Pressure mounts on FDA to loosen drug and device regulations

The 21st Century Cures Act, which passed the House Energy and Commerce Committee unanimously last week, is intended to boost medical research in the U.S. and revise rules over drug and medical device safety (click here for yesterday’s post on the implications the Act could have on physician reporting requirements). The legislation will likely head to a full vote in the House in June with the Senate taking it up by year’s end.

The legislation would increase funding for the National Institutes of Health (NIH) by $10 billion over five years. The NIH would be allowed to use some of that money for the Obama Administration’s $215 million medicine initiative, which would create a database of genomic information obtained from one million U.S. patients who volunteer to participate. Costs would be offset by changing the timing of specific government payments to Medicare Part D plans. Apart from this potential use of Medicare to partially offset increased federal spending, critics question whether the measure, as detailed, would provide the touted boosts to research or instead open the way for companies to sell drugs and devices that are not fully tested.

Pressure on the FDA to revise its drug and device approvals regulation is not limited to congressional chambers or the public sector, as industry is currently challenging regulations perceived to be onerous to off-label usage of drugs. Currently, a patient is free to mention to another patient that a drug’s clinical trial is an effective treatment for an illness or disease. The drug’s manufacturer, however, is not free to tell an individual or a doctor the same thing without facing criminal charges or civil liability. Drug manufacturers would like this to change. Earlier this month, Amarin Pharma filed a lawsuit against the Food and Drug Administration, hoping to resolve this issue in favor of the drug manufacturers.

Prior to the lawsuit, an FDA advisory panel refused to approve Vascepa (Amarin’s flagship therapy) for treatment of “mixed dyslipidenia” after the completion of a Phase III clinical study that  seemed to show that Vascepa safely reduced triglycerides levels in patients with mixed dyslipidemia. The FDA advisory panel made its recommendation because it reached the conclusion that any approval was postponed until results of a longer clinical trial of Vascepa that is focusing on whether the reduction of triglycerides levels in fact has positive cardiovascular benefits were available. In Amarin’s situation, it cannot say anything about the current clinical trial of its drug Vascepa and the effectiveness of the drug for off-label treatments.

The FDA has argued that the off-label ban is a necessary part of the agency’s drug approval process, which requires manufacturers to demonstrate efficacy for each intended use. Although the FDA investigates and the government regularly prosecutes pharmaceutical companies and their representatives that promote off-label uses of their drugs, once approved, doctors can prescribe it for any use, which the FDA recognizes as proper.

The debate continues regarding whether the FDA’s current policy hinders or protects public health. The government does have a substantial interest, but does a ban on off-label promotion of truthful statements about a drug advance said public health? In theory, drug manufacturers should always be mindful of statements made about a drug’s efficacy, whether for profit or defense, in future litigation. Certainly the FDA has a lot of work cut out for it if the 21st Century Cures Act passes or if the Amarin litigation forces it to revise its thinking on off-label usage.