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Misalignment between U.S. disease burden and novel therapy innovation may contribute to an increase in U.S. disease prevalence, morbidity, and premature mortality, and may exacerbate existing health disparities. Closing existing gaps between clinical therapy innovation and unmet population health needs through public and private actions may help reduce health disparities, improve health outcomes, and lower societal costs.
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Consensus
·2025
The United States is a global leader in biomedical research, generating therapeutic breakthroughs that advance the health of the nation and the world. The public and private sectors contribute to this advancement by funding biomedical research and development. The current level of investment in phar...
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Description
An ad hoc committee of the National Academies of Sciences, Engineering, and Medicine will examine the current degree and patterns of alignment or mismatch between innovation in developing novel therapies and unmet needs associated with US disease burden (including high-impact, low-frequency diseases as well as highly prevalent conditions). The committee will recommend strategies to spur and facilitate increased innovation to address unmet needs and reduce health disparities. In addition to reviewing the published literature and publicly available information sources, the committee will identify and engage appropriate stakeholders, including relevant federal agencies (e.g., NIH, ARPA-H, FDA, CMS), the academic/professional community of researchers and clinicians, private industry, and patient/consumer groups, to gain their perspectives as input to committee deliberations. Based on the information gathered, the committee will identify the challenges, opportunities, and responsibilities in building both public and private capacity for innovation in therapeutic development and ensuring broad, equitable access to safe and effective novel therapies.
The committee will address three overarching questions:
1. How well or poorly is the current pipeline of public and private innovations and investments in developing new therapies aligned with the actual burden of illness, injury, and disability in the US?
2. What changes in policy, finance, regulation, and other influences would help achieve better alignment to address unmet needs and reduce health disparities?
3. Are there unmet needs that should be prioritized within the proposed changes?
Specifically, the committee will:
1. Describe the current US disease burden, with consideration of topics such as:
a. The total burden of illness, injury, and disability in the US by therapeutic area, including high-impact, low-frequency diseases as well as highly prevalent conditions
b. Disease areas with the greatest unmet need for effective therapies (excluding unmet need due to lack of access to existing therapies)
c. The impact of co-morbidities and downstream outcomes for unmet needs on health care spending (e.g., mental health conditions)
d. Variations in burden across different populations (e.g., age, race, gender, socioeconomic status, payer mix)
2. Characterize the degree and patterns of mismatch between US 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. Potential proxies for determining innovation efforts by disease area (e.g., clinical trials, total funding, novelty and effectiveness of new therapies)
c. The areas of mismatch for which remedy could have the greatest impact on US disease burden and health equity.
3. Describe the challenges in better aligning innovations in therapeutic development with disease burden and unmet needs, with consideration of topics such as:
a. Decision-making by for-profit and private sector developers for investments in innovation, including perceptions of potential risks and returns (e.g., drivers of variation that inform measures of investments across therapeutic areas / needs, such as Net Present Value [NPV])
b. The greatest challenges to overcome in the different stages of development for neglected disease areas (e.g., basic and translational science, clinical trials)
c. The role of public investment in biomedical research, and how that investment is leveraged in development of novel therapies
4. Propose strategies informed by population needs, society costs, and assessments of potential risks and returns to better align both public and private investments in innovations in therapeutic development with disease burden and unmet needs. The committee may make recommendations to a variety of actors, such as federal agencies and Congress, the pharmaceutical industry, and private philanthropy, with consideration of topics such as:
a. Ways to better utilize existing policies and programs to incentivize high-value innovation
b. Potential new policies to facilitate greater investment in innovation to address unmet need, as well as to discourage practices that impede innovation or competition
c. The potential role of public-private partnerships and pre-competitive collaboration
d. Potential innovations in regulatory science and practices
e. Lessons learned from rapid innovations during the COVID-19 pandemic
f. Potential new policies to ensure equitable patient access to innovative therapies that effectively address unmet needs
Contributors
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Conflict of Interest Disclosure
Disclosure of Unavoidable Conflicts of Interest Statement: Dr. Scott Howell
The conflict of interest policy of the National Academies of Sciences, Engineering, and Medicine (http://www.nationalacademies.org/coi) prohibits the appointment of an individual to a committee authoring a Consensus Study Report if the individual has a conflict of interest that is relevant to the task to be performed. An exception to this prohibition is permitted if the National Academies determines that the conflict is unavoidable and the conflict is publicly disclosed. A determination of a conflict of interest for an individual is not an assessment of that individual's actual behavior or character or ability to act objectively despite the conflicting interest.
Dr. Scott Howell has a conflict of interest in relation to service on the Committee on Strategies to Better Align Investments in Innovations for Therapeutic Development with Disease Burden and Unmet Needs based on his current role with Blue Line Advisors, LLC, through which he provides ad hoc consulting to companies such as Jazz Pharmaceuticals. He is co-founder of Synapse Sciences, an emerging global drug research network. Dr. Howell owns stock in Novartis and United Health Group.
The National Academies has concluded that for the committee to accomplish the tasks for which it was established, its membership must include at least one person with current expertise and experience in how strategic investments are made by large pharmaceutical companies. As described in his biographical summary, due to Dr. Howell’s experience in market access in the pharmaceutical industry, he understands the strategic investment landscape for commercializing innovative therapeutics to address unmet patient needs and making therapeutics deliverable to patients.
The National Academies has determined that the expertise and experience of Dr. Howell is needed for the committee to accomplish the task for which it has been established. The National Academies could not find another available individual with the equivalent expertise and experience who does not have a conflict of interest. Therefore, the National Academies has concluded that the conflict is unavoidable.
The National Academies believes that Dr. Howell can serve effectively as a member of the committee, and the committee can produce an objective report, taking into account the composition of the committee, the work to be performed, and the procedures to be followed in completing the study.
Disclosure of Unavoidable Conflicts of Interest Statement: Dr. Edith Perez
The conflict of interest policy of the National Academies of Sciences, Engineering, and Medicine (http://www.nationalacademies.org/coi) prohibits the appointment of an individual to a committee authoring a Consensus Study Report if the individual has a conflict of interest that is relevant to the task to be performed. An exception to this prohibition is permitted if the National Academies determines that the conflict is unavoidable and the conflict is publicly disclosed. A determination of a conflict of interest for an individual is not an assessment of that individual's actual behavior or character or ability to act objectively despite the conflicting interest.
Dr. Edith Perez has a conflict of interest in relation to service on Committee on Strategies to Better Align Investments in Innovations for Therapeutic Development with Disease Burden and Unmet Needs based on her role as Chief Medical Officer at Bolt Biotherapeutics, which develops therapeutics to treat numerous cancers. She is on the Board of Directors of Artiva Biotherapeutics, an early-stage immunotherapy company and is a member of the Genentech External Council for Advancing Inclusive Research. Her spouse has stock in Genentech.
The National Academies has concluded that for the committee to accomplish the tasks for which it was established, its membership must include at least one person who has relevant current expertise and experience in all phases of therapeutic development and the biopharmaceutical industry. As described in her biographical summary, due to her current role as Chief Medical Officer and her past roles as a physician, principal investigator, and Vice President and Head of BioOncology at Genentech, Dr. Perez has extensive expertise and experience in basic science research and clinical trials, and the biopharmaceutical industry launching novel therapies.
The National Academies has determined that the expertise and experience of Dr. Perez is needed for the committee to accomplish the task for which it has been established. The National Academies could not find another available individual with the equivalent expertise and experience who does not have a conflict of interest. Therefore, the National Academies has concluded that the conflict is unavoidable.
The National Academies believes that Dr. Perez can serve effectively as a member of the committee, and the committee can produce an objective report, taking into account the composition of the committee, the work to be performed, and the procedures to be followed in completing the study. **In July 2024, Dr. Perez’s role with Bolt Therapeutics changed from chief medical officer to advisor.
Disclosure of Unavoidable Conflicts of Interest Statement: Mr. David Scheer
The conflict of interest policy of the National Academies of Sciences, Engineering, and Medicine (http://www.nationalacademies.org/coi) prohibits the appointment of an individual to a committee authoring a Consensus Study Report if the individual has a conflict of interest that is relevant to the task to be performed. An exception to this prohibition is permitted if the National Academies determines that the conflict is unavoidable and the conflict is publicly disclosed. A determination of a conflict of interest for an individual is not an assessment of that individual's actual behavior or character or ability to act objectively despite the conflicting interest.
Mr. Scheer has a conflict of interest in relation to service on the Committee on Strategies to Better Align Investments in Innovations for Therapeutic Development with Disease Burden and Unmet Needs based on his current role as advisor for Nektar Therapeutics and Twist Biosciences. He is a board member of companies advancing novel therapeutics for unmet needs, including OrphAI Therapeutics and BiologicsMD. He is also co-chair of BioCT, a nonprofit focused on promoting growth of the life sciences in Connecticut.
The National Academies has concluded that for the committee to accomplish the tasks for which it was established, the committee requires expertise in financial investment, including knowledge about the intricacies of commercialization, investment, and sale of novel therapies. Inherent in this expertise is knowledge about the incentives and regulatory policies that influence innovation and the perceived risks and returns on investment of innovative products. As described in his biographical summary, Mr. Scheer has expertise and experience in financial investment and has deep understanding of the many factors that influence the investment, growth, and sale of companies that translate basic science into novel therapeutics.
The National Academies has determined that the expertise and experience of Mr. Scheer is needed for the committee to accomplish the task for which it has been established. The National Academies could not find another available individual with the equivalent expertise and experience who does not have a conflict of interest. Therefore, the National Academies has concluded that the conflict is unavoidable.
The National Academies believes that Mr. Scheer can serve effectively as a member of the committee, and the committee can produce an objective report, taking into account the composition of the committee, the work to be performed, and the procedures to be followed in completing the study.
Committee Membership Roster Comments
Edits were made to the bios of Marcarius M. Donneyong, Holly Fernandez Lynch, Edith A. Perez, Kathryn A. Phillips, Joshua Salomon, and David I. Scheer on 03/22/2024.
Edits were made to the unavoidable COI for Edith A. Perez on 03/22/2024.
Stacey Adam, Mark Olfson, and Wu Zeng were added on 04/22/2024.
Edits were made to the bio of Donald M. Berwick on 05/23/2024.
Edits were made to the bio for Edith A. Perez on 05/28/2024.
Edits were made to the bio for Edith A. Perez on 07/15/2024.
Edits were made to the unavoidable COI for Edith A. Perez on 07/15/2024.
Sponsors
Gates Ventures
Peterson Center on Healthcare
Staff
Alex Helman
Lead
Aja Drain
