FDA Publishes Guidances Aimed at More Inclusive Eligibility Criteria for Cancer Clinical Trials

March 28, 2019 Becky Hurt

Authored By: Amy McKee, VP Regulatory Consulting, Parexel

Clinical trials in oncology historically have enrolled approximately 5% of cancer patients in the United States1. Many barriers to clinical trial participation have been identified, including patient-related barriers such as geography, providers who do not query whether a patient is interested in a clinical trial, fear of side effects, costs to patient, and eligibility criteria. Restrictive eligibility criteria can limit accrual to trials and prolong enrollment periods.

In March 2019, the FDA published four draft guidances and one final guidance that address specific eligibility criteria in cancer clinical trials that can be modernized based on current understanding of comorbidities and past medical history as well as age of patients2 3 4 5 6. These guidances include:

  • Cancer Clinical Trial Eligibility Criteria: Patients with HIV, Hepatitis B Virus, or Hepatitis C Virus Infections (draft)
  • Cancer Clinical Trial Eligibility Criteria: Patients with Organ Dysfunction or Prior or Concurrent Malignancies (draft)
  • Cancer Clinical Trial Eligibility Criteria: Brain Metastases (draft)
  • Cancer Clinical Trial Eligibility Criteria: Minimum Age for Pediatric Patients (draft)
  • Considerations for the Inclusion of Adolescent Patients in Adult Oncology Clinical Trials (final)

Expanding the eligibility criteria for cancer clinical trials may maximize the generalizability of trial results across the patient population in whom the drug is likely to be used upon marketing approval. Some eligibility criteria have become commonly accepted over time without reconsidering the scientific or clinical rationale for such criteria. For example, human immunodeficiency virus (HIV), hepatitis B virus (HBV), and hepatitis C virus (HCV) infections can be managed chronically or cured with current antiviral treatments; thus, inclusion of these patients in clinical trials should be reconsidered in light of the current life expectancies of these patients.

The guidances contain key recommendations for specific comorbidities or conditions and related inclusion or exclusion criteria, including:

  • Renal, cardiac and hepatic function recommendations;

  • Recommendations for patients with brain metastases that are active or treated/stable and for patients with leptomeningeal disease;

  • Inclusion of children and adolescents in clinical trials based on factors such as biologic rationale, preclinical data, predictive biomarkers and evidence from completed clinical studies;

  • Patients with HIV, HBV or HCV;

  • and recommendations for inclusion of patients with prior or concurrent malignancies.

In summary, these guidances provide clear direction from FDA on specific inclusion and exclusion criteria for cancer clinical trials based upon current scientific knowledge and clinical practice. Parexel's subject matter experts, who include former regulators, industry professionals, and physicians, have a broad and deep internal expertise in all phases of oncology drug development as well as Regulatory Affairs experience. Parexel can help you every step of the way throughout your oncology product’s lifecycle.



1 Murthy VH, Krumholz HM, Gross CP. Participation in cancer clinical trials: race-, sex-, and age-based disparities. JAMA. 2004 Jun 9;291(22):2720–6.

2 https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM633136.pdf

3 https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM633137.pdf

4 https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM633132.pdf

5 https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm633138.pdf

6 https://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM609513.pdf

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