Story perspectives

New Oncology Policies Transform Rare Disease Clinical Trials

9/28/2025

Summary

Story summary

New oncology policies shift focus toward rare diseases, changing clinical trial dynamics, per Ryan Haumschild at the PCOC conference in Nashville.
Stakeholders worry policies such as the Inflation Reduction Act will affect innovation, pricing, and patient access.
Haumschild says clinical trials are vital for patients without standard care, and shifting to rare diseases could benefit patients and pharmaceutical investments.

History

Historical Context

The Orphan Drug Act of 1983 spurred the development of treatments for rare diseases, resulting in over 700 approved drugs and more than 4,000 orphan designations by the FDA. This legislation marked a significant shift in pharmaceutical investment strategies. Current oncology policy discussions, including concerns about the Inflation Reduction Act, continue to emphasize the importance of balancing drug affordability with innovation in rare disease research.

1 of 2

Story summary

New oncology policies shift focus toward rare diseases, changing clinical trial dynamics, per Ryan Haumschild at the PCOC conference in Nashville.
Stakeholders worry policies such as the Inflation Reduction Act will affect innovation, pricing, and patient access.
Haumschild says clinical trials are vital for patients without standard care, and shifting to rare diseases could benefit patients and pharmaceutical investments.

1 / 2