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Using Real-World Evidence to Accelerate Safe and Effective Cures

Thursday, June 23, 2016

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https://cdn.bipartisanpolicy.org/wp-content/uploads/2016/06/BPC-Health-Innovation-Safe-Effective-Cures.pdf

Unparalleled advances in science and technology during the past two decades have resulted in some stunning medical breakthroughs, such as the cure for Hepatitis C and new, more effective cancer treatments based on patients’ genetic profiles.

Science is moving swiftly, but there are still significant unmet needs. An estimated 5.3 million Americans suffer from Alzheimer’s disease, for which there is still no prevention or cure. Heart disease remains the leading cause of death in the United States, killing nearly 800,000 people in 2011 alone.

In fact, for the approximately 10,000 known molecular-based diseases, there are approved treatments for only 500 of them. The urgency of finding the next generation of cures is clear.

Modernizing the drug discovery, development, and approval of new cures and treatments plays a critical role in addressing this issue and should be a key priority for the United States. It takes too long and costs too much to bring treatments and cures to patients in need. The most reliable studies suggest that on the average it costs approximately $2 billion and takes more than a decade for a new drug to reach the market.

While the United States has invested more than $1.5 trillion in research and development (R&D) over the past two decades, the level of R&D efficiency (number of drugs approved per billion dollars of R&D spending) has declined.

The digitization of biology and the vast increase in the amount of data captured during routine care creates an unprecedented opportunity to modernize clinical trials.

Both Congress and the administration have taken steps to address this issue. The U.S. House of Representatives nearly unanimously passed the 21st Century Cures Act in 2015. The Senate Health, Education, Labor, and Pensions Committee completed its mark-up of bipartisan legislation earlier this year and is negotiating to advance a medical innovation package in the Senate in 2016. Negotiations between industry and the Food and Drug Administration are now well underway in preparation for the reauthorization of drug and device user fee legislation in 2017. Finally, the administration’s Precision Medicine Initiative is making considerable progress, as is the vice president’s Cancer Moonshot launched earlier this year.

Modernizing the drug discovery, development, and approval process was also the focus of the Bipartisan Policy Center’s July 2015 report, Advancing Medical Innovation for a Healthier America.

The digitization of biology and the vast increase in the amount of electronic data captured during routine care, or by patients themselves, creates an unprecedented opportunity to modernize and augment clinical trials and improve the post-market monitoring process. The need to advance the generation and use of “real-world evidence” was one of the key recommendations of BPC’s July 2015 report. This report highlights opportunities to modernize and improve the current drug development process through the use of real-world evidence, as well as the policy actions needed to realize those opportunities.

KEYWORDS: FOOD AND DRUG ADMINISTRATION, 114TH CONGRESS, SENATE HELP COMMITTEE, ALZHEIMER'S, 21ST CENTURY CURES

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