FDA Issues Draft Guidance on Multi-Regional Clinical Trials

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The FDA issued draft guidance that provides recommendations for companies holding multi-regional clinical trials (MRCT) for cancer drugs.¹ Trials such as these are important in determining the effect a drug or treatment has on a variety of populations, but they can also produce skewed results. According to a press release from FDA, MRCTs are including shrinking proportions of US citizens, which may make the results less effective for determining the impact on the US population.

As FDA is a US agency, its goal is to determine the safety and efficacy of drugs and treatments on the US population.

In a press release, FDA’s director of its Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in FDA’s Center for Drug Evaluation and Research Richard Pazdur, MD, said, “The FDA encourages sponsors to pursue multi-regional clinical trials, but stresses that such trials should be conducted within the appropriate context. It is important that data from multi-regional clinical trials are applicable to patients in the United States who may use the drug and our current standards of oncological care. The new draft guidance, when finalized, will not only support the agency's review of data generated from multi-regional clinical trials, but also help sponsors improve the generalizability and applicability of results from these trials to the U.S. population and to U.S. medical practice."

This is just the latest draft guidance issued by FDA. In July, the agency issued guidance related to companies responding to misinformation about clear and approved medical products.²

In a press release issued at the time, FDA commissioner Robert M. Califf, MD, said, “Regulated industry plays a critical role in ensuring consumers have accurate information about medical products. We've updated our draft guidance to help further ensure industry has clarity and additional flexibility to promptly and proactively issue responsive communications to address misinformation they are seeing. The growing spread of rumors about science and medicine continues to put patients and consumers at risk. We remain steadfast in our commitment to address this public health concern and continue to support and encourage all parties in the public health ecosystem to take an active role."

In June, the agency also issued guidance that provided pharma companies with diversity action plans for clinical trials.³

“Participants in clinical trials should be representative of the patients who will use the medical products," Califf said in a press release issued at the time. "The agency's draft guidance is an important step—and one of many ongoing efforts—to address the participation of underrepresented populations in clinical trials to help improve the data we have about patients who will use the medical products if approved."

Also, in April, FDA issued guidance aimed at ensuring the safety of LDTS.⁴

"LDTs are being used more widely than ever before – for use in newborn screening, to help predict a person's risk of cancer, or aid in diagnosing heart disease and Alzheimer's. The agency cannot stand by while Americans continue to rely on results of these tests without assurance that they work," said Califf said. "The final rule announced today aims to provide crucial oversight of these tests to help ensure that important health care decisions are made based on test results that patients and health care providers can trust."

Sources

1. FDA Issues Draft Guidance on Conducting Multiregional Clinical Trials in Oncology. U.S. Food and Drug Administration. September 16, 2024. https://www.prnewswire.com/news-releases/fda-issues-draft-guidance-on-conducting-multiregional-clinical-trials-in-oncology-302249082.html
2. FDA Updates Guidance to Further Empower Companies to Address the Spread of Misinformation. U.S. Food and Drug Administration. July 8, 2024. Accessed September 16, 2024. https://www.prnewswire.com/news-releases/fda-updates-guidance-to-further-empower-companies-to-address-the-spread-of-misinformation-302190916.html
3. FDA Guidance Provides New Details on Diversity Action Plans Required for Certain Clinical Studies. U.S. Food and Drug Administration. June 26, 2024. Accessed September 16, 2024. https://www.prnewswire.com/news-releases/fda-guidance-provides-new-details-on-diversity-action-plans-required-for-certain-clinical-studies-302183322.html
4. FDA Takes Action Aimed at Helping to Ensure the Safety and Effectiveness of Laboratory Developed Tests. U.S. Food and Drug Administration. April 29, 2024. Accessed September 16, 2024. https://www.prnewswire.com/news-releases/fda-takes-action-aimed-at-helping-to-ensure-the-safety-and-effectiveness-of-laboratory-developed-tests-302130166.html