ACMG Issues Policy Statement to Address Affordability of Treatments in Rare and Ultra-Rare Disorders

Rare Daily Staff

The American College of Medical Genetics and Genomics, a professional membership organization representing all members of a medical genetics team, said a national policy on controlling drug costs was “urgently needed” to ensure their affordability.

In a policy statement published in the journal Genetics in Medicine, the ACMG board highlights a set of points to consider. It suggests making modifications to the Orphan Drug Act to prevent abuses, aligning drug packaging with dosing recommendations to avoid waste, allowing the federal government to negotiate drug prices, and preventing the use of “pay-for-delay where drugmakers provide financial incentives to potential competitors to slow the entry of generic drugs coming to market.

The policy statement does not contain any novel or radical suggestions. Instead, it draws on a growing body of work from professional organizations and seems to pick and choose from those with an eye towards rare and ultra-rare disorders. Among the points it calls for is a cap on patient’s out-of-pocket costs for specialty drugs.

“It will be important to get the costs of rare disease drugs under control as more and more increasingly narrowly targeted molecular treatments in subsets of patients with a disease are becoming available or are in late stage clinical trials,” said Michael Watson, executive director of the American College of Medical Genetics and Genomics. “While the target group is often small, the financial impact of the cost to healthcare may be low. In aggregate, the treatments for many rare genetic diseases will be substantial to society and to patients which can limit their development and accessibility if the problem isn’t addressed.”

The organization said potential solutions should be “affordable, simple, and transparent,” and that cost-containment efforts should consider the burden address on the entire health care system as “high prescription drug prices may be shifted and absorbed in ways that negatively impact patient and prescriber access to important medications.”

September 10, 2018
Photo: Michael Watson, executive director of the American College of Medical Genetics and Genomics

 

X