Bill Overview
Title: Helping Experts Accelerate Rare Treatments Act of 2022
Description: This bill requires studies on processes for approving drugs that treat rare diseases and conditions. The Government Accountability Office (GAO) must contract with an appropriate entity to study the European Union's process for evaluating the safety and efficacy of such drugs, including any flexibilities or mechanisms that are specific to rare diseases or conditions. The GAO must also conduct a study about the Food and Drug Administration's mechanisms for ensuring that patient and physician perspectives are considered when evaluating and approving drugs for rare diseases or conditions. The GAO must report to Congress the results of these studies.
Sponsors: Rep. Tonko, Paul [D-NY-20]
Target Audience
Population: People living with rare diseases worldwide
Estimated Size: 30000000
- Rare diseases, by definition, affect a small percentage of the population.
- In the EU and the US, drugs for rare diseases are often given orphan status to encourage development.
- The bill aims to study and potentially improve processes for drug approval specific to rare diseases.
- Since there are thousands of rare diseases, the cumulative number of individuals affected worldwide is significant, even though each disease affects a small number.
- Individuals with rare diseases often have few treatment options currently, which this bill seeks to address through improved evaluation and approval processes.
Reasoning
- Rare diseases affect fewer than 200,000 individuals in the US, representing a diverse array of conditions.
- The policy primarily impacts individuals suffering from such diseases by potentially improving the availability of treatments through faster approval processes.
- The budget constraint suggests the policy is in the study and planning phase, meaning immediate drug availability changes might not yet occur.
- We have varied interviews in terms of demographics but maintained focus on relevance to the healthcare and rare disease communities.
- Some individuals may not experience direct effects due to the initial focus on research rather than implementation.
Simulated Interviews
Biomedical Researcher (San Francisco, CA)
Age: 32 | Gender: female
Wellbeing Before Policy: 7
Duration of Impact: 10.0 years
Commonness: 4/20
Statement of Opinion:
- I see this policy as a crucial step in improving our processes around rare diseases, something that is very close to the work I do.
- It is a bit early to feel the direct effects, but the long-term potential seems promising.
- Increased collaboration with the EU might bring beneficial insights.
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 | 9 | 7 |
| Year 20 | 9 | 6 |
Rare Disease Advocate (Houston, TX)
Age: 45 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 20.0 years
Commonness: 3/20
Statement of Opinion:
- This policy could potentially streamline much-needed treatments for our community.
- I hope the bill leads to tangible changes rather than just more bureaucracy.
- I'm optimistic, but cautious.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 6 |
| Year 2 | 8 | 6 |
| Year 3 | 8 | 5 |
| Year 5 | 8 | 5 |
| Year 10 | 9 | 4 |
| Year 20 | 9 | 4 |
Pharmaceutical Consultant (Boston, MA)
Age: 28 | Gender: female
Wellbeing Before Policy: 8
Duration of Impact: 5.0 years
Commonness: 5/20
Statement of Opinion:
- As a professional in the pharmaceutical industry, this policy signals future opportunities for innovation.
- Short-term changes may not be drastic, but long-term benefits are likely.
- Collaboration with the EU is a wise move.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 8 | 8 |
| Year 2 | 8 | 8 |
| Year 3 | 9 | 8 |
| Year 5 | 9 | 8 |
| Year 10 | 9 | 8 |
| Year 20 | 9 | 7 |
General Practitioner (Los Angeles, CA)
Age: 50 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 10.0 years
Commonness: 8/20
Statement of Opinion:
- The policy seems promising, but as a physician, I need to see results before getting too hopeful.
- In the short term, my patients might not notice much difference.
- The integration of diverse perspectives in drug approval is encouraging.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 6 |
| Year 2 | 6 | 6 |
| Year 3 | 7 | 6 |
| Year 5 | 7 | 6 |
| Year 10 | 8 | 6 |
| Year 20 | 8 | 6 |
Stay-at-home mom (Chicago, IL)
Age: 39 | Gender: female
Wellbeing Before Policy: 5
Duration of Impact: 20.0 years
Commonness: 2/20
Statement of Opinion:
- This bill could mean hope for new treatments for my child.
- At the moment, the changes are not yet visible.
- I'm supportive of any policy that seeks to improve our situation.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 5 | 5 |
| Year 2 | 6 | 5 |
| Year 3 | 6 | 4 |
| Year 5 | 7 | 4 |
| Year 10 | 8 | 3 |
| Year 20 | 8 | 2 |
Retired Biotech Executive (New York, NY)
Age: 60 | Gender: male
Wellbeing Before Policy: 7
Duration of Impact: 5.0 years
Commonness: 4/20
Statement of Opinion:
- Improving processes for rare disease drug approval is a step in the right direction.
- It's more of a long-term effect rather than immediate impacts.
- Having seen the US and EU landscape, this collaboration may yield beneficial insights.
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 |
University Student (Seattle, WA)
Age: 22 | Gender: female
Wellbeing Before Policy: 8
Duration of Impact: 10.0 years
Commonness: 7/20
Statement of Opinion:
- As a student interested in rare diseases, this could be a crucial step for future research and career opportunities.
- The direct effects aren't very apparent to me right now, but the implications are exciting.
- Increased cooperation with Europe is also beneficial.
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 | 9 | 8 |
| Year 10 | 9 | 8 |
| Year 20 | 9 | 8 |
Patient with rare autoimmune disorder (Miami, FL)
Age: 55 | Gender: female
Wellbeing Before Policy: 5
Duration of Impact: 20.0 years
Commonness: 2/20
Statement of Opinion:
- I am hopeful that this policy might lead to quicker access to new therapies.
- With so few options currently, any effort to improve the situation is welcomed.
- It is just a start, but every little help counts.
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 | 9 | 4 |
| Year 20 | 9 | 4 |
Regulatory Affairs Specialist at a Pharmaceutical Company (Atlanta, GA)
Age: 40 | Gender: male
Wellbeing Before Policy: 7
Duration of Impact: 5.0 years
Commonness: 5/20
Statement of Opinion:
- This policy could streamline processes and help our company get treatments to patients faster.
- I'm intrigued by the potential to integrate European insights.
- Initially, the effects are administrative, but the long-term operational benefits stand out.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 8 | 7 |
| Year 2 | 8 | 7 |
| Year 3 | 9 | 7 |
| Year 5 | 9 | 7 |
| Year 10 | 9 | 7 |
| Year 20 | 8 | 7 |
Blogger, Rare Disease Survivor (Phoenix, AZ)
Age: 33 | Gender: female
Wellbeing Before Policy: 6
Duration of Impact: 20.0 years
Commonness: 3/20
Statement of Opinion:
- This policy gives me hope that more treatments will become available sooner.
- While official changes are not immediate, the promise is there.
- I appreciate the focus on patient perspectives in drug approval.
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 | 6 |
| Year 10 | 9 | 5 |
| Year 20 | 9 | 5 |
Cost Estimates
Year 1: $4500000 (Low: $4000000, High: $5000000)
Year 2: $4500000 (Low: $4000000, High: $5000000)
Year 3: $0 (Low: $0, High: $0)
Year 5: $0 (Low: $0, High: $0)
Year 10: $0 (Low: $0, High: $0)
Year 100: $0 (Low: $0, High: $0)
Key Considerations
- The studies aim to align the EU and US drug approval processes for rare diseases, potentially leading to faster drug availability.
- Potentially transformative for individuals with rare diseases if approval times are reduced.
- The cost of the study is a small fraction compared to the potential economic benefits of streamlined drug approval and increased access to treatments.