> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Senators Ask GAO to Probe Orphan Drug Act
- URL: https://www.fdaweb.com/senators-ask-gao-to-probe-orphan-drug-act/
- Published: 2017-03-06T12:00:00.000Z
- Updated: 2026-09-14T22:10:00.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5138126

Sen. **Chuck Grassley** (R-IA) has expanded his inquiry into potential misuses of FDA’s orphan drug program in the wake of the agency’s recent approval of Marathon Pharmaceuticals’ orphan drug Emflaza (deflazacort) tablets and oral suspension to treat patients age five and older with Duchenne muscular dystrophy ([see story](https://www.fdaweb.com/fda-approves-emflaza-duchenne-drug/)). The company and the agency are feeling some backlash over the $89,000 annual price tag Marathon says it will charge for the decades-old steroidal drug, which is 50-70 times higher than the drug’s price overseas.

  
[Last month](https://www.fdaweb.com/grassley-probing-orphan-drug-program-misuses/), Grassley said he and his staff were examining how the orphan drug provisions appear to be stretched beyond their original intent and “whether the unanticipated uses of the provisions are contributing to high prices for commonly used drugs.” Now, Grassley has joined with Senators **Orrin Hatch** (R-UT) and **Tom Cotton** (R-AR) to ask the Government Accountability Office (GAO) to look into potential abuses of the Orphan Drug Act (ODA). Specifically, the lawmakers have asked GAO to “investigate whether the ODA is still incentivizing product development for diseases with fewer than 200,000 affected individuals, as intended, and provide any regulatory or legislative changes that may be needed in order to preserve the intent of this vital law.”