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21st Century Cures: Myth-Fact

Jul 27, 2015

"Woodcock said that drug-resistant infections have reached the level of a ‘societal crisis’ and that the bill would help spur the development of treatments for patients who might die of their infections otherwise. She said the standards of efficacy remain the same, although the tolerance for uncertainty about the risks and benefits of a treatment increases when there are no other options." – The Washington Post, July 10, 2015

"The 21st Century Cures Act does not reduce safety standards. Nothing could be further from the truth: the FDA’s authority for maintaining the gold standard for safety and effectiveness remains unchanged. Rather, the bill will allow the FDA to utilize today’s rapidly evolving science to support the standard, while explicitly prohibiting the agency from approving a drug based on a lower standard of evidence." – Former FDA Commissioner Mark McLellan, Katie Couric, and Founder and Chairperson of Friends of Cancer Research Dr. Ellen Sigal in The Huffington Post

"The 21st Century Cures legislation makes clear that Congress will continue to demand vigilance and rigor, in addition to efficiency and consistency, from the FDA. In return, a modernized regulatory process can assure every American that they will have access to the medical products they need as rapidly and safely as possible, using the best technology available." – Former FDA Commissioner Andy von Eschenbach and Director of the Manhattan Institute’s Center for Medical Progress Paul Howard in The Morning Consult

MYTH: 21st Century Cures threatens safety standards for patients

FACT: Some critics have raised unsubstantiated and misleading attacks on a variety of provisions included in the bipartisan 21st Century Cures Act (H.R. 6), legislation that was overwhelmingly approved by the House of Representatives in June. These critics have argued that some provisions of H.R. 6 will lower, diminish, or undermine the safety or efficacy standard used by the Food and Drug Administration to approve drugs and devices.

These attacks have been debunked by a number of experts inside and outside of government, including the more than 700 groups supporting the legislation, and also run counter to the initiative’s goal and process. Goal: to help patients live longer and healthier lives. Process: transparent and inclusive, including close work with FDA officials to ensure all policies included in the bill maintain the highest standards of safety and efficacy.

MYTH: H.R. 6 will lead to substandard data being used during the drug review process at FDA.

FACT: Some critics have raised this charge in relation to Section 2021 (Qualification of Drug Development Tools) and Section 2062 (Utilizing Evidence from Clinical Experience). These charges are completely false. 

Section 2021 would codify a structured framework for the submission, review, and qualification of biomarkers and other drug development tools for specific contexts of use that, if qualified, can be relied on by any person for such purposes. The legislation allows the Secretary of Health and Human Services to consult with outside experts and consider their recommendations throughout the review of a qualification package submitted under this framework.

In addition, section 2021 would require the Secretary to make public information related to each submission, all drug development tools qualified, and all surrogate endpoints that were the basis of approval or licensure of a drug or biological product, while maintaining the protections currently in place for confidential commercial or trade secret information contained in an application submitted outside the public qualification process. 

This provision does not in any way alter the safety and efficacy standard used by the FDA during the drug review process. Rather, this section codifies a structure to ensure that there are clear standards and a process in place regarding when data derived from a drug development tool can be used during the drug review process. 

It is important to note that this provision will expand and enhance FDA’s understanding of both the benefit and the risks of a drug by leveraging drug development tools, including taking advantage of 21st Century technological advancements to more precisely judge the benefits and risks of a drug.  Critics of this provision are effectively advocating against a framework that would advance the use of modern and scientifically valid measurements to help understand how drugs will work for patients.

Section 2062 would require the Secretary to establish a program to evaluate the potential use of evidence from clinical experience to help support the approval of a new indication and to help support or satisfy post-approval study requirements. In parallel, FDA would identify and execute pilot demonstrations to extend existing use of the Sentinel System to, among other things, support these efforts.

Again, this section does not in any way alter the standards of evidence required by law or alter the Secretary’s authority to require post-approval studies or clinical trials.

Instead, this provision will advance the inclusion of scientifically validated information on a drug’s label for different indications. It would also advance the collection of post-market safety information for the FDA so the agency has a better understanding of how a drug is working post-approval.  Critics of this provision are effectively advocating against improved labeling and safety information for patients. 

MYTH: The passage of the ADAPT Act within 21st Century Cures "would allow a drug to be tested on humans based on only limited evidence that it is safe and effective." (Source: July 20, New York Times editorial)  

FACT: The increasing prevalence of antibiotic resistance in humans is an ever-increasing public health threat. In 2014, the World Health Organization issued a report that began, "Antimicrobial resistance (AMR) within a wide range of infectious agents is a growing public health threat of broad concern to countries and multiple sectors. Increasingly, governments around the world are beginning to pay attention to a problem so serious that it threatens the achievements of modern medicine. A post-antibiotic era—in which common infections and minor injuries can kill—far from being an apocalyptic fantasy, is instead a very real possibility for the 21st century." (WHO: Antimicrobial Resistance: Global Report on Surveillance, 2014)

Everyone from the President of the United States to global health organizations like the PEW Charitable Trusts have sounded the alarm bell on the issue of drug resistance and called for congressional passage of a pathway that helps support global researchers and developers in their efforts to speed up new drug development to address this public health crisis. The 21st Century Cures Act answers this call with the inclusion of the ADAPT Act. This policy would help support new drug development for new antibiotic drugs that treat unmet medical needs as determined by the FDA.

Arguments that this provision of law lowers the safety and efficacy standards at the FDA lack merit and threaten the wellbeing of patients. This provision clearly states that drugs approved under the pathway would have to meet the same safety and efficacy standards the FDA requires for any other drug under existing law. The FDA would, however, be able to consider the risks and benefits to patients with few, if any, treatment options available when determining candidates for a streamlined approval process. Such considerations shall ensure that patients who might otherwise lack a treatment option are able to now potentially find a treatment or cure. Such careful consideration by the FDA will also allow the FDA to restrict the use of such drugs to those with unmet medical needs. 

MYTH: "The bill reverses part of the transparency push about pharmaceutical companies paying doctors." (Source: July 14, Vox piece)

FACT: American medical professionals are the best in the world because our medical societies and health care system expect that they remain up-to-date on the latest in new treatments and technologies. Much of the funding for these events come from third parties and others interested in educating providers on the latest in medicine. However, some have warned that the Sunshine Act, established in the Patient Protection and Affordable Care Act passed in March 2010, threatens such opportunities in the future because of its potential to dissuade public and private investments that support such educational opportunities for medical professionals.

The 21st Century Cures Act would ensure that our best and brightest can continue to receive the best in medical education.       

MYTH: "The bill would weaken the F.D.A.’s already flimsy regulation of medical devices, posing a threat to future patients who have devices implanted that cannot easily be removed if found defective." (Source: July 20, New York Times editorial

FACT: Once the undisputed leader of global innovation in medicine, the U.S. has been losing ground to Asia and Europe and could lose its place in the coming years if we do not establish ways to improve the process. The 21st Century Cures Act recognizes this and puts in place a number of policies to modernize and improve the process and resources FDA is required to expend. Fundamental to patient care is access to new and life-saving treatments. With a finite number of resources, developer’s ability to invest in new products is in large part dependent upon the efficiency of Washington’s regulatory pathways. It is Congress’ job is to support the search and adoption of new and innovative ways to work with developers so that safe and effective products can be brought to market for effective patient care. 

21st Century Cures supports the ability of developers and the FDA to do their business in the most efficient means possible. New and emerging technologies have opened the door to new ways in which developers can gather and utilize information to support the FDA safety and effectiveness standard for approval. But some valid sources of data are not often recognized by the agency today. 

The 21st Century Cures Act also requires that the FDA conduct regular education of its employees about methods to conduct its review and approval work in the most efficient and least burdensome manner possible.  Such provisions can ensure that both the FDA and industry utilize their time and dollars as efficiently as possible while maintaining the very best in quality and safety standards. 

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