Earlier this year, I talked about the challenges 21st Century Cures Act was facing in Congress. Since then, the Health, Education, Labor and Pensions (HELP) Committee, led by Sen. Lamar Alexander, scrutinized the legislation inch by inch and in December 2016 this bipartisan bill passed the U.S. Senate. On the 13th of December, President Barack Obama signed the bill into law and remarked that with this Act, much-needed medical breakthroughs will become a reality.

The 21st Century Cures Act marks a landmark in the labyrinth that is the U.S. healthcare system. The Act aims to combat heroin and opioid abuse, offer support for research in precision medicine and through the BRAIN Initiative (Brain Research through Advancing Innovative Neurotechnologies), improve mental healthcare, streamline Food and Drug Administration (FDA) design, and further advance the Cancer Moonshot program. A budget of $6.3 billion for the Act includes $1 billion funding for opioid epidemic over a span of two years and $4.8 billion funding for National Institutes of Health (NIH) which will be used to boost biomedical research for over a decade as well as $500 million for the FDA to improve regulatory processes.

With the Cures Act in place, the FDA market authorization process stands to change in some notable ways. For instance, beyond randomized clinical trials, pharmaceutical companies would be increasingly allowed to present real-world evidence such as observational studies and safety monitoring data for drug authorizations. Patient advocacy groups and associations had long been pressurizing the government to encourage innovations and accelerate approvals; allowing patient experience data to be considered for drug reviews gives voice to these patients. On the other hand, some critics argue that this modification undermines the significance of randomized clinical trials, which are considered the gold standard in evaluating drug efficacy and safety.

The Cures Act also directs FDA to develop accelerated reviews for breakthrough medical devices and regenerative medicines. The agency would also be required to conduct shorter trials for new antibiotics and antifungals. Mobile software falling under the administrative, general wellness, health records, medical devices data and support umbrella would be exempted from the scope of FDA. The Act contests that these measures would facilitate faster patient access. It aims to modernize FDA drug development design in a way that everyday changes in biomedical technology are better adapted.

This Act comes well in time for Christmas after having struggled through numerous gatekeepers. It still remains as controversial among some stakeholders as it was at the time of inception. Guaranteed funding was one of the crossroads that divided the Senate. Approximately 55% of the Act’s total budget would be funded through cuts from Prevention and Public Health Fund. Critics are suspicious of this provision and continue to speculate about the assurance of funds as well as the support of the new government in the coming years.

Nonetheless, the Cures Act is a massive reform, probably the biggest since the Affordable Care Act (ACA). The Cures Act appears enterprising and even though the FDA reforms might take some time to be fully implemented, the pharmaceutical industry has a host of relaxed norms to look forward to.

Blog# 1 in series: https://decisionresourcesgroup.com/drg-blog/as-rare-disease-day-approaches-key-legislation-faces-challenges-in-congress/

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