Wednesday, August 23, 2023 5:24:12 PM
by Megan Redshaw, J.D.
Aug 10 2023
The 21st Century Cures Act (Cures Act), signed into law in December 2016, was created to help accelerate medical product development and “bring new innovations and advances” to patients quicker and more efficiently. Yet some researchers suggest the law is being used to bypass the once rigorous and evidenced-based standards for new drug approvals, allowing novel drugs to flood the market without adequate data and public transparency.
According to a research letter published on August 8 in the Journal of the American Medical Association Network Open (JAMA), 24 of the 37 drugs approved in 2022 by the U.S. Food and Drug Administration (FDA) were based on a single study, with only four drugs having more than three studies to support their approval.
“I’m not surprised,” David Gortler, a pharmacologist, pharmacist, and FDA reform advocate at the Ethics and Public Policy Center, told The Epoch Times in an email. As a former senior advisor to the FDA commissioner, Mr. Gortler said he saw the agency grant expedited approval to a medication called aducanumab—used to treat Alzheimer’s disease “based on zero positive studies.”
“They did the same with other monoclonal antibodies for Alzheimer’s disease,” Mr. Gortler said.
According to the research letter, most of the 413 studies evaluating the 37 drugs approved in 2022 were sponsored by the industry—meaning they were manufactured, funded, and analyzed by the company producing the product, seeking FDA approval, and standing to benefit financially from the drug.
Of the studies available for analysis, only 25 percent of study results have been made publicly available, with the results of six percent of those studies published after the FDA had already approved the drug for use.
Furthermore, researchers found that only 55 percent of studies evaluating drugs in 2022 consisted of randomized clinical trials—the “gold standard” of evidence-based medicine—despite the FDA justifying most approvals based on randomized clinical trial data.
For comparison, only 20 percent of medical products in 2016 were approved based on a single study, and 55 percent were approved based on three or more studies, whereas 65 percent of drugs in 2022 were approved based on a single study, with only 11 percent having three or more studies.
“We believe consumers deserve access to the full range of evidence for the drugs they are considering, not just from the selected studies released to the public,” the authors wrote.
The researchers say their results “highlight a trend toward less rigorous standards for novel drug approvals that has evolved over the past few decades” and are consistent with other reports showing a widespread decrease in the number of trials used for drug approvals.
“The authors point to the deterioration of the quality and rigor of the regulatory review and approval of new drugs over time,” Sasha Latypova told The Epoch Times in an email. Ms. Latypova is a retired pharmaceutical industry executive with 25 years of experience in pharmaceutical research and development and co-founder of several organizations that work with pharmaceutical companies to design, execute, collect data, and submit clinical trial data to the FDA.
Ms. Latypova says this trend began with a “fast track” designation implemented in 1988 that increased the number of special regulatory programs available by the FDA and decreased the evidentiary requirements for approval. In the 2000s, Ms. Latypova said many blockbuster drugs became generic medicines, which started a “patent cliff” where industry investments began to focus on narrower niches in an effort to get patent exclusivity—which is more profitable for a pharmaceutical company.
“For example, approvals receiving an ‘orphan’ designation or what is considered rare disease increased to over 50% percent,” Ms. Latypova said. “These products are sometimes approved on as little as a single observational study with fewer than 20 subjects, however, once approved, the drug’s price increased one million to three million dollars per treatment and was fully covered by the taxpayer and private insurance—driving the costs of premiums.”
Thus, the “regulatory requirements are minimal, but the profits are outsized,” she added.
FDA Cures Act Made It Easier for Pharma and Regulatory Agencies to Cut Corners
The FDA, on its website, states the intent of the Cures Act (pdf) passed by Congress in December 2016 was to “incorporate the perspectives of patients into the development of drugs, biological products, and devices in FDA’s decision-making process” and enhance its ability to “modernize clinical trial designs,” including the use of “real world evidence” to speed up the development and review of novel medical products, including emergency and preparedness response countermeasures used to justify rapid authorization of COVID-19 vaccines.
Allowing the FDA to consider real-world evidence instead of randomized trials previously required under its strict methodological standards used to evaluate the safety and efficacy of a drug relaxed requirements for pharmaceutical companies and opened the door for bias.
The Cures Act gave new authority to the FDA to “recruit and retain scientific, technical, and professional experts and it establishes new expedited product development programs” and directed the agency to create one or more intercenter institutes to assist with coordination of activities between the drug, biologics, and device centers to improve the regulation of combination products.
The 312-page Act provided $500 million U.S. tax dollars to help the FDA implement the law over nine years and provided $6.3 billion in funding, mainly to the National Institutes of Health (NIH), a major funder of American universities and research institutions.
According to the National Center for Health Research (NCHR), the Cures Act dramatically benefits pharmaceutical and medical device companies, lowers the standards for drugs and devices, and makes it difficult for patients and physicians to decide whether to try a new treatment without knowing if it is safe or effective. This may explain why the Act was originated and promoted by major pharmaceutical companies, universities, and other organizations that hired more than 1,455 lobbyists to advance the bill.
The NCHR says the bill has had the following effects:
- Allowed anecdotal, unreliable, and easily manipulated health data to be used to approve new drugs.
- Allowed pharmaceutical and device companies to bypass public reporting requirements related to funding and gifts provided to physicians.
- Weakened patient safety by lowering the evidentiary standards required to prove a new drug or medical device is safe and effective.
- Allowed companies to disseminate potentially inaccurate scientific information not evaluated as part of the FDA approval process, opening the door for widespread use of drugs and treatments not FDA-approved.
- Reduced the FDA’s authority to regulate electronic health records systems and other medical software, which, if defective, can lead to deaths and permanent harm.
- Encourages smaller and shorter-term studies that are less likely to measure product safety and effectiveness for excluded parts of the population who may rely on them.
FDA Is Not Enforcing Reporting Requirements for Clinical Trials
Problems with clinical trial reporting go back to a law passed in 2007 requiring companies, universities, and other institutions to publish most clinical trial data in a federal database so that doctors and patients can determine whether a new product is safe or effective, according to a Science analysis. After trial sponsors failed to follow the law, the NIH and FDA attempted in 2017 to enact a final rule explaining the requirements and penalties for failing to disclose clinical trial results. Yet many sponsors ignored the requirements, and federal officials have done “little or nothing” to enforce the law.
The analysis of more than 4,700 clinical trials that should have been published on the NIH database ClinicalTrials.gov under the 2017 rule showed improved compliance rates of most large pharmaceutical companies and some universities. Yet the performance of many other sponsors, including the NIH, was “lackluster.”
ClinicalTrials.gov is an online registry of clinical trials run by the National Library of Medicine at NIH, where researchers, doctors, and patients are supposed to be able to see data on trial outcomes from peer-reviewed publications and can compare results across trials. Yet according to the analysis, thousands of trials are never published, especially when treatments are shown to be ineffective.
The Science analysis showed roughly 67 percent of studies from 30 of the 184 sponsor organizations with at least five trials failed to report any results on ClinicalTrials.gov, reinforcing the 2022 data published in the JAMA research letter.
Perhaps even more concerning is that these organizations consisting of pharmaceutical companies, universities, and medical centers failed to meet a single deadline. Those considered “habitual violators” didn’t report results in 67 percent of their trials and were an average of 268 days late disclosing data past their original deadlines.
These institutions included Harvard University, the University of Minnesota, and Baylor College of Medicine—leading recipients of NIH grants in 2019. Researchers found that The University of Texas MD Anderson Cancer Center and Mayo Clinic both failed to report results on time, or at all, in nearly two-thirds of their clinical trials. Yale University did not report results in 84 percent of its trials.
The NIH is tasked with reporting results when they sponsor studies done by agency staff or certain grantees, and the top four NIH institute sponsors reported results late or not at all in more than six of every ten trials assessed by Science. In addition, the Science analysis found the sponsors violated the reporting law more than 55 percent of the time and identified hundreds of cases where sponsors were credited for reporting results where the results themselves were not publically posted.
Despite the 2017 rule promising “aggressive enforcement and stiff penalties,” the NIH and FDA have not penalized sponsors who have not followed the requirements. The FDA in 2019 said it would not enforce penalties of up to $12,103 a day for failing to report a trial’s results until the agency issues further guidance on how it will exercise its power.
The FDA and NIH did not respond to requests for comment at the time of press.
Megan Redshaw
J.D.
Megan Redshaw is an attorney and investigative journalist with a background in political science. She is also a traditional naturopath with additional certifications in nutrition and exercise science.
https://www.theepochtimes.com/health/fda-approved-65-percent-of-new-drugs-in-2022-based-on-a-single-study-5453802?utm_source=brightnoe&src_src=brightnoe&utm_campaign=bright-2023-08-23&src_cmp=bright-2023-08-23&utm_medium=email&est=ZEY2sBilrl3%2F33K45ssrKxiNDzZ%2Ba0%2BiyRlKL1WP8uXw7pL2Eb3EY%2FnFzg%3D%3D
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