that are expected to have little benefit in terms of saving lives and improving health. The marginal cost part of this equation is relatively straightforward, although it is challenging to get such data in practice, as one can theoretically approach cost as the sum of private and public investment.
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no existing clinically effective therapeutic treatment options, (2) exist ing therapeutic solutions with limited effectiveness, or (3)
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Therefore, to the extent that drug pricing and access affect innovation, the committee considered this to be within its scope. On the other hand, while the committee recognizes that unmet need is often driven by broader social, economic, and behavioral determinants that influence patient access and the appropriate use of existing therapeutics, an adequate assessment of the policies and practices that address these determinants was beyond the scope of this report, as indicated in the statement of task.
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Even though preventatives, medical devices, and such treatments as physical or behavioral therapy were determined to be out of scope for this report, they are often intertwined with therapeutic innovation and it is important to recognize that pharmacological therapies are not the only, or necessarily the best, way to address disease burden or unmet medical need in many cases. Committee Approach The Committee on Strategies to Better Align Investments in Innovations for Therapeutic Development with Disease Burden and Unmet Needs consisted of 16 members with a broad range of expertise, including health economics, data science, epidemiology, health policy, biomedical/ pharmaceutical sciences, therapeutic development (including those with experience in the biopharmaceutical industry and the investment community)
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From page 27... ...
. Cutting government funding for research will impede innovation, limit opportunities for private entities that rely on publicly funded research, and ultimately stall economic activity.
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From page 28... ...
2022. Improving representation in clinical trials and research: Building research equity for women and underrepresented groups.
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