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A view shows the U.S. Food and Drug Administration (FDA) headquarters in Silver Spring, Maryland August 14, 2012. To match special report USA-FDA/CASES REUTERS/Jason Reed/File Photo - RTX2OO3V
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“Right-to-try” warrants FDA oversight for experimental treatments

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While the American Health Care Act failed to garner enough congressional support, efforts promoting healthcare deregulation continue to present challenges to the status quo. All fifty states either have passed or are considering “right-to-try” laws that would allow terminally ill patients to acquire experimental drugs that are in the process of Food and Drug Administration approval.

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Currently, terminally ill patients and their doctors can petition the FDA for access to experimental drugs after a drug manufacturer confirms their willingness to provide the medication. The drug must have passed Phase I clinical trials that demonstrate safety, but not efficacy. Right-to-try laws propose to bypass the FDA and allow patients to acquire pre-approval drugs from manufacturers when they have exhausted all other options. The debate centers on whether patients should need government approval to decide how to manage terminal illness. Evaluating right-to-try laws requires considering which institutions limit experimental drug access and what safeguards are appropriate in individual decision-making.

FDA Approval Process

The FDA allows terminally ill patients to apply for access to experimental drugs currently in clinical trials through the Expanded Access program. However, proponents of new legislation claim that a single application takes physicians 100 hours to complete. They argue that the excessive paperwork and the 30-day delay that the FDA requires before doctors can begin experimental treatment dissuade patients and doctors from petitioning. In response to these complaints, the FDA streamlined the application process in 2015 and moved to provide expedited approval over the phone in case of emergencies. A 2016 report from the NYU School of Medicine found that the new application takes doctors approximately 45 minutes to complete.

While critics argue that less than one-tenth of one percent of terminally ill patients receive pre-approval medication, the concern over access is misplaced. Physicians request experimental drug access from the drug manufacturer before petitioning the FDA. The FDA approves nearly all expanded access applications it receives, indicating that drug manufacturers limit experimental drug access more than government regulation.

Additionally, clinical trials include an institutional review board (IRB) that weighs the risks and benefits of the proposed trial and reviews treatment protocol to ensure the study will protect participant welfare. The same safeguards should apply to patients that are going to receive investigational drugs on an individual basis as a matter of equal access as those in clinical trials. The FDA requires similar IRB review when considering pre-approval access, while right-to-try laws make no mention of panel reviews. With the quickened approval process, the FDA maintains patient safety and provide timely assessment, while new legislature fails to address important oversight concerns.

The Manufacturer Bottleneck

Drug manufacturers are wary of providing pre-approval drug access for a number of reasons. To receive FDA pre-approval access, terminally ill patients must be ineligible to participate in a drug’s clinical trial. Eligibility criteria may include age, gender, health status, and medical history. This provision both ensures that pre-approval access does not delay a trial’s timeline and promotes equal access to potentially life-saving treatments for ineligible patients. During clinical trials, drug manufacturers often stock only as much medication as is necessary for the investigation. Furthermore, manufacturers rarely charge for medication provided under expanded access programs, likely because any financial barrier to treatment would draw criticism from advocacy groups.

A recent industry analysis found that 63.2% of clinical drugs that enter Phase I trials succeed into Phase II, while less than 10% of these drugs ultimately receive FDA approval. From a manufacturer’s standpoint, providing free medication before the FDA has approved its safety and efficacy might have large financial and social implications if the drug is ineffective or has adverse outcomes. If a patient suffers adverse effects after pre-approval drug use, other patients might be unwilling to participate in clinical trials or purchase the medication despite FDA approval. The manufacturer would incur larger financial burden in larger costs to fund their trials and potentially decreased revenue.

Efforts to increase the number of terminally ill patients receiving investigational medication should combat the obstacles and risks manufacturers’ face in this dilemma.

First, do no harm

Consider the unfortunate scenario in which a terminally ill patient suffers because of an investigational drug provided through pre-approval access. Right-to-try laws both reduce oversight and free doctors and drug manufacturers from liability. This combination seems rife for exploitation. The FDA expanded access program ensures that doctors properly weighing the risks and benefits before recommending investigational drug use, and removing this oversight would negatively impact patient safety.

Furthermore, insurance companies and drug manufacturers might find a lucrative opportunity to encourage doctors to prescribe pre-approval drugs to vulnerable populations. Under pressure from state legislatures and advocacy groups to expand access, drug manufacturers might charge fees to cover increased production and offset the additional risk. Insurance companies might step in to cover these expenses and shift the cost to the public by raising premiums or consolidating them in high-risk pools. This situation would allow private companies to benefit while increasing healthcare costs for many Americans.

Despite support from the current administration, right-to-try arguments for bypassing the FDA are misplaced because the agency has quickened the approval process and grants 99 percent of petitions. Drug manufacturers continue to limit drug access because of the scarcity of available medication during clinical trials and large potential risks. The right-to-try movement has made advances across the country in recent years, but proponents must consider its purported values and refine its model legislation to protect patient safety.

Dan Bernstein contributed to this post.

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