Bill Overview
Title: Helping Experts Accelerate Rare Treatments Act of 2022
Description: This bill modifies provisions relating to the approval of drugs for rare diseases and conditions, including by requiring regular reporting on the number and types of applications received and the extent to which external experts are consulted during the review process.
Sponsors: Sen. Casey, Robert P., Jr. [D-PA]
Target Audience
Population: Individuals with rare diseases or conditions globally
Estimated Size: 30000000
- The bill targets the approval processes for drugs designed to treat rare diseases and conditions.
- Rare diseases are conditions that affect a small percentage of the population, often with less than 200,000 people in the United States.
- There are over 7,000 rare diseases, collectively affecting an estimated 300 million people globally.
- The bill includes measures to involve external experts in the review process, enhancing the expertise applied to the approval of these treatments.
- The enhanced approval process could potentially speed up access to new treatments for individuals with rare diseases.
Reasoning
- The bill aims to accelerate the approval process for drugs treating rare diseases, which means individuals with such conditions could have quicker access to necessary treatments, potentially improving their quality of life.
- The financial limitations of the bill suggest not all who might benefit will receive new drugs immediately, hence impact might scale depending on the disease prevalence.
- Not everyone in the target population will be affected immediately; the policy primarily benefits those who have active drug applications during the policy's ten-year span.
- Consulting external experts may improve the accuracy of drug approval, thus reducing potential adverse drug reactions or misclassifications.
- Wellbeing improvements might not appear immediately; many of these drugs can take years to develop and review, and benefits could increase incrementally over time.
Simulated Interviews
Marketing Specialist (New York, NY)
Age: 35 | Gender: female
Wellbeing Before Policy: 5
Duration of Impact: 10.0 years
Commonness: 6/20
Statement of Opinion:
- I think this policy is a good step forward. Knowing that there's a push to get rare disease treatments approved faster gives me hope.
- Involving experts in the process seems like it will improve the quality of decisions made on whether a drug should be approved or not.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 5 |
| Year 2 | 6 | 5 |
| Year 3 | 7 | 5 |
| Year 5 | 7 | 5 |
| Year 10 | 8 | 6 |
| Year 20 | 8 | 6 |
Software Engineer (Austin, TX)
Age: 50 | Gender: male
Wellbeing Before Policy: 4
Duration of Impact: 8.0 years
Commonness: 4/20
Statement of Opinion:
- This policy could make a huge difference for people like me who are waiting for new treatment options.
- I'm cautiously optimistic but worried about budget constraints limiting how soon or whether I see new treatments.
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 | 8 | 4 |
| Year 10 | 9 | 5 |
| Year 20 | 9 | 5 |
Graduate Student in Biology (Salt Lake City, UT)
Age: 28 | Gender: female
Wellbeing Before Policy: 6
Duration of Impact: 10.0 years
Commonness: 3/20
Statement of Opinion:
- I believe the enhanced focus on rare diseases will lead to groundbreaking treatments.
- I'm hopeful but realize the timeline means benefits won't be immediate.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 6 |
| Year 2 | 6 | 6 |
| Year 3 | 6 | 6 |
| Year 5 | 7 | 6 |
| Year 10 | 8 | 7 |
| Year 20 | 9 | 8 |
Retired Teacher (Miami, FL)
Age: 60 | Gender: male
Wellbeing Before Policy: 5
Duration of Impact: 5.0 years
Commonness: 7/20
Statement of Opinion:
- The policy might help younger patients more than those who are already advanced in their conditions.
- I'm worried that the cost and time might limit tangible benefits within my lifetime.
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 | 5 |
| Year 10 | 6 | 5 |
| Year 20 | 6 | 5 |
College Student (Raleigh, NC)
Age: 22 | Gender: female
Wellbeing Before Policy: 4
Duration of Impact: 12.0 years
Commonness: 3/20
Statement of Opinion:
- I've seen treatments for my condition get stuck in the approval process, so this policy gives me hope.
- Involving more experts might make the approval process more rigorous and balanced.
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 | 8 | 4 |
| Year 10 | 9 | 5 |
| Year 20 | 9 | 5 |
Research Scientist (Chicago, IL)
Age: 40 | Gender: male
Wellbeing Before Policy: 7
Duration of Impact: 15.0 years
Commonness: 5/20
Statement of Opinion:
- This is a necessary move to build the pipeline of available treatments for rare diseases.
- The involvement of outside experts is a promising development for generating creative solutions.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 7 |
| Year 2 | 7 | 7 |
| Year 3 | 7 | 7 |
| Year 5 | 8 | 7 |
| Year 10 | 8 | 7 |
| Year 20 | 9 | 8 |
Retired Nurse (Seattle, WA)
Age: 70 | Gender: female
Wellbeing Before Policy: 6
Duration of Impact: 5.0 years
Commonness: 5/20
Statement of Opinion:
- I am skeptical about how much the policy will change for patients who need help urgently.
- This could be beneficial for future generations, but today's patients may not feel much impact.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 6 |
| Year 2 | 6 | 6 |
| Year 3 | 6 | 6 |
| Year 5 | 6 | 6 |
| Year 10 | 7 | 6 |
| Year 20 | 7 | 7 |
Pharmaceutical Consultant (Denver, CO)
Age: 48 | Gender: female
Wellbeing Before Policy: 7
Duration of Impact: 15.0 years
Commonness: 4/20
Statement of Opinion:
- This policy is a critical advancement that could make pharmaceutical approval more efficient.
- It will help streamline the process and might reduce wait times for families in need.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 8 | 7 |
| Year 2 | 8 | 7 |
| Year 3 | 8 | 7 |
| Year 5 | 9 | 7 |
| Year 10 | 9 | 8 |
| Year 20 | 10 | 9 |
Attorney (Portland, OR)
Age: 55 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 8.0 years
Commonness: 6/20
Statement of Opinion:
- While this policy has potential, I worry about its actual implementation amidst budget constraints.
- Streamlining approvals for rare drugs has been needed for a long time.
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 | 8 | 7 |
| Year 20 | 8 | 7 |
Artist (Los Angeles, CA)
Age: 30 | Gender: other
Wellbeing Before Policy: 5
Duration of Impact: 10.0 years
Commonness: 3/20
Statement of Opinion:
- The notion of accelerated approvals for rare diseases is both exciting and necessary.
- I hope this means less delay in gaining access to therapies.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 5 |
| Year 2 | 6 | 5 |
| Year 3 | 7 | 5 |
| Year 5 | 8 | 6 |
| Year 10 | 9 | 6 |
| Year 20 | 9 | 6 |
Cost Estimates
Year 1: $5000000 (Low: $4000000, High: $7000000)
Year 2: $4800000 (Low: $3500000, High: $6800000)
Year 3: $4600000 (Low: $3400000, High: $6500000)
Year 5: $4500000 (Low: $3000000, High: $6000000)
Year 10: $4200000 (Low: $2500000, High: $5500000)
Year 100: $4200000 (Low: $2500000, High: $5500000)
Key Considerations
- This bill focuses primarily on regulatory and administrative adjustments which may have varying impacts on different stakeholders.
- Diverse engagement of external experts can bring about nuanced benefits in terms of regulatory expediency and quality control.
- Efficacy in implementation will pivot on inter-agency coordination and support from the healthcare sector.