Rare Disease Caucus Co-Chair Hatch Speaks on Targeted Therapies After Committee Passage
Washington, D.C.—Senator Orrin Hatch, R-Utah, member and former chairman of the Senate HELP Committee, and Senate co-chair of the Congressional Rare Disease Caucus spoke about the Advancing Targeted Therapies for Rare Diseases Act after it passed out of committee.
“As a long-time champion for helping those with rare diseases, I am encouraged by the amazing progress we are making in precision medicine,” Senator Hatch said. “If we want to realize the full potential of cutting-edge advances and address serious unmet needs of many patients, the Senate must pass the Advancing Targeted Therapies for Rare Diseases Act. This legislation will incentivize drug development, help overcome the difficulties of conducting conventional trials for many rare diseases, conserve FDA’s limited resources, bring needed therapies to patients faster, and make treatments available where there otherwise would be no other options.”
Senator Hatch is a cosponsor of the Advancing Targeted Therapies for Rare Diseases Act together with Senators Bennet (D-CO), Burr (R-NC) and Warren (D-MA). This bill clarifies that the FDA has the authority to allow innovators to rely on the clinical trial work they’ve done getting a previous targeted therapy approved when seeking approval for a subsequent targeted therapy with a similar chemical backbone that targets a different genetic subtype of the same disease. Many therapies can be easily altered to target a different genetic mutation without altering the overall chemistry of the drug. This would not change the FDA’s standards for the safety or effectiveness of the drug, but rather allow the FDA approval process to be more efficient.
For a one-pager on the bill, click here.
