The Food and Drug Administration is changing the way it approves medicines known as ‘orphan drugs’ after revelations that drugmakers may be abusing a law intended to help patients with rare diseases.
Critics say vulnerable patients are being manipulated and the goals promoted are skewed by the pharma benefactors who want faster government approval for new products.
Acting on a request from three influential U.S. senators, the government’s accountability arm confirmed that it will investigate potential abuses of the Orphan Drug Act.
Building on weeks of mounting pressure to address high prescription drug prices, three influential U.S. senators have asked the government’s accountability arm to probe the orphan drug program for potential misuse.
Robert Califf, the just-departed commissioner of the U.S. Food and Drug Administration, offers his take on the Trump administration’s plans to speed up drug approvals and dramatically reduce regulations at the agency.
The Orphan Drug Act has opened the door to almost unlimited price tags for drugs to treat rare diseases — burdening insurers, government programs, and families for whom obtaining the medication is often a matter of life and death.
The system intended to help those with rare diseases is being manipulated by drugmakers to maximize profits and to protect niche markets for medicines that millions of people already take — and it’s all legal.
The lure of the pharmaceutical industry — along with a slow hiring process — has the Food and Drug Administration’s drug approval team struggling to hire and retain staff.