Bill Overview
Title: Orphan Drug COVID–19 Mitigation Act of 2022
Description: This bill extends by 180 days the relevant periods of market exclusivity for drugs for rare diseases or conditions (i.e., orphan drugs) for which applications were submitted during the COVID-19 emergency period.
Sponsors: Rep. Gottheimer, Josh [D-NJ-5]
Target Audience
Population: People affected by rare diseases worldwide
Estimated Size: 30000000
- The bill specifically mentions 'drugs for rare diseases or conditions,' which are referred to as orphan drugs. These are generally targeted towards patients with rare diseases.
- The COVID-19 pandemic has potentially affected a wide range of pharmaceutical applications and developments, causing delays and disruptions in drug availability.
- Orphan drugs typically serve a very small segment of the population, specifically those who have rare diseases which by definition impact fewer than 200,000 individuals in the United States.
- There are an estimated 7,000 different types of rare diseases worldwide, affecting about 300 million people globally according to various health organizations.
- The total number of people who might directly benefit from this bill includes not only those currently on orphan drugs but also those awaiting new drugs that could be delayed without the extension of market exclusivity.
Reasoning
- The target population for this policy is people affected by rare diseases or conditions in the United States, numbering around 30 million individuals.
- The bill aims to mitigate disruptions in the availability of essential treatments for these individuals by extending market exclusivity, encouraging ongoing development, and preventing shortages due to COVID-19-related delays.
- Given the budget constraints, it is essential to ensure that the benefits reach a significant portion of the affected population and assess whether the extensions will indeed improve access to the necessary medications.
- The majority of the beneficiaries will be those who have direct reliance on orphan drugs, with a possible spillover of benefits to the healthcare system and relatives relying on these patients' well-being.
- Indirect beneficiaries include healthcare providers and the pharmaceutical industry, though their 'self-reported well-being' will largely reflect economic outcomes rather than health-related quality of life metrics.
Simulated Interviews
Pharmaceutical researcher (New York, NY)
Age: 45 | Gender: female
Wellbeing Before Policy: 7
Duration of Impact: 3.0 years
Commonness: 15/20
Statement of Opinion:
- I think the extension is beneficial because it provides more time for companies like us to ensure our treatments reach patients without disruption.
- On a personal level, knowing that more patients can receive timely medication is rewarding.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 8 | 6 |
| Year 2 | 7 | 5 |
| Year 3 | 7 | 5 |
| Year 5 | 6 | 4 |
| Year 10 | 6 | 4 |
| Year 20 | 5 | 3 |
Patient with rare condition (Los Angeles, CA)
Age: 35 | Gender: male
Wellbeing Before Policy: 5
Duration of Impact: 5.0 years
Commonness: 2/20
Statement of Opinion:
- This extension might help keep my medication accessible, which is crucial for managing my condition.
- I'm worried about whether prices will rise during the exclusivity period.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 5 |
| Year 2 | 7 | 4 |
| Year 3 | 7 | 4 |
| Year 5 | 7 | 3 |
| Year 10 | 6 | 2 |
| Year 20 | 5 | 2 |
Healthcare policy analyst (Chicago, IL)
Age: 50 | Gender: female
Wellbeing Before Policy: 6
Duration of Impact: 10.0 years
Commonness: 10/20
Statement of Opinion:
- The market exclusivity might provide stability in drug availability, but we must ensure it doesn't stifle innovation.
- There are concerns about price increases during this exclusivity period, potentially limiting access.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 6 |
| Year 2 | 6 | 5 |
| Year 3 | 6 | 5 |
| Year 5 | 6 | 4 |
| Year 10 | 6 | 4 |
| Year 20 | 5 | 3 |
Software engineer (Austin, TX)
Age: 28 | Gender: male
Wellbeing Before Policy: 5
Duration of Impact: 2.0 years
Commonness: 8/20
Statement of Opinion:
- While it's important for companies to innovate, my primary concern is my cousin's uninterrupted access to necessary drugs.
- The policy seems beneficial, but it must be monitored to ensure patient needs are prioritized.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 4 |
| Year 2 | 6 | 4 |
| Year 3 | 5 | 4 |
| Year 5 | 5 | 3 |
| Year 10 | 4 | 3 |
| Year 20 | 4 | 3 |
Biotech entrepreneur (Seattle, WA)
Age: 42 | Gender: female
Wellbeing Before Policy: 8
Duration of Impact: 4.0 years
Commonness: 3/20
Statement of Opinion:
- The extension provides our company the space to navigate unforeseen delays and challenges during drug launches.
- This policy helps us ensure sustainability and continuity.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 9 | 7 |
| Year 2 | 9 | 6 |
| Year 3 | 8 | 6 |
| Year 5 | 8 | 5 |
| Year 10 | 7 | 4 |
| Year 20 | 6 | 3 |
Retired physician (Houston, TX)
Age: 60 | Gender: male
Wellbeing Before Policy: 7
Duration of Impact: 5.0 years
Commonness: 12/20
Statement of Opinion:
- This policy should help ensure continuity in care for young patients, which is crucial for long-term health outcomes.
- Hopefully, it also encourages long-term investments into rare disease treatments.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 8 | 6 |
| Year 2 | 8 | 5 |
| Year 3 | 8 | 5 |
| Year 5 | 7 | 4 |
| Year 10 | 7 | 4 |
| Year 20 | 6 | 3 |
Pharmaceutical executive (San Francisco, CA)
Age: 52 | Gender: other
Wellbeing Before Policy: 8
Duration of Impact: 3.0 years
Commonness: 5/20
Statement of Opinion:
- The extension helps our team manage logistical hurdles during drug approval processes.
- Given the impact of COVID-19, this cushion is necessary for strategic planning.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 8 | 7 |
| Year 2 | 8 | 6 |
| Year 3 | 7 | 5 |
| Year 5 | 7 | 4 |
| Year 10 | 6 | 4 |
| Year 20 | 5 | 4 |
Patient advocate (Miami, FL)
Age: 31 | Gender: female
Wellbeing Before Policy: 6
Duration of Impact: 2.0 years
Commonness: 7/20
Statement of Opinion:
- Ensuring uninterrupted access to drugs is pivotal, but we need transparency in pricing during this exclusivity period.
- This policy is promising if effectively monitored.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 5 |
| Year 2 | 6 | 5 |
| Year 3 | 6 | 5 |
| Year 5 | 5 | 4 |
| Year 10 | 4 | 4 |
| Year 20 | 4 | 3 |
Retired engineer (Phoenix, AZ)
Age: 75 | Gender: male
Wellbeing Before Policy: 5
Duration of Impact: 5.0 years
Commonness: 11/20
Statement of Opinion:
- This extension is reassuring as it should help ensure my granddaughter continues to receive her necessary treatments.
- I just hope it leads to lower drug prices over time.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 4 |
| Year 2 | 6 | 4 |
| Year 3 | 6 | 3 |
| Year 5 | 6 | 3 |
| Year 10 | 5 | 3 |
| Year 20 | 5 | 2 |
Healthcare economist (Boston, MA)
Age: 40 | Gender: female
Wellbeing Before Policy: 7
Duration of Impact: 3.0 years
Commonness: 13/20
Statement of Opinion:
- The extension provides an opportunity for further study on long-term impacts of market exclusivity.
- I am cautiously optimistic, as any disruptions in orphan drug access can be concerning.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 8 | 6 |
| Year 2 | 7 | 5 |
| Year 3 | 7 | 4 |
| Year 5 | 6 | 3 |
| Year 10 | 5 | 3 |
| Year 20 | 5 | 2 |
Cost Estimates
Year 1: $200000000 (Low: $150000000, High: $250000000)
Year 2: $210000000 (Low: $160000000, High: $260000000)
Year 3: $220000000 (Low: $170000000, High: $270000000)
Year 5: $230000000 (Low: $180000000, High: $280000000)
Year 10: $250000000 (Low: $200000000, High: $300000000)
Year 100: $300000000 (Low: $250000000, High: $350000000)
Key Considerations
- The exclusivity extension benefits pharmaceutical companies but limits competitive market dynamics, potentially affecting drug pricing elsewhere.
- Target beneficiaries primarily comprise patients with rare diseases; hence, access and affordability considerations are crucial.
- Legal and industry responses to changes in exclusivity, including impacts on research and development incentives, could vary.
- The healthcare system, including insurers and government providers, may incur extended high costs without generics.