The seemingly effortless passage of the 21st Century Cures Act in both the House and the Senate quickly received overwhelming praises from the biopharma industry. Now that President Obama has already signed it into law, the proposed changes made to the clinical research and drug approval process still await detailed guidance from regulatory authorities.
Though the passage of the Act—regarded as the most significant healthcare reform law in the U.S. since the Affordable Care Act—seemed easy, given the constant fight between the two Parties, it is the result of huge lobbying effort. According to data gathered by the Center for Responsive Politics, an organization dedicated to checking campaign contributions, all together 420 organizations lobbied on this bill, including almost every major pharmaceutical and medical device company—Roche, Sanofi, Merck & Co., Pfizer, AstraZeneca, GlaxoSmithKline, Boston Scientific, Medtronic, you name it. So, it is no surprise that the Act offers new clinical tools that favor the industry.
Even before it reached the Senate, concerns that the Act would allow more real-world evidence based on patient experience into regulatory decision-making process and more leeway for surrogate endpoints into clinical trials were raised among patient safety advocates, who argued that it will corrode the FDA’s oversight over the drug approval process and jeopardize current approval standards.
“There are provisions to this bill that weaken the authority of the FDA, that lower the standards of evidence for the efficacy of drugs and reduce the ability of the FDA to protect the safety of drugs and medical devices,” Steve Nissen, head of the cardiovascular division at the Cleveland Clinic said, as quoted by NBC News in a story after the House passed the bill in 2015. The National Center for Health Research, a strong opponent to the Act, was also quoted as saying that “to be a 'cure,' a treatment should actually help patients, not just enrich stockholders.”
But John Lewis, SVP of policy & public affairs at the Association of Clinical Research Organizations dismissed such concerns as “either don’t understand, or have intentionally mischaracterized, not only the intent of Cures but the actual legislative language.”
“We interpret Cures as providing the FDA with greater discretion in what types of evidence it reviews and how it evaluates evidence but see nothing in the bill that directly lowers standards for efficacy and safety if properly implemented,” he added.
The provision can be especially helpful for personalized medicine and those meant for treatment of life-threatening rare diseases, where randomized clinical trials might find it difficult to collect sufficient data. And the Act is very articulate about setting guidelines on circumstances where clinical experience can be relied on as a supplement to clinical trials and the appropriate standards and methodologies for collecting and analyzing such evidence.
What’s more, the Act will also remove differences between two regulations—the HHS Human Subject Regulations and the FDA Human Subject Regulations--protecting human subjects in researches, and promotes that single or central independent institutional review boards should be more broadly used.
To Lewis, the new Act doesn’t necessarily mean a revolution to the CRO industry, as many CROs have already been using innovative clinical trial designs, “so additional guidance in this area will only bring about more efficiencies and breakthroughs,” he said. But he admitted that more work still needs to be done in this emerging area.
Another potential perk for the CRO industry comes from more financial support for the NIH. About $4.8 billion additional funding, subject to annual appropriations, will go to NIH over 10 years, including $1.8 billion for the Cancer Moonshot initiative. Since NIH itself sometimes outsources and funds clinical researches, it means more business opportunities for CROs.
However, Lewis thinks that the much bigger issue for CROs is the planned enhancement to the clinical trial database to make it more user-friendly and searchable. The vision of ACRO is an improved version of clinicaltrials.gov being integrated with electronic health records to help recruit patient for clinical trials.
Delays in patient recruitment has been a big obstacle to conducting clinical trials. About two-thirds of clinical studies fail to meet patient enrollment goals, according to a 2011 report by the Tufts Center for the Study of Drug Development. “[T]here is no excuse why a patient or their physician could not find a clinical trial through the registry if they are willing to participate,” said Lewis.
As instructed by the Act, HHS will hold a meeting with stakeholders within 90 days after the enactment of the Act to “receive advice on enhancements to the clinical trial registry data bank,” including improvements to its usability, functionality and search capability.
Now that the bill has already been made official with President Obama's signature, Lewis said the organization will keep a close eye on future guidance and regulations that will come out of it.
Editor's Note: This story has been updated at the news that President Obama has signed the Act into law.