As the dust settles on Cures, what will it mean for biopharma R&D?

congress

At almost 1,000 pages, the 21st Century Cures Act is quite literally a weighty piece of legislation that will have far-reaching consequences for pharma in 2017 and beyond.

After months of political wrangling, Cures was eventually signed into law by President Obama as his term in office was drawing to a close, and provides $4.8 billion for three of his administration's flagship projects: Vice President Joe Biden's cancer moonshot, the BRAIN Initiative, and the Precision Medicine Initiative. Another $1 billion is earmarked to fight the opioid epidemic, and the FDA gets an extra $500 million.

Behind the headline dollar figures lie some pretty drastic changes for pharma R&D. Cures aims to speed up the times it takes new medicines to get to patients, embed patient groups more firmly in the FDA review process, and—more controversially—allow urgently needed new drugs to be approved using different standards from those used at the moment.

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Among the measures laid out in Cures is greater use of surrogate rather than clinical efficacy measures in trials, making them smaller, quicker and cheaper to run, and the adoption of new forms of evidence for efficacy such as real-world data from patient registries and electronic health records.

Regulatory shortcuts for regenerative medicines such as stem cell therapies are also in play, with companies now able to apply for a new "regenerative advanced therapy" designation for their candidates that automatically gets them on the FDA's accelerated approval track, for example. Meanwhile, Cures also introduces a new vehicle for the FDA to approve new antibiotics and antifungals—and possibly other drugs—using smaller studies. If approved, the new medicines would be labelled as being rested in a "limited population."

There has been no shortage of praise for the legislation from industry and patient groups, who are hailing it as an important modernization of FDA's approval process that will—in the words of the Biotechnology Innovation Organization (BIO)—"help expedite the development of the next generation of breakthrough medicines to save lives and reduce suffering for millions of patients, while helping to lower other healthcare costs."

Critics have made their voices heard too however. Consumer rights group Public Citizen insists that the law is an "early Christmas present" for the pharma, biotech and device industries, weakening the FDA's approval standards and undermining patient safety. It says it is particularly concerned about allowing real-world evidence in drug approvals—favored by current FDA Commissioner Robert Califf—and clauses in the Act allowing companies to submit summaries of study data, rather than full clinical trial records.

Many of these concerns will play out as the bill is implemented through 2017 and beyond, but let's not forget the FDA won't play a passive role in the process. Some of the provisions in Cures will require it to develop new guidance and regulatory tools, but most—such as accelerated approval—are covered by existing authorities and the agency retains significant leeway when interpreting the new legislation.

In the meantime, drug sponsors will still have to work with the agency to work out how these new clinical tools can be incorporated into registration trials in practice. The Association of Clinical Research Organizations (ACRO) says it does not believe Cures will lower approval standards, but gives the FDA "greater discretion in what types of evidence it reviews and how it evaluates evidence."

Califf insists that with Cures the FDA can "speed the discovery, development, and delivery of medical products to prevent and cure disease and improve health while sustaining the evidence framework that enables assurance to the public of the safety and effectiveness of medical products."

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