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