Bill Overview
Title: RARE Act
Description: This bill specifies that the seven-year market exclusivity period for drugs for rare diseases or conditions (i.e., orphan drug exclusivity period) prohibits the approval of the same drug for the same approved use or indication with respect to the disease or condition. Current law grants a seven-year period of market exclusivity for an approved orphan drug, during which the Food and Drug Administration (FDA) may not approve an application from another manufacturer for the same drug for the same disease or condition. The FDA's regulations provide that this exclusivity is specific to the same approved use or indication of the drug, rather than all uses or indications, for the disease or condition. However, in Catalyst Pharmaceuticals, Inc. v. Becerra, a court held that exclusivity did extend to all uses or indications for the disease or condition. The bill provides statutory authority for the FDA's regulations.
Sponsors: Sen. Baldwin, Tammy [D-WI]
Target Audience
Population: Individuals with rare diseases impacted by orphan drug exclusivity laws
Estimated Size: 30000000
- This bill concerns orphan drugs, which are drugs developed explicitly for rare diseases or conditions.
- Rare diseases affect a relatively small percentage of the population, but there are about 7,000 known rare diseases worldwide.
- Globally, it is estimated that about 300 million people are affected by rare diseases.
- Orphan drugs are crucial for these populations because there may be limited alternative treatments available.
- The bill affects the pharmaceutical industry because it impacts how companies can compete in developing drugs for rare diseases, thus potentially affecting drug availability.
- The bill addresses the market exclusivity period which affects when similar drugs can be made available after the first drug is approved.
Reasoning
- The RARE Act impacts individuals with rare diseases by clarifying market exclusivity for orphan drugs, which could influence the timing and availability of alternative treatments.
- The policy may benefit populations whose treatment is protected under exclusivity, ensuring sustained market availability and potentially more stable treatment regimens.
- On the downside, it may discourage other manufacturers from developing competing drugs in the exclusivity period, which could perpetuate high drug prices and limited treatment innovation.
- The US target population is about 30 million individuals with rare diseases, but the impact level and duration vary based on the specific disease and treatment availability.
- Budget and program size limit how expansively policy effects can be simulated, thus focusing on diverse perspectives within the target group.
Simulated Interviews
Teacher (New York, NY)
Age: 45 | Gender: female
Wellbeing Before Policy: 4
Duration of Impact: 7.0 years
Commonness: 8/20
Statement of Opinion:
- The RARE Act could mean I have a guaranteed treatment option for seven years without changes.
- I'm worried about medication costs staying high due to reduced competition.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 5 | 4 |
| Year 2 | 5 | 4 |
| Year 3 | 5 | 4 |
| Year 5 | 5 | 3 |
| Year 10 | 4 | 3 |
| Year 20 | 3 | 2 |
Software Developer (Austin, TX)
Age: 32 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 5.0 years
Commonness: 12/20
Statement of Opinion:
- The RARE Act might slow down the development of new treatments in some cases.
- Exclusivity is beneficial for companies developing new drugs, but not always for patients needing alternatives.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 6 |
| Year 2 | 6 | 5 |
| Year 3 | 6 | 5 |
| Year 5 | 6 | 5 |
| Year 10 | 5 | 4 |
| Year 20 | 4 | 3 |
Retired (Boston, MA)
Age: 60 | Gender: female
Wellbeing Before Policy: 4
Duration of Impact: 10.0 years
Commonness: 10/20
Statement of Opinion:
- Secure access to my current medication is crucial, and the RARE Act provides that.
- There is a need for more treatments, but not at the cost of losing the current ones.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 5 | 4 |
| Year 2 | 6 | 4 |
| Year 3 | 6 | 3 |
| Year 5 | 5 | 3 |
| Year 10 | 4 | 2 |
| Year 20 | 4 | 2 |
Biomedical Researcher (San Francisco, CA)
Age: 26 | Gender: other
Wellbeing Before Policy: 7
Duration of Impact: 5.0 years
Commonness: 14/20
Statement of Opinion:
- The RARE Act may stabilize current markets but may slightly limit the fast evolution of new treatments.
- The research environment remains key to driving drug development incentives.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 7 |
| Year 2 | 7 | 6 |
| Year 3 | 6 | 6 |
| Year 5 | 6 | 5 |
| Year 10 | 5 | 4 |
| Year 20 | 4 | 4 |
Lawyer (Atlanta, GA)
Age: 55 | Gender: male
Wellbeing Before Policy: 5
Duration of Impact: 7.0 years
Commonness: 9/20
Statement of Opinion:
- The Act reduces regulatory ambiguity, meaning better planning for treatments.
- However, more competition might decrease costs, which the Act could hinder.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 5 |
| Year 2 | 6 | 5 |
| Year 3 | 6 | 4 |
| Year 5 | 5 | 4 |
| Year 10 | 4 | 3 |
| Year 20 | 3 | 2 |
Retired Nurse (Miami, FL)
Age: 72 | Gender: female
Wellbeing Before Policy: 3
Duration of Impact: 10.0 years
Commonness: 7/20
Statement of Opinion:
- Stability in drug provision is crucial at my age, so I see benefits in the RARE Act.
- I'm concerned about costs and future options if competitors are limited.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 4 | 3 |
| Year 2 | 4 | 3 |
| Year 3 | 5 | 3 |
| Year 5 | 4 | 2 |
| Year 10 | 3 | 2 |
| Year 20 | 3 | 2 |
Hospital Pharmacist (Denver, CO)
Age: 39 | Gender: male
Wellbeing Before Policy: 6
Duration of Impact: 7.0 years
Commonness: 11/20
Statement of Opinion:
- The RARE Act clarifies rules which can help me manage patient expectations better.
- However, it might encourage a stagnant market for some drugs, affecting patient care.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 7 | 6 |
| Year 2 | 7 | 6 |
| Year 3 | 6 | 5 |
| Year 5 | 6 | 4 |
| Year 10 | 5 | 4 |
| Year 20 | 5 | 3 |
College Student (Seattle, WA)
Age: 22 | Gender: female
Wellbeing Before Policy: 5
Duration of Impact: 10.0 years
Commonness: 9/20
Statement of Opinion:
- The stability the RARE Act provides is reassuring for what I currently need.
- I fear price rises without competitive pressure.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 6 | 5 |
| Year 2 | 6 | 4 |
| Year 3 | 6 | 4 |
| Year 5 | 5 | 3 |
| Year 10 | 4 | 3 |
| Year 20 | 4 | 2 |
Biotech Investor (Chicago, IL)
Age: 50 | Gender: male
Wellbeing Before Policy: 7
Duration of Impact: 5.0 years
Commonness: 13/20
Statement of Opinion:
- From a business standpoint, the RARE Act secures investments in drug exclusivity.
- Balances industry incentives but might slow innovation without competition.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 8 | 7 |
| Year 2 | 8 | 7 |
| Year 3 | 7 | 6 |
| Year 5 | 7 | 6 |
| Year 10 | 6 | 5 |
| Year 20 | 5 | 4 |
Freelance Writer (Portland, OR)
Age: 28 | Gender: other
Wellbeing Before Policy: 3
Duration of Impact: 7.0 years
Commonness: 8/20
Statement of Opinion:
- Having a longer exclusivity period ensures I get the medication I need.
- However, we need a balance that encourages diverse treatment options.
Wellbeing Over Time (With vs Without Policy)
| Year | With Policy | Without Policy |
|---|---|---|
| Year 1 | 4 | 3 |
| Year 2 | 4 | 3 |
| Year 3 | 3 | 2 |
| Year 5 | 3 | 2 |
| Year 10 | 2 | 2 |
| Year 20 | 2 | 1 |
Cost Estimates
Year 1: $5000000 (Low: $2000000, High: $8000000)
Year 2: $5000000 (Low: $2000000, High: $8000000)
Year 3: $5000000 (Low: $2000000, High: $8000000)
Year 5: $5000000 (Low: $2000000, High: $8000000)
Year 10: $5000000 (Low: $2000000, High: $8000000)
Year 100: $5000000 (Low: $2000000, High: $8000000)
Key Considerations
- Support for individuals with rare diseases remains a critical focus of the legislation.
- The clarity in exclusivity periods can stabilize pharmaceutical investment decisions.
- Potential reduction in drug variety and competition due to extended exclusivity could impact market dynamics.
- The legal costs saved may foster more efficient use of FDA and judicial resources.