Bill Overview
Title: RARE Act of 2022
Description: The bill reauthorizes through FY2028 the National Institutes of Health Rare Disease Regional Centers of Excellence and expands activities to facilitate research and increase awareness of rare diseases and conditions. (Rare diseases and conditions are those with a prevalence of fewer than 200,000 individuals in the United States.) The Centers for Disease Control and Prevention may expand infrastructure and activities to track the epidemiology of up to four rare diseases and conditions and incorporate this information into a National Rare Disease or Condition Surveillance System. The Agency for Healthcare Research and Quality must expand its activities to increase health care providers' awareness of rare diseases and conditions. The National Academies of Sciences, Engineering, and Medicine, or another appropriate entity, must update the 2010 report Rare Diseases and Orphan Products: Accelerating Research and Development.
Sponsors: Rep. Carson, Andre [D-IN-7]
Target Audience
Population: People with rare diseases
Estimated Size: 28000000
- The bill targets rare diseases, which are defined as diseases affecting fewer than 200,000 people in the United States.
- There are approximately 7,000 different rare diseases affecting an estimated 25 million to 30 million Americans collectively.
- Globally, rare diseases affect around 300 million people, based on current estimates.
Reasoning
- The population of individuals with rare diseases in the United States is estimated to be between 25 million and 30 million. However, each individual rare disease affects fewer than 200,000 people, meaning the impact of increased research, surveillance, and awareness could vary widely depending on the particular disease.
- The policy is likely to have a more pronounced impact on individuals who have rare diseases that are prioritized in tracking and research efforts. Individuals with diseases not covered may see little to no direct impact initially.
- The commonness score for each simulated individual gives an idea of how widespread their situation is, reflecting the diversity within the category of rare diseases.
Simulated Interviews
student (Raleigh, NC)
Age: 8 | Gender: female
Wellbeing Before Policy: 6
Duration of Impact: 15.0 years
Commonness: 3/20
Statement of Opinion:
- The policy would help my doctors understand my condition better and could lead to improved treatments.
- Raising awareness might make it easier for my family to find specialists.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 6 |
| Year 2 | 7 | 6 |
| Year 3 | 8 | 6 |
| Year 5 | 8 | 6 |
| Year 10 | 9 | 5 |
| Year 20 | 9 | 5 |
software engineer (San Francisco, CA)
Age: 34 | Gender: male
Wellbeing Before Policy: 5
Duration of Impact: 10.0 years
Commonness: 4/20
Statement of Opinion:
- Increased research can potentially improve my quality of life through better understanding and treatment options.
- Awareness programs could reduce stigma and misunderstanding in professional and personal environments.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 5 |
| Year 2 | 7 | 5 |
| Year 3 | 7 | 5 |
| Year 5 | 8 | 5 |
| Year 10 | 8 | 4 |
| Year 20 | 7 | 4 |
retired nurse (Houston, TX)
Age: 50 | Gender: female
Wellbeing Before Policy: 7
Duration of Impact: 10.0 years
Commonness: 5/20
Statement of Opinion:
- This policy could advance understanding of auto-immune related rare diseases.
- It may also aid in the development of more precisely targeted therapies.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 7 |
| Year 2 | 8 | 7 |
| Year 3 | 8 | 7 |
| Year 5 | 8 | 7 |
| Year 10 | 9 | 6 |
| Year 20 | 8 | 6 |
retired factory worker (Des Moines, IA)
Age: 65 | Gender: male
Wellbeing Before Policy: 4
Duration of Impact: 5.0 years
Commonness: 2/20
Statement of Opinion:
- I hope this leads to better and more approved treatment paths.
- Any enhanced infrastructure for data collection is crucial for a condition like mine.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 5 | 4 |
| Year 2 | 6 | 4 |
| Year 3 | 7 | 4 |
| Year 5 | 7 | 4 |
| Year 10 | 7 | 3 |
| Year 20 | 6 | 2 |
college student (Boston, MA)
Age: 23 | Gender: female
Wellbeing Before Policy: 6
Duration of Impact: 20.0 years
Commonness: 1/20
Statement of Opinion:
- The increased awareness and research make me hopeful for break-throughs.
- Better training for physicians means fewer misdiagnoses.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 6 |
| Year 2 | 8 | 6 |
| Year 3 | 8 | 6 |
| Year 5 | 9 | 5 |
| Year 10 | 9 | 5 |
| Year 20 | 8 | 5 |
graphic designer (Miami, FL)
Age: 42 | Gender: other
Wellbeing Before Policy: 5
Duration of Impact: 10.0 years
Commonness: 2/20
Statement of Opinion:
- Research advancement is needed to improve treatment options.
- Hope that awareness initiatives will help mitigate the lengthy diagnostic odyssey.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 5 |
| Year 2 | 7 | 5 |
| Year 3 | 8 | 5 |
| Year 5 | 8 | 5 |
| Year 10 | 9 | 4 |
| Year 20 | 7 | 4 |
freelance writer (Seattle, WA)
Age: 29 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 5.0 years
Commonness: 8/20
Statement of Opinion:
- Policy efforts like this can help raise awareness and fuel necessary advancements.
- Crucial to counteract misinformation and assumptions about sleep disorders.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 6 |
| Year 2 | 7 | 6 |
| Year 3 | 7 | 6 |
| Year 5 | 8 | 6 |
| Year 10 | 7 | 5 |
| Year 20 | 6 | 5 |
retired librarian (Springfield, IL)
Age: 72 | Gender: female
Wellbeing Before Policy: 4
Duration of Impact: 5.0 years
Commonness: 3/20
Statement of Opinion:
- I hope that more focus will be given to multiple myeloma similarly.
- Support networks often lack information; hopefully, this will change.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 5 | 4 |
| Year 2 | 5 | 4 |
| Year 3 | 6 | 4 |
| Year 5 | 6 | 4 |
| Year 10 | 6 | 3 |
| Year 20 | 5 | 3 |
legal consultant (New York, NY)
Age: 55 | Gender: male
Wellbeing Before Policy: 7
Duration of Impact: 10.0 years
Commonness: 2/20
Statement of Opinion:
- Focus on research could help in earlier diagnosis and better management strategies.
- More clinical awareness can only be a positive for those diagnosed.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 7 |
| Year 2 | 7 | 7 |
| Year 3 | 8 | 7 |
| Year 5 | 9 | 6 |
| Year 10 | 9 | 5 |
| Year 20 | 8 | 5 |
small business owner (Denver, CO)
Age: 48 | Gender: female
Wellbeing Before Policy: 5
Duration of Impact: 5.0 years
Commonness: 4/20
Statement of Opinion:
- Good to know there's potential for more information sharing and support.
- Insurance hurdles require comprehensive groundwork and understanding.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 5 | 5 |
| Year 2 | 5 | 5 |
| Year 3 | 6 | 5 |
| Year 5 | 6 | 4 |
| Year 10 | 6 | 3 |
| Year 20 | 5 | 3 |
Cost Estimates
Year 1: $60000000 (Low: $50000000, High: $70000000)
Year 2: $60000000 (Low: $50000000, High: $70000000)
Year 3: $60000000 (Low: $50000000, High: $70000000)
Year 5: $60000000 (Low: $50000000, High: $70000000)
Year 10: $60000000 (Low: $50000000, High: $70000000)
Year 100: $60000000 (Low: $50000000, High: $70000000)
Key Considerations
- There is inherent uncertainty in cost estimates due to the innovativeness and complexity of rare disease research and tracking efforts.
- Population health benefits might not immediately reflect in healthcare cost reductions within the forecast period but would accrue over time.
- Collaboration across multiple agencies and stakeholders will be essential to achieve the desired impact and optimize resource allocation.