Bill Overview
Title: Modernizing the Accelerated Approval Pathway Act of 2022
Description: This bill modifies provisions relating to the accelerated approval of drugs for serious or life-threatening diseases or conditions, including by requiring the Food and Drug Administration (FDA) to publish its reasons when it determines a postapproval study to be unnecessary. The bill also establishes a pilot program and an FDA council to support the development of drugs for rare diseases and ensure the appropriate use of the accelerated approval pathway, respectively.
Sponsors: Sen. Collins, Susan M. [R-ME]
Target Audience
Population: People with serious, rare, or life-threatening diseases that could benefit from drugs approved through accelerated pathways
Estimated Size: 30000000
- The bill impacts entities involved in the drug development process, particularly those developing drugs for serious or life-threatening conditions.
- Patients with rare diseases will be directly affected as the bill's pilot program is designed to support drug development for these conditions.
- The accelerated approval pathway primarily affects those with serious or life-threatening diseases who potentially depend on quicker access to new treatments.
- Globally, serious and life-threatening diseases, including cancer and rare diseases, affect millions of people.
- The establishment of a council to ensure appropriate use of accelerated approval pathways can potentially impact global drug approval standards, indirectly affecting patients worldwide.
Reasoning
- The policy targets the development of drugs for rare and serious diseases, which affects a niche, critical segment of the U.S. population, primarily those suffering from rare or life-threatening conditions.
- The budget constraints suggest that the policy focuses on increasing efficiency in drug approval processes rather than expanding patient services directly, affecting the industry more initially but with downstream effects on patients.
- A spectrum of those affected includes pharmaceutical researchers, patients with serious diseases, caregivers, and health policy analysts, each experiencing different levels of impact.
- Given the size of the target population and the high cost associated with drug development, the policy's immediate and direct impacts are limited but with potential for large indirect benefits especially in small but high-need areas like rare diseases.
- People not directly impacted include those not suffering from serious diseases or not involved in the pharmaceutical industry.
Simulated Interviews
Clinical Researcher (Boston, MA)
Age: 58 | Gender: female
Wellbeing Before Policy: 7
Duration of Impact: 5.0 years
Commonness: 6/20
Statement of Opinion:
- The new act streamlines parts of my research process, which could lead to getting life-saving drugs to patients faster.
- There have been frustrations in the past with how long approvals can take, so this is definitely a step in the right direction.
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 | 9 | 8 |
| Year 10 | 9 | 8 |
| Year 20 | 8 | 7 |
Biotech Entrepreneur (Seattle, WA)
Age: 40 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 10.0 years
Commonness: 5/20
Statement of Opinion:
- Streamlining the approval process is essential for the survival of smaller biotech firms like mine.
- I am cautiously optimistic but hope the policy effectively reduces bureaucratic hurdles.
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 | 10 | 7 |
| Year 20 | 9 | 7 |
Patient with Cystic Fibrosis (Houston, TX)
Age: 30 | Gender: female
Wellbeing Before Policy: 5
Duration of Impact: 20.0 years
Commonness: 3/20
Statement of Opinion:
- Anything that speeds up the availability of treatments matters to patients like me who have limited options.
- I've seen too many treatments stall in approval when patients desperately need them.
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 | 6 |
| Year 10 | 9 | 6 |
| Year 20 | 10 | 7 |
Oncologist (Phoenix, AZ)
Age: 65 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 10.0 years
Commonness: 8/20
Statement of Opinion:
- The accelerated approval could transform cancer treatment, helping me offer better options to my patients.
- I'm hopeful but also realistic about the practical implementation hurdles.
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 | 7 |
| Year 10 | 9 | 7 |
| Year 20 | 8 | 7 |
Healthcare Policy Analyst (New York, NY)
Age: 25 | Gender: other
Wellbeing Before Policy: 7
Duration of Impact: 5.0 years
Commonness: 4/20
Statement of Opinion:
- This act could set important precedents for how quickly new therapies reach patients.
- It'll be pivotal to track its success and ensure it doesn't compromise safety.
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 |
Caregiver to a child with a rare disease (Chicago, IL)
Age: 50 | Gender: female
Wellbeing Before Policy: 5
Duration of Impact: 15.0 years
Commonness: 5/20
Statement of Opinion:
- I hope this act leads to breakthroughs that can dramatically change my child's quality of life.
- Access to new drugs faster could mean everything to families like ours.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 5 |
| Year 2 | 7 | 6 |
| Year 3 | 8 | 6 |
| Year 5 | 9 | 7 |
| Year 10 | 9 | 8 |
| Year 20 | 9 | 8 |
Pharmaceutical Company Executive (San Francisco, CA)
Age: 39 | Gender: female
Wellbeing Before Policy: 7
Duration of Impact: 10.0 years
Commonness: 6/20
Statement of Opinion:
- This policy could cut down on delays that impact our bottom line and speed access to innovations.
- It could redefine competitive strategies in pharmaceutical sectors.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 7 |
| Year 2 | 8 | 7 |
| Year 3 | 9 | 7 |
| Year 5 | 9 | 8 |
| Year 10 | 10 | 8 |
| Year 20 | 9 | 7 |
Lobbyist for Rare Disease Charities (Miami, FL)
Age: 45 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 5.0 years
Commonness: 5/20
Statement of Opinion:
- The bill could enhance advocacy impacts, finally translating into tangible improvements for affected communities.
- Detailed and transparent FDA communications in this context are crucial.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 6 |
| Year 2 | 8 | 6 |
| Year 3 | 8 | 7 |
| Year 5 | 8 | 7 |
| Year 10 | 8 | 7 |
| Year 20 | 8 | 7 |
Retired Teacher with early-stage Alzheimer's (Columbus, OH)
Age: 60 | Gender: female
Wellbeing Before Policy: 4
Duration of Impact: 10.0 years
Commonness: 7/20
Statement of Opinion:
- I'm hopeful this means potential treatments won't take as long to reach people like me.
- Every delay weighs heavily when you're racing against time.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 5 | 4 |
| Year 2 | 6 | 5 |
| Year 3 | 7 | 5 |
| Year 5 | 7 | 6 |
| Year 10 | 8 | 6 |
| Year 20 | 8 | 7 |
Public Health Researcher (Denver, CO)
Age: 33 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 5.0 years
Commonness: 3/20
Statement of Opinion:
- Improved drug approval processes can shift public health trends over the long term.
- Concerns remain about policy accommodating broader systemic health inequities.
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 | 7 |
| Year 20 | 8 | 7 |
Cost Estimates
Year 1: $75000000 (Low: $50000000, High: $100000000)
Year 2: $70000000 (Low: $45000000, High: $95000000)
Year 3: $60000000 (Low: $40000000, High: $80000000)
Year 5: $50000000 (Low: $35000000, High: $70000000)
Year 10: $40000000 (Low: $25000000, High: $55000000)
Year 100: $0 (Low: $0, High: $0)
Key Considerations
- The scalability of the pilot program for rare diseases depends on its initial success and stakeholder engagement.
- Coordination with international regulatory entities may be needed, affecting the program's design and implementation.
- Ongoing evaluation mechanisms should be in place to assess the program's effectiveness in achieving its stated goals.
- Political and public support for the program's expansion could influence funding and implementation strategies.