The 21st Century Cures Act doesn't go far enough in accelerating the development and delivery of new medical treatments, a group of former lawmakers and patient advocates said Monday.
A new report released Monday by a Washington D.C.-based think tank, the Bipartisan Policy Center, found that $1.5 trillion invested in research and development over the past 20 years resulted in fewer drugs actually coming to market compared to the 1950s.
“It's clearly time to take action to significantly advance medical innovation in the United States,” said former Senate Majority Leader Dr. Bill Frist, a senior fellow at the center, on Monday.
The report focuses on four key policy issues aimed at reducing the length of time and the cost it takes to develop new medical drugs and devices, a process which the report found can take more than a decade and up to $2 billion to complete.
Some of the recommendations made in the report called upon Congress to establish policies that sought to modernize the regulatory framework by which new drugs are tested and approved.
A number of those concerns were addressed when the House passed the proposed 21st Century Cures Act, but the report calls for the additional step of requiring the FDA to develop a framework that would allow for the incorporation of randomized studies that fell outside of the traditional Phase III clinical trial model to be considered for a product's approval.
The report also called for policies that encouraged the development of more public- and private-sector partnerships, and to greater use of patient-reported outcomes. Partnerships between the biopharmaceutical industry and other stakeholders toward the development of new drugs have become increasingly popular as a means of addressing concerns of increasingly high costs of breakthrough treatments.
According to the report, private-sector investment in medical innovation in the U.S. declined by nearly $13 billion between 2007 and 2012, resulting in a decline in the U.S. share of the world's total R&D investment from 50% to 44% over that period.
“We are all well aware of the ability of the government to either incentivize or stifle industry growth and advancement,” Frist said. “You have to have that partnership of government and the private sector working hand and hand to maximize innovation.”
Much like in the Cures bill, the report calls for greater use of biomarkers to make determinations on the efficacy of drugs, an issue some of the bill's critics have argued raises the risk of an adverse health event.
The report also calls for Congress to increase incentives for drugmakers to develop products for unmet clinical needs.
But unlike the six-month extension of market exclusivity for some drugs proposed in the bill, the report recommends the FDA designate a new “dormant therapy” pathway. This would allow approval for drugs that treat an unmet medical need if they don't contain active ingredients already approved by the FDA.
