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Study Reveals FDA History of Approving Opioids with Limited Scientific Data

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Key Takeaways

  • A new study found the FDA has a history of approving opioids for use with limited data.
  • 81% of the data accepted by the FDA excluded patients who could not tolerate the drug, had side effects, or didn’t experience benefits.
  • Many accepted trials for opioids designed for long-term use were short.

A new study has found that the Food and Drug Administration (FDA) has a history of approving prescription opioids with limited data. Currently, the U.S. is in the midst of an opioid crisis.

The study, which was published in the Annals of Internal Medicine, analyzed data from new drug applications for opioids submitted to the FDA between 1997 and 2018. The researchers analyzed 48 new drug applications, which were largely for new dosages of opioids or new formations.

Of the 39 new drug applications that were approved for treating chronic pain, only 21 had at least one pivotal trial to support its use. Most trials ran for a median of 84 days and enrolled about 299 patients. Researchers discovered that 81% of the drugs were approved based on study designs that excluded patients who could not tolerate the drugs, had early bad side effects, or didn’t experience many immediate benefits.

About 20% of the new drug applications for chronic pain pooled safety reviews, with seven of them reporting non-medical use, and 15 reporting patients who developed tolerance to the medication.

Eight of the nine medications that received new drug approval had trials that ran for only one to two days and enrolled around 329 patients.

“Between 1997 and 2018, the FDA approved opioids on the basis of pivotal trials of short or intermediate duration, often in narrowly defined pain populations of patients who could tolerate the drug,” the researchers wrote in the paper. “Systematic collation of important safety outcomes was rare.”

Opioids Basics

Opioids are a class of drugs that are used to reduce pain, according to the Centers for Disease Control and Prevention (CDC). Opioids include the following:

  • Prescription opioids: These can be prescribed by doctors to treat moderate to severe pain, but can also cause serious risks and side effects. Common prescription opioids include oxycodone (OxyContin), hydrocodone (Vicodin), morphine, and methadone.
  • Fentanyl: A synthetic opioid pain reliever, fentanyl is much more powerful than other opioids. It is approved for treating severe pain, usually pain associated with advanced cancer. Fentanyl is also illegally made and distributed in the U.S.
  • Heroin: This is an illegal opioid. Its use has increased across the U.S.

The Opioid Crisis

The National Institute of Drug Abuse (NIDA) calls opioid misuse a “serious national crisis,” noting that the total economic burden of prescription opioid misuse is $78.5 billion a year. That includes the costs of health care, lost productivity, addiction treatment, and involvement of the criminal justice system.

The U.S. is facing a record number of drug overdose deaths. In 2018, more than 67,000 Americans died of a drug overdose death—of those, almost 70% involved opioids, the CDC says.

The NIDA says the crisis began in the late 1990s when pharmaceutical companies reassured the medical community that patients would not become addicted to prescription opioid pain relievers. As a result, healthcare providers started to prescribe them more.

It soon became apparent that the medications could be highly addictive, and opioids overdose rates increased.

Now, the NIDA says that up to 29% of patients who are prescribed opioids for chronic pain misuse them, and between 8% and 12% developed an opioid use disorder. Up to 6% of those who misuse prescription opioids transition to heroin.

The FDA’s Role in the Crisis

“Despite the scope of America’s ongoing opioid epidemic, little is known regarding the FDA’s approval of new opioid products over the past two decades,” study co-author Caleb Alexander, MD, a professor of epidemiology at the Johns Hopkins Bloomberg School of Public Health, tells Verywell.

Alexander cites many methods used by the FDA as concerning, including that “among the trials of products approved for chronic pain, none extended beyond 84 days, despite the fact that many individuals take these medicines for much longer periods of time.”

Alexander argues that the FDA could more tightly regulate opioids before they’re approved. “The FDA has regulatory flexibility in the requirements they set for market access, and our findings suggest that the agency did not use this to require opioid manufacturers to produce more information about the safety and effectiveness of prescription opioids prior to market entry,” he says.

In the future, Alexander says the FDA can “improve opioid regulation by requiring manufacturers to produce more, and more relevant, information about the systematic safety and efficacy of opioids.” He recommends that the FDA require manufacturers to systematically assess known bad health outcomes associated with opioid use, as well as to stop relying upon ”enriched” trials that are “unlikely to reflecting true product effectiveness.”

 “The FDA should also improve guidance for manufacturers by explicitly providing information about the populations, duration of therapy, and efficacy and safety outcomes that should be measured in trials going forward,” Alexander says.

Finally, Alexander says, “the FDA should also re-label chronic opioids so that the labeling for these important products better reflects the conditions under which they have been studied for regulatory approval.”

What This Means For You

Prescription opioid abuse remains a problem in the U.S., where the FDA has a history of approving drugs based on short-term, limited data. If you’re prescribed opioids for pain, talk to your doctor about the potential for addiction and serious side effects.

In 2018, the FDA released a Strategic Policy Roadmap in which the agency vowed to ensure better prescription practices, support treatment for people who are addicted to opioids, and increase efforts to stop the illegal shipment of opioids into the country. The FDA also says that it plans to take steps to help with alternative treatments for chronic pain.

The FDA has released a public timeline of “selected activities” and “significant events” designed to address opioid misuse and abuse. More recently, the agency has approved Olinvyk (oliceridine), an opioid designed for the management of moderate to severe acute pain in adults.

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  1. Heyward J, Moore TJ, Chen J, Meek K, Lurie P, Alexander GC. Key evidence supporting prescription opioids approved by the U.S. Food and Drug Administration, 1997 to 2018. 29 September 2020. doi:10.7326/M20-0274

  2. Centers for Disease Control and Prevention. Opioid basics. Updated March 16, 2020

  3. National Institute of Drug Abuse. Opioid overdose crisis. Updated May 27, 2020.

  4. Centers for Disease Control and Prevention. Understanding the epidemic. Updated March 19, 2020.

  5. U.S. Food and Drug Administration. Health innovation, safer families: FDA's 2018 strategic policy roadmap. Updated January 2018.

  6. U.S. Food and Drug Administration. FDA approves new opioid for intravenous use in hospitals, other controlled clinical settings. Updated August 7, 2020.