Bill Overview
Title: To amend the Orphan Drug Act to reauthorize a program of grants and contracts for the development of drugs for rare diseases and conditions (commonly referred to as "orphan drugs").
Description: This bill reauthorizes through FY2027 the Food and Drug Administration's Orphan Products Grants Program, which supports clinical research and product development for rare diseases and conditions.
Sponsors: Rep. Bilirakis, Gus M. [R-FL-12]
Target Audience
Population: individuals suffering from rare diseases and conditions
Estimated Size: 30000000
- Rare diseases are defined in the United States as conditions affecting fewer than 200,000 people.
- There are over 7,000 known rare diseases globally.
- Approximately 30 million people in the United States are affected by a rare disease, according to the National Institutes of Health.
- Globally, an estimated 350 million people are affected by rare diseases.
- Orphan drugs are developed specifically to treat these rare diseases, which are often underserved by the pharmaceutical industry due to the limited market size.
- The reauthorization of grants and development programs can affect patients with those conditions by potentially increasing treatment options and improving health outcomes.
Reasoning
- The policy primarily affects those with rare diseases, a population of around 30 million in the US, depending on the condition's prevalence.
- Considering the budget, not every rare disease can be prioritized, and the impact will vary.
- Some individuals from affected populations may not see immediate benefits, as drug development and approval processes can take time.
- The long-term impact may include an increase in available treatments, thus improving Cantril wellbeing scores over time for those with treatable conditions.
Simulated Interviews
Teacher (Chicago, IL)
Age: 42 | Gender: female
Wellbeing Before Policy: 5
Duration of Impact: 15.0 years
Commonness: 4/20
Statement of Opinion:
- I'm hopeful about the policy as it may encourage more research into my child's condition.
- This will only matter if funds are allocated to research the specific disease my child suffers from.
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 | 7 | 5 |
| Year 10 | 7 | 5 |
| Year 20 | 8 | 5 |
Software Developer (Seattle, WA)
Age: 30 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 10.0 years
Commonness: 3/20
Statement of Opinion:
- Every bit of funding helps, but I doubt it will directly affect my condition soon.
- It's a step forward but may not be enough.
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 | 7 | 6 |
| Year 20 | 7 | 6 |
Nurse (Nashville, TN)
Age: 55 | Gender: female
Wellbeing Before Policy: 4
Duration of Impact: 20.0 years
Commonness: 2/20
Statement of Opinion:
- I think the policy will give me more hope for future treatments.
- More research is definitely needed for my condition.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 5 | 4 |
| Year 2 | 6 | 4 |
| Year 3 | 6 | 4 |
| Year 5 | 7 | 4 |
| Year 10 | 7 | 4 |
| Year 20 | 8 | 4 |
Retired Engineer (Dallas, TX)
Age: 67 | Gender: male
Wellbeing Before Policy: 7
Duration of Impact: 0.0 years
Commonness: 10/20
Statement of Opinion:
- Even though it won't affect me directly, I'm glad policies like this exist.
- It's important to support those with rare diseases.
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 | 7 | 7 |
| Year 10 | 7 | 7 |
| Year 20 | 7 | 7 |
College Student (Miami, FL)
Age: 22 | Gender: female
Wellbeing Before Policy: 7
Duration of Impact: 5.0 years
Commonness: 5/20
Statement of Opinion:
- As someone interested in medical research, I see this as positive for future developments.
- It's a start, but implementation is key.
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 | 8 | 7 |
Entrepreneur (Phoenix, AZ)
Age: 38 | Gender: male
Wellbeing Before Policy: 8
Duration of Impact: 0.0 years
Commonness: 10/20
Statement of Opinion:
- I think this policy can catalyze innovation and attract investments in this neglected sector.
- Great for community awareness and potential tech development.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 8 | 8 |
| Year 2 | 8 | 8 |
| Year 3 | 8 | 8 |
| Year 5 | 8 | 8 |
| Year 10 | 8 | 8 |
| Year 20 | 8 | 8 |
Research Scientist (New York, NY)
Age: 49 | Gender: female
Wellbeing Before Policy: 7
Duration of Impact: 20.0 years
Commonness: 1/20
Statement of Opinion:
- This policy should unlock new research avenues, although tangible results may take time.
- Funding is often the bottleneck in making substantial progress.
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 | 7 |
| Year 20 | 9 | 7 |
Lawyer (San Francisco, CA)
Age: 60 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 12.0 years
Commonness: 6/20
Statement of Opinion:
- Policies like these are crucial for improving accessibility and quality of life for many.
- It could have a legislative advantage for more support systems.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 6 |
| Year 2 | 7 | 6 |
| Year 3 | 7 | 6 |
| Year 5 | 7 | 6 |
| Year 10 | 8 | 6 |
| Year 20 | 8 | 6 |
Freelance Writer (Boston, MA)
Age: 35 | Gender: other
Wellbeing Before Policy: 6
Duration of Impact: 5.0 years
Commonness: 7/20
Statement of Opinion:
- This policy is an important step for advocacy and awareness.
- Seeing practical outcomes will take dedication and consistent execution.
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 | 7 | 6 |
| Year 10 | 7 | 6 |
| Year 20 | 7 | 6 |
Pharmaceutical Sales Representative (Houston, TX)
Age: 28 | Gender: male
Wellbeing Before Policy: 7
Duration of Impact: 8.0 years
Commonness: 4/20
Statement of Opinion:
- This should definitely help in bringing more products to market, which is good for business.
- It's aligned with the trends I'm already seeing in the industry.
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 | 8 | 7 |
| Year 20 | 9 | 7 |
Cost Estimates
Year 1: $40000000 (Low: $35000000, High: $50000000)
Year 2: $41000000 (Low: $36000000, High: $51000000)
Year 3: $42000000 (Low: $37000000, High: $52000000)
Year 5: $44000000 (Low: $39000000, High: $54000000)
Year 10: $46000000 (Low: $40000000, High: $56000000)
Year 100: $50000000 (Low: $45000000, High: $60000000)
Key Considerations
- Advancements in orphan drug research could significantly improve life expectancy and quality of life for those affected by rare diseases.
- Programs like these support public health goals by addressing critical unmet needs and often overlooked medical conditions.
- Funding stability is crucial to maintaining momentum in rare disease research and fostering innovation in treatment development.
- The balance between public support and private sector investment in rare disease drug development should be evaluated for effectiveness.