March 12, 2019
“When drug developers design a clinical trial, they identify eligibility criteria to define what types of patients qualify for participation in the trial. They base the eligibility criteria on factors such as the mechanism of action of the drug, characteristics of the disease, the expected toxicities of the investigational drug and the ability to recruit trial participants from the patient population to meet the objectives of the clinical trial. However, in trials testing treatments for cancer, some eligibility criteria have become commonly accepted over time or used as a template across trials without a clear scientific or clinical rationale or justification. In other cases, eligibility criteria can be deliberately restrictive, even though it is not clinically merited. As a result, cancer patients are often unnecessarily restricted from participating in trials,” said FDA Commissioner Scott Gottlieb, M.D. “Overly restrictive eligibility criteria may slow patient accrual, limit patients’ access to clinical trials and lead to trial results that don’t fully represent treatment effects in the patient population that will ultimately receive the drug. Today, the FDA issued new recommendations for broadening cancer trial eligibility criteria that are designed to help address these challenges. A clinical trial that’s more representative of the patient population can maximize the generalizability of the trial results and the ability to understand the therapy’s benefit-risk profile across the patient population likely to receive the drug in clinical practice. For example, broadening eligibility criteria to include pediatric patients, when appropriate, may provide information on safe and effective use in children and may accelerate the development of effective therapies for pediatric patients. Another patient population that’s typically excluded from clinical trials is patients with HIV, Hepatitis B and Hepatitis C Virus infections. Including these patients in clinical trials can be justified in many cases and may accelerate the development of effective therapies in cancer patients that also have these chronic infections. Additionally, patients with brain metastases, organ dysfunction and prior or concurrent malignancies are typically unnecessarily excluded from cancer clinical trials. These are all patient groups that we aim to address with the new policies being released today. These policies will help ensure that the design of clinical trials reflects the diversity of the population that receives drugs in the real world. For far too long, certain patients have been unnecessarily excluded from the chance to be a part of a clinical trial. Today’s policies will help to shift the design of oncology clinical trials to be more representative of the patients that may ultimately benefit from novel treatments.”
Today, the U.S. Food and Drug Administration published four draft guidances and one final guidance regarding cancer trial eligibility criteria. These guidances provide recommendations on how sponsors can safely and effectively broaden the criteria for the inclusion of certain patient populations in clinical trials, when appropriate, for pediatric patients, and patients with Human Immunodeficiency Virus, Hepatitis B and Hepatitis C Virus Infections, brain metastases, prior or concurrent malignancies or organ dysfunction.
The four draft guidances were developed by the FDA with input from stakeholders, including the American Society of Clinical Oncology and Friends of Cancer Research, to encourage inclusiveness in cancer trial enrollment to maximize the generalizability of trial results.
The final guidance, Considerations for the Inclusion of Adolescent Patients in Adult Oncology Clinical Trials, finalizes the draft guidance of the same name issued on June 4, 2018.
- Cancer Clinical Trial Eligibility Criteria: Minimum Age for Pediatric Patients; Draft Guidance for Industry
- Cancer Clinical Trial Eligibility Criteria: Patients with HIV, Hepatitis B Virus or Hepatitis C Virus Infections; Draft Guidance for Industry
- Cancer Clinical Trial Eligibility Criteria: Patients with Organ Dysfunction or Prior or Concurrent Malignancies; Draft Guidance for Industry
- Cancer Clinical Trial Eligibility Criteria: Brain Metastases; Draft Guidance for Industry
- Considerations for the Inclusion of Adolescent Patients in Adult Oncology Clinical Trials; Guidance for Industry
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.