Legislation designed to make new therapies accessible to patients more quickly has been passed by the US Senate, with just a signature by President Obama now required before it comes into law.
The US Senate voted 94 to 5 to approve the 21st Century Cures Act, a wide-ranging and controversial bill that will shake up the operations of the FDA and lead to the introduction of new mechanisms to speed the approval of new pharmaceuticals and medical devices.
As examples, it could allow a drug that has been approved in one indication a truncated approval route for a second, greater reliance on surrogate rather than clinical efficacy measures in trials, and the adoption of new forms of evidence for efficacy such as real-world data.
From those changes stem the controversy, with critics of the bill – including consumer group Public Citizen – arguing that it panders to the pharma industry and lowers the bar for proving safety and efficacy before new medicines can reach the market. There are also concerns about a new accelerated approval pathway for regenerative medicines such as stem cell therapies.
The agency gets an extra $500m over the coming five years to help fund the changes and the Act also commits funding to three major projects introduced under Pres Obama’s administration – the Precision Medicine Initiative, Cancer Moonshot and the BRAIN Initiative seeking treatments for diseases such as dementia and epilepsy as well as traumatic brain injury.
In his weekly address, President Obama said the new bill is “an opportunity to save lives, and an opportunity we just can’t miss”, adding: “I’ll sign it as soon as it reaches my desk.”
The Biotechnology Innovation Organization (BIO), which represents biotech companies, welcomed the news, saying the new act “takes important steps towards placing patients at the centre of the drug development process”.
BIO said it establishes “a framework for incorporating patient views into the development and regulatory review process in a more structured and transparent way, both with respect to patient input for benefit-risk assessments and use of patient experience in data in regulatory decision-making.”