Analysis of 20 Years of Legislative Trends to Predict Future Investment and Access to Orphan Drugs
Author(s)
Jose Medrano, PhD, Aashna Shah, MSc, Nicolette Scinicariello, BA, Sarah Neubert, BA;
Costello Medical, Boston, MA, USA
Costello Medical, Boston, MA, USA
Presentation Documents
OBJECTIVES: Government legislation profoundly shapes the US pharmaceutical industry's investment, pricing, and market access strategies for rare diseases (RDs) and orphan drugs (ODs). The Orphan Drug Act boosted OD availability from 38 to over 600, enhancing market access, while the Inflation Reduction Act has resulted in many companies rethinking investments and pipelines for multi-indication drugs. By examining the frequency, volume, and topics of RD/OD-related laws passed over the last 20 years, this research aims to identify legislative trends that may influence investment and access to ODs.
METHODS: On 3 January 2025, targeted searches of www.congress.gov identified RD/OD-related legislation passed since 3 January 2005 using keywords in bill titles, summaries, and texts. Data on the bill’s presiding president, status, introduction/enactment dates, and how it addresses rare disease community needs were extracted into a predefined grid and analyzed.
RESULTS: Since 2005, 278 RD/OD-related bills were introduced to Congress. Of the 19 that became law, 13 were introduced from 2005-2015, compared to six from 2016-2025. The highest number of laws (six) were passed during Obama's first term, while the lowest (two) were passed during both Trump's first term and Bush's second term. Four laws focused on expanding access to ODs, 12 on promoting RD research, five on expanding diagnosis programs, and six on coordinating care for individuals with RDs.
CONCLUSIONS: Over the past 20 years, 63% of RD/OD-related legislation focused on promoting RD/OD research through funding, awards for clinical trial coordination, and patient recruitment incentives, reflecting a policy-focus on fostering innovation to expand treatment options for individuals with RDs. While this focus may boost future OD availability, the decline in legislative activity over time raises concerns about sustained policy attention. Future research should evaluate the impact of these laws on investment and access to ODs to guide more effective policymaking for the RD community.
METHODS: On 3 January 2025, targeted searches of www.congress.gov identified RD/OD-related legislation passed since 3 January 2005 using keywords in bill titles, summaries, and texts. Data on the bill’s presiding president, status, introduction/enactment dates, and how it addresses rare disease community needs were extracted into a predefined grid and analyzed.
RESULTS: Since 2005, 278 RD/OD-related bills were introduced to Congress. Of the 19 that became law, 13 were introduced from 2005-2015, compared to six from 2016-2025. The highest number of laws (six) were passed during Obama's first term, while the lowest (two) were passed during both Trump's first term and Bush's second term. Four laws focused on expanding access to ODs, 12 on promoting RD research, five on expanding diagnosis programs, and six on coordinating care for individuals with RDs.
CONCLUSIONS: Over the past 20 years, 63% of RD/OD-related legislation focused on promoting RD/OD research through funding, awards for clinical trial coordination, and patient recruitment incentives, reflecting a policy-focus on fostering innovation to expand treatment options for individuals with RDs. While this focus may boost future OD availability, the decline in legislative activity over time raises concerns about sustained policy attention. Future research should evaluate the impact of these laws on investment and access to ODs to guide more effective policymaking for the RD community.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
HPR149
Topic
Health Policy & Regulatory
Topic Subcategory
Approval & Labeling, Reimbursement & Access Policy
Disease
SDC: Rare & Orphan Diseases