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  • BIOETHICS FORUM ESSAY

The 21st Century Cures Act Sparks Values Debate

On December 13th, President Obama signed the 21st Century Cures Act, a bipartisan, multidimensional health research and development bill.  The act allocates $4.8 billion to the National Institutes of Health and $500 million to the Food and Drug Administration over the next 10 years for research and treatment development, and mandates reforms of clinical trials and drug approval and access.  The law provides support for the Obama administration’s major science programs – the Precision Medicine Initiative, BRAIN Initiative, and the Cancer Moonshot Project – and funds initiatives focused on mental health and the opioid epidemic. In addition, the bill introduces reforms for clinical trial regulations, drug and medical device review and approval pathways, and Medicare and Medicaid reimbursements. Many of these provisions are double-edged swords – while advancing treatments, they could reduce regulatory authority, threaten patient safety, and raise other ethical issues.

The White House called the bill “critically important.” Though Donald Trump has not said anything about the act, it is likely to survive in his administration given its strong bipartisan support in Congress (218 Republicans and 174 Democrats voted for it in the House, and 52 Republicans, 41 Democrats and one Independent voted in its favor in the Senate). However, Representative Tom Price, Trump’s nominee for Secretary of Health and Human Services, voted against the bill and is particularly opposed to the mandatory funding of the NIH and FDA programs.

While the act’s mandated reforms could take several years to take shape, it raises social and ethical considerations at this time. Critics cite funding cuts to the Prevention and Public Health Fund, which provides resources to several public health and preventative medicine programs nationwide. Frequently discussed are provisions that bring new drugs and devices to the market faster, with limited information from clinical trials about their safety and effectiveness.  The law would allow manufacturers of drugs for serious rare diseases – specifically, drugs that incorporate a genetically targeted, or “precision” technology– to use data from similar, previously approved drugs for their applications, rather than gathering robust data on the new drug itself. This provision highlights a complicated tradeoff between faster drug approvals and patient safety.

The Cures Act’s passage joins an existing debate over standards of evidence in health care. As scholars have noted, America’s health care industry has moved steadily towards a model in which evidence-based decision-making is given less weight than faster innovation (see recent commentaries on this issue by Hastings Scholars Karen Maschke and Michael Gusmano here and here).  This “evidence gap” has been attributed to political and industry-based pushes to bring life-saving treatments to market faster, which requires relaxing the traditional standard of robust clinical trials and empirical research.  The 21st Century Cures is an example of this effort.

The discussion of an “evidence gap” is itself divisive.  Those who speak of an evidence gap believe that more evidence allows for more informed decision-making, which in turn is indicative of better care.  From this perspective, standards of evidence have become increasingly inadequate and are likely to result in the approval of medical technologies that don’t add value, or that result in harm.  On the contrary, advocates of “faster” developments believe that medical advances are the key to better care and that the evidence thresholds for approving drugs and devices are too high.  In this view, the existing standards are likely to block vital innovations from reaching the market.

Stemming from this dichotomy, an ethical issue arises.  It may seem obvious that physicians, health policy makers, and others working in health care have an ethical obligation (fiduciary or otherwise) to provide only safe treatments to their patients.  And yet, many patients fought for the passage of 21st Century Cures and faster approval pathways for good reason.  Faster drug approval, to these patients, seems synonymous with access to much needed treatments and potential cures.

Of course, the involvement of the pharmaceutical industry further complicates the matter.  It is clear that the industry’s interests were heavily served in the drafting of the law. The patients who need 21st Century Cures most are those suffering from rare diseases. For them, any treatment, even one that is “experimental,” is critically important.  Is the movement towards innovation essential, or exploitative? Advocates of the law assert that its revision of evidence standards is critical both for the health of patients and for the safety of the market.  Yet the FDA has existing accelerated approval pathways for the rare disease drugs that the Act intends to speed up development for.   Opponents believe the law instead represents an effort to advance drugs to market on far lower standards of evidence in favor of private industry’s profits.  Underlying this view is a value judgement of the possible harms of approving drugs that are ineffective or unsafe, versus the harm of not approving a safe and valuable drug when it first becomes available.  Does delaying the approval of treatments evoke a sense of medical paternalism, or ethically responsible care? Who should weigh these risks – the government, industry, or the public?  Is it ethical for the government or industry to make such judgements, if it is the public who must bear their consequences?

Another provision of the law has piqued the interests of bioethicists, specifically those focused in the genome sciences.  Sections 2012 and 2013 provide protections for research subjects whose biological data could be identifiable. These provisions are a welcome step forward for researchers and regulators alike – they are an active shift towards ensuring safer and more ethical genomic research, and in turn, could provide more participants for such research.

The text of the law provides several circumstances in which identifiable information from research participants cannot be disclosed. Matters concerning insurance were not explicitly mentioned.  Depending on the interpretation of the provision (which I assume a court will be asked to give at some point), insurance could be either included or excluded.  This is important, when combined with the Genetic Information Nondiscrimination Act of 2008 (GINA).  GINA protects people’s genetic information by prohibiting insurance companies and employers from accessing data about people’s health or disease predispositions.  A hole in this law, which some bioethicists have argued against, is that life insurance companies may still use genetic information in their formulation of quotes.  By not explicitly including insurance, 21st Century Cures upholds this loophole.  Some believe this is sensible – the life insurance market could not survive without an understanding of its risk pool, for which genetic predisposing to late-onset diseases are critically important.  Others believe it exposes people to unethical discrimination about their genetic information. This debate, relatively dormant since the passage of GINA, is alive once more.

The questions highlighted by 21st Century Cures draw on a wider debate of the interactions of government, industry, and academia in science research and health care.  Under the Trump administration, the law will provide critical funding of NIH, the FDA, and major life science research initiatives.  I sincerely hope that these efforts continue with a true commitment to and consideration of good science, robust evidence, and normative values.

Rachel Zacharias is a project manger and research assistant at The Hastings Center.

Published on: December 22, 2016
Published in: Clinical Trials and Human Subjects Research, Health and Health Care, Medicine & Business, Pharmaceutics

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